FDA Approves Second CAR T-Cell Therapy for Multiple Myeloma

Ciltacabtagene Autoleucel (Carvykti) Approved for Relapsed/Refractory Myeloma

If you’re an adult with multiple myeloma whose disease has progressed after multiple rounds of treatment, you may have a new option for therapy. The FDA recently approved a new chimeric antigen receptor (CAR) T-cell therapy. Ciltacabtagene autoleucel (Carvykti) is the second CAR T-cell therapy available for multiple myeloma following last year’s approval of idecabtagene vicleucel (Abecma).

The treatment is similar to other CAR T therapies. Ciltacabtagene autoleucel, which is also referred to as cilta-cel, involves genetically engineering patients’ T cells in a laboratory. The modified cells are then infused back into the patient’s blood where they will bind to and kill cancer cells.

The FDA based its approval on data from a study with 97 patients who had received a median of six prior treatment regimens. According to the data, 99% did not respond to their last line of treatment.

However, 98% of patients treated with cilta-cel responded to therapy, according to two-year follow-up data presented at the 2021 American Society of Hematology (ASH) Annual Meeting. With 18 months of follow-up, the median duration of response in patients was 21.8 months, according to the study.

“I think it brings a lot of hope to patients with myeloma,” said Gurbakhash Kaur, MD, an oncologist and assistant professor of internal medicine at the University of Texas Southwestern Medical Center in Dallas. She was not involved with the cilta-cel clinical trial.

Side Effects of Cilta-Cel

Like other CAR T therapies, cilta-cel comes with its share of side effects and risks.

Nearly 95% of patients enrolled in the trial experienced what is known as cytokine release syndrome, or CRS. CRS is an inflammatory that typically causes flu-like symptoms, including fever, difficulty breathing, and lowered blood pressure. These symptoms typically clear up within two weeks but can sometimes be severe.

Neurotoxicity was another side effect that affected one in five patients during clinical trials. The symptoms include confusion, headaches, delirium, trouble speaking, and other cognitive or memory problems.

When considering whether this or other CAR T therapy is right for you, you and your doctors will need to decide whether you are able to withstand these side effects. They’ll also have to determine whether your body has enough T cells to collect.

How Might This New Therapy Affect Patient Access to CAR T?

Accessing CAR T therapy has been a frequent point of concern for physicians and patients alike. Not all treatment centers offer it, which can create challenges for patients. Some patients may need to travel and remain close to their treatment center for weeks after the infusion, which can be a significant barrier to access.

Staying within close distance to the center that performed CAR T therapy is often necessary so your care team can monitor you after the procedure for 28 days. In some instances, they may need to intervene if you experience potentially serious side effects after the procedure.

While the approval means myeloma patients have a second CAR T therapy option, Dr. Kaur said it may only slightly improve barriers. “It’s still a therapy that’s offered at selective institutions,” she said. “It bridges the gap slightly, but I don’t think it bridges the gap completely.”