T-cell therapy for relapsed/refractory multiple myeloma

This study aimed to investigate the safety of a T-cell therapy, bb2121, for patients with relapsed or unresponsive multiple myeloma.  

This study concluded that bb2121 shows antitumor activity in these patients.  

Early studies indicate that bb2121 has potential to treat relapsed or unresponsive multiple myeloma (MM). Bb2121 is a chimeric antigen receptor (CAR) T-cell therapy that targets B-cell maturation antigen (BCMA). BCMA allows cell survival and growth in cancer. By targeting BCMA, bb2121 has the potential to stop the growth and expansion of cancer cells. CAR T-cell therapy involves immune cells changes in the laboratory to attack and kill cancer cells.

It was not known how safe and effective bb2121 treatment would be for patients with relapsed or unresponsive MM.  

This study involved 33 patients with relapsed or unresponsive MM. These patients had received at least three previous treatments. Patients were treated with bb2121 infusions. Different doses were administered in the dose-escalation phase. Safety of the treatment was measured.  

The most common severe side effects were hematologic (blood-related) toxic effects. These included neutropenia (low level of neutrophils – white blood cells that fight off infections) in 85% of patients and leukopenia (low level of leukocytes – white blood cells) in 58% of patients. Other hematologic effects were anemia (low level of red blood cells) in 45% of patients and thrombocytopenia (low level of platelets – blood cells involved in clotting) in 45% of patients.  

76% of patients experienced cytokine release syndrome (symptoms include fever, pain, nausea, vomiting, and fatigue). Most of these had a mild case. 42% of patients experienced nerve-related side effects.  

The overall response rate was 85%. 45% of patients had a complete response. The average progression-free survival (survival without cancer getting worse – PFS) was 11.8 months.  

This study concluded that there are a series of side effects related to this treatment. It was also concluded that bb2121 shows promising antitumor activity in these patients. 

This was the first study of its kind. Further, larger studies are needed.