Amy Speck recovered quickly after minimally invasive, robotic surgery to remove a large noncancerous tumor on her spine. The surgical technique was developed at MSK.
Doctors at Memorial Sloan Kettering Cancer Center (MSK) pioneered advances in a variety of cancer treatments in 2024 — including new surgical techniques, innovative cancer vaccines, drugs to boost radiation therapy’s effectiveness, and novel tools to diagnose and detect cancer.
Additionally, the U.S. Food and Drug Administration (FDA) approved 11 drugs in 2024 based on clinical trials in which MSK played a pivotal role. These approvals span new uses for existing drugs, more effective drug combinations, engineered cell therapies, and novel classes of targeted therapies.
“Clinical advances made by MSK researchers this year will benefit patients afflicted with a range of cancers, from some of the most common to the extremely rare,” said MSK President and CEO Selwyn M. Vickers, MD, FACS. “Additionally, our faculty continued to demonstrate their leadership in shepherding investigational drugs through clinical trials — resulting in nearly a dozen new FDA approvals.”
Here are some of the most important developments of 2024, listed in chronological order.
Vaccine Targeting KRAS in Pancreatic and Colorectal Cancer Shows Promise
A new vaccine shows encouraging early results as a potential off-the-shelf treatment for certain patients with pancreatic or colorectal cancer, according to a study co-led by medical oncologist and pancreatic cancer specialist Eileen O’Reilly, MD. The vaccine targets tumors with mutations in the KRAS gene, a driving force in many cancers. The phase 1 trial demonstrated this vaccine is safe and appears to stimulate the patient’s immune system to create cancer-fighting cells. The results were published in Nature Medicine on January 9, 2024.
Advanced Basal Cell Carcinoma Responds to Combination Drug-Radiation Treatment
Basal cell carcinoma, the most common form of skin cancer, can usually be removed with surgery alone. But in rare cases, the tumor may be too large to remove or in a difficult place in the body. For those patients, researchers led by radiation oncologist Christopher Barker, MD, report that combining radiation therapy with a drug called vismodegib (Erivedge®) may control the disease up to 50% longer than radiation alone. The study also suggests that vismodegib sensitizes the cancer cells to radiation and helps patients avoid the long-term side effects associated with continuous drug treatment. Results from a phase 2 clinical trial were published April 17, 2024, in the Journal of Clinical Oncology.
A Potential New Test for Lung Cancer: The Noninvasive E-Nose
Detecting lung cancer as early as possible improves outcomes for patients, but only 1 in 5 patients who should be screened with a low-dose CT scan actually are. A team led by MSK thoracic surgeon Gaetano Rocco, MD, conducted a phase 2 clinical trial to test a screening method known as “E-nose,” which uses technology that can “smell” lung cancer in a person’s breath. The trial found E-nose reliably detected early-stage lung cancer and was highly accurate when compared with more invasive imaging methods. The results were published online in the Journal of Thoracic Oncology in May 2024.
Improving Transplant Success in Patients Without Fully Matched Blood Stem Cell Donors
For blood cancer patients requiring donor cells for stem cell and bone marrow transplants (BMTs), it can be hard to find an exact match. MSK bone marrow transplant specialist and cellular therapist Brian Shaffer, MD, led a team that demonstrated how to improve transplants in patients who don’t have fully matched donors. They found that patients with partially matched donors who received chemotherapy after their BMT had better outcomes than those who received other antirejection drugs. An ongoing clinical trial at MSK is now looking at whether lower doses of chemotherapy are as effective as higher doses at preventing complications after transplant. The results were published July 17, 2024, in the Journal of Clinical Oncology.
Can Exercise Impact Prostate Cancer? A First-of-Its-Kind Clinical Trial
A clinical trial led by MSK exercise scientist Lee Jones, PhD, suggested that specific amounts of endurance exercise before surgery among patients with early-stage prostate cancer can meaningfully improve two key biomarkers associated with better outcomes. The investigators set out to rigorously test controlled amounts of exercise therapy in the same way drugs are tested — with a specific duration, number of times per week, and intensity. The study found that these men were able to consistently complete high levels of exercise therapy and that no serious adverse events were observed. The results of the study were published in JAMA Oncology on July 18, 2024. Based on the findings, a phase 2 clinical trial has already begun to examine how exercise impacts the progression of prostate cancer.
New Imaging Detects Deadly Lung and Prostate Cancers, May Improve Treatment
A new imaging technique developed at MSK shows promise for detecting deadly forms of lung cancer and prostate cancer. The technology uses a radioactive particle that binds to a ligand (molecule) on cancer cells called DLL3, making the cells more visible on PET scans. Detecting these cancers would help identify patients who might respond to a new class of drugs that target DLL3. The imaging agent, called [89Zr]Zr-DFO-SC16.56, was developed in the laboratories of MSK radiochemist Jason S. Lewis, PhD, and physician-scientist Charles M. Rudin, MD, PhD. Results from the first study were published in the August 2024 issue of The Lancet Oncology.
TIL Therapy for Non-Small Cell Lung Cancer May Be Effective
A phase 2 clinical trial of a new type of immunotherapy called TIL therapy found this approach works in some patients with non-small cell lung cancer. The treatment involves removing a specialized type of white blood cell called a tumor-infiltrating lymphocyte (TIL, pronounced “till”) from a patient’s tumor, growing the cells into large numbers in a lab, and then putting them back into the patient so they can seek out and destroy cancer cells. The trial, led by MSK lung cancer specialist Adam Schoenfeld, MD, found the therapy substantially shrank tumors in about one-quarter of patients who had exhausted other treatment options. The results were published in Cancer Discovery on August 2, 2024.
Robotic Neurosurgery at MSK Speeds Recovery From Some Spinal Tumors
Minimally invasive, robotic surgery has been used routinely by MSK surgeons since 2007 to remove tumors from patients’ chest and abdominal cavities. Now MSK spine neurosurgeon Ori Barzilai, MD, is expanding the reach of this tool by using it to remove noncancerous spinal tumors, which are usually removed with open techniques that require large incisions. When surgeons are able to use a robotic device to make small incisions, patients recover much faster. While the approach is still investigational, the procedure appears to be safe and effective. Dr. Barzilai and his colleagues reported outcomes from the first 19 patients to have this type of surgery in the journal Operative Neurosurgery on August 20, 2024.
Avoiding Surgery for HPV Throat Cancer May Be Possible for More Patients
An innovative radiation treatment that is typically given to patients after surgery for HPV-positive head and neck cancers may now help some of those patients avoid surgery altogether. The approach, pioneered by MSK radiation oncologist Nancy Lee, MD, significantly reduces the overall radiation dose to minimize side effects. The results from a phase 2 clinical trial were presented by Dr. Lee at the annual meeting of the American Society of Clinical Oncology. MSK has now opened a randomized phase 3 clinical trial testing the low-dose technique.
Imlunestrant Stops Progression of ER+, HER2- Advanced Breast Cancer That Resists Standard Therapy
An experimental drug stops advanced breast cancer from progressing in some people with tumors that have grown resistant to standard therapy. The drug, imlunestrant, was especially effective when combined with a second drug, abemaciclib (Verzenio®). An international phase 3 clinical trial found that the combination reduced the risk of cancer progression or death by more than 40% compared with imlunestrant alone. The results will change the way many breast cancers are treated, says MSK medical oncologist Komal Jhaveri, MD, FACP, chair of the study. The findings were presented at the San Antonio Breast Cancer Symposium and published simultaneously in the New England Journal of Medicine on December 11, 2024.
New Results From Ongoing Clinical Trials at MSK
Investigational mRNA Vaccine Induced Persistent Immune Response in Phase 1 Trial of Patients With Pancreatic Cancer
An experimental approach to treating pancreatic cancer with the messenger RNA (mRNA)-based therapeutic cancer vaccine candidate autogene cevumeran continues to show potential to stimulate an immune response that may reduce the risk of the disease returning after surgery. The latest results from a phase 1 clinical trial showed that the treatment activated immune cells that persisted in the body up to three years after treatment in certain patients. In addition, a vaccine-induced immune response correlated with reduced risk of the cancer coming back. The findings were presented by pancreatic cancer surgeon-scientist Vinod Balachandran, MD, at the American Association for Cancer Research Annual Meeting on April 7, 2024. A phase 2 clinical trial, sponsored by Genentech in collaboration with BioNTech, will evaluate the efficacy and safety of autogene cevumeran in a larger patient group.
Rectal Cancer Disappears After Experimental Use of Immunotherapy
A groundbreaking therapy developed at MSK for patients whose rectal cancer carries certain genetic mutations continues to show remarkable results. The clinical trial, which now includes 42 patients, uses immunotherapy alone to treat rectal cancer. Those eligible for the trial had tumors with a specific genetic makeup known as mismatch repair-deficient (MMRd) or microsatellite instability (MSI). After being treated with the checkpoint inhibitor dostarlimab (Jemperli), everyone in the trial saw their tumors disappear and avoided surgery. MSK gastrointestinal oncologist Andrea Cercek, MD, who led the clinical trial with gastrointestinal oncologist Luis Diaz, MD, reported the latest results at the American Society of Clinical Oncology annual meeting on June 3, 2024. Based on the trial results, dostarlimab received Breakthrough Therapy Designation from the FDA for rectal cancer on December 16, 2024.
The FDA Approves 11 Drugs in 2024 Based on Significant Contributions From MSK Researchers
(Note: The FDA uses the term “accelerated approval” when granting approval for drugs that treat diseases lacking effective treatments. These criteria are slightly different than for a standard approval. Also, the term “full approval” applies to drugs that had previously been granted “conditional approval.”)
These approvals of therapies for a range of blood cancers and solid tumors are listed in chronological order.
- For metastatic non-small cell lung cancer caused by a genetic error called METex14, the FDA granted approval to the targeted therapy tepotinib (Tepmetko®) on February 15, 2024. Tepotinib was approved based on the findings from a multicenter international phase 2 clinical trial known as VISION. MSK thoracic oncologist Paul Paik, MD, Clinical Director of MSK’s Thoracic Oncology Service, was lead author of the study.
- For a rare blood cancer called mantle cell lymphoma, the FDA approved the CAR T treatment lisocabtagene maraleucel (Breyanzi®). The decision on May 30, 2024, was based on results from a clinical trial led by MSK hematologist-oncologist Lia Palomba, MD.
- For treating adult and pediatric patients with advanced or metastatic thyroid cancers with RET mutations, the FDA granted full approval to the targeted therapy selpercatinib (Retevmo®) on June 12, 2024. MSK head and neck medical oncologist Eric Sherman, MD, was one of the leaders of the clinical trial studying selpercatinib for thyroid cancer.
- For treating cancers anywhere in the body caused by gene changes called NTRK fusions, a targeted drug called repotrectinib (AugtyroTM) received accelerated approval from the FDA on June 13, 2024. The FDA action was based on a global clinical trial led by early drug development specialist Alexander Drilon, MD, Chief of MSK’s Early Drug Development Service.
- For people with advanced colorectal cancer caused by a gene mutation called KRAS-G12C, the FDA granted accelerated approval for the targeted therapy adagrasib (Krazati®) in combination with cetuximab (Erbitux®) on June 21, 2024. The trial that resulted in the approval was led by MSK gastrointestinal medical oncologist and early drug development specialist Rona Yaeger, MD.
- For the treatment of adults with a rare soft tissue cancer called synovial sarcoma, the FDA granted accelerated approval for the immunotherapy afamitresgene autoleucel (Tecelra®) on August 2, 2024. The clinical trial that resulted in the drug’s approval was led by MSK sarcoma specialist and immunotherapy expert Sandra D’Angelo, MD.
- For brain tumors called low-grade diffuse gliomas that carry an IDH gene mutation, the FDA approved the drug vorasidenib (Voranigo®) on August 6, 2024. MSK’s Ingo Mellinghoff, MD, FACP, Chair of the Department of Neurology, led the clinical trial demonstrating the drug’s potential.
- For certain breast cancers, the FDA approved a new three-drug combination. The treatment combines a new targeted drug, inavolisib (Itovebi®), with two older drugs, fulvestrant (Faslodex®) and palbociclib (Ibrance®). The approval on October 10, 2024, was based on a trial co-led by MSK medical oncologist and breast cancer specialist Komal Jhaveri, MD.
- For children and adults with leukemia that has come back after treatment and carries a molecular change called a KMT2A translocation, the FDA approved the drug revumenib (Revuforj®). The decision on November 15, 2024, was based on results of a clinical trial led by Eytan Stein, MD, Chief of the Leukemia Service at MSK.
- For treating advanced and metastatic bile duct cancer (cholangiocarcinoma) and other biliary tract cancers with high levels of HER2, the FDA granted accelerated approval to zanidatamab (Ziihera®). The approval on November 20, 2024, was based on a trial co-led by MSK gastrointestinal medical oncologist and early drug development specialist James J. Harding, MD.
- For treating patients with advanced pancreatic cancer or non-small cell lung cancer that has an alteration called an NRG1 gene fusion, the FDA granted accelerated approval to zenocutuzumab (Bizengri®). The December 4, 2024, decision was based on a phase 2 clinical trial led by MSK early drug development specialist and gynecologic medical oncologist Alison Schram, MD.