Multiple sclerosis, or MS, is an autoimmune disorder that affects the brain and spinal cord. The immune system destroys the patient's own myelin, the protective membrane wrapped about the nerves, thus disrupting communication with the central nervous system. About 50 percent of patients are unable to walk 25 years after their diagnosis.
Rather than suppress the immune system with a high dose of drugs, a technique called autologous hematopoietic stem cell transplantation (HSCT) hopes to reset the immune system. Hematopoietic simply refers to blood, and autologous means using one’s own cells, instead of those from a donor. So what happens is, after low-dose immunosuppressive drugs are administered to dampen the body’s response, stem cells harvested from the patient’s blood are infused back.
Northwestern’s Richard Burt and colleagues examined the outcomes of 145 MS patients who were treated with this stem cell transplant during trials between 2003 and 2014. Specifically, the patients—who ranged in age from 18 to 60 years old—have relapsing-remitting multiple sclerosis: Symptom flare-ups are followed by periods with little or no symptoms. The patients were periodically tested (2.5 years on average) with the Expanded Disability Status Scale (EDSS), which measures the function of the brainstem and sensory and visual systems, among other things.
The team found significant improvement in 41 patients tested after two years—that’s about 50 percent of the patients who were tested at that interval. For patients tested at four years, they found similar improvement in 23 patients, or 64 percent of the patients tested. Patients who received HSCT showed improvement in physical and cognitive function as well as quality of life, the researchers report. Furthermore, MRI imaging showed a reduction in the volume of brain lesions.
After four years, the relapse-free survival was 80 percent. And the four-year progression-free survival was 87 percent. However, the EDSS score did not improve in patients with secondary-progressive MS or in those who've had MS for more than 10 years.
The results of this preliminary study (observational and without a control group) were published this week in JAMA, the Journal of the American Medical Association. Burt’s team is currently conducting a larger study to compare HSCT with drugs to manage MS that have already been approved by the U.S. Food and Drug Administration. There are currently no therapies for relapsing-remitting multiple sclerosis that can significantly reverse the disability.