CAR T-cell Therapy for Multiple Sclerosis

Your immune system is like a security team protecting you from danger. But what if it thinks the danger is coming from inside your own body?

This happens in autoimmune diseases like multiple sclerosis (MS), in which your immune system mistakes normal cells in your brain and spinal cord as threats.

A new type of immunotherapy, called CAR T-cell therapy, can retrain a part of the immune system to tame this overreaction. Already FDA-approved to treat some cancers, this therapy is showing promise in patients with MS in clinical trials.

Neurologist Claire Riley, MD, Director of Columbia’s Multiple Sclerosis Center, explains what MS is, how CAR T-cell therapy works, and why this emerging treatment is offering new hope to more patients.

What Is Multiple Sclerosis (MS)?

MS is an autoimmune disease in which the body perceives threats in the nerves of the brain, eyes, and spinal cord, even though there is no danger. The immune system reacts by sending white blood cells, specifically B and T immune cells, to attack the coverings of the nerves, which damages the nerves themselves and impairs their ability to communicate with each other. This leads to problems with balance, coordination, memory, and vision. It can also cause numbness, tingling, dizziness, mood changes, and pain. For some people who have a progressive form of MS, these symptoms get worse over time.

What Is CAR T-cell Therapy?

Originally developed to treat cancer, CAR T-cell therapy “trains” a patient's T cells to recognize and attack a threat. The patient's own T cells are collected from their blood and genetically modified to find and kill a specific target, then they are infused back into the patient. This therapy has already been shown to be especially effective in treating certain kinds of blood cancers.

"When CAR T-cell therapy works for cancer, it seems to be quite a durable effect. Our hope is that this kind of immune reset may be long-lasting for our MS patients," says Dr. Riley.

How CAR T-cell Therapy Can Be Used To Treat MS

Currently, a highly effective MS treatment involves creating antibodies in a lab that attack B cells. When it works, it works well to control relapses, but long-term use may be complicated by infections—and for patients with progressive MS, the relief can be inadequate. The antibodies circulate in the blood but can't cross the barrier that protects the brain and spinal cord, where most of the damage occurs.

CAR T-cell therapy for MS modifies T cells to recognize and kill the B cells that are promoting damage. Because CAR T-cell therapy uses the patient's own cells, they can cross the barrier and get to where they may be needed the most. 

CAR T-cell Therapy at Columbia

Columbia is paving the way for using CAR T-cell therapy to treat both cancer and autoimmune diseases. Columbia offers CAR T-cell therapy for adults with recurrent or persistent diffuse large B-cell lymphoma, and for children or young adults with recurrent or persistent acute lymphoblastic leukemia (at NewYork-Presbyterian Morgan Stanley Children’s Hospital).

A clinical trial at Columbia is studying how patients with MS who have not found relief from other treatments respond to CAR T-cell therapy. “At this stage, we’re primarily evaluating the safety of the therapy, but we’re also seeing how well it works. So far, these treatments have been well tolerated,” says Dr. Riley.

“If the trials continue to yield positive results, CAR T-cell therapy could become a powerful option for MS patients, particularly those who have not responded well to traditional treatments,” says Dr. Riley. “We’re at the beginning of this journey, but the potential is enormous.”