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Modifying patients’ T-cells with mRNA to express a chimeric antigen receptor (rCAR-T therapy) a novel approach to treat myasthenia gravis and other autoimmune diseases.
Cartesian Therapeutics, a clinical-stage biotechnology company pioneering cell therapies for autoimmune diseases, announced today the publication of a landmark paper in The Lancet Neurology. The study describes Descartes-08, a cutting-edge RNA CAR-T (rCAR-T) therapy administered to patients with generalized myasthenia gravis (MG), a debilitating autoimmune neurological disease. The data demonstrate marked and long-lasting clinical improvement in patients with MG. This is the first clinical trial using rCAR-T to treat autoimmunity, and the first successful Phase 2 trial using an engineered cell therapy to treat autoimmunity.
“Conventional, DNA-engineered CAR T-cells are highly effective in treating several blood cancers, but associated toxicities and the need for lymphodepletion chemotherapy limit their use beyond oncology,” said Dr. Miloš Miljković, Chief Medical Officer at Cartesian. “To expand the capabilities of cell therapy to new areas and improve safety, we engineered CAR T-cells with RNA and used them to treat patients with MG.”
The study enrolled 14 patients with generalized MG. Patients received 6 infusions of rCAR-T therapy, administered as an outpatient treatment, without lymphodepletion. Patients were followed for a median duration of 6 months (range: 3–9 months). rCAR-T was well tolerated, with no dose-limiting toxicities, cytokine release syndrome, or neurotoxicity. Clinical improvements were marked and long-lasting across four validated MG disease scoring systems. Strikingly, at 6 months, the average improvements were about 3-fold greater than thresholds considered clinically meaningful. Clinical benefit was sustained long-term for most patients, even months after completing the course of therapy. Three patients achieved complete or near-complete eradication of all…
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