Top cancer treatment advances at MSK in 2025
Memorial Sloan Kettering Cancer Center
Doctors at Memorial Sloan Kettering Cancer Center (MSK) pioneered advances in a variety of cancer treatments in 2025 — including new approaches to immunotherapy, targeted therapy, and surgical imaging. Highlights this year also included a stem cell–based therapy for Parkinson’s disease and several clinical trials looking at the benefits of integrative medicine.
Additionally, in 2025, the U.S. Food and Drug Administration (FDA) approved 11 drugs based on clinical trials in which MSK played a pivotal role.
“MSK’s clinical research teams made great progress in 2025, discovering new treatments to help cancer patients everywhere live better and longer,” says MSK President and CEO Selwyn M. Vickers, MD, FACS. “When patients choose to participate in one of our more than 1,500 interventional clinical trials, they have the opportunity to receive the latest therapies for their cancer and also give hope to future generations.”
Here are some of the most important developments of the past year, listed in chronological order.
Latest Research Into DIPG Pediatric Brain Cancer Treatment Using Novel Drug Delivery Technique
Researchers from MSK and Weill Cornell Medicine reported hopeful results from a phase 1 study that tested a novel drug delivery method for an extremely challenging pediatric brain cancer. The technique, called convection-enhanced delivery (CED), slowly infuses drugs directly into the brain, bypassing the blood-brain barrier and targeting specific regions.
Results from the study, led by MSK pediatric neurosurgeon Mark Souweidane, MD, indicated that CED appears safe and effective at distributing a drug throughout a rare and particularly deadly pediatric brain tumor called diffuse intrinsic pontine glioma (DIPG).
The median survival rate for DIPG is typically 8-12 months, but several children in the trial have lived for more than three years after treatment, according to findings published February 19 in Neuro-Oncology. Additional research is planned.
Promising New Treatment for Myelofibrosis Blood Cancer Using a Combination Targeted Therapy
An international phase 3 clinical trial of a new drug combination for treating the rare blood cancer myelofibrosis found that adding a second, experimental drug to standard treatment was more effective than the standard treatment alone and did not significantly increase side effects.
This study looked at adding an experimental drug called pelabresib to the current treatment, the drug ruxolitinib (Jakafi®). Both are targeted therapies. Pelabresib blocks the action of proteins involved in inflammation and cancer; ruxolitinib blocks a protein called JAK. The combination-drug approach was based on ongoing research from the lab of MSK leukemia specialist and physician-scientist Ross Levine, MD.
MSK enrolled the most patients in the double-blind, placebo-controlled trial, led by MSK leukemia specialist Raajit Rampal, MD, PhD. The results were published March 10 in Nature Medicine.
Potential Treatment for Parkinson’s Using Investigational Cell Therapy Shows Early Promise
A stem cell–based therapy developed at MSK may lead to a new treatment for advanced Parkinson’s disease, according to results from a phase 1 clinical trial. The cell therapy was developed in the laboratories of Lorenz Studer, MD, Director of MSK’s Center for Stem Cell Biology, and Viviane Tabar, MD, Chair of MSK’s Department of Neurosurgery.
The treatment involved creating nerve cells (neurons) derived from embryonic stem cells and transplanting them into the brains of 12 Parkinson’s patients. The injected cells produce a chemical called dopamine, which coordinates movement. Parkinson’s patients have unusually low levels of dopamine, and as a result suffer from tremors, slowness, stiffness, and walking or balance problems.
After 18 months, the injected cells had taken hold in the brain with no serious side effects, reported the researchers. Notably, some patients’ symptoms appeared to stabilize or improve. Based on these preliminary results, published in Nature on April 16, the treatment will proceed directly to a phase 3 clinical trial in a larger patient group.
Using Only Immunotherapy Successfully Treats Several Cancers With MMRd Mutation, Improves Quality of Life
An approach pioneered at MSK that caused rectal cancer tumors with a specific genetic mutation to disappear in 100% of clinical trial participants has been expanded to treat other types of cancer, including stomach (gastric), colon, esophageal, urothelial, and others.
The new study saw nearly 80% of patients with several types of cancer successfully treated using only immunotherapy. Those who responded positively to treatment did not require surgery to remove an organ tumor and did not have to undergo chemotherapy or radiation, allowing them to maintain a better quality of life. The trial, which included 103 patients who had a tumor mutation called mismatch repair deficiency (MMRd), was led by MSK gastrointestinal oncologist Andrea Cercek, MD, and gastrointestinal oncologist Luiz Diaz Jr., MD.
The results were presented at the American Association of Cancer Research (AACR) Annual Meeting and simultaneously published in The New England Journal of Medicine on April 27.
Potential New Treatment for KRAS-GI2D Lung Cancer Reported in First-of-Its-Kind Clinical Trial
For the first time, a drug that takes aim at the cancer-causing mutation called KRAS-G12D is showing promise in a phase 1 trial for patients with non-small cell lung cancer (NSCLC). The drug is an experimental targeted therapy called zoldonrasib.
Analysis of the first 18 trial participants found that 11 of them (61%) had their tumors shrink substantially. Patients in the trial had previously received chemotherapy, immunotherapy, or both and had not responded.
The findings on zoldonrasib were presented at the AACR meeting on April 27 by MSK thoracic medical oncologist Kathryn Arbour, MD. The results were part of a larger trial led by MSK gastrointestinal medical oncologist and early drug development specialist Rona Yaeger, MD, which is studying zoldonrasib in a range of cancers caused by KRAS-G12D. Clinical research in all of these cancers is ongoing.
Immunotherapy After Surgery Helps Prevent Cancer Recurrence in MMRd Patients With ctDNA, MSK Study Shows
A small pilot study led by researchers at MSK has found that a simple blood test can help doctors identify which patients are most likely to benefit from immunotherapy after cancer surgery to remove tumors — potentially preventing the disease from coming back.
After surgery, the patients — whose tumors had mismatch repair deficiency (MMRd) — underwent a liquid biopsy to look for circulating tumor DNA (ctDNA) still present in the bloodstream. Those who tested positive were given pembrolizumab (Keytruda®), a type of immunotherapy.
Of those patients, 85% had complete clearance of ctDNA after treatment, 64% were free of recurrence two years later, and 92% were still alive at two years. The phase 1 results were presented by physician-scientist Yelena Janjigian, MD, on April 27 at the AACR meeting. There are plans to confirm these findings in a larger, randomized study.
New Drug Combination Improves Survival for ER+, HER2- Breast Cancer
A phase 3 clinical trial of patients with advanced breast cancer that was estrogen receptor (ER) positive but negative for a protein called HER2 has shown that a new three-drug combination enabled them to survive an average of 26% longer than patients who received standard therapy.
The treatment combined a new targeted therapy, inavolisib (Itovebi®), with two older breast cancer drugs — the hormone therapy fulvestrant (Faslodex®) and the targeted therapy palbociclib (Ibrance®). Inavolisib targets mutations in the gene PIK3CA, found in up to 40% of ER-positive, HER2-negative breast cancers. Adding inavolisib appears to be critical to making the three-drug therapy effective.
Medical oncologist and breast cancer specialist Komal Jhaveri, MD, helped lead the trial and was corresponding author of the study, which was presented May 31 at the annual meeting of the American Society of Clinical Oncology (ASCO) and published simultaneously in The New England Journal of Medicine.
New Imaging Technique Could Reduce Side Effects of Prostate Cancer Surgery
Surgery for prostate cancer can cause damage to surrounding nerves, risking side effects such as erectile dysfunction and incontinence. Now a new way to light up nerves during the operation may help surgeons avoid this nerve damage, preserving the patient’s quality of life.
The technology uses a fluorescent imaging agent called rizedisben (Illuminare-1), which binds to myelin, an insulating sheath around the nerves. When doctors shine a special blue light on the surgical area, the agent glows and increases the visibility of the nerves. A phase 1 clinical trial to study the agent was led by MSK urologic surgeon Timothy Donahue, MD, and suggests the nerve-illuminating agent is safe and effective.
When given to patients undergoing robotic-assisted surgery for prostate cancer, rizedisben created lasting illumination of important nerves in the pelvis for several hours, according to results reported in JAMA Surgery on July 2.
Controlling Weight Gain During Breast Cancer, Including New Research Into Ozempic and Related Weight-Loss Drugs
Most people experience weight gain following a breast cancer diagnosis, a focus of ongoing research by MSK breast medical oncologist Sherry Shen, MD.
Recently Dr. Shen led a team that analyzed the medical records of 75 breast cancer patients at MSK who received weight-loss drugs such as Ozempic and Wegovy. On average, patients lost around 5% of their body weight after a year, a finding that suggests these drugs could be beneficial for many breast cancer patients.
The research included a wide range of patients in terms of age, breast cancer type, stage of breast cancer, treatments given, and other variables. Pre- and post-menopausal women were included in the analysis, published in the journal Oncology on August 12. Dr. Shen and her colleagues plan to continue studying the effectiveness and implication of weight-loss drugs for this group of patients.
New Targeted Therapy, Ulixertinib, Shows Promise for Histiocytosis, a Rare Blood Cancer
Research from MSK found that a targeted drug called ulixertinib can benefit patients with histiocytosis. The first-ever trial of the drug found that in four out of five patients, their tumors shrunk substantially. This small study was led by MSK neuro-oncologist and early drug development specialist Eli Diamond, MD.
Histiocytosis is a collection of rare blood cancers that can affect patients of any age, including children, and can cause tumors in many parts of the body. Dr. Diamond, an internationally recognized leader in treating histiocytosis, published the study results October 23 in Cancer Cell. Based on these findings, ulixertinib is being tested in a bigger phase 2 clinical trial now open at MSK and two other hospitals.
CAR T Cell Therapy for Light Chain (AL) Amyloidosis Achieves Strong Results
Dramatic results from a phase 1 clinical trial show that a chimeric antigen receptor (CAR) T cell therapy appears to be effective at stopping AL amyloidosis in patients who have stopped responding to other treatments. Light chain (AL) amyloidosis is a rare and serious blood disease that develops when irregular immune plasma cells produce abnormal “light chain” proteins. These proteins misfold and accumulate in tissues and organs. When untreated, AL amyloidosis can cause organ failure and death.
Although therapies for AL amyloidosis have improved over the past decade, there have been no treatments approved for patients when the disease develops resistance or returns after other therapies fail.
According to the study — led by MSK cellular therapist Heather J. Landau, MD, the treatment appears to be safe and to work fast. In patients receiving the therapy, key biomarkers returned to normal levels within one to two weeks, and no lingering cancer cells could be detected. This research was presented at the 2025 American Society of Hematology Annual Meeting (ASH) on December 7.
Advancing the Science of Integrative Medicine
MSK is studying the potential benefits of various integrative medicine techniques, through a series of controlled clinical trials, which found:
- Participating in integrative therapy classes online could benefit cancer patients in active treatment, not only by reducing fatigue, anxiety, and depression but also by lowering hospitalization rates, according to a study led by integrative medicine specialist Jun Mao, MD, MSCE, and published in npg Digital Medicine on January 14. Read more.
- Music therapy for cancer-related anxiety is as effective as cognitive behavioral therapy and produces long-lasting benefits, according to a clinical trial led by integrative medicine specialist Kevin Liou, MD, who presented at the ASCO meeting on June 1. Read more.
- Acupuncture can alleviate cancer-related cognitive impairment — often called “brain fog” or “chemo-brain” — according to a study led by Dr. Mao and featured at the annual San Antonio Breast Cancer Symposium on December 12. Read more.
Updated Results From Ongoing Clinical Trials at MSK
In Early-Phase Pancreatic Cancer Clinical Trial, Investigational mRNA Vaccine Induces Sustained Immune Activity in Small Patient Group
A phase 1 clinical trial of an experimental approach for treating pancreatic cancer with a messenger RNA (mRNA) vaccine continues to show promise. The therapeutic cancer vaccine, autogene cevumeran, activated tumor-specific immune cells that persisted in the body up to nearly four years after treatment in some patients. In addition, patients with a vaccine-induced immune response had a reduced risk of the cancer coming back, in a trial led by MSK hepatopancreatobiliary surgeon Vinod Balachandran, MD, Director of MSK’s Olayan Center for Cancer Vaccines. The updated data was published February 19 in Nature. A phase 2 study is underway.
Vaccine Targeting KRAS in Pancreatic and Colorectal Cancer Shows Promise
The latest results from a phase 1 study of a new vaccine that targets tumors with KRAS mutations showed that the vaccine helps some patients live longer without their cancer returning. The clinical trial, co-led by MSK medical oncologist and pancreatic cancer specialist Eileen O’Reilly, MD, is studying ELI-002 2P, a potential “off-the-shelf” treatment for patients with pancreatic or colorectal cancer. Unlike individualized cancer vaccines, which are tailored to each patient, off-the-shelf vaccines can be mass-produced and stored for immediate use. Updated findings were published on August 11 in Nature Medicine.
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