Peter A. Merkel, MD, MPH, on Approaching Clinical Trial Design for CAR-T in Autoimmune Disease
The chief of the Division of Rheumatology and professor of medicine and professor of epidemiology at Penn Medicine discussed the panel he participated in at the inaugural Cell Therapy for Autoimmune Disease Summit.
“This probably needs to work moderately to extremely well for it to be worth it economically and in a risk-benefit standpoint. But I think there are real opportunities here to take our treatments to another level to help our patients. But caution is appropriate. The number of patients treated to date are few. We need to be careful, do this safely, and be self-critical as we go forward. These are a series of experiments.”
A significant unmet need exists for many patient populations with autoimmune diseases like lupus nephritis, as some patients in these groups are simply refractory to all currently available therapeutic options for their respective disease. In recent years, treating specialists in autoimmune disease and the investigator community have taken an interest in CD19-directed chimeric antigen receptor T-cell (CAR-T) therapies, which are currently only FDA-approved for hematological cancer indications, as a potential new option to address unmet needs in autoimmune disease. As such, many companies have begun development of CAR-T products for autoimmune disease indications, some of which have now entered early-stage clinical trials. Alongside this burgeoning area of interest, the first Cell Therapy for Autoimmune Disease Summit was held from November 28-30, 2023, in Philadelphia, Pennsylvania, to bring together investigators and industry leaders to discuss opportunities and challenges.
Peter A. Merkel, MD, MPH, the chief of the Division of Rheumatology and a professor of medicine and professor of epidemiology at Penn Medicine, spoke at one of the sessions held at the conference, a panel discussion entitled “Improving Clinical Endpoints & Optimizing Trial Design”. Following the session, CGTLive™ interviewed Merkel to learn more about his thoughts on how CAR-T should be evaluated for this therapeutic area. Merkel went over the key points discussed in the session and emphasized the need to balance potential benefit and potential risks during this clinical research.
Considerations for Administering Hemoglobinopathy Gene Therapies From a Pharmacist’s Perspective
April 20th 2024Alexis Kuhn, PharmD, BCOP, a pediatric oncology pharmacist at Mayo Clinic, discussed the incorporation of the recently FDA-approved gene therapies for SCD and TDT into the work of pharmacists.
