Researchers at the Icahn School of Medicine at Mount Sinai have made a groundbreaking discovery in treating multiple myeloma, a blood cancer that often relapses or becomes resistant to initial treatments. A new study found that one-third of patients with relapsed or refractory multiple myeloma remained in remission for at least five years after receiving a single infusion of the CAR-T cell therapy cilta-cel.
The treatment, which has shown more durable responses in earlier follow-ups, targets cancer cells by reprogramming immune cells to recognize and attack them. In this study, researchers analyzed data from 97 patients who had undergone multiple prior lines of therapy and received a single cilta-cel infusion between July 2018 and October 2019.
The results showed that nearly all (31) of the patients achieved complete remission by year five or later, with many remaining progression-free for at least five years without any maintenance therapy. Patients who remained in remission had lower tumor burden, higher hemoglobin and platelet counts, and more favorable immune responses.
While safety concerns were minimal, two second primary malignancies and four grade 3 or higher infections were reported among the long-term responders. The study’s findings suggest that cilta-cel may offer a potentially curative treatment option for some patients with multiple myeloma.
Clinical trials are currently underway to assess cilta-cel in earlier lines of therapy, with the goal of expanding treatment-free survival to more patients. This breakthrough could bring new hope to patients struggling with this difficult-to-treat cancer.
Source: https://medicalxpress.com/news/2025-06-dose-car-therapy-potentially-curative.html