CAR T-Cell Therapy: Could a Cancer Breakthrough Cure Autoimmune Diseases?

An innovative treatment that has shown remarkable success against cancer is now being adapted by physician scientists to target some of the most common autoimmune diseases.

Alicia Lieberman, MD, MS, a rheumatologist and researcher specializing in cellular therapies for autoimmune disease at Roswell Park Comprehensive Cancer Center is leading work to determine whether CAR T-cell therapy, which transforms a patient’s own healthy cells to target and kill the cancerous cells, could do the same to these diseases.

“It’s time for a new revolution,” Dr. Lieberman says. “We’re rethinking our approach to be able to offer an effective and safe immune reset.”

Autoimmune diseases including lupus, multiple sclerosis (MS) and rheumatoid arthritis are chronic, lifelong conditions where the body mistakenly attacks itself, causing inflammation, problems with mobility and memory. Symptoms can vary from burning and itchy skin to swollen joints to dizziness, and require medications to manage.

Along with collaborator Shernan Holtan, MD, Chief of Blood and Marrow Transplant at Roswell Park, the goal is to develop cellular therapies as a one-and-done approach to treatment where one infusion could train patients’ bodies to be stronger to fight back against autoimmune disease within weeks. The team is looking at both standard-of-care options already in use — such as stem cell transplant — and investigational options available only through clinical trials.

“The long-term hope is that people don’t need medications for the rest of their lives and could potentially be cured,” Dr. Holtan says.

According to the National Institutes of Health, nearly one in 10 Americans lives with an autoimmune disease. Denise Herkey-Jarosch, a 54-year-old Buffalo native, was diagnosed with MS when she was 24 and has struggled to balance lifestyle changes and medications to control the disease.

“I still have permanent damage to different body parts that I have to still live with and manage,” says Herkey-Jarosch. “Having a medical campus here in Western New York so you can have that synergy of all those clinicians and patients and researchers so they can collaborate and share information is just brilliant. I’m thrilled this research is happening.”

Dr. Lieberman’s current focuses are addressing the needs of patients living with systemic lupus, lupus nephritis, systemic sclerosis and MS. She is looking at bone marrow transplants (also known as stem cell transplants) as a way to treat forms of MS and systemic sclerosis, and also hopes to study the effect of CAR T-cell therapy for rheumatoid arthritis, myositis, Sjogren’s disease and some pediatric autoimmune diseases.

“The nervous system can be particularly difficult as far as repair after damage, but we are actually getting some early signals that there can be some reversal of damage and restoration of function,” says Dr. Lieberman.

CAR T-cell therapy involves extracting T cells, a type of immune cell, from a patient’s blood and scientists engineer the cells in a specialized lab to recognize and kill cancer cells, B cells. The cells are then duplicated by the millions and replaced in the patient's body through an IV infusion.

Dr. Lieberman says B cells in autoimmune diseases express the same markers seen in some cancers, leading her to believe the transformed T cells would also be able to target and eliminate the over-activated B cells that cause the development and progression of many of these conditions.

The cells are processed in Roswell Park’s expanded Good Manufacturing Practice Engineering & Cell Manufacturing Facility (GEM), one of the largest facilities of its kind in the United States. Its 20 sterile “clean rooms” house unique resources available at the only National Cancer Institute-designated comprehensive cancer center in Upstate New York.

For more information about Dr. Lieberman’s current CAR T-cell study or other Roswell Park clinical studies, please call 1-800-ROSWELL (1-800-767-9355) or send an e-mail to [email protected].