Interim results are promising from on ongoing phase 1 clinical trial testing an allogeneic or “off-the-shelf” chimeric antigen receptor T cell (CAR-T) therapy for patients with multiple myeloma who have not responded to existing treatments or who have relapsed.
The therapy, P-BCMA-ALL01, demonstrated a 91% overall response rate for the 23 patients participating in the phase 1 trial. Vanderbilt-Ingram Cancer Center accrued the most participants of the study’s 15 sites. Bhagirathbhai Dholaria, MD, associate professor of Medicine, characterized the data as “remarkable” at the 21st International Myeloma Society Annual Meeting in Rio de Janeiro on Sept. 27. He shared the results of the trial, which enrolled patients who had been heavily pretreated with other therapies.
CAR-T treatment is a type of immunotherapy, but currently it is only available by autologous donation, in which a patient’s T cells are harvested, reengineered in a laboratory to attack cancer cells and then infused back into the patient’s body — an expensive and time-consuming process. An allogeneic option would use T cells from healthy donors and be available off-the-shelf so that a cancer patient could start treatment sooner. The Food and Drug Administration has not approved an allogeneic CAR-T therapy, but the agency has given the P-BCMA-ALL01 therapy regenerative medicine advanced therapy designation through the 21st Century Cures Act to accelerate development.
P-BCMA-ALL01 shows early promise of being an effective treatment for multiple myeloma patients who have no other options.
“The high overall response rate of 91% is remarkable because most study participants in my center had rapidly proliferative refractory disease in contrast with those treated in the pivotal clinical trials of FDA-approved autologous CAR-T therapies,” Dholaria said. “Such patients treated in the current trial of P-BCMA-ALL01 would not have qualified for standard of care autologous CAR-T therapy. All patients in the phase 1 trial have been treated quickly once enrolled with no waiting for manufacturing and with no need for apheresis or bridging therapy, demonstrating key advantages of allogeneic CAR-T cell therapy.”
The median time from enrollment in the study to the start of treatment was one day. Safety results reported in the interim data indicated no dose-limiting toxicities. No patients experienced graft-versus-host disease or movement disorders of the nervous system.
The trial data had diverse representation with 33% of participants from racial minority groups. Nearly half were 64 or older.
Recruitment continues for this ongoing trial. For more information about this study and other clinical trials at Vanderbilt-Ingram, visit https://www.vicc.org/clinical-trials or call 615-343-8010.
