Peter A. Merkel, MD, MPH, on the Challenges of Bringing Cell Therapy to Autoimmune Disease

This is the third part of an interview with Peter A. Merkel, MD, MPH. For the first part, click here.

“I think there’s an excitement, but a cautious optimism about this area. I think we are certainly hopeful that some forms of cell-based therapy will help some forms of autoimmune disease and hopefully substantially. We need to do this right, we need to move it forward, but I think we're very enthused. We need to see how this plays out and this will take some time.”

In late 2022 and throughout 2023, a number of companies gained FDA clearance for and/or launched clinical trials for chimeric antigen receptor T-cell (CAR-T) therapies and other cell therapy products in a myriad of autoimmune disease indications, including systemic lupus erythematosus, lupus nephritis, cutaneous systemic sclerosis, myasthenia gravis, and idiopathic inflammatory myopathy. Although CAR-T products now have a well-established safety and efficacy profile in oncology, suitable for patients with relapsed/refractory cancers, the risk-to-benefit ratio of CAR-T in autoimmune diseases is yet to be determined. As such, a large amount of clinical work remains to be done in order to bring these products to the field of autoimmune disease. The inaugural Cell Therapy for Autoimmune Disease Summit was held from November 28-30, 2023, in Philadelphia, Pennsylvania, with the intention of bringing together experts working on the early stages of this ongoing transition.

At the conference, CGTLive® spoke with Peter A. Merkel, MD, MPH, the chief of the Division of Rheumatology and a professor of medicine and professor of epidemiology at Penn Medicine, to learn about what he sees as the main challenges in bringing CAR-T and other cell therapy types to autoimmune disease. Merkel pointed out that because many of the aforementioned autoimmune diseases are rare, finding participants who meet criteria for clinical trials may be difficult, especially as multiple companies are launching CAR-T clinical trials for the same autoimmune disease indications. He also discussed other potential challenges such as unknown safety risks in these indications, the costliness of cell therapy products, and the difficulty of designing informative clinical trials.