The University of Cincinnati Gardner Neuroscience Institute has joined an international Phase 1 clinical trial testing CAR-T cell therapy for multiple sclerosis (MS), marking a significant step forward in treating this debilitating autoimmune condition. The institution is among the first U.S. sites to participate in this groundbreaking study that could revolutionize MS treatment approaches.
Novel Approach to MS Treatment
MS occurs when the immune system mistakenly attacks the myelin that protects and covers nerve fibers, disrupting communication between the brain and the rest of the body and leading to a wide range of symptoms. The CAR-T cell therapy being tested represents a fundamentally different approach to addressing this immune dysfunction.
"CAR-T cell therapy begins by collecting a type of immune cell —T cells — from the patient's own blood," explained Dr. Aram Zabeti, the site's principal investigator, associate professor of neurology at UC's College of Medicine and director of the Waddell Center for MS. "These cells are then sent to a specialized lab, where they're genetically modified to recognize and eliminate the malfunctioning immune cells that drive MS. Once engineered, they are infused back into the patient."
Addressing Current Treatment Limitations
The innovative therapy aims to overcome significant limitations of existing MS treatments. Dr. Zabeti, who is also a UC Health physician, noted that current MS therapies primarily target inflammatory cells in the bloodstream and don't effectively reach the central nervous system, where much of the immune activity in MS occurs.
"Engineered CAR-T cells are designed to find and eliminate those harmful immune cells wherever they are: in the blood, the brain or other tissues," he said. This targeted approach could potentially provide more comprehensive treatment coverage than conventional therapies.
Broad Therapeutic Potential
The treatment shows promise for addressing multiple forms of the disease. "This treatment has the potential to help patients with all forms of MS, whether relapsing or progressive," Dr. Zabeti continued. The trial is structured to include both patient populations, reflecting the therapy's potential broad applicability.
Trial Progress and Patient Enrollment
The research team has already achieved an important milestone in patient recruitment. "We've already successfully screened our first patient in the progressive MS group, and we still have a few openings in both cohorts of the trial," Dr. Zabeti reported. This progress indicates the trial is actively enrolling participants across different MS subtypes.
The Phase 1 trial represents a critical early step in evaluating the safety and preliminary efficacy of this novel therapeutic approach, potentially paving the way for larger studies if initial results prove promising.
