Rolling back the clock in a multiple sclerosis (MS) patient means getting back some of the lost physical function, cognitive function, and overall quality of life that this disease steals away. This, surely, is the dream and hope of every MS patient who watches his or her abilities slip away as this terrible disease progresses. New clinical research, sponsored by the National Institute of Allergy and Infectious Diseases, National Institutes of Health (NIH), tries to realize this dream of reclaimed abilities with the use of stem cell therapy.
It’s not quite time to celebrate yet, however, since this stem cell research remains at the experimental stage and – although there are tantalizing benefits seen in patients – this therapy also brings substantial risks. Allow me to explain: stem cells can be harvested from a patient’s own bone marrow or blood and then these cells are reintroduced to a patient’s body after chemotherapy or radiation (or both) has destroyed the person’s immune cells. The goal is for the stem cells to “reboot” the immune system. In the process, however, there have been serious and even fatal side effects in past research.
In the NIH study, 24 people with MS received high-dose immunosuppressive therapy and then were transplanted with their own hematopoietic stem cells. During the next three years, most of these patients (78.4%) achieved remission and experienced neurological improvements. In addition, there were no new brain lesions or progression of disability in the patients who responded to the stem cell therapy. Although there were some complications related to the immunosuppression at the beginning of the trial, there were few side effects as the study continued and no deaths.
In a late-breaking study that was very recently published, there are even more encouraging results in 123 patients with relapsing-remitting multiple sclerosis and 28 patients with secondary progressive MS. The immune systems of these patients were altered in a different way (e.g., a nonmyeloablative regimen) that left some of the bone marrow intact. During four years of follow up, 80% of patients were relapse-free and there were decreases in brain lesions and neurological improvements.
For a patient who fails to respond to conventional medications, stem cell therapy could offer the only hope for improved quality of life.
Stephen C. Vogt, Pharm.D.
President and CEO
BioPlus Specialty Pharmacy | www.bioplusrx.com
Burt RK, Balabanov R, Han X, et al. Association of nonmyeloablative hematopoietic stem cell transplantation with neurological disability in patients with relapsing-remitting multiple sclerosis. JAMA 2015;313(3):275-84.
Stem cells may halt progression of multiple sclerosis. Pharmacy Practice News January 5, 2015.
Press release. Report on remission in patients with MS 3 years after stem cell transplant. JAMA Neurology Releases December 29, 2014.