First use of CAR-T therapy in patient with stiff-person syndrome

CAR-T therapy

KYV-101, a fully human anti-CD19 chimeric antigen receptor (CAR) T-cell product candidate, has been used to treat a 69-year-old patient suffering from treatment-refractory stiff-person syndrome (SPS).

The therapy was given as part of a named-patient use in Germany for critically ill individuals who fail conventional therapies.

SPS is a rare, progressive neurological autoimmune disorder causing debilitating muscle stiffness in the torso, arms, and legs, impacting the ability to walk or move.

The treatment was well tolerated and led to a significant improvement in walking distance and 40% reduction in GABAergic medications.

“It is extremely encouraging to see this patient improving the self-reported, uninterrupted walking distance from less than 50 meters to several kilometres within three months after treatment,” said Simon Faissner, Professor for Translational Neuroimmunology at the Department of Neurology, Ruhr University Bochum, St Josef Hospital, in Germany, and lead co-author.

“These dramatic improvements – if confirmed by further studies – may eventually provide renewed hope for a much-needed paradigm shift in the treatment of debilitating autoimmune diseases.”

KYV-101 is a product candidate for use in B cell-driven autoimmune diseases. It is currently also being evaluated in Phase I/II and Phase II trials across two broad areas of autoimmune disease: rheumatology and neurology.

“On the heels of recent case reports of the use of KYV-101 in multiple sclerosis and myasthenia gravis, we are excited to see positive outcomes of KYV-101 in a patient suffering from SPS,” said Peter Maag, Chief Executive Officer of Kyverna. “These data underscore the dedication of the Kyverna village to patient care and scientific advancement.”

In further news, the US Food and Drug Administration (FDA) has now cleared Kyverna’s Investigational New Drug (IND) application to evaluate KYV-101 in patients with treatment-refractory SPS in the Phase II KYSA-8 clinical trial.

Diana Spencer, Senior Digital Content Editor, DDW

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