A groundbreaking study on CAR-T therapy for autoimmune disease has been published in the prestigious scientific journal Cell (journal), marking a major milestone in the field of immunotherapy. On October 16, 2025, IASO Biotherapeutics announced that its fully human anti-BCMA CAR-T therapy, Equecabtagene Autoleucel (Eque-cel), demonstrated promising results in treating progressive multiple sclerosis (PMS).
The study, titled “Anti-BCMA CAR-T Cells in Progressive Multiple Sclerosis,” reports the first global evidence demonstrating the safety and efficacy of CAR-T cell therapy for patients with progressive forms of Multiple Sclerosis. It is also the first time that a BCMA-targeted CAR-T therapy has been reported in Cell as a successful treatment for an autoimmune disease.
The trial enrolled five patients with progressive multiple sclerosis, including one patient with primary progressive MS (PPMS) and four patients with secondary progressive MS (SPMS). The average age of disease onset was 38.2 years, and the mean baseline Expanded Disability Status Scale (EDSS) score was 6.2.
Following a single infusion of Eque-cel, patients showed remarkable clinical improvements. All participants experienced significant improvement in EDSS scores, the Nine-Hole Peg Test (9-HPT), and the Timed 25-Foot Walk (T25FW). Additionally, cerebrospinal fluid oligoclonal bands (OCBs) completely disappeared, and Kappa free light chain (κFLC) levels declined significantly. Follow-up MRI scans showed no new or enlarged gadolinium-enhancing T1 lesions or T2 hyperintense lesions.
The treatment also demonstrated a favorable safety profile. Eighty percent of patients experienced only transient Grade 1 cytokine release syndrome (CRS), while no Grade 2 or higher CRS events were observed. Importantly, no cases of immune effector cell-associated neurotoxicity syndrome (ICANS) or other neurological toxicities were reported.
Professor Wang Wei, the principal investigator of the study, noted that the publication represents an important step forward in the development of cell therapy for neurological autoimmune diseases. According to Wang, the research team was the first to apply B-cell-targeted CAR-T therapy to multiple sclerosis and other refractory neuro-immune disorders, demonstrating for the first time the safety and effectiveness of this approach in progressive MS. Encouragingly, 83% of refractory patients achieved long-term clinical remission without the need for ongoing medication.
Wang added that the findings not only challenge current treatment paradigms but also reveal new mechanisms of immune regulation within the central nervous system microenvironment. This discovery could open new directions for immunotherapy targeting chronic neuroinflammatory diseases.
Autoimmune diseases remain a key focus area for the company. Clinical trials of Eque-cel for autoimmune indications have already been approved in both China and the United States, covering multiple sclerosis, myasthenia gravis, and systemic lupus erythematosus. The company plans to continue expanding its global development efforts and accelerate the clinical translation of innovative cell therapies to provide transformative treatment options for patients with autoimmune disorders.
About Multiple Sclerosis
Multiple Sclerosis is a chronic inflammatory disease of the central nervous system that leads to demyelination and neuronal damage. It is one of the most common causes of non-traumatic disability among young adults between the ages of 18 and 40.
According to analyses cited by Frost & Sullivan, there were approximately 3.07 million people living with multiple sclerosis worldwide in 2023, including around 400,000 in the United States and about 50,000 in China. The disease occurs significantly more often in women than in men, with an overall female-to-male ratio of approximately 3:1.
MS is characterized by infiltration of lymphocytes into the central nervous system, leading to destruction of the myelin sheath and axonal injury. These pathological changes cause neurological symptoms and progressive physical disability. Clinical manifestations vary depending on the location of lesions within the central nervous system and may include sensory disturbances, visual impairment, motor dysfunction, coordination problems, spasticity, fatigue, pain, and cognitive deficits.
Approximately 85% to 90% of patients initially experience a relapsing-remitting course, in which symptoms flare up and then partially recover. However, over time, incomplete recovery from relapses leads many patients to develop secondary progressive multiple sclerosis, characterized by gradual and irreversible accumulation of neurological disability.
About Equecabtagene Autoleucel (Eque-cel)
Equecabtagene Autoleucel (Eque-cel) is a CAR-T cell therapy targeting B-cell maturation antigen (BCMA). The therapy is produced by genetically modifying a patient’s own T cells using a lentiviral vector.
The CAR structure includes a fully human single-chain variable fragment (scFv), a CD8α hinge and transmembrane domain, the 4-1BB co-stimulatory domain, and a CD3ζ activation domain. Through rigorous molecular structure screening and extensive in-vitro and in-vivo functional evaluation, Eque-cel has demonstrated rapid and potent anti-disease activity along with durable persistence in the body, enabling deep and long-lasting clinical responses.
