Breakthrough in Multiple Myeloma Treatment: Single-Dose CAR-T Therapy Shows Long-Term Promise

A groundbreaking multi-institutional study led by the Icahn School of Medicine at Mount Sinai has revealed that a single infusion of the CAR-T cell therapy cilta-cel can lead to prolonged remission in patients with relapsed or refractory multiple myeloma. Remarkably, about one-third of these patients remained in remission for at least five years without additional treatment. This represents one of the longest follow-ups for CAR-T therapy in multiple myeloma to date.

Multiple myeloma is a type of blood cancer that usually responds well to initial therapies but often relapses or develops resistance over time, especially in patients who have undergone multiple prior treatments. Historically, these patients have faced median progression-free survival of less than six months and overall survival close to one year.

The study analyzed data from 97 patients who had previously received at least three lines of therapy and were nearly all refractory to three classes of drugs. Each patient received a single dose of cilta-cel between July 2018 and October 2019 across various clinical sites. Over a median follow-up period of approximately five years, 33% of the patients remained alive and free of disease progression, with most achieving complete remission.

Additional assessments showed that patients in remission tended to have lower tumor burdens at baseline, higher counts of healthy blood cells, and more favorable immune cell profiles. Safety outcomes aligned with previous reports, with no new major adverse effects observed. Long-term responders experienced some secondary health issues, including a few malignancies and neurological events, but overall, the therapy demonstrated a manageable safety profile.

These findings suggest that cilta-cel has the potential to be a curative option for a subset of patients with advanced multiple myeloma. Current studies are exploring its effectiveness earlier in treatment lines to broaden the benefits of long-term remission.

This research underscores the promise of CAR-T therapies in transforming outcomes for patients with difficult-to-treat blood cancers and highlights the ongoing efforts to make such treatments more broadly available.