The modified CAR-T therapy offers the potential for a longer-lasting reduction in MG symptoms and was well-tolerated without significant adverse effects, the National Institutes of Health (NIH) said.

The study, published in the Lancet Neurology, was supported by a small business grant from the National Institute of Neurological Disorders and Stroke (NINDS), a part of the NIH, and sponsored by Cartesian Therapeutics, Gaithersburg, MD.

"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," Emily Caporello, director of the NINDS Small Business Program, said in a statement.

MG 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.

Three patients receiving Descartes-08 showed complete or near-complete elimination of their symptoms, which continued six months following treatment. Two others no longer required chronic intravenous immunoglobulin treatment, which is currently used in some severe MG cases.

FDA approves Roche bispecific antibody for people with relapsed or refractory diffuse large B-cell lymphoma
The U.S. Food and Drug Administration (FDA) has approved Roche’s Columvi (glofitamab-gxbm), a bispecific antibody, for the treatment of adult patients...

Targeted erdafitinib therapy clinical trials achieve benefits across multiple cancer types
Three clinical trials demonstrated positive results from targeted erdafitinib therapy for patients with multiple tumor types harboring FGFR alterations....

Advanced lung cancer treatment can stop after two years: Study
A large, retrospective cohort study found that indefinite immunotherapy did not improve survival in patients with advanced lung cancer.

Single-cell RNA sequencing data reveals T cells linked to cancer immunotherapy resistance
Single-cell RNA sequencing data has revealed an immune signature associated with non-responses to checkpoint inhibitors that may be a driver of resistance...

Merck acquires Chord Therapeutics to expand neuroinflammatory pipeline
Merck has acquired Chord Therapeutics to expand its neurology pipeline and develop Chord's lead drug candidate, CRD1 (cladribine), for the treatment of...