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New cancer treatment may reawaken the immune system

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Immunotherapy is a promising strategy to treat cancer by stimulating the body’s own immune system to destroy tumor cells, but it only works for a handful of cancers. MIT researchers have now discovered a new way to jump-start the immune system to attack tumors, which they hope could allow immunotherapy to be used against more types of cancer.

Their novel approach involves removing tumor cells from the body, treating them with chemotherapy drugs, and then placing them back in the tumor. When delivered along with drugs that activate T cells, these injured cancer cells appear to act as a distress signal that spurs the T cells into action.

“When you create cells that have DNA damage but are not killed, under certain conditions those live, injured cells can send a signal that awakens the immune system,” says Michael Yaffe, who is a David H. Koch Professor of Science, the director of the MIT Center for Precision Cancer Medicine, and a member of MIT’s Koch Institute for Integrative Cancer Research.

In mouse studies, the researchers found that this treatment could completely eliminate tumors in nearly half of the mice.

Yaffe and Darrell Irvine, who is the Underwood-Prescott Professor with appointments in MIT’s departments of Biological Engineering and Materials Science and Engineering, and an associate director of the Koch Institute, are the senior authors of the study, which appears today in Science Signaling . MIT postdoc Ganapathy Sriram and Lauren Milling PhD ’21 are the lead authors of the paper.

T cell activation

One class of drugs currently used for cancer immunotherapy is checkpoint blockade inhibitors, which take the brakes off of T cells that have become “exhausted” and unable to attack tumors. These drugs have shown success in treating a few types of cancer but do not work against many others.

Yaffe and his colleagues set out to try to improve the performance of these drugs by combining them with cytotoxic chemotherapy drugs, in hopes that the chemotherapy could help stimulate the immune system to kill tumor cells. This approach is based on a phenomenon known as immunogenic cell death, in which dead or dying tumor cells send signals that attract the immune system’s attention.

Several clinical trials combining chemotherapy and immunotherapy drugs are underway, but little is known so far about the best way to combine these two types of treatment.

The MIT team began by treating cancer cells with several different chemotherapy drugs, at different doses. Twenty-four hours after the treatment, the researchers added dendritic cells to each dish, followed 24 hours later by T cells. Then, they measured how well the T cells were able to kill the cancer cells. To their surprise, they found that most of the chemotherapy drugs didn’t help very much. And those that did help appeared to work best at low doses that didn’t kill many cells.

The researchers later realized why this was so: It wasn’t dead tumor cells that were stimulating the immune system; instead, the critical factor was cells that were injured by chemotherapy but still alive.

“This describes a new concept of immunogenic cell injury rather than immunogenic cell death for cancer treatment,” Yaffe says. “We showed that if you treated tumor cells in a dish, when you injected them back directly into the tumor and gave checkpoint blockade inhibitors, the live, injured cells were the ones that reawaken the immune system.”

The drugs that appear to work best with this approach are drugs that cause DNA damage. The researchers found that when DNA damage occurs in tumor cells, it activates cellular pathways that respond to stress. These pathways send out distress signals that provoke T cells to leap into action and destroy not only those injured cells but any tumor cells nearby.

“Our findings fit perfectly with the concept that ‘danger signals’ within cells can talk to the immune system, a theory pioneered by Polly Matzinger at NIH in the 1990s, though still not universally accepted,” Yaffe says.  

Tumor elimination

In studies of mice with melanoma and breast tumors, the researchers showed that this treatment eliminated tumors completely in 40 percent of the mice. Furthermore, when the researchers injected cancer cells into these same mice several months later, their T cells recognized them and destroyed them before they could form new tumors.

The researchers also tried injecting DNA-damaging drugs directly into the tumors, instead of treating cells outside the body, but they found this was not effective because the chemotherapy drugs also harmed T cells and other immune cells near the tumor. Also, injecting the injured cells without checkpoint blockade inhibitors had little effect.

“You have to present something that can act as an immunostimulant, but then you also have to release the preexisting block on the immune cells,” Yaffe says.

Yaffe hopes to test this approach in patients whose tumors have not responded to immunotherapy, but more study is needed first to determine which drugs, and at which doses, would be most beneficial for different types of tumors. The researchers are also further investigating the details of exactly how the injured tumor cells stimulate such a strong T cell response.

The research was funded, in part, by the National Institutes of Health, the Mazumdar-Shaw International Oncology Fellowship, the MIT Center for Precision Cancer Medicine, and the Charles and Marjorie Holloway Foundation.

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  • Published: 24 March 2021

Advancing Cancer Therapy

Nature Cancer volume  2 ,  pages 245–246 ( 2021 ) Cite this article

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Cancer therapies have evolved considerably in recent decades, substantially improving the quality of life and survival of patients with cancer. In this issue, we launch our Series on Cancer Therapy, exploring current paradigms and recent advances and challenges in this field, through specially commissioned articles.

The earliest evidence of cancer treatment can be traced back to an ancient Egyptian medical text, written around 3000 BC and known widely as the ‘Edwin Smith Papyrus’, that described the cauterization of breast tumors for which, according to the text, there was no cure . The situation is very different now, as, depending on breast cancer subtype, stage and demographic factors, the 5-year survival rates for this disease can surpass 90% in developed countries. For cancer types that are responsive to therapy, including certain subtypes of breast, blood and prostate malignancies, patients now face the management of a chronic disease, rather than a fatal one, owing to the rapid advances in clinical oncology over recent decades. Similarly, the prognosis for several other cancer types has also been improving. For example, patients with melanoma, which used to be considered a deadly disease, have much better prospects thanks to the breakthroughs in targeted and immune-based therapies.

These advances reflect the focus placed on cancer research and oncology by governments, funders and research institutes across the globe over the past several decades. In the USA, 2021 marks the 50-year anniversary of the signing of the National Cancer Act into law, which marked the beginning of a concerted effort to address cancer as a leading cause of death in the USA at the federal level. The National Cancer Program that arose from this initiative resulted in a profound institutional reorganization within the National Institutes of Health, with the overarching goal of developing the infrastructures required ‘for the treatment, cure, and elimination of cancer’. Other countries and international agencies also adopted cancer-focused initiatives over the years, including, for example, the PRIME scheme of the European Medicines Agency, which supports the development of medicines that target an unmet medical need, including cancer, through accelerated planning, evaluation and approval processes.

Thus, substantial progress has been made across first-line cancer therapy modalities. Surgery continues to be a first-line treatment for many cancer types, but it now includes precision and minimally invasive surgery, molecular imaging support and, more recently, robot- or artificial intelligence–assisted surgical procedures. The clinical use of one of the most widely used treatment modalities, chemotherapy, has been improved through better dosing regimens, neoadjuvant or adjuvant administration, and combination therapies. Similarly, radiation oncology has been advanced through precision radiotherapy. First-line recommendations depend on the cancer type and stage at diagnosis, and have continued to be modified as new therapeutic modalities have become available. The advent of targeted therapy and immunotherapy has revolutionized the treatment of cancer, especially with the development and availability of sophisticated diagnostic and molecular characterization technologies. Among these, ‘-omics’ techniques stand out for increasingly enabling a more precise and granular molecular characterization of cancer types and subtypes and the identification of biological correlates of response to specific therapies, thereby enriching the roster of biomarkers at the disposal of clinicians.

Targeted therapies have swiftly taken a prominent position in cancer research and clinical oncology in recent decades, thanks to the molecular insights into oncogenic processes and mechanisms gained from fundamental research and technological development. A key example of how basic research on oncogenic alterations translated into substantial clinical benefits for a large number of patients is BCR-ABL1 tyrosine-kinase inhibitors for chronic myeloid leukemia. The first BCR-ABL1 tyrosine-kinase inhibitor was discovered through drug screens in 1992, and in 2001 it became the first-line therapy with long-term remission rates for BCR-ABL–driven chronic myeloid leukemia 1 ; second-generation tyrosine-kinase inhibitors, rationally designed to circumvent acquired resistance, earned approval from the US Food and Drug Administration as frontline therapies only a decade later. More recently, the announcement of the two first-in-class inhibitors of the mutant kinase KRAS G12C was a milestone in the decades-long efforts to study and treat tumors bearing these, up-to-now considered undruggable, KRAS mutations 2 . However, not every effort in precision oncology and targeted therapy is yielding similarly positive results, especially given the issue of adaptive and acquired resistance, a complication of therapy that a large part of the cancer-research community is striving to address. It should also be noted that advances in sophisticated cancer therapeutics are sometimes associated with a high financial burden for patients, a pressing societal issue tied to the complexities of addressing the challenge of cancer 3 .

In light of the progress made so far and the goals and challenges ahead, we are pleased to launch in this issue of Nature Cancer a Series on Cancer Therapy comprising specially commissioned Review, Perspective, News and Comment articles and a collection of relevant primary research articles published in Nature Cancer . The series is housed in a dedicated page on the Nature Cancer website and will be continually updated with additional content from key opinion leaders discussing novel therapeutic opportunities, the path to drug discovery, and how these advances are transforming clinical practice.

Our series launches with two Review articles that focus on different but important aspects of cancer treatment. Whereas substantial achievements have been witnessed in the treatment of primary tumors, progress has been more modest for metastatic disease. Yibin Kang and colleagues discuss the clinical challenge of treating metastatic disease, and how preclinical and mechanistic knowledge accumulated over the years is being translated into tangible clinical benefits for disseminated disease 4 . The authors also discuss the challenges of running clinical trials for metastatic disease, and the different degrees of success of clinical trials in the metastatic setting. In a separate Review, Frank McCormick and colleagues discuss the multiple and complex links between oncogenic KRAS—one of the most frequently mutated and, as noted above, hard-to-target cancer drivers—and metabolism, highlighting the potentially targetable vulnerabilities that arise at the interface of the two 5 . Although various aspects of targeting KRAS-dependent cancer metabolism have been explored extensively in preclinical settings, ongoing and future clinical trials will hopefully shed light on the translatability of these approaches to the clinic.

Despite the many milestones achieved in cancer treatment, much remains to be addressed. In future issues we will present additional pieces focusing on a breadth of topics under this theme, including key pathways deregulated in cancer, such as EGFR or PI3K, and ongoing clinical approaches for preventing and bypassing therapy resistance. Future issues will also discuss progress in radiotherapy, immunotherapy and therapy combinations, as well as new therapeutic modalities, such as bispecific antibodies, and innovative drug-development approaches through the implementation of artificial intelligence.

Through this selection of commissioned and primary research publications, we aim to underscore how much cancer therapy has advanced over the past several decades, which goals need to be prioritized, and the challenges that should be overcome to continue improving quality of life and outcomes for patients with cancer. We thank our authors and referees for their valuable contributions and hope that our readers will find this Series on Cancer Therapy informative and inspiring.

Druker, B. J. et al. N. Engl. J. Med. 344 , 1038–1042 (2001).

Article   CAS   Google Scholar  

Bar-Sagi, D., Knelson, E. H. & Sequist, L. V. Nat. Cancer 1 , 25–27 (2020).

Article   Google Scholar  

Gruber, K. Nat. Cancer 1 , 1136–1139 (2020).

Esposito, M. et al. Nat. Cancer https://doi.org/10.1038/s43018-021-00181-0 (2021).

Mukhopadhyay, S. et al. Nat. Cancer https://doi.org/10.1038/s43018-021-00184-x (2021).

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research about cure for cancer

Cancer therapy approved by FDA uses body’s own cells as a ‘living drug’

The treatment supercharges the body’s immune system to kill a kind of skin cancer, opening the door to targeting more common tumors.

research about cure for cancer

After undergoing surgery, radiation and three different therapies, Scott Goedeke faced a tough reality: The cancer that first surfaced on the roof of his mouth had spread to a lymph node in his neck. So the 58-year-old health-care consultant agreed to an experimental treatment that would deploy his own cells to destroy it.

“I have to do this,” he recalled thinking at the time.

His medical team at the Siteman Cancer Center in St. Louis extracted one tumor, identified the cells that could attack the disease and multiplied them in a lab. Billions of cells were then infused back into his body in the hope of supercharging his body’s natural defenses, overwhelming the cancer.

Six weeks later, the tumor had shrunk significantly.

Now this first-of-its-kind cancer therapy has crossed a major milestone. On Friday, the U.S. Food and Drug Administration approved its use to treat adults with a skin cancer like Goedeke’s — melanoma that has spread or can’t be removed with surgery, after other approaches have failed. Though the condition is uncommon, cancer experts say winning FDA approval could mark the beginning of a potent new weapon against far more common tumors.

Shares of Iovance Biotherapeutics, the California-based company that makes the therapy, surged more than 30 percent on Tuesday, the first full day of trading following the FDA’s approval.

“The concept that the FDA has now acknowledged is that you can use a patient’s own cells as a living drug to treat their disease, and that to me is a very exciting step forward,” said Steve Rosenberg, a senior investigator for the National Cancer Institute who has helped pioneer the newly approved therapy since the 1980s. “It’s the start of a new era in the development of a new approach to treating cancer,” he said, describing ongoing research regarding more common deadly tumors such as those in breast, pancreatic and colon cancer.

Scientists have for decades worked to develop cancer treatments that exploit the body’s own immune system to seek out and destroy malignant tumors. This field has produced breakthroughs in recent years, including drugs that help the body recognize and attack cancer cells. Another tool, approved by the FDA in 2017, extracts the body’s cancer-killing cells and programs them to attack a particular kind of blood-based tumor.

The new individualized therapy, called tumor-infiltrating lymphocytes, enlists the relatively small number of the body’s T cells that see a tumor as a threat and produces a lab-grown army of them. Of the 73 patients treated in a clinical trial, 31.5 percent of them had their tumors decrease in size or disappear after the cell treatment, the FDA said.

The FDA approval “represents the culmination of scientific and clinical research efforts leading to a novel T cell immunotherapy for patients with limited treatment options,” Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said in a statement Friday.

Iovance Biotherapeutics has priced the therapy, branded Amtagvi, at $515,000 per patient. The FDA approval is “the first step in realizing Iovance’s ambition to usher in the next generation of cell therapy by bringing this breakthrough to patients with advanced solid tumors,” Iovance interim chief executive Frederick Vogt said in a statement Friday.

The FDA action is also a signal to the pharmaceutical industry that there is a commercial path to success, said Jason Bock, chief executive of CTMC, a company spun out of MD Anderson Cancer Center that contracts with biotech firms to help manufacture cell therapies. The approval, he said, “was 40 years in the making” for “one of the most complex therapeutics we’ve ever tried to develop.”

Goedeke, the cancer patient, went through chemotherapy to prepare his body for the cell therapy. He remembers the day his cells were infused back into his body — Jan. 25, 2023 — at Siteman Cancer Center. They came in a special container. The procedure took perhaps half an hour, and was followed by other infusions to help the body put the new cells to work.

After he was discharged from the hospital, Goedeke met with his oncologist to discuss the results of the latest scan for cancer. These visits were always stressful, and Goedeke’s wife joined him.

George Ansstas, a Washington University oncologist at Siteman, broke the news six weeks after the therapy: Goedeke was a “responder” to the experimental therapy.

“I don’t think I can overstate the relief and exhilaration,” Goedeke said. “We couldn’t get out to the car fast enough to share the news.”

Of the FDA’s approval, he said, “I’m super happy other patients will be able to access this.”

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Cancer Treatments and Research

Learn more about the progress made in improving cancer survival rates

Cancer Treatment Development

Radiotherapy, immunotherapy, targeted therapy.

  • Combination Therapies

Diagnostics

Considerable progress has been made in reducing cancer rates and improving cancer survival in the United States since the 1990s. A greater understanding of the immune system , genetics , and cancer pathology has opened the doors to an ever-increasing range of new cancer treatments and diagnostic tools.

Advances in cancer care have been highly specific in terms of the diagnostic and treatment modalities that are recommended for each type of cancer. This article will describe these key treatments as well as the process of cancer treatment development.

sanjeri / Getty Images

Throughout the years, there have been discoveries of drugs and treatment methods that prove to be more successful or reliable than previous ones. These treatment methods are discovered in different ways.

Some are found in nature through the testing and studying of plants, fungi, and animals. Others are found through the study of cancer cells and existing drugs or procedures. But before any type of treatment method is used on patients, there is an important process that ensures its safety and effectiveness.

New cancer drugs typically go through stages of clinical research. These stages are:

  • Preclinical research : Preclinical research aims to ensure a form of treatment is safe for human use. Laboratory studies that include animal research and in vitro studies , or experiments usually done in test tubes and Petri dishes, are common in this research stage.
  • Clinical research : After preclinical research is successful, clinical research focuses on testing the form of therapy on humans. This clinical research stage can be lengthy (up to 10 years or more) as the discovered treatment goes through phases of clinical trials .
  • Post-clinical research : Post-clinical research involves studying a therapy that has gone through the clinical research phase and received approval for human use. This involves collecting data on effectiveness and safety in real-world use.

Advances in and refinement of cancer surgery—including the use of targeted drugs and other medications before and after surgery—that can improve outcomes for cancer patients continue to emerge.

Studies comparing the outcomes of different surgical methods have helped guide doctors in selecting the technique that is most likely to result in a better long-term prognosis.

Video-Assisted Thoracoscopic Surgery (VATS) Lobectomy for Lung Cancer

During a lobectomy , a portion of a lobe of a lung that is affected by cancer is removed.

The minimally invasive technique known as VATS lobectomy, done with general anesthesia , often involves a shorter recovery time than open surgery for lung cancer . The American College of Chest Physicians identifies VATS lobectomy as the preferred method for treating early-stage lung cancer.

During the procedure, a thoracoscope, which is a small tube with a light and camera attached to the end, is inserted between the ribs through a small incision. The affected lung tissue is then removed using special tools.

Open Surgery for Cervical Cancer

In a clinical trial between 2008 and 2013, 631 women were enrolled to compare the efficacy of open surgery with that of minimally invasive surgery for the treatment of cervical cancer .

Postoperative quality of life for both groups was similar. But open surgery resulted in lower rates of cancer recurrence and higher disease-free survival.

Another study found that patients with early-stage cervical cancer who had minimally invasive surgery experienced higher recurrence rates than those who had open surgery, making open surgery a better option for some patients.

Radiation therapy is used as an adjunct to cancer treatment. More effective and targeted radiotherapies are being used to treat early and advanced cancers.

Stereotactic Ablative Radiotherapy (SABR) for Metastatic Cancer

A study demonstrated that patients receiving SABR in addition to standard of care showed improved survival compared with patients receiving palliative standard of care.  

SABR for Inoperable Early-Stage Lung Cancer

For patients who are not surgical candidates, SABR offers an alternative. This approach was shown to have excellent local control and well tolerated in a cohort of 273 patients.

Immunotherapy uses the body's immune system to fight cancer. Immunotherapy can boost or change how the immune system works so it can find and attack cancer cells.  

Molecular testing, which can help select patients most suitable for immunotherapy, has opened the door to this newer form of treatment. Some of the early and commonly used immunotherapy agents are vaccines, including the first FDA-approved cancer vaccine, sipuleucel-T, for prostate cancer .

Below are some breakthrough agents grouped by category:

  • Monoclonal antibodies , such as Trodelvy for metastatic triple-negative breast cancer
  • Oncolytic virus therapy , including Imlygic for inoperable melanoma
  • CAR T-cell therapy , such as CD22 for acute lymphoblastic leukemia relapse
  • Cancer vaccines , such as Provenge for prostate cancer

Targeted therapy is when drugs are directed at specific proteins or genes that promote cancer cell growth. It is designed to attack cancer cells directly.

Some of the targeted drugs commonly used to treat cancer are Tagrisso (osimertinib), Tarceva (erlotinib), and Iressa (gefitinib) for lung cancer, and Kadcyla (ado-trastuzumab), Tykerb (lapatinib), and Afinitor (everolimus) for breast cancer.

Kinase Inhibitors

Dysregulation of protein kinases is involved in many types of cancer, and this protein is the target of several cancer drugs.

Drugs like Rozlytrek (entrectinib) and Tabrecta (capmatinib) are used to treat metastatic non-small cell lung cancer .

  • Rozlytrek (entrectinib) is used to treat non-small cell lung cancer that is positive for ROS1 and the neurotrophic receptor tyrosine kinases (NTRK) fusion-positive solid tumors. It inhibits cell-proliferation while targeting ROS1, a receptor tyrosine kinase.
  • Tabrecta (capmatinib) is a tyrosine kinase inhibitor that can help to shrink tumors involving a MET mutation. The MET gene produces a receptor tyrosine kinase, which is involved in cell proliferation and cell survival.

Kinase Inhibitor

Our bodies contain enzymes called kinases, which help to regulate functional processes such as cell signaling and cell division. A kinase inhibitor blocks the action of kinases.

PARP Inhibitors

Drugs, such as Zejula, are used to treat ovarian cancer . The drug inhibits the enzymatic activity of enzyme poly (ADP-ribose) polymerase (PARP). In a study of 533 patients who had recurring ovarian cancer, Zejula increased the time experienced without symptoms compared with standard therapy.

Combination Therapies 

Combination therapy means using two forms of cancer therapy in conjunction. Newer classes of drugs are being combined with traditional chemotherapy to improve outcomes. This approach becoming the standard of care for treating some types of cancer.

One recent example is the combination of Tecentriq and Avastin in the treatment of liver cancer.

It is an ongoing area of critical research to develop better and more accurate diagnostic and screening techniques. Below are some next-generation technologies that are being developed. However, keep in mind these techniques (aside from ctDNA) have yet to be approved by the FDA.

Artificial Intelligence Mammograms

In a study that involved 28,296 independent interpretations, AI performance was comparable to radiologists' diagnostic ability for detecting breast cancer.

Liquid Biopsy for Breast Cancer

A liquid biopsy can detect circulating levels of cell-free DNA (cfDNA) and circulating tumor DNA (ctDNA).

In a meta-analysis that included 69 published research studies. with 5,736 breast cancer patients, researchers determined that the status of ctDNA mutation predicts disease recurrence and adverse survival results. They also found that the levels of cfDNA can predict metastasis of the axillary lymph node.

Monarch Robotic Endoscopy for Lung Cancer

This may be advantageous for patients with external lung lesions that need biopsy prior to surgery, radiation, targeted therapies, or immunotherapy.  

Genomic Cancer Screening in Embryos

A polygenic risk score used by genomic prediction accurately distinguished which person in a set of siblings will inherit a medical condition. The accuracy was cited between 70% and 90%, depending upon the condition.  

At-Home Urine Test for Prostate Cancer

A convenient, at-home urine test can be used to detect extracellular vesicle-derived RNA to provide prognostic information for men under active surveillance for prostate cancer.   

A Word From Verywell

Cancer research that is investigating better treatments and diagnostic tools is ongoing. Even if you have advanced metastatic cancer, it may be comforting to know that newer treatments are being studied and approved every year. As treatments become better and better, your chances of survival and remission will also improve. If you have been diagnosed with cancer, it may also help to seek a cancer support group to boost your mental well-being and resilience.

American Society of Clinical Oncology: Cancer.Net. How are cancer drugs discovered and developed .

Cancer.net Improvements in Surgery for Cancer: The 2020 Advance of the Year.

Berfield KS, Farjah F, Mulligan MS. Video-assisted thoracoscopic lobectomy for lung cancer . Ann Thorac Surg. 2019 Feb;107(2):603-609. doi: 10.1016/j.athoracsur.2018.07.088

Frumovitz M, Obermair A, Coleman RL, Pareja R, Lopez A, Ribero R. Quality of life in patients with cervical cancer after open versus minimally invasive radical hysterectomy (Lacc): a secondary outcome of a multicentre, randomised, open-label, phase 3, non-inferiority trial . Lancet Oncol . 2020 Jun;21(6):851-860. doi: 10.1016/S1470-2045(20)30081-4

Kim SI, Cho JH, Seol A, et al. Comparison of survival outcomes between minimally invasive surgery and conventional open surgery for radical hysterectomy as primary treatment in patients with stage IB1-IIA2 cervical cancer .  Gynecol Oncol . 2019;153(1):3-12. doi:10.1016/j.ygyno.2019.01.008

Palma DA, Olson R, Harrow S, Gaede S, Louie A, Haasbeek C. Stereotactic ablative radiotherapy versus standard of care palliative treatment in patients with oligometastatic cancers (Sabr-comet): a randomised, phase 2, open-label trial. Lancet. 2019 May 18;393(10185):2051-2058. doi: 10.1016/S0140-6736(18)32487-5

Murray L, Ramasamy S, Lilley J, et al. Stereotactic Ablative Radiotherapy (SABR) in Patients with Medically Inoperable Peripheral Early Stage Lung Cancer: Outcomes for the First UK SABR Cohort .  Clin Oncol (R Coll Radiol) . 2016;28(1):4-12. doi:10.1016/j.clon.2015.09.007

American Cancer Society. Immunotherapy .

Sastre J, Sastre-Ibañez M. Molecular diagnosis and immunotherapy . Curr Opin Allergy Clin Immunol . 2016 Dec;16(6):565-570. doi: 10.1097/ACI.0000000000000318

Vansteenkiste JF, Van De Kerkhove C, Wauters E, Van Mol P. Capmatinib for the treatment of non-small cell lung cancer.   Expert Rev Anticancer Ther . 2019;19(8):659-671. doi:10.1080/14737140.2019.1643239

Matulonis UA, Walder L, Nøttrup TJ, et al. Niraparib Maintenance Treatment Improves Time Without Symptoms or Toxicity (TWiST) Versus Routine Surveillance in Recurrent Ovarian Cancer: A TWiST Analysis of the ENGOT-OV16/NOVA Trial .  J Clin Oncol . 2019;37(34):3183-3191. doi:10.1200/JCO.19.00917

Breast Cancer Research Foundation. How Combination Therapies Are Changing the Landscape of Breast Cancer Care .

Finn RS, Qin S, Ikeda M, et al. Atezolizumab plus Bevacizumab in Unresectable Hepatocellular Carcinoma .  N Engl J Med . 2020;382(20):1894-1905. doi:10.1056/NEJMoa1915745

Rodriguez-Ruiz A, Lång K, Gubern-Merida A, et al. Stand-Alone Artificial Intelligence for Breast Cancer Detection in Mammography: Comparison With 101 Radiologists .  J Natl Cancer Inst . 2019;111(9):916-922. doi:10.1093/jnci/djy222

Alimirzaie S, Bagherzadeh M, Akbari MR. Liquid biopsy in breast cancer: A comprehensive review . Clin Genet . 2019 Jun;95(6):643-660. doi: 10.1111/cge.13514

Murgu SD. Robotic assisted-bronchoscopy: technical tips and lessons learned from the initial experience with sampling peripheral lung lesions. BMC Pulm Med. 2019 May 9;19(1):89. doi: 10.1186/s12890-019-0857-z

Lello L, Raben TG, Hsu SDH. Sibling validation of polygenic risk scores and complex trait prediction.   Sci Rep 10 ,  13190 (2020). doi.org/10.1038/s41598-020-69927-7

Connell SP, Hanna M, McCarthy F, et al. A Four-Group Urine Risk Classifier for Predicting Outcome in Prostate Cancer Patients [published online ahead of print, 2019 May 20].  BJU Int . 2019;124(4):609-620. doi:10.1111/bju.14811

10 new breakthroughs in the fight against cancer

A technician viewing cells on a microscope and another using a pipette at the National Cancer Institute.

Medical advances are continuing to help the world fight cancer. Image:  Unsplash/National Cancer Institute

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This article was originally published in May 2022, and most recently updated in January 2024 .

  • Cancer is one of the world’s biggest killers, with around 10 million deaths per year due to the disease.
  • Scientists are using artificial intelligence, DNA sequencing, precision oncology and other technologies to improve treatment and diagnosis.
  • The Centre for the Fourth Industrial Revolution India, a collaboration with the World Economic Forum, hopes to accelerate 18 cancer interventions.

Cancer kills around 10 million people a year and is a leading cause of death globally, according to the World Health Organization.

Breast, lung and colon cancer are among the most common. Death rates from cancer were falling before the pandemic . But COVID-19 caused a big backlog in diagnosis and treatment .

There is some good news, however. Medical advances are accelerating the battle against cancer. Here are 10 recent developments.

Test to identify 18 early-stage cancers

Researchers in the US have developed a test they say can identify 18 early-stage cancers. Instead of the usual invasive and costly methods, Novelna's test works by analyzing a patient's blood protein. In a screening of 440 people already diagnosed with cancer, the test correctly identified 93% of stage 1 cancers in men and 84% in women. The researchers believe the findings "pave the way for a cost-effective, highly accurate, multi-cancer screening test that can be implemented on a population-wide scale". It's early days, however. With such a small sample screening and a lack of information on co-existing conditions, the test is currently more of "a starting point for developing a new generation of screening tests for the early detection of cancer".

The seven-minute cancer treatment jab

England's National Health Service (NHS) is to be the first in the world to make use of a cancer treatment injection , which takes just seven minutes to administer, rather than the current time of up to an hour to have the same drug via intravenous infusion. This will not only speed up the treatment process for patients, but also free up time for medical professionals. The drug, Atezolizumab or Tecentriq, treats cancers including lung and breast, and it's expected most of the 3,600 NHS patients in England currently receiving it intravenously will now switch to the jab.

Precision oncology

Precision oncology is the “ best new weapon to defeat cancer ”, the chief executive of Genetron Health, Sizhen Wang, says in a blog for the World Economic Forum. This involves studying the genetic makeup and molecular characteristics of cancer tumours in individual patients. The precision oncology approach identifies changes in cells that might be causing the cancer to grow and spread. Personalized treatments can then be developed. The 100,000 Genomes Project, a National Health Service initiative, studied more than 13,000 tumour samples from UK cancer patients , successfully integrating genomic data to more accurately pin-point effective treatment. Because precision oncology treatments are targeted – as opposed to general treatments like chemotherapy – it can mean less harm to healthy cells and fewer side effects as a result.

Artificial intelligence fights cancer

In India, World Economic Forum partners are using emerging technologies like artificial intelligence (AI) and machine learning to transform cancer care. For example, AI-based risk profiling can help screen for common cancers like breast cancer, leading to early diagnosis. AI technology can also be used to analyze X-rays to identify cancers in places where imaging experts might not be available. These are two of 18 cancer interventions that The Centre for the Fourth Industrial Revolution India, a collaboration with the Forum , hopes to accelerate.

Infographic of sequenced DNA of cancer tumours.

Greater prediction capabilities

Lung cancer kills more people in the US yearly than the next three deadliest cancers combined. It's notoriously hard to detect the early stages of the disease with X-rays and scans alone. However, MIT scientists have developed an AI learning model to predict a person's likelihood of developing lung cancer up to six years in advance via a low-dose CT scan. Trained using complex imaging data, 'Sybil' can forecast both short- and long-term lung cancer risk, according to a recent study. "We found that while we as humans couldn't quite see where the cancer was, the model could still have some predictive power as to which lung would eventually develop cancer," said co-author Jeremy Wohlwend.

Clues in the DNA of cancer

At Cambridge University Hospitals in England, the DNA of cancer tumours from 12,000 patients is revealing new clues about the causes of cancer, scientists say. By analyzing genomic data, oncologists are identifying different mutations that have contributed to each person’s cancer. For example, exposure to smoking or UV light, or internal malfunctions in cells. These are like “fingerprints in a crime scene”, the scientists say – and more of them are being found. “We uncovered 58 new mutational signatures and broadened our knowledge of cancer,” says study author Dr Andrea Degasperi, from Cambridge’s Department of Oncology.

Liquid and synthetic biopsies

Biopsies are the main way doctors diagnose cancer – but the process is invasive and involves removing a section of tissue from the body, sometimes surgically, so it can be examined in a laboratory. Liquid biopsies are an easier and less invasive solution where blood samples can be tested for signs of cancer. Synthetic biopsies are another innovation that can force cancer cells to reveal themselves during the earliest stages of the disease.

The application of “precision medicine” to save and improve lives relies on good-quality, easily-accessible data on everything from our DNA to lifestyle and environmental factors. The opposite to a one-size-fits-all healthcare system, it has vast, untapped potential to transform the treatment and prediction of rare diseases—and disease in general.

But there is no global governance framework for such data and no common data portal. This is a problem that contributes to the premature deaths of hundreds of millions of rare-disease patients worldwide.

The World Economic Forum’s Breaking Barriers to Health Data Governance initiative is focused on creating, testing and growing a framework to support effective and responsible access – across borders – to sensitive health data for the treatment and diagnosis of rare diseases.

The data will be shared via a “federated data system”: a decentralized approach that allows different institutions to access each other’s data without that data ever leaving the organization it originated from. This is done via an application programming interface and strikes a balance between simply pooling data (posing security concerns) and limiting access completely.

The project is a collaboration between entities in the UK (Genomics England), Australia (Australian Genomics Health Alliance), Canada (Genomics4RD), and the US (Intermountain Healthcare).

CAR-T-cell therapy

A treatment that makes immune cells hunt down and kill cancer cells was recently declared a success for leukaemia patients. The treatment, called CAR-T-cell therapy, involves removing and genetically altering immune cells, called T cells, from cancer patients. The altered cells then produce proteins called chimeric antigen receptors (CARs). These recognize and can destroy cancer cells. In the journal Nature, scientists at the University of Pennsylvania announced that two of the first people treated with CAR-T-cell therapy were still in remission 12 years on.

Fighting pancreatic cancer

Pancreatic cancer is one of the deadliest cancers. It is rarely diagnosed before it starts to spread and has a survival rate of less than 5% over five years. At the University of California San Diego School of Medicine, scientists developed a test that identified 95% of early pancreatic cancers in a study. The research, published in Nature Communications Medicine , explains how biomarkers in extracellular vesicles – particles that regulate communication between cells – were used to detect pancreatic, ovarian and bladder cancer at stages I and II.

Have you read?

Cancer: how to stop the next global health crisis, how to improve access to cancer medicines in low and middle-income countries, why is cancer becoming more common among millennials, a tablet to cut breast cancer risk.

A drug that could halve the chance of women developing breast cancer is being tested out by England's National Health Service (NHS). It will be made available to almost 300,000 women seen as being at most risk of developing breast cancer, which is the most common type of cancer in the UK . The drug, named anastrozole, cuts the level of oestrogen women produce by blocking the enzyme aromatase . It has already been used for many years as a breast cancer treatment but has now been repurposed as a preventive medicine. “This is the first drug to be repurposed through a world-leading new programme to help us realize the full potential of existing medicines in new uses to save and improve more lives on the NHS," says NHS Chief Executive Amanda Pritchard.

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A new RNA editing tool could enhance cancer treatment

The new study found that an RNA-targeting CRISPR platform could tune immune cell metabolism without permanent genetic changes, potentially unveiling a relatively low-risk way to upgrade existing cell therapies for cancer.

An artistic rendering representing how the MEGA platform developed by these researchers controls the CAR T cell, like a puppet. Pulling on multiple strings at the same time – that is, targeting multiple genes and pathways simultaneously – rewires cellular programs to enhance CAR T cell fitness and function.

An artistic rendering representing how the MEGA platform developed by these researchers controls the CAR T cell, like a puppet. Pulling on multiple strings at the same time – that is, targeting multiple genes and pathways simultaneously – rewires cellular programs to enhance CAR T cell fitness and function. (Image credit: Gerardo Sotillo)

Cell therapies for cancer can be potentially enhanced using a CRISPR RNA-editing platform, according to a new study published Feb. 21 in Cell . The new platform, Multiplexed Effector Guide Arrays, or MEGA, can modify the RNA of cells, which allowed Stanford University researchers to regulate immune cell metabolism in a way that boosted the cells’ ability to target tumors.

Lead author and Stanford graduate student Victor Tieu was interested in improving chimeric antigen receptor (CAR) T cell therapy. In this cancer treatment, T cells – a type of white blood cell – are engineered with the CAR protein, a receptor that allows the cells to better track down cancer cells. While CAR T therapy has successfully treated blood cancers, including lymphomas and multiple myeloma, the engineered immune cells haven’t stacked up well against solid cancers such as pancreatic and lung cancers.

That’s because solid tumors have a bulkier structure for the immune cells to penetrate – the cells grow exhausted before they can make headway in destroying tumors. T cells evolved to fire up quickly and attack viruses, which means they often burn through their energy stores too soon when fighting cancer. “We were really interested in how we can make those cells better to improve clinical outcomes,” said Tieu. “A lot of the tools that we have right now just aren’t that good.”

The researchers tested their tool on CAR T cells in lab cultures with tumor cells and in mice with cancer. “Our finding is that it performs 10 times better, in terms of reducing the tumor growth and in terms of sustaining long term T cell proliferation,” said senior author Stanley Qi , Stanford associate professor of bioengineering and institute scholar at Sarafan ChEM-H .

Stopping cell exhaustion

Previous research efforts to improve CAR T cell therapy have used CRISPR-Cas9 to edit the cells’ DNA. However, this gene-editing platform comes with risks because it permanently deletes bits of DNA, which can have unintended consequences and even cause the T cells themselves to turn cancerous.

So the Stanford team pursued a different route, exploring whether CRISPR-Cas13d – which uses a molecular scissor that cuts RNA, not DNA – could enable reversible changes to gene expression in T cells. Unlike Cas9, Cas13d can easily target multiple genes at the same time – in the paper, the researchers demonstrated they could make 10 edits at once to human T cells. “RNA is the next layer up from DNA, so we’re not actually touching any of the genetic code,” said Tieu. “But we’re still able to get big changes in gene expression that are able to change the behavior of the cell.”

To see whether this tool could successfully improve CAR T cell function, they identified 24 genes that could be involved in the T cell exhaustion. They then tested 6,400 paired gene combinations in culture, with different genes turned down using the MEGA tool, and identified new gene pairings that worked especially well together to boost anti-tumor function.

Turning T cells into marathon runners

In another experiment, the team tuned a set of metabolic genes in the T cells to tilt the cells from sprinters to marathon runners, giving them the endurance to chip away at tumors. They compared these MEGA CAR T cells to non-engineered T cells and CAR T cells, both in lab cultures with tumor cells and in mice with cancer. After three weeks, they tested the extent of the tumors as well as how the T cells were surviving.

At first, the MEGA cells lagged in their anti-cancer activity. “Initially, I was like, ‘Oh, these cells are worse,’” said Tieu. But, after some time, these cells persevered against the tumor cells while the CAR T and regular T cells wore themselves out, leading to the 10-fold improvement in tumor growth reduction and T cell proliferation.

The secret was shifting how the cells spent their sugar, away from a fast-burning glycolysis process toward favoring oxidative phosphorylation. “We were able to use this technology to engineer the mRNAs in this sugar-usage pathway inside the T cells that regulate their choice of which sugar molecule to use,” said Qi. As a result, “We were able to really sustain the persistence of these T cells, so the T cell could live longer in the tumor site, and also exert much better performance.”

Not only did the MEGA platform allow for fine-tuning genes regulating T cell metabolism, the tune-up could also be regulated with a drug. When an antibiotic called trimethoprim was present, it turned on the RNA changes, tamping down on the cells’ glycolysis metabolism and turning them into endurance athletes in their attack on the tumor cells. When the drug was gone, the cells reverted to their original gene expression. This drug-based control mechanism “allows you to create a safety switch” for immunotherapy treatments, said co-author Crystal Mackall , the Ernest and Amelia Gallo Family Professor and a professor of pediatrics and of medicine at Stanford.

While the platform is still in its early stages, the researchers hope that it can eventually prove useful in clinical settings. Tieu plans to continue development of the platform toward this goal. “It would be really cool to try to push this to an actual clinical product,” he said. “I think there’s a lot of potential to really improve CAR T cell therapy in ways that people couldn’t have done before.”

Additional Stanford co-authors include senior research scientist Elena Sotillo; graduate students Jeremy Bjelajac, Crystal Chen, and Justin Guerrero; life sciences researchers Meena Malipatlolla, Peng Xu, and Patrick Quinn; MD candidate Chris Fisher; and postdoctoral scholar Dorota Klysz. Mackall is also a member of Stanford Bio-X , the Maternal & Child Health Research Institute (MCHRI) , and the Stanford Cancer Institute . Qi is also a member of Stanford Bio-X, the Cardiovascular Institute , MCHRI, the Stanford Cancer Institute, and the Wu Tsai Neurosciences Institute and a Chan Zuckerberg Biohub–San Francisco Investigator.

This research was funded by the National Science Foundation; the National Cancer Institute; Stanford Bio-X; the Virginia and D.K. Ludwig Fund for Cancer Research; St. Baldrick’s Empowering Pediatric Immunotherapies for Childhood Cancer; the Parker Institute for Cancer Immunotherapy, which supports the Stanford University Cancer Immunotherapy Program; the Li Ka Shing Foundation; and the California Institute for Regenerative Medicine.

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Cancer research highlights from 2023

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By Mayo Clinic staff

Researchers at Mayo Clinic Comprehensive Cancer Center spent 2023 studying the biology of cancer and new ways to predict, prevent, diagnose and treat the disease. Their discoveries are creating hope and transforming the quality of life for people with cancer today and in the future. Here are some highlights from their research over the past year:

Mayo Clinic researchers link ovarian cancer to bacteria colonization in the microbiome.

A specific colonization of microbes in the reproductive tract is commonly found in people with ovarian cancer, according to a study from the Mayo Clinic  Center for Individualized Medicine . Published in  Scientific Reports  and led by  Marina Walther-Antonio, Ph.D. , a Mayo Clinic researcher, and Abigail Asangba, Ph.D., the discovery strengthens the evidence that the bacterial component of the microbiome — a community of microorganisms that also consists of viruses, yeasts and fungi — is an important indicator for early detection, diagnosis and prognosis of ovarian cancer . The study also suggests that a higher accumulation of pathogenic microbes plays a role in treatment outcomes and could be a potential indicator for predicting a patient's prognosis and response to therapy.  Read more .

Artificial intelligence is forging a new future for colorectal cancer and other digestive system diseases.

Colonoscopy remains the gold standard in detecting and preventing colorectal cancer , but the procedure has limitations. Some studies suggest that more than half of post-colonoscopy colon cancer cases arise from lesions missed at patients' previous colonoscopies. In 2022, Michael Wallace, M.D. , a Mayo Clinic gastroenterologist, published the results  of an international, multicenter study testing the impact of adding artificial intelligence (AI) to routine colonoscopies. His team, including James East, M.D. , a Mayo Clinic gastroenterologist, and other researchers from the U.S., the U.K., Italy, Germany and Ireland, found that incorporating AI into colonoscopies reduced the risk of missing polyps by 50%.  Read more .

A big step forward: Bringing DNA sequencing data to routine patient care.

The Tapestry study , an extensive genomic sequencing clinical research study, aims to complete exome sequencing (sequencing the protein-coding regions of a genome) for 100,000 Mayo Clinic patients. The results will be integrated into patients’ electronic health records for three hereditary conditions, and the amassed data will contribute to a research dataset stored within the Mayo Clinic Cloud on the Omics Data Platform. The overall hope of Tapestry is to accelerate discoveries in individualized medicine to tailor prevention, diagnosis and treatment to a patient's unique genetic makeup. It is poised to advance evidence that exome sequencing, when applied to a diverse and comprehensive general population, can proficiently identify carriers of genetic variants that put them at higher risk for a disease, allowing them to take preventive measures.  Read more .

Patients with multiple tumors in one breast may not need a mastectomy.

Patients who have multiple tumors in one breast may be able to avoid a mastectomy if surgeons can remove the tumors while leaving enough breast tissue, according to research led by the  Alliance in Clinical Trials in Oncology  and  Mayo Clinic Comprehensive Cancer Center . Patients would receive breast-conserving therapy — a  lumpectomy  followed by whole-breast  radiation therapy — rather than mastectomy . The study is published in the  Journal of Clinical Oncology . Historically, women with multiple tumors in one breast have been advised to have a mastectomy. Now, patients can be offered a less invasive option with faster recovery, resulting in better patient satisfaction and cosmetic outcomes, says  Judy Boughey, M.D. , lead author, Mayo Clinic breast surgical oncologist and the W.H. Odell Professor of Individualized Medicine. Read more .

Staging pancreatic cancer early with minimally invasive surgery shows positive results in patient prognosis.

A study published in the  Journal of the American College of Surgeons  reveals that performing a minor surgical procedure on patients newly diagnosed with  pancreatic cancer  helps to identify cancer spread early and determine the stage of cancer. The researchers add that the surgery ideally should be performed before the patient begins chemotherapy. "This is an important study because it supports that staging laparoscopy may help determine a patient's prognosis and better inform treatment so that patients avoid unhelpful or potentially harmful surgical therapy," says  Mark Truty, M.D. , a Mayo Clinic surgical oncologist who led the research.  Read more .

Mayo Clinic study reveals proton beam therapy may shorten breast cancer treatment.

In a trial published in  The Lancet Oncology , Mayo Clinic Comprehensive Cancer Center researchers uncovered evidence supporting a shorter treatment time for people with breast cancer . The study compared two separate dosing schedules of pencil-beam scanning proton therapy , known for its precision in targeting cancer cells while preserving healthy tissue to reduce the risk of side effects. The investigators found that both 25-day and 15-day proton therapy schedules resulted in excellent cancer control while sparing surrounding non-cancerous tissue. Further, complication rates were comparable between the two study groups. "We can now consider the option of 15 days of therapy for patients based on the similar treatment outcomes observed," says  Robert Mutter, M.D. , a Mayo Clinic radiation oncologist and physician-scientist. Read more .

Harnessing the immune system to fight ovarian cancer.

Mayo Clinic research is biomanufacturing an experimental, cell-based ovarian cancer vaccine and combining it with immunotherapy to study a "one-two punch" approach to halting ovarian cancer progression. This research begins with a blood draw from people with advanced  ovarian cancer  whose tumors have returned after standard surgery and chemotherapy. White blood cells are extracted from the blood, biomanufactured to become dendritic cells and returned to the patient. Dendritic cells act as crusaders that march through the body, triggering the immune system to recognize and fight cancer. "We're building on an earlier phase 1 clinical trial  that showed promising results  in terms of survival after the dendritic cell-based vaccine," says  Matthew Block, M.D., Ph.D. , co-principal investigator and Mayo Clinic medical oncologist. "Of the 18 evaluable patients in the phase 1 study, 11 had cancer return, but seven of them — 40% — have been cancer-free for almost 10 years. We typically expect 90% of patients in this condition to have the cancer return."  Read more .

New gene markers detect Lynch syndrome-associated colorectal cancer.

Researchers from Mayo Clinic Comprehensive Cancer Center and Mayo Clinic Center for Individualized Medicine have discovered new genetic markers to identify Lynch syndrome-associated colorectal cancer with high accuracy. Studies are underway to determine if these genetic markers are in stool samples and, if so, how this could lead to a non-invasive screening option for people with  Lynch syndrome . The research was published in Cancer Prevention Research , a journal of the American Association for Cancer Research. "This is an exciting finding that brings us closer to the reality that clinicians may soon be able to offer a non-invasive cancer screening option to patients with the highest risk of getting cancer," says  Jewel Samadder, M.D. , co-lead author of the paper and a Mayo Clinic gastroenterologist. Read more .

Mayo Clinic prepares to biomanufacture a new CAR-T cell therapy for B-cell blood cancers.

Mayo Clinic research has developed a new type of  chimeric antigen receptor-T cell therapy (CAR-T cell therapy)  aimed at killing B-cell blood cancers that have returned and are no longer responding to treatment. This pioneering technology, designed and developed in the lab of  Hong Qin, M.D., Ph.D. , a Mayo Clinic cancer researcher, killed B-cell tumors grown in the laboratory and tumors implanted in mouse models. The preclinical findings are published in  Cancer Immunology, Immunotherapy . "This study shows our experimental CAR-T cell therapy targets several blood cancers, specifically chronic lymphocytic leukemia," says Dr. Qin. "Currently, there are six different CAR-T cell therapies approved for treatment of relapsed blood cancers. While the results are impressive, not everyone responds to this treatment. Our goal is to provide novel cell therapies shaped to each patient's individual need."  Read more .

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This experimental drug could change the field of cancer research

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Sacha Pfeiffer

Jonaki Mehta

Jonaki Mehta

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The new treatment is categorized as immunotherapy. skaman306/Getty Images hide caption

The new treatment is categorized as immunotherapy.

A tiny group of people with rectal cancer just experienced something of a scientific miracle: their cancer simply vanished after an experimental treatment.

In a very small trial done by doctors at New York's Memorial Sloan Kettering Cancer Center, patients took a drug called dostarlimab for six months. The trial resulted in every single one of their tumors disappearing. The trial group included just 18 people, and there's still more to be learned about how the treatment worked. But some scientists say these kinds of results have never been seen in the history of cancer research.

Dr. Hanna Sanoff of the University of North Carolina's Lineberger Comprehensive Cancer Center joined NPR's All Things Considered to outline how this drug works and what it could mean for the future of cancer research. Although she was not involved with the study, Dr. Sanoff has written about the results.

This interview has been lightly edited

On her first reaction to the results: I mean, I am incredibly optimistic. Like you said in the introduction, we have never seen anything work in 100 percent of people in cancer medicine.

On how the drug works to treat cancer: This drug is one of a class of drugs called immune checkpoint inhibitors. These are immunotherapy medicines that work not by directly attacking the cancer itself, but actually getting a person's immune system to essentially do the work. These are drugs that have been around in melanoma and other cancers for quite a while, but really have not been part of the routine care of colorectal cancers until fairly recently.

On the kinds of side effects patients experienced: Very, very few in this study - in fact, surprisingly few. Most people had no severe adverse effects at all.

On how this study could be seen as 'practice-changing': Our hope would be that for this subgroup of people - which is only about five percent to 10 percent of people who have rectal cancer - if they can go on and just get six months of immunotherapy and not have any of the rest of this - I don't even know the word to use. Paradigm shift is often used, but this really absolutely is paradigm-shifting.

On why the idea of being able to skip surgery for cancer treatment is so revolutionary: In rectal cancer, this is part of the conversation we have with someone when they're diagnosed. We are very hopeful for being able to cure you, but unfortunately, we know our treatments are going to leave you with consequences that may, in fact, be life-changing. I have had patients who, after their rectal cancer, have barely left the house for years - and in a couple of cases, even decades - because of the consequences of incontinence and the shame that's associated with this.

On next steps for the drug: What I'd really like us to do is get a bigger trial where this drug is used in a much more diverse setting to understand what the real, true response rate is going to be. It's not going to end up being 100 percent. I hope I bite my tongue on that in the future, but I can't imagine it will be 100 percent. And so when we see what the true response rate is, that's when I think we can really do this all the time.

This piece was reported by Sacha Pfeiffer, produced by Jonaki Mehta and edited by Kathryn Fox. It was adapted for the web by Manuela Lopez Restrepo.

  • cancer treatment

FDA approves groundbreaking treatment for advanced melanoma

The Food and Drug Administration on Friday approved a new cancer therapy that could one day transform the way a majority of aggressive and advanced tumors are treated.

The treatment, called Amtagvi, from Iovance Biotherapeutics , is for metastatic melanoma patients who have already tried and failed other drugs. It’s known as TIL therapy and involves boosting the number of immune cells inside tumors, harnessing their power to fight the cancer.

It’s the first time a cellular therapy has been approved to treat solid tumors. The drug was given a fast-track approval based on the results of a phase 2 clinical trial. The company is conducting a larger phase 3 trial to confirm the treatment’s benefits. The therapy’s list price — the price before insurance and other potential discounts — is $515,000 per patient. 

“This is going to be huge,” said Dr. Elizabeth Buchbinder, a senior physician at Dana-Farber Cancer Institute in Boston. Melanoma is “not one of those cancers where there’s like 20 different” possible treatments, she said. “You start running out of options fast.” 

Dan Bennett, 59, credits TIL therapy with allowing him to beat the slim odds of long-term survival of stage 4 melanoma. His daughter, Faith Bennett, 29, first noticed a suspicious mole on Bennett's neck in 2011.

Friday’s approval is only for melanoma, the deadliest form of skin cancer , but experts say it holds promise for treating other solid tumors, which account for 90% of all cancers. 

“It is our hope that future iterations of TIL therapy will be important for lung cancer, colon cancer , head and neck cancer, bladder cancer and many other cancer types,” said Dr. Patrick Hwu, chief executive of the Moffitt Cancer Center in Tampa, Florida. Moffitt has been involved with Iovance’s clinical trials of TIL therapy.

TIL stands for tumor-infiltrating lymphocytes, which are immune cells that exist within tumors . But there are nowhere nearly enough of those cells to effectively fight off cancer cells. TIL therapy involves, in part, extracting some of those immune cells from the patient’s tumor and replicating them billions of times in a lab, then reinfusing them back into the patient. 

It’s similar to CAR-T cell therapy, where healthy cells are taken out of a person’s body and then modified in a lab to fight cancers. That’s usually used for hard-to-treat blood cancers such as leukemia and lymphoma. With TIL therapy, the cells used are already programmed to recognize cancer — no lab modifications needed — they just need a boost in numbers to fight it. 

Like CAR-T, TIL therapy is a one-time treatment, though the entire process can take up to eight weeks. The TIL cells are first harvested from the tumor through a minimally invasive procedure and then grown and multiplied in the lab, a process that takes 22 days, according to Iovance. 

While that’s happening, patients are given chemotherapy to clear out their immune cells to make room for the billions of new melanoma-fighting TIL cells. Once the TIL cells are reinfused back into the body, patients get a drug called interleukin-2 to further stimulate those cells. 

Hwu said that most side effects in patients undergoing TIL therapy are not from the reinfusion of cells, but from the chemotherapy and the interleukin-2. These can include nausea and extreme fatigue, and patients are also vulnerable to other illnesses because the body is depleted of disease-fighting white blood cells. 

Putting billions of cells back into the body is not entirely risk-free, however, said Dr. William Dahut, chief scientific officer of the American Cancer Society. It’s possible that the body’s immune system could overreact in what’s known as a cytokine storm, which can cause flu-like symptoms, low blood pressure and organ damage.   “There are risks for immune-related side effects, which could be serious,” he said.

Common side effects associated with Amtagvi can include abnormally fast heart rate, fluid buildup, rash, hair loss and feeling short of breath, the FDA said.

Those side effects can be managed, said Dr. Steven Rosenberg, chief of the surgery branch at the National Cancer Institute. “They’re a small price to pay for a growing cancer that would otherwise be lethal.”

Overall, Dahut said the approval of TIL therapy is “meaningful.”

“What’s nice about this is that patients will receive a wide variety of tumor fighting lymphocytes that will be able to have the capacity to overcome resistance and actually be a living therapy over time, too, to target additional cancer cells should they develop,” Dahut said.

In addition to melanoma, Dahut said that TIL therapy is most likely to be useful in cancers that respond to drugs that “take the brakes off the immune system,” called checkpoint inhibitors .

“Those would be things like non-small cell lung cancer, kidney cancer, maybe bladder cancer, that we know are responsive to immune-based therapies to begin with,” he said. “Many of those patients relapse, so another immune-based therapy that works in a different way, seems to me, the most likely way for this to be effective.”

Much more research is needed, and it may be years before TIL therapy is approved for other types of cancer.

One of Iovance’s clinical trials investigating TIL therapy for non-small cell lung cancer was forced to pause when a participant died. While the death is under investigation, the company said it may have been the result of either chemotherapy or interleukin 2 — therapies meant to knock down each patients’ immune system before they can get the reinfusion of their TIL cells. 

The therapy is not expected to work for every metastatic melanoma patient. Clinical trial data that Iovance submitted to the FDA showed that tumors shrank in about a third of patients who received TIL therapy. 

Of those patients, about half saw their tumors shrink for at least one year, Dr. Friedrich Graf Finckenstein, chief medical officer of Iovance Biotherapeutics. “Some of these patients even had their tumor completely disappear,” he said. 

Another study, conducted in the Netherlands , did a head-to-head analysis of TIL therapy and another form of immunotherapy, called ipilimumab. Twenty percent of the patients who received TIL had complete remissions, compared with 7% of patients who got ipilimumab. Iovance was not involved with the Dutch trial.

The goal of the therapy, Hwu said, “is to get rid of the cancer and have it stay away. These immune cells stay in the body and live in the body for decades.”

The technology has been in development and studied for nearly 40 years. It was Rosenberg who pioneered TIL therapy — first describing how it could shrink melanoma tumors in the New England Journal of Medicine in 1988 .

“I’ve been waiting for a very long time to see this given to patients, because I know that it can cure some patients that have metastatic melanoma that cannot be affected by any other treatment,” Rosenberg said.

It’s worked so far for Dan Bennett, 59, of Clermont, Florida. Bennett was diagnosed with melanoma in 2011 after his daughter noticed a suspicious mole on his neck that had changed color. 

Despite surgery, chemotherapy and radiation, his cancer kept returning. In 2014, his doctors at Moffitt recommended he try TIL therapy.

“At first, we were pretty leery about it because it was unproven,” Bennett said. Ten years later, Bennett is convinced the TIL therapy is the reason he has survived so long with stage 4 melanoma, which usually has a five-year survival rate of 22.5% . 

“I would recommend any experimental drug if it’s your last opportunity,” he said. “You owe it to yourself and your family to do whatever you can to stay alive and to be a productive member of society.”

Buchbinder, the Dana-Faber doctor, was not involved with Iovance’s TIL therapy trial for melanoma, but she is scheduled to begin similar trials with other drugmakers. 

“We literally have patients right now waiting for approval because they are hoping they’ll be able to go on it,” Buchbinder said. “It is definitely a practice-changing therapy.”

research about cure for cancer

Erika Edwards is a health and medical news writer and reporter for NBC News and "TODAY."

research about cure for cancer

Anne Thompson is NBC News’ chief environmental affairs correspondent. 

Marina Kopf is an associate producer with the NBC News Health and Medical Unit.

ScienceDaily

New treatment for a rare and aggressive cancer improves survival rates in breakthrough clinical trial

An innovative treatment significantly increases the survival of people with malignant mesothelioma, a rare but rapidly fatal type of cancer with few effective treatment options, according to results from a clinical trial led by Queen Mary University of London.

The phase 3 clinical trial, led by Professor Peter Szlosarek at Queen Mary and sponsored by Polaris Pharmaceuticals, has unveiled a breakthrough in the treatment of malignant pleural mesothelioma (MPM), a rare and often rapidly fatal form of cancer with limited therapeutic options.

Mick's journey with mesothelioma: "I have five grandchildren and two great-grandchildren now -- I wouldn't want to miss all that."

The ATOMIC-meso trial, a randomised placebo-controlled study of 249 patients with MPM, found that a treatment -- which combines a new drug, ADI-PEG20, with traditional chemotherapy -- increased the median survival of participants by 1.6 months, and quadrupled the survival at 36 months, compared to placebo-chemotherapy.

The findings are significant, as MPM has one of the lowest 5-year survival rates of any solid cancer of around 5-10%. This innovative approach marks the first successful combination of chemotherapy with a drug that targets cancer's metabolism developed for this disease in 20 years.

MPM is a rare, aggressive cancer that affects the lining of the lungs and is associated with exposure to asbestos. It's usually treated with potent chemotherapy drugs, but these are seldom able to halt the progression of the disease.

The premise behind this new drug treatment is elegant in its simplicity -- starving the tumour by cutting off its food supply. All cells need nutrients to grow and multiply, including amino acids like arginine. ADI-PEG20 works by depleting arginine levels in the bloodstream. For tumour cells that can't manufacture their arginine due to a missing enzyme, this means their growth is thwarted.

The ATOMIC-meso trial is the culmination of 20 years of research at Queen Mary's Barts Cancer Institute that began with Professor Szlosarek's discovery that malignant mesothelioma cells lack a protein called ASS1, which enables cells to manufacture their own arginine. He and his team have since dedicated their efforts to using this knowledge to create an effective treatment for patients with MPM.

Professor Szlosarek said: "It's truly wonderful to see the research into the arginine starvation of cancer cells come to fruition. This discovery is something I have been driving from its earliest stages in the lab, with a new treatment, ADI-PEG20, now improving patient lives affected by mesothelioma. I thank all the patients and families, investigators and their teams, and Polaris Pharmaceuticals for their commitment to defining a new cancer therapy."

Dr Tayyaba Jiwani, Science Engagement Manager at Cancer Research UK, said: "This study shows the power of discovery research which allows us to dig deep into the biology of mesothelioma to uncover vulnerabilities that we can now target with ADI-PEG20.

"Cancer Research UK is delighted to have funded the early stages of this research, including a preliminary clinical trial which established the safety and effectiveness of this drug."

There are ongoing studies assessing ADI-PEG20 in patients who have sarcoma or glioblastoma multiforme (a type of brain tumour) and other cancers dependent on arginine. The success of this novel chemotherapy in MPM also suggests that the drug may be of benefit in the treatment of multiple other types of cancer. 

Mick's journey with mesothelioma

Mick worked in a factory boiler room in the 1970s, where he was exposed to asbestos. In 2018, he visited his doctor after he began to feel unwell and had lost three stone in weight. He became anaemic and was eventually diagnosed with mesothelioma.

"It was a bit of a shock: I was given four months to live," Mick explains. His doctor referred him to Professor Szlosarek, who enrolled him in the ATOMIC-meso trial. "I always believed in Peter. I said: 'I'm in it to win it -- you're not getting rid of me.' And here I am five years later."

For two years, Mick visited St Bartholomew's Hospital every week, accompanied by his wife, Jackie, or one of his children or grandchildren. "I'd have two injections of the new treatment -- one in each arm. I didn't have any serious side effects," Mick explains. "I met many of the other people on the trial. Over time, some of them disappeared. But I kept going."

Mick was awarded compensation from his former employer responsible for the asbestos exposure that ultimately led to his mesothelioma. Around 80% of mesothelioma cases are caused by workplace exposure.

Two and a half years after Mick enrolled on the ATOMIC-meso trial, his mesothelioma returned and he received a second course of treatment, this time immunotherapy. He experienced more side effects with this therapy, including encephalitis. But his cancer remains under control, and recently he was able to celebrate his 80th birthday. Professor Szlosarek and his team plan to study why certain patients, such as Mick, benefit so greatly from ADI-PEG20, in the hope of discovering how to extend this benefit to more people.

Mick says: "This trial has changed the lives of people with mesothelioma, allowing us to live longer. I have five grandchildren and two great-grandchildren now -- I wouldn't want to miss all that."

  • Mesothelioma
  • Lung Cancer
  • Breast Cancer
  • Colon Cancer
  • Diseases and Conditions
  • Skin Cancer
  • Brain Tumor
  • Breast cancer
  • Malignant melanoma
  • Lung cancer
  • Ovarian cancer
  • Rocky Mountain spotted fever
  • Monoclonal antibody therapy

Story Source:

Materials provided by Queen Mary University of London . Note: Content may be edited for style and length.

Journal Reference :

  • Peter W. Szlosarek, Benjamin C. Creelan, Thomas Sarkodie, Luke Nolan, Paul Taylor, Olga Olevsky, Federica Grosso, Diego Cortinovis, Meenali Chitnis, Amy Roy, David Gilligan, Hedy Kindler, Dionysis Papadatos-Pastos, Giovanni L. Ceresoli, Aaron S. Mansfield, Anne Tsao, Kenneth J. O’Byrne, Anna K. Nowak, Jeremy Steele, Michael Sheaff, Chiung-Fang Shiu, Chih-Ling Kuo, Amanda Johnston, John Bomalaski, Marjorie G. Zauderer, Dean A. Fennell, Igor I. Rybkin, Christian D. Rolfo, Melanie MacKean, Nicola Steele, Kevin Franks, Paul Shaw, Michael J. Lind, Sunil Upadhyay, Thomas John, Christos Karapetis, Ratnesh Srivastav, Manlio Mencoboni, Antonio Chella, Nicoletta Zilembo, Filippo de Marinis, Maria Giulia Stella, Inn-Wen Chong, Chin-Chou Wang. Pegargiminase Plus First-Line Chemotherapy in Patients With Nonepithelioid Pleural Mesothelioma . JAMA Oncology , 2024; DOI: 10.1001/jamaoncol.2023.6789

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Ivermectin, a potential anticancer drug derived from an antiparasitic drug

Mingyang tang.

a Anhui Key Laboratory of Infection and Immunity, Bengbu Medical College, Bengbu, Anhui Province 233030, China

b Clinical Medical Department, Bengbu Medical College, Bengbu, Anhui Province 233030, China

Xiaodong Hu

c Department of Histology and Embryology, Bengbu Medical College, Bengbu, Anhui Province 233030, China

d Department of Microbiology and Parasitology, Bengbu Medical College, Bengbu, Anhui Province 233030, China

Chenying Yu

Fuying cheng, jiangyan li.

e School of Fundamental Sciences, Bengbu Medical College, Bengbu, Anhui Province 233030, China

Graphical abstract

Ivermectin has powerful antitumor effects, including the inhibition of proliferation, metastasis, and angiogenic activity, in a variety of cancer cells. This may be related to the regulation of multiple signaling pathways by ivermectin through PAK1 kinase. On the other hand, ivermectin promotes programmed cancer cell death, including apoptosis, autophagy and pyroptosis. Ivermectin induces apoptosis and autophagy is mutually regulated. Interestingly, ivermectin can also inhibit tumor stem cells and reverse multidrug resistance and exerts the optimal effect when used in combination with other chemotherapy drugs.

An external file that holds a picture, illustration, etc.
Object name is ga1_lrg.jpg

Ivermectin is a macrolide antiparasitic drug with a 16-membered ring that is widely used for the treatment of many parasitic diseases such as river blindness, elephantiasis and scabies. Satoshi ōmura and William C. Campbell won the 2015 Nobel Prize in Physiology or Medicine for the discovery of the excellent efficacy of ivermectin against parasitic diseases. Recently, ivermectin has been reported to inhibit the proliferation of several tumor cells by regulating multiple signaling pathways. This suggests that ivermectin may be an anticancer drug with great potential. Here, we reviewed the related mechanisms by which ivermectin inhibited the development of different cancers and promoted programmed cell death and discussed the prospects for the clinical application of ivermectin as an anticancer drug for neoplasm therapy.

1. Introduction

Ivermectin(IVM) is a macrolide antiparasitic drug with a 16-membered ring derived from avermectin that is composed of 80% 22,23-dihydroavermectin-B1a and 20% 22,23-dihydroavermectin-B1b [ 1 ]. In addition to IVM, the current avermectin family members include selamectin, doramectin and moxidectin [ [2] , [3] , [4] , [5] ] ( Fig. 1 ). IVM is currently the most successful avermectin family drug and was approved by the FDA for use in humans in 1978 [ 6 ]. It has a good effect on the treatment of parasitic diseases such as river blindness, elephantiasis, and scabies. The discoverers of IVM, Japanese scientist Satoshi ōmura and Irish scientist William C. Campbell, won the Nobel Prize in Physiology or Medicine in 2015 [ 7 , 8 ]. IVM activates glutamate-gated chloride channels in the parasite, causing a large amount of chloride ion influx and neuronal hyperpolarization, thereby leading to the release of gamma-aminobutyric acid (GABA) to destroy nerves, and the nerve transmission of muscle cells induces the paralysis of somatic muscles to kill parasites [ 9 , 10 ]. IVM has also shown beneficial effects against other parasitic diseases, such as malaria [ 11 , 12 ], trypanosomiasis [ 13 ], schistosomiasis [ 14 ], trichinosis [ 15 ] and leishmaniasis [ 16 ].

Fig. 1

The chemical structures of ivermectin and other avermectin family compounds in this review.

IVM not only has strong effects on parasites but also has potential antiviral effects. IVM can inhibit the replication of flavivirus by targeting the NS3 helicase [ 17 ]; it also blocks the nuclear transport of viral proteins by acting on α/β-mediated nuclear transport and exerts antiviral activity against the HIV-1 and dengue viruses [ 18 ]. Recent studies have also pointed out that it has a promising inhibitory effect on the SARS-CoV-2 virus, which has caused a global outbreak in 2020 [ 19 ]. In addition, IVM shows potential for clinical application in asthma [ 20 ] and neurological diseases [ 21 ]. Recently scientists have discovered that IVM has a strong anticancer effect.

Since the first report that IVM could reverse tumor multidrug resistance (MDR) in 1996 [ 22 ], a few relevant studies have emphasized the potential use of IVM as a new cancer

treatment [ [23] , [24] , [25] , [26] , [27] ]. Despite the large number of related studies, there are still some key issues that have not been resolved. First of all, the specific mechanism of IVM-mediated cytotoxicity in tumor cells is unclear; it may be related to the effect of IVM on various signaling pathways, but it is not very clear overall. Second, IVM seems to induce mixed cell death in tumor cells, which is also a controversial issue. Therefore, this review summarized the latest findings on the anticancer effect of IVM and discussed the mechanism of the inhibition of tumor proliferation and the way that IVM induces tumor programmed cell death to provide a theoretical basis for the use of IVM as a potential anticancer drug. As the cost of the research and development of new anticancer drugs continues to increase, drug repositioning has become increasingly important. Drug repositioning refers to the development of new drug indications that have been approved for clinical use [ 28 ]. For some older drugs that are widely used for their original indications and have clinical data and safety information, drug repositioning allows them to be developed via a cheaper and faster cycle and to be used more effectively in clinical use clinically [ 29 ]. Here, we systematically summarized the anticancer effect and mechanism of IVM, which is of great significance for the repositioning of IVM for cancer treatment.

2. The role of IVM in different cancers

2.1. breast cancer.

Breast cancer is a malignant tumor produced by gene mutation in breast epithelial cells caused by multiple carcinogens. The incidence of breast cancer has increased each year, and it has become one of the female malignant tumors with the highest incidence in globally. On average, a new case is diagnosed every 18 seconds worldwide [ 30 , 31 ]. After treatment with IVM, the proliferation of multiple breast cancer cell lines including MCF-7, MDA-MB-231 and MCF-10 was significantly reduced. The mechanism involved the inhibition by IVM of the Akt/mTOR pathway to induce autophagy and p-21-activated kinase 1(PAK1)was the target of IVM for breast cancer [ 32 ]. Furthermore, Diao’s study showed that IVM could inhibit the proliferation of the canine breast tumor cell lines CMT7364 and CIPp by blocking the cell cycle without increasing apoptosis, and the mechanism of IVM may be related to the inhibition of the Wnt pathway [ 33 ].

Triple-negative breast cancer (TNBC) refers to cancer that is negative for estrogen receptor, progesterone receptor, and human epidermal growth factor receptor 2(HER2) and is the most aggressive subtype of breast cancer with the worst prognosis. In addition, there is also no clinically applicable therapeutic drug currently [ 34 , 35 ]. A drug screening study of TNBC showed that IVM could be used as a SIN3-interaction domain (SID) mimic to selectively block the interaction between SID and paired a-helix2. In addition, IVM regulated the expression of the epithelial mesenchymal-transition (EMT) related gene E-cadherin to restore the sensitivity of TNBC cells to tamoxifen, which implies the possibility that IVM functions as an epigenetic regulator in the treatment of cancer[ 36 ].

Recent studies have also found that IVM could promote the death of tumor cells by regulating the tumor microenvironment in breast cancer. Under the stimulation of a tumor microenvironment with a high level of adenosine triphosphate (ATP) outside tumor cells, IVM could enhance the P2 × 4/ P2 × 7/Pannexin-1 mediated release of high mobility group box-1 protein (HMGB1) [ 37 ]. However, the release of a large amount of HMGB1 into the extracellular environment will promote immune cell-mediated immunogenic death and inflammatory reactions, which will have an inhibitory effect on the growth of tumor cells. Therefore, we believe that the anticancer effect of IVM is not limited to cytotoxicity, but also involves the regulation of the tumor microenvironment. IVM regulates the tumor microenvironment and mediates immunogenic cell death, which may be a new direction for research exploring anticancer mechanisms in the future.

2.2. Digestive system cancer

Gastric cancer is one of the most common malignant tumors worldwide. In the past year, more than one million patients with gastric cancer have been diagnosed worldwide [ 38 ]. Nambara’s study showed that IVM could significantly inhibit the proliferation of gastric cancer cells in vivo and in vitro and that the inhibitory effect of IVM depended on the expression of Yes-associated protein 1(YAP1)[ 39 ]. The gastric cancer cell lines MKN1 and SH-10-TC have higher YAP1 expression than MKN7 and MKN28 cells, so MKN1 and SH-10-TC cells are sensitive to IVM, while MKN7 and MKN28 are not sensitive to IVM.YAP1 plays an oncogenic role in tumorigenesis, indicating the possibility of the use of IVM as a YAP1 inhibitor for cancer treatment [ 40 ].

In a study that screened Wnt pathway inhibitors, IVM inhibited the proliferation of multiple cancers, including the colorectal cancer cell lines CC14, CC36, DLD1, and Ls174 T, and promoted apoptosis by blocking the Wnt pathway [ 41 ]. After intervention with IVM, the expression of caspase-3 in DLD1 and Ls174 T cells increased, indicating that IVM has an apoptosis-inducing effect and inhibits the expression of the downstream genes AXIN2, LGR5, and ASCL2 in the Wnt/β-catenin pathway. However, the exact molecular target of IVM that affects the Wnt/β-catenin pathway remains to be explored.

Hepatocellular carcinoma is the fourth leading cause of cancer death worldwide. Approximately 80% of cases of liver cancer are caused by hepatitis B virus (HBV) and hepatitis C virus (HCV) infection [ 42 ]. IVM could inhibit the development of hepatocellular carcinoma by blocking YAP1 activity in spontaneous liver cancer Mob1b -/- mice [ 43 ].Cholangiocarcinoma is a malignant tumor that originates in the bile duct inside and outside the liver. Intuyod's experiment found that IVM inhibited the proliferation of KKU214 cholangiocarcinoma cells in a dose- and time-dependent manner [ 44 ]. IVM halted the cell cycle in S phase and promoted apoptosis. Surprisingly, gemcitabine-resistant KKU214 cells showed high sensitivity to IVM, which suggested that IVM shows potential for the treatment of tumors that are resistant to conventional chemotherapy drugs.

2.3. Urinary system cancer

Renal cell carcinoma is a fatal malignant tumor of the urinary system derived from renal tubular epithelial cells. Its morbidity has increased by an average of 2% annually worldwide and the clinical treatment effect is not satisfactory [ [45] , [46] , [47] ]. Experiments confirmed that IVM could significantly inhibit the proliferation of five renal cell carcinoma cell lines without affecting the proliferation of normal kidney cells, and its mechanism may be related to the induction of mitochondrial dysfunction [ 48 ]. IVM could significantly reduce the mitochondrial membrane potential and inhibit mitochondrial respiration and ATP production. The presence of the mitochondrial fuel acetyl-L-carnitine (ALCAR), and the antioxidant N-acetyl-L-cysteine (NAC), could reverse IVM-induced inhibition. In animal experiments, the immunohistochemical results for IVM-treated tumor tissues showed that the expression of the mitochondrial stress marker HEL was significantly increased, and the results were consistent with those of the cell experiments.

Prostate cancer is a malignant tumor derived from prostate epithelial cells, and its morbidity is second only to that of lung cancer among men in Western countries [ 49 ]. In Nappi's experiment, it was found that IVM could enhance the drug activity of the anti-androgen drug enzalutamide in the prostate cancer cell line LNCaP and reverse the resistance of the prostate cancer cell line PC3 to docetaxel [ 50 ]. Interestingly, IVM also restored the sensitivity of the triple-negative breast cancer to the anti-estrogen drug tamoxifen [ 36 ], which also implies the potential for IVM to be used in endocrine therapy. Moreover, IVM was also found to have a good inhibitory effect on the prostate cancer cell line DU145 [ 51 ].

2.4. Hematological cancer

Leukemia is a type of malignant clonal disease caused by abnormal hematopoietic stem cells [ 52 ]. In an experiment designed to screen potential drugs for the treatment of leukemia, IVM preferentially killed leukemia cells at low concentrations without affecting normal hematopoietic cells [ 51 ]. The mechanism was related to the increase in the influx of chloride ions into the cell by IVM, resulting in hyperpolarization of the plasma membrane and induction of reactive oxygen species (ROS) production. It was also proven that IVM has a synergistic effect with cytarabine and daunorubicin on the treatment of leukemia. Wang's experiment found that IVM could selectively induce mitochondrial dysfunction and oxidative stress, causing chronic myeloid leukemia K562 cells to undergo increased caspase-dependent apoptosis compared with normal bone marrow cells [ 53 ]. It was also confirmed that IVM inhibited tumor growth in a dose-dependent manner, and dasatinib had improved efficacy.

2.5. Reproductive system cancer

Cervical cancer is one of the most common gynecological malignancies, resulting in approximately 530,000 new cases and 270,000 deaths worldwide each year. The majority of cervical cancers are caused by human papillomavirus (HPV) infection [ 54 , 55 ]. IVM has been proven to significantly inhibit the proliferation and migration of HeLa cells and promote apoptosis [ 56 ]. After intervention with IVM, the cell cycle of HeLa cells was blocked at the G1/S phase, and the cells showed typical morphological changes related to apoptosis.

Ovarian cancer is a malignant cancer that lacks early clinical symptoms and has a poor therapeutic response. The 5-year survival rate after diagnosis is approximately 47% [ 27 , 57 ]. In a study by Hashimoto, it found that IVM inhibited the proliferation of various ovarian cancer cell lines, and the mechanism was related to the inhibition of PAK1 kinase [ 58 ]. In research to screen potential targets for the treatment of ovarian cancer through the use of an shRNA library and a CRISPR/Cas9 library, the oncogene KPNB1 was detected. IVM could block the cell cycle and induce cell apoptosis through a KPNB1-dependent mechanism in ovarian cancer [ 59 ]. Interestingly, IVM and paclitaxel have a synergistic effect on ovarian cancer, and combined treatment in in vivo experiments almost completely inhibited tumor growth. Furthermore, according to a report by Zhang, IVM can enhance the efficacy of cisplatin to improve the treatment of epithelial ovarian cancer, and the mechanism is related to the inhibition of the Akt/mTOR pathway [ 60 ].

2.6. Brain glioma

Glioma is the most common cerebral tumor and approximately 100,000 people worldwide are diagnosed with glioma every year. Glioblastoma is the deadliest glioma, with a median survival time of only 14-17 months [ 61 , 62 ]. Experiments showed that IVM inhibited the proliferation of human glioblastoma U87 and T98 G cells in a dose-dependent manner and induced apoptosis in a caspase-dependent manner [ 63 ]. This was related to the induction of mitochondrial dysfunction and oxidative stress. Moreover, IVM could induce apoptosis of human brain microvascular endothelial cells and significantly inhibit angiogenesis. These results showed that IVM had the potential to resist tumor angiogenesis and tumor metastasis. In another study, IVM inhibited the proliferation of U251 and C6 glioma cells by inhibiting the Akt/mTOR pathway [ 64 ].

In gliomas, miR-21 can regulate the Ras/MAPK signaling pathway and enhance its effects on proliferation and invasion [ 65 ]. The DDX23 helicase activity affects the expression of miR-12 [ 66 ]. IVM could inhibit the DDX23/miR-12 signaling pathway by affecting the activity of DDX23 helicase, thereby inhibiting malignant biological behaviors. This indicated that IVM may be a potential RNA helicase inhibitor and a new agent for of tumor treatment. However, here, we must emphasize that because IVM cannot effectively pass the blood-brain barrier [ 67 ], the prospect of the use of IVM in the treatment of gliomas is not optimistic.

2.7. Respiratory system cancer

Nasopharyngeal carcinoma is a malignant tumor derived from epithelial cells of the nasopharyngeal mucosa. The incidence is obviously regional and familial, and Epstein-Barr virus (EBV) infection is closely related [ 68 ]. In a study that screened drugs for the treatment of nasopharyngeal cancer, IVM significantly inhibited the development of nasopharyngeal carcinoma in nude mice at doses that were not toxic to normal thymocytes [ 69 ]. In addition, IVM also had a cytotoxic effect on a variety of nasopharyngeal cancer cells in vitro, and the mechanism is related to the reduction of PAK1 kinase activity to inhibit the MAPK pathway.

Lung cancer has the highest morbidity and mortality among cancers [ 70 ]. Nishio found that IVM could significantly inhibit the proliferation of H1299 lung cancer cells by inhibiting YAP1 activity [ 43 ]. Nappi's experiment also proved that IVM combined with erlotinib to achieved a synergistic killing effect by regulating EGFR activity and in HCC827 lung cancer cells [ 50 ]. In addition, IVM could reduce the metastasis of lung cancer cells by inhibiting EMT.

2.8. Melanoma

Melanoma is the most common malignant skin tumor with a high mortality rate. Drugs targeting BRAF mutations such as vemurafenib, dabrafenib and PD-1 monoclonal antibodies, including pembrolizumab and nivolumab have greatly improved the prognosis of melanoma [ 71 , 72 ]. Gallardo treated melanoma cells with IVM and found that it could effectively inhibit melanoma activity [ 73 ]. Interestingly, IVM could also show activity against BRAF wild-type melanoma cells, and its combination with dapafinib could significantly increase antitumor activity. Additionally, it has been confirmed that PAK1 is the key target of IVM that mediates its anti-melanoma activity, and IVM can also significantly reduce the lung metastasis of melanoma in animal experiments. Deng found that IVM could activate the nuclear translocation of TFE3 and induce autophagy-dependent cell death by dephosphorylation of TFE3 (Ser321) in SK-MEL-28 melanoma cells [ 74 ]. However, NAC reversed the effect of IVM, which indicated that IVM increased TFE3-dependent autophagy through the ROS signaling pathway.

3. IVM-induced programmed cell death in tumor cells and related mechanisms

3.1. apoptosis.

IVM induces different programmed cell death patterns in different tumor cells ( Table 1 ). As shown in Table 1 , the main form of IVM induced programmed cell death is apoptosis. Apoptosis is a programmed cell death that is regulated by genes to maintain cell stability. It can be triggered by two activation pathways: the endogenous endoplasmic reticulum stress/mitochondrial pathway and the exogenous death receptor pathway [ 75 , 76 ]. The decrease in the mitochondrial membrane potential and the cytochrome c is released from mitochondria into the cytoplasm was detected after the intervention of IVM in Hela cells [ 56 ].Therefore, we infer that IVM induces apoptosis mainly through the mitochondrial pathway. In addition, morphological changed caused by apoptosis, including chromatin condensation, nuclear fragmentation, DNA fragmentation and apoptotic body formation were observed. Finally, IVM changed the balance between apoptosis-related proteins by upregulating the protein Bax and downregulating anti-apoptotic protein Bcl-2, thereby activating caspase-9/-3 to induce apoptosis [ 48 , 53 , 63 ] ( Fig. 2 ).

Summary of IVM promotes programmed cell death.

Fig. 2

Mechanisms of IVM-induced mitochondria-mediated apoptosis.

Cancer cells exposure to IVM can be induced to generate ROS generation and reduce membrane potential of mitochondria. Moreover, IVM can up-regulate Bax and down-regulate Bcl-2, promote releasing of cytochrome C into the cytosol, and activate the signaling cascade of caspases–9/3. Finally, activated PARP and caspase-3 trigger apoptosis.

3.2. Autophagy

Autophagy is a lysosomal-dependent form of programmed cell death. It utilizes lysosomes to eliminate superfluous or damaged organelles in the cytoplasm to maintain homeostasis. It is characterized by double-layered or multilayered vacuolar structures containing cytoplasmic components, which are known as autophagosomes [ 77 ]. In recent years, many studies have shown that autophagy is a double-edged sword in tumor development. On the one hand, autophagy can help tumors adapt to the nutritional deficiency of the tumor microenvironment, and to a certain extent, protect tumor cells from chemotherapy- or radiotherapy- induced injury. On the other hand, some autophagy activators can increase the sensitivity of tumors to radiotherapy and chemotherapy by inducing autophagy, and excessive activation of autophagy can also lead to tumor cell death [ [78] , [79] , [80] , [81] ]. Overall, the specific environment of tumor cells will determine whether autophagy enhances or inhibits tumor development and improving autophagy activity has also become a new approach in cancer therapy. Programmed cell death mediated by autophagy after IVM intervention and the enhancement of the anticancer efficacy of IVM by regulating autophagy are interesting topics. Intervention with IVM in the breast cancer cell lines MCF-7 and MDA-MB-231 significantly increased intracellular autophagic flux and the expression of key autophagy proteins such as LC3, Bclin1, Atg5, and the formation of autophagosomes can be observed [ 32 ]. However, after using the autophagy inhibitors chloroquine and wortmannin or knocking down Bclin1 and Atg5 by siRNA to inhibit autophagy, the anticancer activity of IVM significantly decreased. This proves that IVM mainly exerts an antitumor effect through the autophagy pathway. In addition, researchers also used the Akt activator CA-Akt to prove that IVM mainly induces autophagy by inhibiting the phosphorylation of Akt and mTOR ( Fig. 3 ). The phenomenon of IVM-induced autophagy has also been reported in glioma and melanoma [ 64 , 74 ]. All of the above findings indicate the potential of IVM as an autophagy activator to induce autophagy-dependent death in tumor cells.

Fig. 3

Mechanisms of IVM-induced PAK1/Akt/mTOR-mediated autophagy.

IVM promotes degradation of PAK1 by ubiquitination/proteasome pathway, thereby inhibiting the Akt/mTOR signaling pathway. Subsequently, the inactivation Akt/mTOR signaling cannot inhibit the formation of the Beclin-1 complex, thus inducing the formation autophagosome. Overall, IVM can induce autophagy through PAK1/Akt/mTOR pathway to represses the growth of cancer cells independent of apoptosis. (Ub:Ubiquitination, P:Phosphorylation)

3.3. Cross talk between IVM-induced apoptosis and autophagy

The relationship between apoptosis and autophagy is very complicated, and the cross talk between the two plays a vital role in the development of cancer [ 82 ]. Obviously, the existing results suggest that IVM-induced apoptosis and autophagy also exhibit cross talk. For example, it was found in SK-MEL-28 melanoma cells that IVM can promote apoptosis as well as autophagy [ 74 ]. After using the autophagy inhibitor bafilomycin A1 or siRNA to downregulate Beclin1, IVM-induced apoptosis was significantly enhanced, which suggested that enhanced autophagy will reduce IVM-induced apoptosis and that IVM-induced autophagy can protect tumor cells from apoptosis. However, in breast cancer cell experiments, it was also found that IVM could induce autophagy, and enhanced autophagy could increase the anticancer activity of IVM [ 37 ]. The latest research shows that in normal circumstances autophagy will prevent the induction of apoptosis and apoptosis-related caspase enzyme activation will inhibit autophagy. However, in special circumstances, autophagy may also help to induce apoptosis or necrosis [ 83 ]. In short, the relationship between IVM-induced apoptosis and autophagy involves a complex regulatory mechanism, and the specific molecular mechanism needs further study. We believe that deeper exploration of the mechanism can further guide the use of IVM in the treatment of cancer.

3.4. Pyroptosis

Pyroptosis is a type of inflammatory cell death induced by inflammasomes. The inflammasome is a multimolecular complex containing pattern recognition receptor (PRR), apoptosis-associated speck-like protein containing a CARD (ASC), and pro-caspase-1. PRR can identify pathogen-associated molecular patterns (PAMPs) that are structurally stable and evolutionarily conserved on the surface of pathogenic microorganisms and damage-associated molecular patterns (DAMPs) produced by damaged cells [ 84 , 85 ]. Inflammasomes initiate the conversion of pro-caspase-1 via self-shearing into activated caspase-1. Activated caspase-1 can cause pro-IL-1β and pro-IL-18 to mature and to be secreted. Gasdermin D(GSDMD)is a substrate for activated caspase-1 and is considered to be a key protein in the execution of pyroptosis [ 86 , 87 ]. In an experiment by Draganov, it was found that the release of lactate dehydrogenase (LDH) and activated caspase-1 was significantly increased in breast cancer cells after IVM intervention [ 37 ]. In addition, characteristic pyroptosis phenomena such as cell swelling and rupturing were observed. The authors speculated that IVM may mediate the occurrence of pyroptosis via the P2 × 4/P2 × 7/NLRP3 pathway ( Fig. 4 ), but there is no specific evidence to prove this speculation. Interestingly, in ischemia-reperfusion experiments, IVM aggravated renal ischemia via the P2 × 7/NLRP3 pathway and increased the release of proinflammatory cytokines in human proximal tubular cells [ 88 ]. Although there is currently little evidences showing that IVM induces pyroptosis, it is important to investigate the role of IVM in inducing pyroptosis in other cancers in future studies and realize that IVM may induce different types of programmed cell death in different types of cancer.

Fig. 4

Mechanisms of IVM-induced P2 × 4/P2 × 7/NLRP3-mediated pyroptosis.

IVM can promote ROS release in cancer cells by P2 × 4/P2 × 7 receptors. Cellular ROS can activate NLRP3 Inflammasome including ASC, NLRP3 and pro-caspase-1 assemble. Subsequently, NLRP3 Inflammasome initiates pro-caspase-1 to self-shear into mature caspase-1. On the one hand, activated caspase-1 induces the secretion of pro-inflammatory cytokines IL-1β and IL-18. On the other hand, caspase-1 activated by GSDMD triggers pyroptosis independent of apoptosis.

4. Anticancer effect of IVM through other pathways

4.1. cancer stem cells.

Cancer stem cells (CSCs) are a cell population similar to stem cells with characteristics of self-renewal and differentiation potential in tumor tissue [ 89 , 90 ]. Although CSCs are similar to stem cells in terms of function, because of the lack of a negative feedback regulation mechanism for stem cell self-renewal, their powerful proliferation and multidirectional differentiation abilities are unrestricted, which allows CSCs to maintain certain activities during chemotherapy and radiotherapy [ [90] , [91] , [92] ]. When the external environment is suitable, CSCs will rapidly proliferate to reactivate the formation and growth of tumors. Therefore, CSCs have been widely recognized as the main cause of recurrence after treatment [ 93 , 94 ]. Guadalupe evaluated the effect of IVM on CSCs in the breast cancer cell line MDA-MB-231 [ 95 ]. The experimental results showed that IVM would preferentially targeted and inhibited CSCs-rich cell populations compared with other cell populations in MDA-MB-231 cells. Moreover, the expression of the homeobox protein NANOG, octamer-binding protein 4 (OCT-4) and SRY-box 2 (SOX-2), which are closely related to the self-renewal and differentiation ability of stem cells in CSCs, were also significantly inhibited by IVM. This suggests that IVM may be used as a potential CSCs inhibitor for cancer therapy. Further studies showed that IVM could inhibit CSCs by regulating the PAK1-STAT3 axis [ 96 ].

4.2. Reversal of tumor multidrug resistance

MDR of tumor cells is the main cause of relapses and deaths after chemotherapy [ 97 ]. ATP binding transport family-mediated drug efflux and overexpression of P-glycoprotein (P-gp) are widely considered to be the main causes of tumor MDR [ [98] , [99] , [100] ]. Several studies have confirmed that IVM could reverse drug resistance by inhibiting P-gp and MDR-associated proteins [ [101] , [102] , [103] ]. In Didier's experiments testing the effect of IVM on lymphocytic leukemia, IVM could be used as an inhibitor of P-gp to affect MDR [ 22 ]. In Jiang's experiment, IVM reversed the drug resistance of the vincristine-resistant colorectal cancer cell line HCT-8, doxorubicin-resistant breast cancer cell line MCF-7 and the chronic myelogenous leukemia cell line K562 [ 104 ]. IVM inhibited the activation of EGFR and the downstream ERK/Akt/NF-kappa B signaling pathway to downregulate the expression of P-gp. Earlier, we mentioned the role of IVM in docetaxel-resistant prostate cancer [ 50 ] and gemcitabine-resistant cholangiocarcinoma [ 44 ]. These results indicated the significance of applying IVM for the treatment of chemotherapy patients with MDR.

4.3. Enhanced targeted therapy and combined treatment

Targeted treatment of key mutated genes in cancer, such as EGFR in lung cancer and HER2 in breast cancer, can achieve powerful clinical effects [ 105 , 106 ]. HSP27 is a molecular chaperone protein that is highly expressed in many cancers and associated with drug resistance and poor prognosis. It is considered as a new target for cancer therapy [ 107 ]. Recent studies have found that IVM could be used as an inhibitor of HSP27 phosphorylation to enhance the activity of anti-EGFR drugs in EGFR/HER2- driven tumors. An experiment found that IVM could significantly enhance the inhibitory effects of erlotinib and cetuximab on lung cancer and colorectal cancer [ 50 ]. Earlier, we mentioned that IVM combined with conventional chemotherapeutic drugs such as cisplatin [ 60 ], paclitaxel [ 59 ], daunorubicin and cytarabine [ 51 ], or with targeted drugs such as dasatinib [ 53 ] and dapafenib [ 73 ] shows great potential for cancer treatment. The combination of drugs can effectively increase efficacy, reduce toxicity or delay drug resistance. Therefore, combination therapy is the most common method of chemotherapy. IVM has a variety of different mechanisms of action in different cancers, and its potential for synergistic effects and enhanced efficacy in combination therapy was of particular interest to us. Not only does IVM not overlap with other therapies in term of its mechanism of action, but the fact that of IVM has multiple targets suggests that it is not easy to produce IVM resistance. Therefore, continued study and testing of safe and effective combination drug therapies is essential to maximize the anticancer effects of IVM.

5. Molecular targets and signaling pathways involved in the anticancer potential of IVM

As mentioned above, the anticancer mechanism of IVM involves a wide range of signaling pathways such as Wnt/β-catenin, Akt/mTOR, MAPK and other possible targets such as PAK1 and HSP27, as well as other mechanisms of action ( Table 2 ). We found that IVM inhibits tumor cell development in a PAK1-dependent manner in most cancers. Consequently, we have concentrated on discussing the role of PAK1 kinase and cross-talk between various pathways and PAK1 to provide new perspectives on the mechanism of IVM function.

Summary of the anticancer mechanism of IVM

As a member of the PAK family of serine/threonine kinases, PAK1 has a multitude of biological functions such as regulating cell proliferation and apoptosis, cell movement, cytoskeletal dynamics and transformation [ 108 ]. Previous studies have indicated that PAK1 is located at the intersection of multiple signaling pathways related to tumorigenesis and is a key regulator of cancer signaling networks ( Fig. 5 ). The excessive activation of PAK1 is involved in the formation, development, and invasion of various cancers [ 109 , 110 ]. Targeting PAK1 is a novel and promising method for cancer treatment, and the development of PAK1 inhibitors has attracted widespread attention [ 111 ]. IVM is a PAK1 inhibitor in a variety of tumors, and it has good safety compared to that of other PAK1 inhibitors such as IPA-3. In melanoma and nasopharyngeal carcinoma, IVM inhibited cell proliferation activity by inhibiting PAK1 to downregulate the expression of MEK 1/2 and ERK1/2 [ 69 , 73 ]. After IVM intervention in breast cancer, the expression of PAK1 was also significantly inhibited, and the use of siRNA to downregulate the expression of PAK1 in tumor cells significantly reduced the anticancer activity of IVM. Interestingly, IVM could inhibit the expression of PAK1 protein but did not affect the expression of PAK1 mRNA [ 32 ].The proteasome inhibitor MG132 reversed the suppressive effect of IVM, which indicated that IVM mainly degraded PAK1 via the proteasome ubiquitination pathway. We have already mentioned that IVM plays an anticancer role in various tumors by regulating pathways closely related to cancer development. PAK1 is at the junction of these pathways. Overall, we speculate that IVM can regulate the Akt/mTOR, MAPK and other pathways that are essential for tumor cell proliferation by inhibiting PAK1 expression, which plays an anticancer role in most cancers.

Fig. 5

PAK1 cross regulate multiple signal pathways.

RAS activation directly initiates PAK1, MAPK and PI3K/Akt pathway. PAK1 allocates crosstalk between the PI3K and MAPK pathways. PAK1 can induce MEK1/2 and ERK1/2 activation by RAF and increase PI3K/Akt signaling by PDK1. PAK1 can also activate pro-inflammatory pathways by facilitating nuclear activation of NF-kappa B. In addition, PAK1 facilitates Wnt/β-catenin signaling, make β-catenin accumulate in the cytoplasm and translocate to the nucleus. Moreover, Akt can inhibit β-catenin transfer into nucleus.

6. Summary and outlooks

Malignant tumors are one of the most serious diseases that threaten human health and social development today, and chemotherapy is one of the most important methods for the treatment of malignant tumors. In recent years, many new chemotherapeutic drugs have entered the clinic, but tumor cells are prone to drug resistance and obvious adverse reactions to these drugs. Therefore, the development of new drugs that can overcome resistance, improve anticancer activity, and reduce side effects is an urgent problem to be solved in chemotherapy. Drug repositioning is a shortcut to accelerate the development of anticancer drugs.

As mentioned above, the broad-spectrum antiparasitic drug IVM, which is widely used in the field of parasitic control, has many advantages that suggest that it is worth developing as a potential new anticancer drug. IVM selectively inhibits the proliferation of tumors at a dose that is not toxic to normal cells and can reverse the MDR of tumors. Importantly, IVM is an established drug used for the treatment of parasitic diseases such as river blindness and elephantiasis. It has been widely used in humans for many years, and its various pharmacological properties, including long- and short-term toxicological effects and drug metabolism characteristics are very clear. In healthy volunteers, the dose was increased to 2 mg/Kg, and no serious adverse reactions were found, while tests in animals such as mice, rats, and rabbits found that the median lethal dose (LD50) of IVM was 10-50 mg/Kg [ 112 ] In addition, IVM has also been proven to show good permeability in tumor tissues [ 50 ]. Unfortunately, there have been no reports of clinical trials of IVM as an anticancer drug. There are still some problems that need to be studied and resolved before IVM is used in the clinic.

(1) Although a large number of research results indicate that IVM affects multiple signaling pathways in tumor cells and inhibits proliferation, IVM may cause antitumor activity in tumor cells through specific targets. However, to date, no exact target for IVM action has been found. (2) IVM regulates the tumor microenvironment, inhibits the activity of tumor stem cells and reduces tumor angiogenesis and tumor metastasis. However, there is no systematic and clear conclusion regarding the related molecular mechanism. Therefore, in future research, it is necessary to continue to explore the specific mechanism of IVM involved in regulating the tumor microenvironment, angiogenesis and EMT. (3) It has become increasingly clear that IVM can induce a mixed cell death mode involving apoptosis, autophagy and pyroptosis depending on the cell conditions and cancer type. Identifying the predominant or most important contributor to cell death in each cancer type and environment will be crucial in determining the effectiveness of IVM-based treatments. (4) IVM can enhance the sensitivity of chemotherapeutic drugs and reduce the production of resistance. Therefore, IVM should be used in combination with other drugs to achieve the best effect, while the specific medication plan used to combine IVM with other drugs remains to be explored.

Most of the anticancer research performed on the avermectin family has been focused on avermectin and IVM until now. Avermectin family drugs such as selamectin [ 36 , 41 , 113 ], and doramectin [ 114 ] also have anticancer effects, as previously reported. With the development of derivatives of the avermectin family that are more efficient and less toxic, relevant research on the anticancer mechanism of the derivatives still has great value. Existing research is sufficient to demonstrate the great potential of IVM and its prospects as a novel promising anticancer drug after additional research. We believe that IVM can be further developed and introduced clinically as part of new cancer treatments in the near future.

Declaration of Competing Interest

The authors report no declarations of interest.

Acknowledgments

This work was supported by the Science Research Innovation Team Project of Anhui Colleges and Universities (2016-40), the Bengbu City Natural Science Foundation (2019-12), the Key Projects of Science Research of Bengbu Medical College (BYKY2019009ZD) and National University Students’ Innovation and Entrepreneurship Training Program (201910367001).

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Cancer experts call on philanthropists to help fund ‘golden age’ of research

More than 50 top researchers sign letter asking for philanthropic support to transform cancer prevention, diagnosis and treatment

Leading cancer experts from around the world are calling on wealthy individuals and philanthropists to dig into their deep pockets to accelerate a new golden age of cancer research.

More than 50 senior scientists from the UK, Europe, North America and Asia, including three Nobel laureates, say advances in artificial intelligence and other technologies have created a “unique opportunity” to transform cancer prevention, diagnosis and treatment in the next 10 years.

In a “Letter to the World”, the researchers called cancer a “defining health issue of our time” and argued that it deserves the same “massive global response” that swung into action during the Covid pandemic to produce tests, vaccines and treatments for the virus.

“As leading representatives of the global scientific and research community, we know we’re standing at a tipping point that could transform how we understand and overcome cancer,” they wrote. With philanthropic support, the researchers said, the field could turn ideas in the lab into clinical tools much faster, and improve or save millions of lives.

Globally, 18 million people are diagnosed with cancer each year and 10 million die from the disease. The number of cases is expected to rise 50% by 2040, according to the International Agency for Research on Cancer. Recently, scientists have noted a sharp rise in cases among the under-50s .

Sir Paul Nurse, the director of the Francis Crick Institute in London and winner of the 2001 Nobel prize in medicine, said technological leaps meant cancer research could now be carried out much more quickly. In the next decade, he said, therapies for childhood cancers could be “revolutionised”, while tests and personal data should enable the earlier detection of tumours and more personalised treatments.

But according to Cancer Research UK, scientists in the field face a £1bn funding shortfall over the next decade that threatens to jeopardise progress. “If we are to continue making huge leaps in how we prevent, diagnose and treat cancer, we need the funds,” said Nurse, a signatory of the letter, which is urging philanthropists to donate to the global effort.

The letter coincides with the launch of CRUK’s More Research, Less Cancer campaign which aims to raise £400m in philanthropic funding. The charity estimates that 110,000 deaths could be avoided over the next 20 years if UK cancer death rates are reduced by 15% in that time.

Britain has some of the worst five-year survival rates among rich countries for breast, lung and colon cancer, three of the most common forms of the disease. The poor performance is largely driven by late diagnoses and delays in treatment. In England, waiting times for cancer patients were the worst on record last year, with less than two-thirds starting treatment within 62 days of cancer being suspected.

Researchers hope survival rates will improve if a raft of new technologies prove themselves. Next generation blood tests can detect more than a dozen cancers early on , while AI is increasingly being used to flag patients most at risk of specific cancers.

Another signatory of the letter, Prof Sir Peter Ratcliffe, the winner of the 2019 Nobel prize in medicine who holds posts at the Crick and the University of Oxford, said new computational tools showed enormous potential. “When combined with new analytical methods operating at the molecular level there is the ability to transform the way we think about cancer and the design of cancer therapeutics,” he said.

CRUK said philanthropic donations raised by its campaign would support work at the Crick and the global Cancer Grand Challenges research initiative.

Prof Caroline Dive, interim director at the CRUK Manchester Institute, said: “We are at a really crucial time for cancer research. I don’t think it’s any exaggeration to say that we are moving into a golden age, where discoveries from the past few decades have set us up to make real progress.”

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Could somebody be hiding the cure for cancer?

25th March 2020

A stubborn myth that we often see popping up on social media is that there’s already a cure for cancer, but it’s being hidden from the public for some reason. A myth is exactly what this is. Read on to get the full explanation from our experts.

Billions have been spent on cancer research over many decades, but we still haven’t cured cancer. Our experts explain why that is — and why we still urgently need to fund more cancer research.

One survey suggests that over a quarter of Americans believe this to be absolutely true, while a further 1 in 7 believe that it might be true.

Could there really be a big conspiracy? Is it possible that pharmaceutical companies are hiding the cure for cancer to make a profit from cancer drugs?

We want to shed some light on the issue and explain why it’s simply not true - there is no ‘hidden cancer cure’.

Because we already know we’re not looking for a single cure for cancer.

Cancer is a name for a group of over 200 distinct diseases. Types of cancer vary considerably in their causes and the way in which they grow and spread - the sheer complexity of cancer makes a single cure incredibly unlikely.

We may not find a single cure, but we do have the tools and treatments to cure many people already. Cancer survival rates have doubled in the last 40 years and continue to improve. Half of all people diagnosed with cancer in the UK in 2019 will survive their disease for 10 years or longer. That’s astonishing progress – and it’s been achieved by cancer research that has been carried out over years.

Because it wouldn’t be profitable for ‘Big Pharma’ to hide a cure for cancer.

Apart from the scientific improbability of a universal cancer cure, it wouldn’t make a whole lot of economic sense to hide a cure, either. Even if a potential silver bullet existed, it would take decades to test it on each type and stage of cancer. This kind of testing requires vast amounts of money.

What would the benefit of hiding a cure be? Big pharmaceutical companies invest billions in the development of new drugs. If one of them had struck gold and found a magic bullet, they’d want to claim those expenses back.

Because it wouldn’t be possible to keep something like a cure for cancer secret.

The sheer scale of the operation would be mind-boggling. Think of the huge amount of people involved in the research and manufacturing of the drug. Could that number of people really keep such a secret? 

Dr Robert Grimes published a great paper in which he studied the mathematical likelihood of conspiracy theories. He created a model using real uncovered medical conspiracies to estimate how long it would take for something like this to be uncovered, depending on the amount of people involved.

Dr Grimes estimated that, if only the biggest pharmaceutical companies were involved in the conspiracy, there would still be around 714,000 people who knew something . And with that many people involved; his calculations show that it would only take around 3.17 years for someone to blab.

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In 2023 we said “Yes!” to 30 new pioneering ideas from outstanding cancer researchers around the world

Because cancer researchers want to create new treatments that help people..

A lot of people believe in a ‘Big Pharma’ conspiracy because the companies involved are exactly that – companies that exist to make money for their shareholders. But these companies aren’t faceless, they’re made up of people. And whether people are rich, famous or a board member of a pharmaceutical company, cancer doesn’t care, it affects everyone.

Everyone knows somebody that has been affected by the disease – it doesn’t make sense that some of those people would be willing to risk their lives and the lives of their loved ones by hiding new cures.

Because we are all human and we all have the same goal.

At Worldwide Cancer Research, we are surrounded by colleagues, scientists, doctors, patients and supporters who are dedicated to their quests to conquer cancer.

Suggesting that there’s some kind of conspiracy to make money by holding back a lifesaving cure for cancer insults the people who contribute every day to finding new ways to prevent, diagnose and treat the disease. 

One day we will stop the suffering caused by cancer.

Not because of a miracle drug, or because there’s a secret cure out there already, but because hundreds of thousands of people are working together every day, and all over the world, to find new cures.

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How CRISPR Is Changing Cancer Research and Treatment

July 27, 2020 , by NCI Staff

Wrench and socket on a graphic of DNA

CRISPR is a highly precise gene editing tool that is changing cancer research and treatment.

Ever since scientists realized that changes in DNA cause cancer , they have been searching for an easy way to correct those changes by manipulating DNA . Although several methods of gene editing have been developed over the years, none has really fit the bill for a quick, easy, and cheap technology.

But a game-changer occurred in 2013, when several researchers showed that a gene-editing tool called CRISPR could alter the DNA of human cells like a very precise and easy-to-use pair of scissors. 

The new tool has taken the research world by storm, markedly shifting the line between possible and impossible. As soon as CRISPR made its way onto the shelves and freezers of labs around the world, cancer researchers jumped at the chance to use it.

“CRISPR is becoming a mainstream methodology used in many cancer biology studies because of the convenience of the technique,” said Jerry Li, M.D., Ph.D., of NCI’s Division of Cancer Biology .

Now CRISPR is moving out of lab dishes and into trials of people with cancer. In a small study, for example, researchers tested a cancer treatment involving immune cells that were CRISPR-edited to better hunt down and attack cancer. 

Despite all the excitement, scientists have been proceeding cautiously, feeling out the tool’s strengths and pitfalls, setting best practices, and debating the social and ethical consequences of gene editing in humans. 

How Does CRISPR Work?

Like many other advances in science and medicine, CRISPR was inspired by nature. In this case, the idea was borrowed from a simple defense mechanism found in some microbes, such as bacteria. 

To protect themselves against invaders like viruses, these microbes capture snippets of the intruder’s DNA and store them away as segments called CRISPRs, or clustered regularly interspersed short palindromic repeats. If the same germ tries to attack again, those DNA segments (turned into short pieces of RNA ) help an enzyme called Cas find and slice up the invader’s DNA. 

After this defense system was discovered, scientists realized that it had the makings of a versatile gene-editing tool. Within a handful of years, multiple groups had successfully adapted the system to edit virtually any section of DNA, first in the cells of other microbes, and then eventually in human cells.

Graphic showing how Cas and a guide RNA work together to find and cut the target DNA.

CRISPR consists of a guide RNA (RNA-targeting device, purple) and the Cas enzyme (blue). When the guide RNA matches up with the target DNA (orange), Cas cuts the DNA. A new segment of DNA (green) can then be added.

In the laboratory, the CRISPR tool consists of two main actors: a guide RNA and a DNA-cutting enzyme, most commonly one called Cas9. Scientists design the guide RNA to mirror the DNA of the gene to be edited (called the target). The guide RNA partners with Cas and—true to its name—leads Cas to the target. When the guide RNA matches up with the target gene's DNA, Cas cuts the DNA. 

What happens next depends on the type of CRISPR tool that’s being used. In some cases, the target gene's DNA is scrambled while it's repaired, and the gene is inactivated . With other versions of CRISPR, scientists can manipulate genes in more precise ways such as adding a new segment of DNA or editing single DNA letters . 

Scientists have also used CRISPR to detect specific targets, such as DNA from cancer-causing viruses and RNA from cancer cells . Most recently, CRISPR has been put to use as an experimental test to detect the novel coronavirus .

Why Is CRISPR a Big Deal?

Scientists consider CRISPR to be a game-changer for a number of reasons. Perhaps the biggest is that CRISPR is easy to use, especially compared with older gene-editing tools. 

“Before, only a handful of labs in the world could make the proper tools [for gene editing]. Now, even a high school student can make a change in a complex genome ” using CRISPR, said Alejandro Chavez, M.D., Ph.D., an assistant professor at Columbia University who has developed several novel CRISPR tools.

CRISPR is also completely customizable. It can edit virtually any segment of DNA within the 3 billion letters of the human genome, and it’s more precise than other DNA-editing tools. 

And gene editing with CRISPR is a lot faster. With older methods, “it usually [took] a year or two to generate a genetically engineered mouse model , if you’re lucky,” said Dr. Li. But now with CRISPR, a scientist can create a complex mouse model within a few months, he said. 

Another plus is that CRISPR can be easily scaled up. Researchers can use hundreds of guide RNAs to manipulate and evaluate hundreds or thousands of genes at a time. Cancer researchers often use this type of experiment to pick out genes that might make good drug targets . 

And as an added bonus, “it’s certainly cheaper than previous methods,” Dr. Chavez noted.

What Are CRISPR’s Limitations?

With all of its advantages over other gene-editing tools, CRISPR has become a go-to for scientists studying cancer. There’s also hope that it will have a place in treating cancer, too. But CRISPR isn’t perfect, and its downsides have made many scientists cautious about its use in people.

A major pitfall is that CRISPR sometimes cuts DNA outside of the target gene—what’s known as “off-target” editing. Scientists are worried that such unintended edits could be harmful and could even turn cells cancerous , as occurred in a 2002 study of a gene therapy . 

“If [CRISPR] starts breaking random parts of the genome, the cell can start stitching things together in really weird ways, and there’s some concern about that becoming cancer,” Dr. Chavez explained. But by tweaking the structures of Cas and the guide RNA, scientists have improved CRISPR’s ability to cut only the intended target, he added. 

Another potential roadblock is getting CRISPR components into cells. The most common way to do this is to co-opt a virus to do the job. Instead of ferrying genes that cause disease, the virus is modified to carry genes for the guide RNA and Cas. 

Slipping CRISPR into lab-grown cells is one thing; but getting it into cells in a person's body is another story. Some viruses used to carry CRISPR can infect multiple types of cells, so, for instance, they may end up editing muscle cells when the goal was to edit liver cells. 

Researchers are exploring different ways to fine-tune the delivery of CRISPR to specific organs or cells in the human body. Some are testing viruses that infect only one organ, like the liver or brain. Others have created tiny structures called  nanocapsules that are designed to deliver CRISPR components to specific cells.

Because CRISPR is just beginning to be tested in humans, there are also concerns about how the body—in particular, the immune system —will react to viruses carrying CRISPR or to the CRISPR components themselves. 

Some wonder whether the immune system could attack Cas (a bacterial enzyme that is foreign to human bodies) and destroy CRISPR-edited cells. Twenty years ago, a patient died after his immune system launched a massive attack against the viruses carrying a gene therapy he had received. However, newer CRISPR-based approaches rely on viruses that appear to be safer than those used for older gene therapies.

Another major concern is that editing cells inside the body could accidentally make changes to sperm or egg cells that can be passed on to future generations. But for almost all ongoing human studies involving CRISPR, patients’ cells are removed and edited outside of their bodies. This “ ex vivo ” approach is considered safer because it is more controlled than trying to edit cells inside the body, Dr. Chavez said.

However, one ongoing study is testing CRISPR gene editing directly in the eyes of people with a genetic disease that causes blindness, called Leber congenital amaurosis.

The First Clinical Trial of CRISPR for Cancer

The first trial in the United States to test a CRISPR-made cancer therapy was launched in 2019 at the University of Pennsylvania. The study, funded in part by NCI, is testing a type of immunotherapy in which patients’ own immune cells are genetically modified to better “see” and kill their cancer. 

The therapy involves making four genetic modifications to T cells , immune cells that can kill cancer. First, the addition of a synthetic gene gives the T cells a claw-like protein (called a receptor ) that “sees” NY-ESO-1, a molecule on some cancer cells.

Then CRISPR is used to remove three genes: two that can interfere with the NY-ESO-1 receptor and another that limits the cells’ cancer-killing abilities. The finished product, dubbed NYCE T cells, were grown in large numbers and then infused into patients. 

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The first trial of CRISPR for patients with cancer tested T cells that were modified to better "see" and kill cancer. CRISPR was used to remove three genes: two that can interfere with the NY-ESO-1 receptor and another that limits the cells’ cancer-killing abilities. 

“We had done a prior study of NY-ESO-1–directed T cells and saw some evidence of improved response and low toxicity ,” said the trial’s leader, Edward Stadtmauer, M.D., of the University of Pennsylvania. He and his colleagues wanted to see if removing the three genes with CRISPR would make the T cells work even better, he said. 

The goal of this study was to first find out if the CRISPR-made treatment was safe. It was tested in two patients with advanced multiple myeloma and one with metastatic sarcoma . All three had tumors that contained NY-ESO-1, the target of the T-cell therapy. 

Initial findings suggest that the treatment is safe . Some side effects did occur, but they were likely caused by the chemotherapy patients received before the infusion of NYCE cells, the researchers reported. There was no evidence of an immune reaction to the CRISPR-edited cells. 

Only about 10% of the T cells used for the therapy had all four of the desired genetic edits. And off-target edits were found in the modified cells of all three patients. However, none of the cells with off-target edits grew in a way that suggested they had become cancer, Dr. Stadtmauer noted.

The treatment had a small effect on the patients’ cancers. The tumors of two patients (one with multiple myeloma and one with sarcoma) stopped growing for a while but resumed growing later. The treatment didn't work at all for the third patient. 

It's exciting that the treatment initially worked for the sarcoma patient because “ solid tumors have been a much more difficult nut to crack with cellular therapy," Dr. Stadtmauer said. "Perhaps [CRISPR] techniques will enhance our ability to treat solid tumors with cell therapies.”

Although the trial shows that CRISPR-edited cell therapy is possible, the long-term effects still need to be monitored, Dr. Stadtmauer continued. The NYCE cells are “safe for as long as we’ve been watching [the study participants]. Our plan is to keep monitoring them for years, if not decades,” he said. 

More Studies of CRISPR Treatments to Come 

While the study of NYCE T cells marked the first trial of a CRISPR-based cancer treatment, there are likely more to come. 

“This [trial] was really a proof-of-principle, feasibility, and safety thing that now opens up the whole world of CRISPR editing and other techniques of [gene] editing to hopefully make the next generation of therapies,” Dr. Stadtmauer said. 

Other clinical studies of CRISPR-made cancer treatments are already underway. A few trials are testing CRISPR-engineered CAR T-cell therapies , another type of immunotherapy. For example, one company is testing CRISPR-engineered CAR T cells in people with B cell cancers and people with multiple myeloma .

There are still a lot of questions about all the ways that CRISPR might be put to use in cancer research and treatment. But one thing is for certain: The field is moving incredibly fast and new applications of the technology are constantly popping up. 

“People are still improving CRISPR methods,” Dr. Li said. “It’s quite an active area of research and development. I’m sure that CRISPR will have even broader applications in the future.”

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