CAR-T Therapy Offers New Hope for Autoimmune Diseases

After 20 years as a clinical pharmacist specializing in bone marrow transplantation, David Frame, PharmD, is an expert in dialing down the body’s immune response. That’s essential in therapies where you are basically replacing the patient’s immune system. Unchecked, the transplanted cells will attack not only the disease, but the host. 

Frame, a Clinical Associate Professor in the Clinical Pharmacy Department at the College of Pharmacy, is excited about a new way to try to turn down the immune system when it is the cause of the disease, not the cure. 

Chimeric antigen receptor T-cell (CAR-T) therapy is a rapidly growing treatment for advanced cancers that now shows promise for treating a wide range of autoimmune diseases, from scleroderma and psoriasis to gut conditions like Crohn’s disease and possibly lupus. Although the treatment is still investigational, Frame has witnessed remarkable results.

“A couple of months ago, we had a patient who had terrible scleroderma, a disease that hardens your skin. His skin was so tight he had very limited movement of his extremities,” he recalls. The patient couldn’t walk, but after two months in a CAR-T research protocol, “he actually walked out of here. That was more than I thought CAR-T would be able to do for autoimmune disorders,” says Frame, a Clinical Pharmacist at University Hospital Inpatient Pharmacy Services for Michigan Medicine.

How CAR-T Works

CAR-T is an approved therapy for some leukemias, lymphomas, and multiple myeloma.A form of personalized immunotherapy, CAR-T reengineers a patient’s own immune cells to better recognize and kill cancer cells. T-cells are skimmed from a patient’s blood and modified to add a receptor designed to recognize a specific protein on cancer cells. The modified cells are grown in large quantities in the lab. 

After a short course of chemotherapy to kill off some of the natural T-cells, the patient receives an infusion of the CAR-T cells, which hunt down and destroy the cancer cells. The modified cells persist and multiply, acting as a living drug within the body to keep the cancer in check. This approach has been used successfully with lymphomas and several B-cell cancers. In the last few years, it has also been used for multiple myeloma. 

“It’s growing really rapidly; I think we’re doing around 120 of these procedures at Michigan Medicine this year,” Frame says.

Hope for Autoimmune Diseases

CAR-T “could really be a huge game changer in the autoimmune field,” Frame said, because there are few effective treatments for many of these diseases. He cautions that it is still in its early stages.“There is a lot of fine-tuning and learning to be done,” he noted.  Compared to stem cell treatment, “CAR-T has toxicities that we don’t see with stem cell treatment, but we have definitely seen real evidence that it can potentially be extremely effective.” 

Frame and his fellow pharmacists have played a significant role in establishing clinical practice guidelines to manage and minimize the side effects of these new therapies. He has also helped write research protocols for new trials designed to enhance the effect of CAR-T cells. 

Frame, who pursued a research career in biochemistry before opting for a PharmD and a clinical role with teaching responsibilities, is delighted to be able to contribute to research that builds on his clinical knowledge of what his patients experience. “A part of my role is to recognize ways therapy can improve and to help to develop research that makes those changes come to fruition.”