Severe disease, strict endpoints in CAR T trials 'could be a way through the regulators'
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SAN DIEGO — Regulatory trials of chimeric antigen receptor T-cell therapies for autoimmune diseases could be successful with a focus on patients with severe disease and strict endpoints, according to a speaker.
After reviewing his team’s recent successes in using CAR T-cell therapies for autoimmune diseases at the 2024 Congress of Clinical Rheumatology West, Georg Schett, MD, vice president of research and head of internal medicine at Friedrich-Alexander University Erlangen-Nürnberg, in Germany, was asked for his views on potential regulatory paths forward.
While noting multiple potential scenarios, Schett envisioned a design involving patients with particularly severe disease and a high bar for efficacy. He compared this to trials in hematology-oncology.
“You take actually very severe patients, and you have a one-arm study and have a stringent endpoint, which is drug-free remission,” Schett told attendees. “I think that would be a way through the regulators, and that would be a more hematology-oncology-driven way.”
That said, Schett acknowledged the challenges in setting up a comparison group for such a trial.
“I have never clearly understood how I would use a comparator arm in such patients, because you would put them at substantial risk for progression,” he said. “You would need a comparator arm as best-of-care — whatever you define best-of-care — with the risk being that the patient progresses. You would need to have an early escape possibility, potentially, to not harm the patient too much.”
Schett also noted that it would be challenging to recruit patients for such a study.
“My personal hope is that it will go in the hematology-oncology way and stay to actually very severe patients with a very stringent endpoint, because then the spontaneous resolution is very low,” he said. “I mean, these patients — I don’t think that they have a big placebo response, honestly.”
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Schett G. CAR T cell therapy in autoimmune disease. Presented at The Congress of Clinical Rheumatology West; Sept. 26-29, 2024. (hybrid meeting).
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