Niels van de Donk, MD, PhD, Hematologist at the VU University Medical Center in Amsterdam, discusses new results from Cohort B of the phase 2 CARTITUDE-2 study evaluating the efficacy and safety of ciltacabtagene autoleucel (cilta-cel) in patients with multiple myeloma who had early relapse after initial therapy. These data were recently presented at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting.

Multiple myeloma is a rare blood cancer associated with uncontrolled growth of plasma cells. Abnormal plasma cells – also known as myeloma cells – interfere with the production of healthy blood cells in the bone marrow. Myeloma cells also produce inactive clones of abnormal antibodies that may negatively affect the bones and kidneys. Symptoms of multiple myeloma may include: bone pain (particularly in the chest and spine), frequent infections, weakness or numbness in the legs, fatigue, confusion, excessive thirst, and constipation. While the disease is treatable, relapses are common and some patients are refractory to early therapies.

As Dr. van de Donk explains, safety and efficacy were assessed in the CARTITUDE-2 study and the primary endpoint was minimal residual disease (MRD) negativity at 105. As of January 2022, 19 multiple myeloma patients who experienced early relapse after front-line therapy received cilta-cel as part of Cohort B. Overall response rate (ORR) was 100.0%, 90% achieved complete response (CR) or better, and 95% achieved Very Good Partial Response (VGPR) or better. Median time to first response and best response were 0.95 months and 5.1 months, respectively. Of patients who were MRD-evaluable (n = 15), 14 (93%) achieved MRD 10-5 negativity during this study. Median duration of response was not reached and 12-month event-free rate was 88.9%. The 12-month progression-free survival rate was 90%. Median time to onset of cytokine release syndrome (CRS) was 8 days and occurred in 16 (84.2%) patients. CRS resolved in all patients. ICANS (grade 1) occurred in  one patient. Movement/neurocognitive adverse events (grade 3) occurred in one patient, which was previously reported. One patient died post cilta-cel due to Parkinson’s disease on day 158. 

Overall, these results indicate that a single cilta-cel infusion can lead to deep and durable responses in a functionally high-risk multiple myeloma patient population with a manageable safety profile. 

To learn more about multiple myeloma and other rare cancers, visit https://checkrare.com/diseases/cancers/