Cell-Based Therapy for Secondary Progressive Type of MS

Linda Agnello, 56, recalls the days when she was able to go for long walks with her beloved husband, Steve, and golden retriever, Calleigh, and freely take on projects at the school where she teaches music. Now, she must first calculate how much energy and time each activity will require and if her body will cooperate. She — like many other people living with multiple sclerosis (MS) — has had to adjust her life to her condition. She has also had to cope with side effects from existing medications.

People with MS urgently need new treatments to halt the condition, and hope is on the horizon. Shepherd Center is one of 30 sites in the United States and Canada participating in a clinical trial to study whether an investigational therapy using a patient’s own immune cells can help stabilize or stop the progression of secondary progressive multiple sclerosis (SPMS). People with SPMS have moved beyond the initial period of relapsing-remitting MS, and their condition has begun to worsen more steadily.

“This is a group of individuals with MS who have not had many treatment options available, so there is a lot of excitement about this study,” says Carlyn Kappy, RD, LD, CCRP the study coordinator at Shepherd.

Eligible study participants are being randomized to receive either placebo or the investigational T-cell immunotherapy called Tcelna™, which is manufactured by Opexa Therapeutics, Inc. Encouraging results from earlier Tcelna trials in MS patients, including some with SPMS, prompted the U.S. Food and Drug Administration to grant it fast-track designation for SPMS.

To develop the therapy, Opexa isolates the “bad” T-cells (myelin reactive T-cells) from a patient’s blood sample and expands them to generate enough reactive T-cells to treat a patient for one year (five doses). The myelin reactive T-cells are thought to cause the inflammation that is the hallmark sign of MS. During the final dose preparation, the myelin reactive T-cells are modified to make the cells unable to replicate. The modified reactive T- cells are then reintroduced to the patient via an injection, which should stimulate the body’s immune system to reduce the number reactive T-cells.

“We are essentially vaccinating them against their own MS,” explains Ben Thrower, M.D., medical director of the Andrew C. Carlos Multiple Sclerosis Institute at Shepherd Center. “The hope is that the body will recognize these cells and trigger an immune response.”

If this new therapy works, it would give patients a customized therapy from the start.
“The immunology of MS is so different from one person to the next, so it’s a little bit of trial and error to find the right fit,” Dr. Thrower says. “This new approach would allow us to treat an individual based on their unique disease.”

Tcelna is also thought to have a lower risk of side effects because, unlike many other therapies used to treat this type of MS, it does not suppress the immune system. In the early 1990s, doctors believed the best way to manage the overactive immune response typically seen in MS was to crush it with chemotherapy and later with immune modulators, including beta interferons, but there were a lot of side effects.

“Existing medications allow us to treat symptoms of MS, but they may also put the patient at risk for other health problems and conditions,” Dr. Thrower explains. “Newer medications have taken us a step in the right direction, but we still find many patients on immune modulators don’t respond very well.”

So far, six Shepherd Center patients have been screened to take part. Participants must be able to walk to some extent on their own with or without an assistive device be between ages 18 and 60 and will need to stop taking any other treatments to slow disease progression if they decide to pursue the trial. Patients are given just five shots of the treatment or placebo a year. Researchers will evaluate the percentage of brain change at 24 months, disease progression, and changes in disability and annual relapse rates.

Although Linda, a Shepherd Center patient, is not participating in the study, she is hopeful this approach will be effective. “Folks with long-term MS have been waiting a very long time for an effective medication for MS,” she says. “We’re running out of options.”

Dr. Thrower notes a lot of excitement about this study. “People with secondary progressive MS often feel left out [of clinical trials],” he says. “With this study, we are closer than ever to a tailored therapy.”

The National Multiple Sclerosis Society reports more than 2 million people have MS worldwide; of these, an estimated 25 to 35 percent may have SPMS.

An initial analysis of the study results is expected in 2016. For information, contact
Carlyn Kappy, RD, LD, CCRP, at 404-367-2620 or carlyn_kappy@shepherd.org

Written by Amanda Crowe, MA, MPH
Photography by Gary Meek