A groundbreaking study using stem cells to treat multiple sclerosis (MS) has been approved by the Food and Drug Administration. If successful, scientists believe it may one day lead to a cure for the autoimmune system disease.
Researchers at the Tisch MS Research Center of New York say that approval of early clinical Phase I trials by the FDA comes after more than a decade of research.
“As important a milestone as the FDA investigational new drug approval is, it marks the true beginning and not the end of our clinical research of stem cell therapy,” wrote Tisch researchers in a statement announcing the trial.
“In this and subsequent studies, we hope to define the optimal therapeutic dose and dosing frequency of stem cells, the best route of administration, whether or not stem cells should be used in combination with other disease modifying treatments, and which patients are most likely to benefit from this treatment.”
About 20 progressive MS patients, recruited from an existing study will be initially enrolled.
The new regenerative strategy will involve using mesenchymal stem cell-derived neural progenitor cells (MSC-NPs), which will be harvested from the bone marrow of MS patients. The stem cells will then be injected into the cerebrospinal fluid that surrounds the spinal cords of the patients.
Researchers say the primary objective of the trial will be to determine the safety of stem cell injections. The secondary objective is to observe the effectiveness of the treatment during the three year study.
Injections of MSC-NP cells will be performed over a six month period. Study participants will have follow-up visits up to 27 months after the final injection.
Multiple sclerosis is a chronic disease which attacks the body’s central nervous system and destroys the myelin sheath that protects the nerve cells. An estimated 400,000 Americans have the disease and more than 2 million worldwide. 200 more people are diagnosed with the disease every week.
Symptoms may be mild, such as numbness in the limbs, or severe, such as paralysis or loss of vision. The progress, severity and specific symptoms of MS are unpredictable and vary from one person to another.
Early episodes of inﬂammation can cause nerve damage, which worsens as the disease progresses. Initial relapses are often followed by gradual progression of the disease after several years.
Although therapies for the inﬂammatory phase are available, none has been shown to slow the progression of MS in the absence of relapses.
Previous testing of stem cell therapy in both animals and humans has shown promise.
In one study involving mice, injected MSC-NPs migrated to areas of demyelination, where they seemed to influence the rate of repair through effects on endogenous progenitors in the spinal cord.
Another study in humans found that bone marrow derived MSC-NPs from MS patients were a feasible source of stem cells for clinical application.
In a small pilot study, seven patients who received MSC-NP infusions between 2005 and 2007 experienced reduced brain inflammation, repair of the insulating layer of myelin that forms around nerves, along with improved protection of the neuronal structure and function of the brain.
Enrollment in the clinical trial will begin after approval of the protocol has been obtained from a review board and funding has been secured. A definitive date for enrollment will be announced at that time.