An experimental therapy from Kyverna Therapeutics has yielded significant benefits for patients with crippling autoimmune disorders that affect the nerves and muscles, researchers say.
The treatment, a CAR T-cell therapy known as mivocabtagene autoleucel, or miv-cel, significantly improved walking ability and other goals of the late-stage trial in patients with Stiff-person Syndrome, with the benefits persisting one year after the treatment, Kyverna said.
Stiff-person Syndrome causes muscle rigidity and increased sensitivity to sound, touch and emotional stimuli that can trigger muscle spasms.
The company expects to present detailed data from the trial at the joint ACTRIMS-ECTRIMS meeting later this month in Toronto.
In 2022, singer Celine Dion announced that she suffers from the disease.
CAR T-cell therapies involve removal of a patient's immune system T cells, modifying and multiplying them in the lab to prime them to attack or address a specific condition before reinfusing them into the patient.
The first approved CAR T therapies were designed to treat blood cancers
The new trial results "are compelling, particularly given the severe burden of stiff-person syndrome and the absence of approved therapies," study leader Dr. Amanda Piquet, director and Celine Dion Foundation Endowed Chair in Autoimmune Neurology at the University of Colorado Anschutz School of Medicine, said in a statement.
“The sustained improvements observed in mobility, stiffness and other disease-specific measures, together with a well-tolerated profile, underscore its potential to deliver significant, long-lasting benefit to patients with Stiff-Person Syndrome," she said of the miv-cel results.
In a separate follow-up study of a mid-stage trial in patients with generalized myasthenia gravis, miv-cel significantly improved symptoms and ability to accomplish activities of daily living, the researchers reported at the recent American Association of Neuromuscular & Electrodiagnostic Medicine Annual Meeting in Orlando.
Myasthenia gravis causes fluctuating losses of strength in skeletal muscles.
A late-stage trial of miv-cel in patients with myasthenia gravis is underway, with enrollment expected to be completed in mid-2027, the researchers said.