What's new in use of gene therapy to treat autoimmune disease?

A while back, we wrote about the expansion of gene therapy, specifically CAR-T therapy, to treat lupus patients. Traditionally used to treat cancer, CAR-T therapy modified the genes of immune T-cells to attack cancerous cells.

Last year, a study was published on CAR-T's success in treating 5 lupus patients. This year, a new study reported success in adapting CAR-T therapy to treat patients with myasthenia gravis, an autoimmune disease that affects the nervous system. 14 participants with the disease received varying doses of the modified CAR-T therapy called Descartes-08. Out of these participants, 3 went into complete or near-complete remission, which continued for 6 months after the treatment. 2 other participants showed significant improvement such that they no longer required IVIG infusions.

Moreover, Descartes-08 is different from traditional CAR-T therapy in its target molecules. Traditional CAR-T modifies the DNA of T-cells, which is copied every time the cells divide and can lead to amplified and long-term side effects. Descartes-08, however, modifies the messenger RNA of the cells, which does not duplicate when the cells divide. Thus, this treatment requires multiple dosages over a period of time (whereas traditional CAR-T consisted of only 1 dose).

Researchers are now pursuing a larger-scale, randomized study for Descartes-08. If successful, this could offer a treatment alternative for autoimmune disease patients without the conventional risks associated with CAR-T therapy.

To support our work, please join our registry or share it with a friend who has an autoimmune condition. Donations are also greatly appreciated!