Does gene therapy offer a solution for autoimmune diseases?

With a better understanding of our genomes, scientists have increasingly looked to personalized medicine or gene therapy to target diseases. Recently, gene therapy was used to effectively treat 5 lupus patients in a groundbreaking study.

The technology used is called CAR T-cell therapy. T cells are our body's immune cells that help fight off infections and destroy cancer cells. Traditionally, CAR T-cell therapy has been used to fight cancer. A patient's blood is drawn to isolate the T cells, which are then genetically modified to attack the cancerous tumor cells. When the T cells are reinjected into the patient's body, the modified T cells have helped cancer patients achieve remission.

Recently, scientists used the same CAR T-cell technology to modify T cells to attack B cells, which create antibodies. In patients with autoimmune diseases like lupus, B cells incorrectly produce antibodies that signal the body to attack its cells. In this study, when patients were injected with the modified T cells, their B cells completely disappeared in a few days and the patient's symptoms improved.

But what is more incredible is that, after a few months, when B cells began to grow back, they did not produce the defective antibodies that previously led to lupus activity. The immune system had effectively reset itself, and the study patients continued to be in drug-free remission 8 to 12 months after the treatment. This opens up a whole new avenue for exploring treatments for autoimmune diseases. However, this type of personalized gene therapy is still very expensive today.

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