The First Cell-based Immunotherapy for Multiple Myeloma 

The FDA approved the first CAR T-cell therapy to treat certain adult patients with multiple myeloma.  

The U.S. Food and Drug Administration (FDA) has approved idecabtagene vicleucel (Abecma) to treat adult patients with relapsed or refractory multiple myeloma. This is the first-ever cell-based gene therapy approved for multiple myeloma and is indicated for adult patients who have had four or more prior treatments that include an agent that modulates the immune response and one that blocks protein degradation as well as a monoclonal antibody designed to lock on to cancerous blood cells and enable the delivery of a substance to kill them. 

Idecabtagene vicleucel is a type of immunotherapy called CAR T-cell therapy. It involves extracting a patient’s T cells, which are a part of the body’s immune system, and then genetically modifying them in the lab and infusing them back into the patient.   

The efficacy of idecabtagene vicleucel was evaluated in 100 patients as part of a multicenter study. The overall response rate among these patients was 72 percent and the complete response rate was 28 percent. An estimated 65 percent of the patients who had a complete response continued to experience a complete response for at least 12 months.  

Idecabtagene vicleucel has the potential to induce a life-threatening inflammatory reaction or other toxic side effects. As such, the manufacturer is required to conduct a follow-up study on patients who receive the treatment. 

Multiple myeloma is a cancer of plasma cells, which is a type of white blood cell. Tumors form in the bone or soft tissues of the body. It is more common among Black Americans than any other racial or ethnic group in the country. According to federal statistics, it was estimated that there would be close to 35,000 new cases diagnosed in the U.S. in 2021.  

This FDA approval was rendered on March 26, 2021.