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MSK Researchers Spearhead Advances in Cancer Treatment, Leading to 11 FDA Approvals in 2024

• Memorial Sloan Kettering Cancer Center (MSK) made significant strides in cancer treatment in 2024, including novel vaccines, drugs enhancing radiation, and advanced diagnostics. • A vaccine targeting KRAS mutations in pancreatic and colorectal cancers showed promising early results in stimulating the immune system. • Imlunestrant, combined with abemaciclib, demonstrated a significant reduction in cancer progression or death in ER+, HER2- advanced breast cancer patients resistant to standard therapy. • MSK played a pivotal role in clinical trials leading to 11 FDA approvals in 2024, spanning new uses for existing drugs, effective drug combinations, and novel targeted therapies.

Memorial Sloan Kettering Cancer Center (MSK) researchers have spearheaded numerous advances in cancer treatment throughout 2024, resulting in 11 FDA approvals and innovative approaches to combatting a range of malignancies. These breakthroughs span from novel surgical techniques and cancer vaccines to drugs designed to boost the effectiveness of radiation therapy and advanced diagnostic tools.

Vaccine Targeting KRAS Mutations Shows Promise

Early results from a phase 1 trial of a new vaccine targeting tumors with mutations in the KRAS gene, a key driver in pancreatic and colorectal cancers, have been encouraging. The study, co-led by medical oncologist Eileen O’Reilly, MD, demonstrated the vaccine's safety and its ability to stimulate the patient’s immune system to create cancer-fighting cells. The findings were published in Nature Medicine, marking a potential off-the-shelf treatment option for patients with these specific mutations.

Combination Drug-Radiation Treatment for Advanced Basal Cell Carcinoma

For patients with advanced basal cell carcinoma, a phase 2 clinical trial led by radiation oncologist Christopher Barker, MD, revealed that combining radiation therapy with vismodegib (Erivedge®) can control the disease up to 50% longer than radiation alone. The study, published in the Journal of Clinical Oncology, suggests that vismodegib sensitizes cancer cells to radiation, potentially helping patients avoid long-term side effects associated with continuous drug treatment.

E-Nose: A Potential New Test for Lung Cancer

A phase 2 clinical trial led by thoracic surgeon Gaetano Rocco, MD, explored the use of an “E-nose,” a noninvasive screening method that can “smell” lung cancer in a person’s breath. The trial found that the E-nose reliably detected early-stage lung cancer with high accuracy compared to more invasive imaging methods. These results, published in the Journal of Thoracic Oncology, highlight the potential for improved early detection of lung cancer.

Improving Transplant Success with Partially Matched Donors

Brian Shaffer, MD, led a team that demonstrated improved outcomes in blood cancer patients receiving stem cell and bone marrow transplants (BMTs) from partially matched donors. The study, published in the Journal of Clinical Oncology, found that patients who received chemotherapy after their BMT had better outcomes than those treated with other antirejection drugs. An ongoing clinical trial at MSK is investigating whether lower doses of chemotherapy are as effective as higher doses in preventing post-transplant complications.

Exercise and Prostate Cancer: A Clinical Trial

A clinical trial led by exercise scientist Lee Jones, PhD, indicated that specific amounts of endurance exercise before surgery among patients with early-stage prostate cancer can improve key biomarkers associated with better outcomes. Published in JAMA Oncology, the study rigorously tested controlled amounts of exercise therapy, demonstrating that men could consistently complete high levels of exercise therapy without serious adverse events. A phase 2 clinical trial is now underway to examine how exercise impacts the progression of prostate cancer.

Novel Imaging for Lung and Prostate Cancers

A new imaging technique developed at MSK shows promise for detecting aggressive forms of lung and prostate cancer. The technology, which uses a radioactive particle that binds to DLL3 on cancer cells, making them more visible on PET scans, was developed in the laboratories of radiochemist Jason S. Lewis, PhD, and physician-scientist Charles M. Rudin, MD, PhD. The results from the first study were published in The Lancet Oncology.

TIL Therapy for Non-Small Cell Lung Cancer

A phase 2 clinical trial of TIL therapy, led by lung cancer specialist Adam Schoenfeld, MD, found that this immunotherapy approach is effective in some patients with non-small cell lung cancer. The trial, published in Cancer Discovery, showed that TIL therapy substantially shrank tumors in about one-quarter of patients who had exhausted other treatment options.

Robotic Neurosurgery for Spinal Tumors

Spine neurosurgeon Ori Barzilai, MD, is expanding the use of minimally invasive, robotic surgery to remove noncancerous spinal tumors. Outcomes from the first 19 patients to have this type of surgery were reported in Operative Neurosurgery, demonstrating that the procedure is safe and effective, leading to faster patient recovery.

Avoiding Surgery for HPV Throat Cancer

An innovative radiation treatment pioneered by radiation oncologist Nancy Lee, MD, may help some patients with HPV-positive head and neck cancers avoid surgery altogether. Results from a phase 2 clinical trial were presented at the American Society of Clinical Oncology annual meeting. MSK has now opened a randomized phase 3 clinical trial testing the low-dose technique.

Imlunestrant for Advanced Breast Cancer

An international phase 3 clinical trial found that the combination of imlunestrant and abemaciclib (Verzenio®) reduced the risk of cancer progression or death by more than 40% compared with imlunestrant alone in patients with ER+, HER2- advanced breast cancer resistant to standard therapy. The findings, presented at the San Antonio Breast Cancer Symposium and published in the New England Journal of Medicine, are expected to change the way many breast cancers are treated.

mRNA Vaccine for Pancreatic Cancer

Vinod Balachandran, MD, presented the latest results from a phase 1 clinical trial of the mRNA-based therapeutic cancer vaccine candidate autogene cevumeran at the American Association for Cancer Research Annual Meeting. The results showed that the treatment activated immune cells that persisted in the body up to three years after treatment in certain patients, and a vaccine-induced immune response correlated with reduced risk of cancer recurrence. A phase 2 clinical trial will evaluate the efficacy and safety of autogene cevumeran in a larger patient group.

Dostarlimab for Rectal Cancer

Andrea Cercek, MD, and Luis Diaz, MD, reported the latest results from a clinical trial using immunotherapy alone to treat rectal cancer with mismatch repair-deficient (MMRd) or microsatellite instability (MSI) at the American Society of Clinical Oncology annual meeting. After being treated with the checkpoint inhibitor dostarlimab (Jemperli), everyone in the trial saw their tumors disappear and avoided surgery. Based on the trial results, dostarlimab received Breakthrough Therapy Designation from the FDA for rectal cancer.

FDA Approvals Based on MSK Research

In 2024, the FDA approved 11 drugs based on significant contributions from MSK researchers. These approvals include:
  • Tepotinib (Tepmetko®) for metastatic non-small cell lung cancer caused by METex14 (February 15, 2024).
  • Lisocabtagene maraleucel (Breyanzi®) for mantle cell lymphoma (May 30, 2024).
  • Selpercatinib (Retevmo®) for thyroid cancers with RET mutations (June 12, 2024).
  • Repotrectinib (AugtyroTM) for cancers with NTRK fusions (June 13, 2024).
  • Adagrasib (Krazati®) in combination with cetuximab (Erbitux®) for colorectal cancer caused by KRAS-G12C (June 21, 2024).
  • Afamitresgene autoleucel (Tecelra®) for synovial sarcoma (August 2, 2024).
  • Vorasidenib (Voranigo®) for low-grade diffuse gliomas with an IDH gene mutation (August 6, 2024).
  • Inavolisib (Itovebi®) with fulvestrant (Faslodex®) and palbociclib (Ibrance®) for certain breast cancers (October 10, 2024).
  • Revumenib (Revuforj®) for leukemia with a KMT2A translocation (November 15, 2024).
  • Zanidatamab (Ziihera®) for bile duct cancer (cholangiocarcinoma) and other biliary tract cancers with high levels of HER2 (November 20, 2024).
  • Zenocutuzumab (Bizengri®) for pancreatic cancer or non-small cell lung cancer with an NRG1 gene fusion (December 4, 2024).
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[1]
Top Cancer Treatment Advances at MSK in 2024
mskcc.org · Dec 17, 2024

MSKCC advanced cancer treatments in 2024 include new surgical techniques, vaccines, drugs enhancing radiation, and diagn...

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