Four people who were successfully treated for rectal cancer in a clinical trial at MSK join the trial’s two principal investigators. All participants in the trial saw their tumors disappear using immunotherapy, without surgery, radiation, or chemotherapy. (Left to right) Sascha Roth, Dr. Luis Diaz, Imtiaz Hussain, Dr. Andrea Cercek, Avery Holmes, and Nisha Varughese.
Doctors at Memorial Sloan Kettering Cancer Center (MSK) pioneered advances in many types of cancer treatment for patients in 2022. Clinical trials reported results of cutting-edge treatments, including new types of immunotherapy, radiation therapy, and experimental targeted drugs. In many cases, these trials resulted in new standards of care that have the potential to benefit people with cancer all over the world.
“MSK has one of the largest cancer clinical trials programs in the country,” says MSK President and CEO Selwyn M. Vickers, MD, FACS. “Patients who choose to participate in a trial at MSK can receive the most advanced cancer treatment available, sometimes long before it’s offered anywhere else.”
Here are some of the most important developments in 2022, grouped by cancer type and chronological order:
A Combination Therapy Developed at MSK Benefits People With Rare Stomach Cancer
The targeted therapy imatinib (Gleevec®) is a standard treatment for a rare stomach cancer called gastrointestinal stromal tumor (GIST). But patients often develop resistance to this drug. A phase 2 clinical trial led by MSK scientist and medical oncologist Ping Chi, MD, PhD, found that adding another targeted drug called binimetinib (Mektovi®) resulted in a significantly better response than what’s usually seen in patients treated with imatinib alone. The trial was based on lab research Dr. Chi conducted more than a decade ago, when she was a postdoctoral research fellow. Results were published online January 18, 2022, in the Journal of Clinical Oncology.
Major Advances in Prostate Cancer
- On March 23, 2022, the U.S. Food and Drug Administration (FDA) approved the targeted radiodrug lutetium Lu 177 vipivotide tetraxetan (Pluvicto™) for patients with metastatic castration-resistant prostate cancer that is positive for a protein called prostate-specific membrane antigen (PSMA). MSK medical oncologist Michael Morris, MD, helped design, execute, and analyze the clinical trial that showed the effectiveness of the drug and led to its approval. The approval was the latest advance in the field of theranostics, which uses radioactive substances to visualize cancer cells and destroy them without harming normal cells.
- A new treatment known as focal therapy has emerged for prostate cancer considered to be “intermediate risk” — mostly, small tumors confined to one area of the prostate. Researchers at MSK have been developing an advanced focal therapy approach called high-intensity focused ultrasound (HIFU), which is guided by magnetic resonance imaging (MRI). On June 14, 2022, a team led by MSK urologic cancer surgeon Behfar Ehdaie, MD, published a phase 2 clinical trial in Lancet Oncology that found this less-invasive method worked well for 88% of the 101 patients in the study.
- A technique called MRI-guided radiotherapy takes multiple MRI images during radiation treatments to adjust for tumor movement in real time. This treatment, developed by radiation oncologist and prostate cancer specialist Michael Zelefsky, MD, enables doctors to obliterate tumors in just five treatment sessions without harming nearby cells. At the annual meeting of the American Society for Radiation Oncology (ASTRO) in October 2022, MSK radiation oncologist and prostate cancer specialist Victoria Brennan, MBBCh, reported results from a small study that demonstrated the treatment is safe and effective
Improved Treatments for Bladder Cancer
- Bladder removal (radical cystectomy) is an effective treatment for bladder cancer, but patients are often worried about how this surgery will affect their quality of life. A new study led by urologic surgeon Bernard Bochner, MD, showed that patients who have their bladders removed at MSK recover well. A survey of 411 people who had a radical cystectomy found no significant decrease in patients’ quality of life — even within the first three months after the operation. This research was published in the March 2022 issue of European Urology.
- A new combination therapy showed positive results treating people with metastatic bladder cancer. MSK genitourinary oncologist Jonathan Rosenberg, MD, presented results of a clinical trial that tested the effectiveness of the antibody-drug conjugate enfortumab vedotin (Padcev®) combined with the checkpoint inhibitor pembrolizumab (Keytruda®), at the annual meeting of the European Society for Medical Oncology (ESMO) on September 12, 2022. The treatment shrank tumors in about 65% of patients
A Surgical Milestone Protects Fertility in Young People With Cancer
The first-known uterine transposition surgery in the United States was reported by MSK in April 2022, performed by gynecologic surgeon Mario Leitao, MD, and gynecologic surgeon Jennifer Mueller, MD. This groundbreaking procedure involves temporarily moving the uterus up to the abdominal wall to avoid damage from radiation to the pelvis. It was offered to a young woman being treated for colorectal cancer, with the goal of preserving her fertility. This complex surgery requires many specialists and a highly coordinated treatment plan. It has since been performed on five other patients at MSK.
Rectal Cancer Disappears After Experimental Use of Immunotherapy
A small but historic trial led by MSK medical oncologist Andrea Cercek, MD, and medical oncologist Luis Diaz, Jr., MD, found that for patients with certain gene mutations, immunotherapy completely eliminated their rectal cancer, enabling them to avoid debilitating side effects from the standard treatment of radiation, surgery, and chemotherapy. After more than two years, all 14 patients in the trial remained cancer free. The checkpoint inhibitor drug dostarlimab (Jemperli) was given to patients with stage 2 or 3 rectal cancer whose tumors were classified as mismatch repair-deficient (MMRd) or microsatellite instability (MSI). Results from the trial were presented at the American Society of Clinical Oncology (ASCO) meeting on June 5, 2022, and published in The New England Journal of Medicine (NEJM) the same day.
An mRNA Vaccine for Pancreatic Cancer Shows Promising Results
Even before messenger RNA (mRNA) vaccines helped turn the tide against COVID-19, MSK researchers were using mRNA vaccine technology to treat cancer. MSK pancreatic cancer surgeon and scientist Vinod Balachandran, MD, is leading the only clinical trial to test mRNA vaccines for pancreatic cancer. The key to these vaccines appears to be proteins in the pancreatic tumors that alert the immune system to keep the cancer at bay. The vaccines are custom-made for every person, and the hope is that they will stimulate the production of immune cells that recognize and destroy pancreatic cancer cells. Dr. Balachandran presented positive findings from this trial on June 5, 2022, at the ASCO meeting.
Innovative Drug Targets HER2, a Common Driver in Breast Cancer and Lung Cancer
- On August 5, 2022, the FDA approved the first targeted therapy for patients with HER2-low breast cancer that has spread to other parts of the body and is unable to be surgically removed. The drug, trastuzumab deruxtecan (T-DXd or Enhertu®), was approved based on an international clinical trial led by MSK breast medical oncologist Shanu Modi, MD. Dr. Modi’s transformational study showed that T-DXd is able to block HER2 even when levels of the protein are low — the case for about 55% of people with breast cancer. Dr. Modi presented the findings at the ASCO meeting on June 5, 2022, and the results were published in NEJM on the same day.
- On August 11, 2022, the FDA also approved T-DXd for non-small cell lung cancers caused by mutations in the HER2 gene. The trial that resulted in the approval was led by MSK medical oncologist Bob Li, MD, PhD, MPH. This drug is an antibody-drug conjugate consisting of two parts: an antibody that homes in on cancer cells with a potent dose of chemotherapy. Because the antibody delivers the chemotherapy payload directly to its target, it reduces the collateral damage of side effects seen with traditional chemotherapy. This antibody-drug conjugate is more effective than other drugs that target HER2.
Molecular Testing Leads to Personalized Treatment for People With Rare Sarcomas
MSK conducted the two largest studies ever to analyze the genetics of sarcoma tumors, which are rare cancers of the bones and soft tissues that are particularly difficult to diagnose and treat. The two papers, published June 15, 2022, in Nature Communications and led by medical oncologist and sarcoma expert Mrinal Gounder, MD, and Benjamin Nacev, MD, reported the findings from the analysis of thousands of sarcoma tumors. These specialized molecular tests can suggest personalized cancer treatments, which can be more effective than standard chemotherapy and have fewer side effects.
New CAR T Cell Therapy for Multiple Myeloma Shows Promise
With CAR T therapy, a patient’s own immune cells are engineered to attack cancer cells. Results from a phase 1 clinical trial showed that a new CAR T cell therapy appears to be safe and effective in treating multiple myeloma, a form of blood cancer. This therapy targets a protein called GPRC5D. The trial included 17 people with multiple myeloma for whom other treatments did not work. It was led by MSK multiple myeloma specialist Sham Mailankody, MBBS. Overall, 71% of the patients responded to the therapy, meaning their cancer shrank or disappeared, although some had significant side effects. Among 12 patients who received a lower dose, which had manageable side effects, the response rate was 58%. The study was reported in the September 29, 2022, issue of NEJM.
FDA Approves New Immunotherapy Combination To Treat Advanced Liver Cancer
On October 24, 2022, the FDA approved a combination of the immunotherapy drugs durvalumab (Imfinzi®) and tremelimumab (Imjudo®) for people with unresectable hepatocellular cancer, the most common type of liver cancer. The approval was based on results of the clinical trial HIMALAYA involving 1,117 patients, led by MSK gastrointestinal medical oncologist Ghassan Abou-Alfa, MD. The results from the study showed after three years, about 30.7% of the patients who received the new combination immunotherapy were still alive, compared with 20.2% of the people who received only the kinase inhibitor drug sorafenib (Nexavar®). In the group given durvalumab alone, 24.7% were still alive. In other words, the combination immunotherapy lowered the risk of death by 22%.
FDA Approves a Targeted Drug for Rare Blood Disorders Called Histiocytic Neoplasms
On November 1, 2022, the FDA approved a drug called cobimetinib (Cotellic®) for treating adult patients with a rare group of blood disorders called histiocytosis or histiocytic neoplasms. These diseases include Erdheim-Chester disease, Rosai-Dorfman disease, and Langerhans cell histiocytosis. The clinical trial that led to this approval was conducted solely at MSK and was led by neuro-oncologist Eli Diamond, MD. Based on research conducted in the lab of hematologic oncologist Omar Abdel-Wahab, MD, MSK investigators determined that cobimetinib, a targeted drug already approved to treat melanoma, was likely to be an effective treatment for histiocytosis.
International Study Shows Liquid Biopsies May Improve Lung Cancer Survival
An international study led by MSK investigators found that liquid biopsies may improve survival in people with lung cancer. Liquid biopsies are blood tests seeking to detect small amounts of cancer DNA floating in the blood, which can reveal the targeted drugs that are most likely to be effective for each patient. The study followed more than 1,000 people who were being treated for non-small cell lung cancer and had liquid biopsies. Of those who could be matched with targeted therapies based on molecular analysis, there was a 37% reduction in mortality. This research was published in Nature Medicine on November 10, 2022, and was led by Dr. Li and medical oncology fellow Justin Jee, MD, PhD.