FDA approves the first CAR T-cell therapy for Multiple Myeloma

Multiple myeloma has been a notoriously difficult blood cancer to manage.  Relapses are common despite the advances that have been made in treatments over the years.  Idecabtagene vicleucel (Ide-cel) a new chimeric antigen receptor (CAR) T-cell therapy, has just been approved by the FDA for relapsed refractory multiple myeloma.  This is the first CAR T-cell therapy for myeloma and offers real hope to patients.

Ide-cel targets B-cell maturation antigen (BCMA) protein which is expressed on the majority cancer cells in myeloma.  Ide-cel was tested in a trial (KarMMa) enrolling 140 patients with relapsed and refractory myeloma1.  Patients had to have had at least three previous treatments including proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody.

Of the 140 patients enrolled, 128 received ide-cel treatment.  Seventy-three per cent of patients had a response to treatment at the medium follow-up of 13.3 months.  Thirty-three per cent had a complete response or better!  Twenty-six per cent of patients achieved minimal residual disease1.

KarMMa trial results for efficacy1

Outcome Per cent of patients
Response to treatment 73%
Complete response 33%
Achieved minimal residual disease 26%

As with other CAR T-cell therapies there are side effects to the treatment.  The common toxic side effects were neutropenia, anaemia and thrombocytopenia.  Cytokine release syndrome was also common occurring in 84 per cent of patients.

This is incredibly exciting news for myeloma patients.  The future is looking brighter.




  1. Munshi NC, Anderson LD Jr, Shah N, Madduri D, Berdeja J, Lonial S, Raje N, Lin Y, Siegel D, Oriol A, Moreau P, Yakoub-Agha I, Delforge M, Cavo M, Einsele H, Goldschmidt H, Weisel K, Rambaldi A, Reece D, Petrocca F, Massaro M, Connarn JN, Kaiser S, Patel P, Huang L, Campbell TB, Hege K, San-Miguel J. Idecabtagene Vicleucel in Relapsed and Refractory Multiple Myeloma. N Engl J Med. 2021 Feb 25;384(8):705-716. doi: 10.1056/NEJMoa2024850. PMID: 33626253.