An experimental treatment that uses a patient's own stem cells may offer new hope for people with multiple sclerosis.
In a small clinical trial, patients experienced long-term disease remission after undergoing a transplant of their own hematopoietic stem cells. This type of cell is responsible for the formation of blood in the body and are typically derived from bone marrow. The patients also took high-dose immunosuppressive drugs.
The paper, published Monday in JAMA Neurology, reports on the third year of a five-year study. A total of 24 patients with active relapsing-remitting MS were enrolled in the trial. With this type of MS, patients have points when their disease is active followed by periods when they do not experience any symptoms.
The researchers found that nearly 79 percent of the patients who underwent the procedure sustained full neurologic function for the three years following the treatment and symptoms of their disease did not progress. Additionally, patients in that time period did not develop any new lesions related to their disease.
More than 90 percent of patients did not experience disease progression, while 86 percent did not have any periods of relapse. Though a small number of patients did have side effects from the immunosuppressive drugs, they were no different than the side effects typically experienced by MS patients taking the drugs who haven't undergone stem cell therapy.
"Longer follow-up is needed to determine the durability of the response," the authors write in the study. "Careful comparison of the results of this investigation and other ongoing studies will be needed to identify the best approaches for high-dose immunosuppressive therapies for MS and plan the next clinical studies."
The authors of an accompanying editorial say the research indicates this type of therapy has potential to work on patients who do not experience disease remission with medications alone, such as immunosuppressive drugs and anti-inflammatory drugs such as corticosteroids.
However, they add that "the jury is still out regarding the appropriateness and indication" of stem cell transplants for MS patients. Stem cell therapy is not approved by the U.S. Food and Drug Administration for the treatment of MS. The National Multiple Sclerosis Society currently funds 15 research projects on stem cell therapies that have the potential to prevent disease activity and repair nerve damage.
"Stem cell transplant for multiple sclerosis (MS) has been a subject of great interest to scientists, physicians, and patients," the editorial notes. "The status of stem cell therapy is a common question posed by patients at their annual MS visit."
MS is a progressive disease that damages the central nervous system. It affects about 250,000 to 300,000 people in the U.S., and 2.3 million worldwide. It is classified as an autoimmune condition, in which a person's own immune system attacks myelin, that fatty coating that covers and protects nerves of the spine and brain.
Symptoms of MS can range from mild to severe, and may include numbness and tingling, loss of vision, chronic fatigue, balance and coordination problems, and sometimes a decline in memory and thinking skills. Sometimes damage from the disease can be permanent and lead to disability such as paralysis. Though there is no cure for this chronic condition, treatment for the disease has come a long way.
In the last few years a number of new drugs for MS have been approved by the FDA that have expanded options for a disease that often robs a person of essential functioning and disrupts quality of life. There are currently 12 FDA-approved drugs on the market that reduce symptoms and slow progression of the disease. Some have been found to also reverse nerve damage.
Advances in genomics have also provided new clues for how best to treat and manage the disease. In 2011, scientists completed the largest gene study to date that compared DNA from nearly 10,000 people with MS to DNA from more than 17,000 healthy individuals. The researchers were able to confirm 23 previously known genetic links and identified 29 genes and five genes that contribute to MS.
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