In-body engineered CAR-T cells show promise for multiple sclerosis and other autoimmune disorders
A small trial found that a single injection of a virus delivering CAR-T instructions boosted patients' own immune cells and improved symptoms in multiple sclerosis and related autoimmune diseases.
In a phase-small clinical trial, sixteen individuals suffering from multiple sclerosis or other autoimmune illnesses were given a single intravenous dose of a lentivirus designed by Shenzhen Genocury Biotech. The virus carried genetic code for chimeric antigen receptors, prompting the patients' own T cells to become CAR-T cells that target autoantibody-producing B cells. Over a six-month follow-up, participants generated increasing numbers of these CAR-T cells, which depleted pathogenic B cells and lowered autoantibody levels, suggesting an immune system reset.
Clinical outcomes included improved motor and cognitive abilities, reduced fatigue for those with multiple sclerosis, and enhanced muscle strength and lower inflammation for other conditions. Experts such as David Simon and Bing Du highlighted the approach's speed and cost advantages over traditional ex-vivo CAR-T manufacturing, while lead investigator Dai-Shi Tian cautioned that efficacy is not yet definitively proven. The study, published in TheNewEnglandJournalofMedicine, calls for larger trials to confirm safety and lasting benefit.
Why it matters
It demonstrates a potentially faster, cheaper way to treat autoimmune diseases by reprogramming the immune system inside the body.
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