A groundbreaking international study, with participation from an Israeli hospital, has shown that a form of personalized immunotherapy may offer a potential cure for multiple myeloma, a deadly and previously incurable blood cancer.
Researchers presented the findings this week at the American Society of Clinical Oncology’s annual meeting in Chicago and published them simultaneously in the Journal of Clinical Oncology. After five years of follow-up, one-third of patients remained free of cancer following CAR-T cell therapy using the drug Carvykti, a treatment that re-engineers a patient’s own immune cells to target malignant cells.
The study included 97 patients with advanced-stage multiple myeloma who had exhausted nearly all standard treatment options. Most had undergone a median of six different therapies with little success before being enrolled in the clinical trial.
Prof. Yael Cohen, head of the multiple myeloma unit at Tel Aviv Sourasky Medical Center (Ichilov Hospital), said the therapy showed unprecedented results. “This is the first time a scientific article explicitly discusses the possibility of curing multiple myeloma,” she said. “These are patients who previously had almost no remaining treatment options.”
According to Cohen, about 98% of participants responded to the therapy, with half remaining disease-free after nearly three years and one-third still cancer-free five years later. In oncology, five-year remission is a benchmark often used to indicate a potential cure.
CAR-T therapy involves harvesting T cells from a patient’s immune system and modifying them in a lab to recognize and destroy cancer cells. The altered cells are multiplied and infused back into the patient. Once inside the body, they reactivate upon encountering cancer cells and begin destroying them.
“Seeing such durable responses in patients with aggressive, treatment-resistant myeloma is something we have never seen before,” Cohen said.
Multiple myeloma affects plasma cells in the bone marrow, where they multiply uncontrollably, weakening bones and increasing the risk of fractures. Roughly 550 new cases are diagnosed in Israel each year, and about 36,000 in the United States.
Dr. Peter Voorhees of Atrium Health Levine Cancer Institute in North Carolina, who led the study, said multiple myeloma typically becomes resistant to treatment over time. “This therapy appears to have eradicated every last cancer cell in some patients,” he said.
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The trial was funded by Johnson & Johnson, which partnered with China-based Legend Biotech, the company that developed the Carvykti therapy. Israeli medical centers, including Ichilov, participated in testing the treatment and reported similarly strong results among their patients.
While more data is needed, Cohen said the findings mark a major turning point. “Today, leading conferences on myeloma are openly discussing the possibility that some patients might be cured,” she said. “This brings enormous hope.”