Positive study results from a preliminary phase II clinical trial for a new potential therapeutic intervention for the treatment of patients with relapsing remitting multiple sclerosis (RRMS) were recently released at the American Academy of Neurology’s Annual Meeting in Philadelphia by Dr. Rhonda Voskuhl, M.D., from the University of California, Los Angeles.
The trial was designed to test the efficacy of a combination therapy of estriol (an estrogen hormone) and copaxone, an FDA-approved drug that treats the relapses in patients with RRMS, against a combination of copaxone and a placebo. A total of 158 volunteers participated in this trial, which spanned a period of two years.
From previous laboratory tests conducted on possible therapeutic options that could be used to treat the condition, the estrogen hormone estriol has been found to be the most beneficial. The idea of utilizing estriol in a combination therapy came about because symptoms of MS are seen to be reduced considerably during pregnancy, a time when estrogen levels are high. Estriol is the only estrogen hormone secreted during pregnancy, which paved the paved for this choice of therapy in the study
Estriol (8 mg/ day) was administered in combination with copaxone, and tested against a placebo. The results showed that, over a period of 12 months, the effect of combination therapy was far more prominent in treating relapses than that achieved in the combination therapy using a placebo. However, after 24 months, expected higher success rates were not met. These mixed results will spur on further research before the combination therapy can be approved and prescribed to patients.
Based on these observations, Walter Koroshetz, M.D., deputy director of NINDS, concluded that, “While these results are encouraging, the results of this Phase II study should be considered preliminary as a larger study would be needed to know whether benefits outweigh the risks for persons affected by MS. At present, we cannot recommend estrogen as part of standard therapy for MS. We encourage patients to talk with their doctors before making any changes to their treatment plans.” “The findings presented by Dr. Voskuhl suggest that there may be benefits to supplementing Copaxone therapy with estrogen. A longer study, with more patients, would be necessary to definitively validate these provocative, although early, findings,” he added.
Multiple sclerosis refers to a rare clinical condition where there is an immune response mediated by the body’s own immune system against the myelin sheath — the insulatory covering of nerve fibers — in the central nervous system (CNS) causing inflammation, interruption, and progressive damage to the conduction of nerve impulses. This leads to neurological damage and cognitive issues accompanied by physiological and emotional stress.
Patients often suffer symptomatic attacks due to demyelination (relapses) with intervals of total or partial recovery of symptoms (remissions), a condition referred to as RRMS. This is the most common form of MS and affects nearly 80% of the people who are diagnosed.
Approximately 2 – 2.5 million patients are affected by Multiple Sclerosis each year globally, with the figures in United States being almost 400,000. MS is regarded as the second most common cause of neurodegeneration in patients aged between 20-50, with trauma being the leading cause.
The study was funded by the National Institute of Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health; and the National Multiple Sclerosis Society.