Evaluating the effectiveness and safety of ciltacabtagene autoleucel for the treatment of patients with unresponsive multiple myeloma.

This study investigated the effectiveness and safety of ciltacabtagene autoleucel (cilta-cel), a chimeric antigen receptor (CAR) T-cell therapy for patients with previously treated relapsed or refractory (r/r) multiple myeloma (MM). The data showed that a single dose of cilta-cel was safe and effective for the treatment of these patients.

Multiple myeloma (MM) is a type of cancer that comes from blood cells called plasma cells. A high number of patients with MM experience relapse (the tumor grows after treatment) or are refractory (not responsive to the treatment) to standard treatment. Currently, the treatment strategies for r/r MM are based on the different combinations of conventional drugs and novel drugs.

CAR T-cell therapy is a type of immunotherapy that uses modified T cells to recognize and destroy cancer cells. Cilta-cel is a type of CAR T-cell immunotherapy in which a person’s own T-cells are collected from the blood, modified in a laboratory, and reintroduced to the body to fight cancer. The effectiveness and safety outcomes of cilta-cel therapy for patients with previously treated r/r MM are still unknown.

The study involved 97 patients with heavily pre-treated r/r MM. All patients received a single cilta-cel dose of 0.75 X 10⁶ CAR-T cells per kg. The average follow-up time was 12.4 months.

Overall, 97% of the patients responded to the treatment. The complete response rate (complete disappearance of cancer cells) was 67%. Patients achieved their first response within 1 month of administering cilta-cel treatment.

After 12 months, the overall survival rate was 89%. After 12 months, 77% of the patients were alive without cancer progression.

Of the 57 patients who were evaluated for minimal residual disease (MRD; any cancer cells remaining after treatment that can lead to relapse), 93% were MRD negative (no residual cancer cells).

All 97 patients experienced side effects. 95% of the patients experienced cytokine release syndrome (CRS). CRS is a side effect of cilta-cel caused by the immune system becoming highly active. CRS can cause fever, nausea, skin rash, an increase in heartbeat, low blood pressure, and trouble breathing. CRS resolved in all except 1 patient. 58% of patients experienced infections. 6 patients died due to side effects related to the treatment. 

This study concluded that a single dose of cilta-cel was effective with manageable toxicity for the treatment of patients with unresponsive MM.

The sample size of this study was small and the follow-up period was rather short. This study was sponsored by Janssen and Legend Biotech, the manufacturers of cilta-cel.