Introduction
Multiple sclerosis is an autoimmune disease that affects 2.3 million people worldwide. Current treatment is expensive and limited, but new research is being done every day. One promising trial sponsored by the National Institute of Allergy and Infectious Diseases (NIAID) compares stem cell experimental therapies to current treatments.
Multiple Sclerosis Overview
Multiple Sclerosis (MS) is an autoimmune disease that affects the central nervous system. This means that the body attacks its own nervous cells, causing them to degenerate. It affects women more than men, Caucasian people more than other races, and people in hotter climates than colder climates. Symptoms include motor and sensory disturbances like blurred vision, difficulty walking, numbness and tingling, bowel and bladder issues, and mental changes.
MS comes in 3 varieties: relapsing-remitting, secondary-progressive, and primary- progressive. In relapsing-remitting, the symptoms may appear for weeks or months and then resolve, repeating this pattern. Primary-progressive is when the symptoms appear and gradually worsen. Secondary-progressive is, in a way, a mixture of both. Exacerbations occur and there is a continuous worsening of symptoms in between exacerbations.
Current Treatments of Multiple Sclerosis
Standard treatment of MS depends on the type of MS, but typically revolves around immune suppression. Because MS is caused by the body’s immune system attacking the central nervous system, the treatment of MS is suppression of that immune system. In relapsing- remitting and secondary-progressive, exacerbations are treated with high-dose glucocorticoids. Second-line therapy for exacerbations is plasmapheresis. To prevent exacerbations in these types of MS, therapy includes different types of immune suppression. Primary-progressive has no established standard treatment. Working with a neurologist who specializes in MS can help all MS patients.
Stem Cell Trials
There have been many studies on stem cells in MS. The stem cell experiment sponsored by NIAID approaches this immune therapy from a different angle. Their experiment uses a treatment called Autologous Hematopoietic Stem Cell Transplantation. Stem cells are naturally occurring cells that can turn into many different types of other cells. In the NIAID experiment, they used chemical agents to remove the patient’s immune cells that were attacking their central nervous system. They then used some of the patient’s own stem cells to grow more stem cells, which were then infused back into the patient. The stem cells were used to repopulate the patient’s immune system. Essentially, this experiment took out the bad immune cells that were causing the inflammatory, autoimmune symptoms of MS and replaced them with new, functioning immune cells.
Stem Cells Compared to Standard Treatments
NIAID, in collaboration with the Blood and Marrow Transplant Clinical Trials Network (BMT-CTN), is specifically sponsoring a trial that compares these stem cell therapies to standard treatment. The trial is called the BEst Available Therapy versus autologous hematopoietic stem cell transplant for Multiple Sclerosis, or BEAT-MS. Jeffrey A. Cohen, MD, a professor of neurology at the Cleveland Clinic Lerner College of Medicine, is leading the trial. He is also the director of the Experimental Therapeutics Program in the Mellen Center for Multiple Sclerosis Treatment and Research at the Cleveland Clinic.
The trial will enroll 156 adults ages 18 to 55 years across the United States and United Kingdom. Participants will receive either the stem cell therapy or the standard treatments. It is a blinded experiment, meaning that the neurologists assessing the patients will not know which treatment type the patients receive. The researchers will determine the immune function in both treatment arms, time between MS relapses, the effect of the treatments on disease activity, the cost-effectiveness, and the quality of life of participants in both treatment arms.
Stem cell therapy research on treating people with MS is in the beginning stages, as shown by this trial. As such, researchers have to simplify the variables as much as possible. Right now, the trial will just address people with relapsing-remitting MS. Eventually, researchers would likely expand research to include multiple types of MS in stem cell therapies.
Future Impact
The implications of this study would be life changing for many MS patients. For patients suffering from relapsing-remitting MS, stem cell therapy would halt the progress of the destruction of the nerve cells, eliminate the need to take lifelong medications, and allow these patients to have an immune system they need to fight off other illnesses. If the authors of the study find that stem cell therapy is more effective both in terms of disease outcomes and cost, insurance companies would be more likely to cover the treatment and pharmaceutical companies would be more willing to invest in stem cell research.
Summary
While millions of people are affected by MS, the standard treatments still do not meet many of their needs. The BEAT-MS trial sponsored by the NIAID and BMT-CTN is a promising new development in the field of stem cell transplant research. Stem cell research has long shown promise in treating autoimmune diseases, but big trials are still in the beginning stages. If you would like to learn more about how stem cell transplants can help you with your autoimmune disease, contact us at the Stem Cell Transplant Institute today.
