A potentially safer, simpler way to create CAR T cells in the body could bring this powerful therapy to the masses.

The immune system is one of our most powerful defenses, protecting us from infections, cancer, and other threats. But it’s a double-edged sword. Sometimes all that firepower is turned against healthy tissue. The resulting autoimmune diseases can be devastating. Take multiple sclerosis, an insidious disease that gradually eats away at the insulating coating around delicate nerve fibers, scrambling the electrical signals that control our bodies. The disease often strikes in young or middle adulthood, and there’s no cure.

Now a new clinical trial turns the immune system’s arsenal against the cells driving the autoimmune attack. Called CAR T cell therapy, the approach has already had success tackling previously untreatable blood cancers. Normally, CAR T cells are isolated and genetically engineered in specialized facilities. But in the new trial, with a single injection, researchers delivered a virus carrying genetic instructions to reprogram T cells in the body.

In 16 patients with autoimmune disorders affecting the nervous system, the treatment had few severe side effects. It also seemed to hit a kind of immune reset button. Follow-up tests suggested that the treatment restored parts of patients’ immune systems to normal, and there were no signs the friendly fire had returned. Across three different diseases, symptoms and molecular markers improved for over six months.

“These findings provide proof-of-concept that in vivo [in the body] CAR T-cell generation is associated with manageable side effects and may be effective for treating refractory neurologic autoimmune disorders,” wrote the team.

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