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NIH Record - National Institutes of Health

Potential Treatment for Rare Autoimmune Disorder Adapted from CAR-T Therapy

Evidence from a small-scale clinical trial suggests that a variation of the advanced blood cancer immunotherapy known as CAR-T could be adapted to treat myasthenia gravis, an autoimmune disorder of the nervous system. 

The modified CAR-T therapy, short for chimeric antigen receptor T-cell, used by scientists offers the potential for a longer-lasting reduction in myasthenia gravis symptoms and was well-tolerated without significant adverse effects. The study, published in The Lancet Neurology, was supported by a small business grant from NINDS and sponsored by Cartesian Therapeutics of Gaithersburg, Md.

Blue colorized image of a T cell

Colorized scanning electron micrograph of a T lymphocyte (also known as a T cell) (blue).

Photo: NIAID

“Repurposing a groundbreaking therapy such as CAR-T to potentially treat a neurological disorder shows the versatility of immunotherapies in instances where there are limited to no treatment options,” said Dr. Emily Caporello, director of the NINDS Small Business Program.

Myasthenia gravis is a chronic autoimmune disorder most often caused when the body’s immune system attacks a protein found where nerve cells communicate with muscles. The disease is marked by muscle weakness that worsens after periods of activity and can improve somewhat after rest. Current treatments focus on controlling symptoms, primarily muscle weakness.

In the study, 14 people with generalized myasthenia gravis received varying doses of a modified form of CAR-T therapy, known as Descartes-08, targeting the cells responsible for producing myasthenia gravis-causing antibodies. The ideal dosage was determined to be once weekly for six weeks. Early data on the effectiveness of the treatment are promising, but additional clinical studies are needed to evaluate the therapy’s efficacy.

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