CAR T Cell Therapy for Autoimmune Blistering Disease | Research at Penn

Penn Dermatologist Aimee S. Payne, MD, PhD, and her research team at the Perelman School of Medicine at the University of Pennsylvania discuss their innovative investigational approach to engineering chimeric antigen receptor (CAR) T cells to treat pemphigus -- rare, potentially deadly autoimmune blistering disease that occurs when the immune system mistakenly attacks desmoglein.

“Using desmoglein, the target of immune attack, and pemphigus as a decoy, we genetically engineered chimeric autoantibody receptor T cells that lure in and kill only the disease-causing cells,” explains Christoph Ellebrecht, MD, University of Pennsylvania postdoctoral fellow explains. “Desmoglein CAR T cells eliminated disease in a pemphigus mouse model without any detectable collateral damage to other tissues.”

“Our past success in inducing long-lasting remissions of cancer with genetically engineered T cells suggests that the highly powerful and specific nature of this new CAR technology may offer the hope for safe and lasting remissions of autoimmunity,” said Michael Milone, MD, PhD.

Both Drs. Payne and Milone are recipients of 2017 National Clinical Research Achievement Awards for this immunotherapy research. The team looks forward to further developing this technology to bring it to phase III clinical trials.

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