Bruce Cree, MD, PhD, MAS, on Selecting the Right Neurologic Autoimmune Disease Patient Populations for CAR-T

This is the fourth part of an interview with Bruce Cree, MD, PhD, MAS. For the first part, click here.

“Don't target the patients that are doing well on [an approved] therapy or haven't been treated yet with the best possible [approved] therapy. The group to focus on—and this is just my opinion, of course—is the patients who have refractory disease for whom there really are no other therapeutic options. That's the unmet need. That's what you have to go after.”

When it comes to neurologic diseases that are known or thought to be autoimmune in nature, such as myasthenia gravis and multiple sclerosis, there are a range of patient experiences. Some patients are able to experience consistent benefit from available FDA-approved treatment options, but others have disease that is refractory to these treatments. Substantial unmet need remains for the latter group, and chimeric antigen receptor T-cell (CAR-T) therapy products, several of which are now in clinical development for neurologic autoimmune indications by various companies and institutions, may provide a new effective treatment option for these patients. On the other hand, because of its high cost and potential risks, CAR-T therapy is unlikely to be a good option for patients who are experiencing benefit from currently available treatments, or who have not tried all approved treatment options.

CGTLive® recently sat down with Bruce Cree, MD, PhD, MAS, a professor of neurology and the clinical research director of the University of California San Francisco (UCSF) Multiple Sclerosis Center, to ask about advice for colleagues considering CAR-T clinical trials as a potential option for their patients. Cree gave examples of various situations where CAR-T may be a good or not-so-good option for patients with specific neurologic autoimmune diseases, emphasizing that it could be a great choice in situations where patients are refractory to standard of care therapies for their disease and have no FDA-approved treatment options remaining. He also called attention to the importance of carrying out randomized controlled clinical trials for CAR-T therapies in autoimmune diseases to determine whether these therapies truly provide benefit in these indications.