IASO Biotherapeutics (IASO Bio) is a biopharmaceutical company focused on the discovery, development, manufacturing, and commercialization of innovative cell therapies. In late October, officials announced that the journal Cell had published results from a study evaluating its independently developed fully human anti-BCMA CAR-T cell therapy, Equecabtagene Autoleucel (Eque-cel), for the treatment of progressive multiple sclerosis (PMS). CAR-T cell therapy is a form of immunotherapy.

“This landmark study provides the world's first clinical evidence confirming the safety and efficacy of a CAR-T therapy in PMS,” according to officials of IASO Bio. “It is also the first paper Cell has ever published on the successful application of BCMA-targeted CAR-T therapy for an autoimmune disease, representing a pivotal advancement in this field.”

The study, called Anti-BCMA CAR-T Cells in Progressive Multiple Sclerosis, was published in Volume 188, Issue 23 of Cell and was based on an investigator-initiated trial (NCT04561557) led by Professor Wei Wang's team from the Department of Neurology at Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology in Wuhan, China. The study enrolled five patients with PMS including one with primary progressive MS and four with secondary progressive MS, with a mean age at disease onset of 38.2 years and a baseline mean Expanded Disability Status Scale score of 6.2.

Following a single infusion of Eque-cel, all five patients demonstrated significant improvements in EDSS scores, Nine-Hole Peg Test , and Timed 25-Foot Walk tests. Complete disappearance of cerebrospinal fluid oligoclonal bands and significant decrease in kappa free light chain levels were observed. Follow-up MRI scans revealed no new T1 gadolinium-enhancing lesions or new/enlarging T2 lesions. In terms of safety, only transient grade 1 cytokine release syndrome occurred in 80% of patients, with no cases of ≥grade 2 CRS. No immune effector cell-associated neurotoxicity syndrome or other neurotoxic reactions were observed.

“We are delighted to see our team's research published in Cell, a significant milestone in the field of cell therapy,” according to the principal investigator of this study, Professor Wei Wang from Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology. “Our work has pioneered the application of anti-BCMA CAR-T therapy for multiple sclerosis and other refractory neuroimmune diseases, providing the world's first clinical validation of both its safety and efficacy in patients with progressive MS. Notably, 83% of the refractory patients achieved long-term clinical remission without requiring ongoing medication. This study not only challenges the existing treatment paradigm but also reveals novel regulatory mechanisms within the central nervous system immune microenvironment, paving new directions for immunotherapy in chronic neuroinflammatory diseases. Building on the remarkable efficacy of Eque-cel in clearing plasma cells and B cells, we are actively expanding the application of this therapy to a broader range of autoimmune diseases, including neuromyelitis optica spectrum disorders and myasthenia gravis, where we have already observed encouraging clinical outcomes. We are optimistic that this CAR-T therapy will offer a novel and powerful treatment option for more patients suffering from autoimmune diseases."

“Congratulations to Professor Wei Wang and his team as the principal investigators for this accomplishment,” said Jinhua Zhang, Founder, Chairwoman, and CEO of IASO Biotherapeutics, which became a Hacienda tenant in November 2024. “This achievement further highlights IASO Bio's pioneering innovation capabilities in the field of CAR-T cell therapy. Autoimmune diseases are the key therapeutic focus for IASO Bio. Currently, Eque-cel has obtained multiple clinical trial approvals for autoimmune indications in both China and the U.S., including multiple sclerosis, myasthenia gravis, systemic lupus erythematosus, and other autoimmune disorders. Moving forward, we remain committed to driving breakthroughs in autoimmune disease treatment through innovation, with the goal of delivering more advanced therapeutic options to patients worldwide.”

For more information about IASO Biotherapeutics, please visit www.iasobio.com.