A revolutionary cancer treatment that could reverse autoimmune diseases

Causing a secondary cancer may be an acceptable risk when treating a life-threatening cancer, but perhaps not in an autoimmune setting, says Matt Lunning, MD, medical director of gene and cell therapy at Nebraska Medicine in Omaha. How to balance the risks between the effects of autoimmune diseases, the severity of which can range widely, and the difficult-to-measure risks of future side effects or cancers remains a major open question.

Researchers are already working on second- and third-generation versions of CAR T, which they expect to be safer for both cancer and autoimmunity. For example, James Howard, a neuromuscular neurologist at the University of North Carolina at Chapel Hill, is testing a technology from a company called Cartesian Therapeutics that encodes CAR using mRNA molecules, the short-lived genetic messenger used in COVID-19 vaccines, rather than long-lived DNA. CAR T cells only have to wipe out B cells as long as the mRNA lasts, and then they lose their abilities to target B cells. With no chance of long-term survival of the genetically modified T cells, there should be no risk of cancer.

Another advantage of the Cartesian approach: Doctors inject these T cells in sufficient numbers that they don’t need to multiply in the patient’s body, which Howard believes reduces the risk of inflammation. In a recent trial, 15 people with autoimmune diseases received CAR T therapy; Two-thirds of the participants saw an improvement in their symptoms, and none experienced serious long-term side effects.

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Aside from side effects, the other major challenge facing CAR T therapy is its price tag, which runs into the hundreds of thousands of dollars including hospital stays, cell engineering, and other expenses.

The treatment would likely be cheaper and simpler if scientists could eliminate the need to personally engineer each patient’s cells and use donor cells instead, or if they could cut out the step of engineering and growing the cells in a lab. Löning says he’s looking forward to upcoming procedures that would modify a person’s T cells inside their body rather than genetically engineering them in a lab.