Stopping MS – Emerging Therapies

Stuart SchlossmanMS Drug Therapies

Many studies were presented showing continued benefit and safety of available therapies, and additional findings from novel therapies proceeding through the development pipeline.
New form of Avonex for relapsing MS: Results were presented from the second year of an international, phase 3 trial of peginterferon beta-1a in relapsing MS, a new form of Avonex® designed to stay in the body longer than the standard form. Previously reported results after the one-year placebo-controlled portion of the trial suggested that peginterferon injected under the skin every two or four weeks was effective in reducing relapse rates and also reduced the risk of progression of disability. For the second year, all participants received therapy including those previously on placebo, and results suggested that effectiveness and safety were maintained. Trial sponsor Biogen Idec has submitted peginterferon for regulatory approval. (Abstract S4.005)
Pregnancy hormone estriol: Dr. Rhonda Voskuhl (University of California, Los Angeles) presented preliminary results of a clinical trial of the pregnancy hormone estriol combined with Copaxone® in relapsing-remitting MS. This study was inspired by the observation that MS relapses are less frequent during later pregnancy, a time when estriol is at high levels. In this trial of 164 women, the investigators determined that oral estriol plus Copaxone reduced the rate of relapses after one-year by 47% compared to women taking Copaxone alone, and also showed significant positive benefits in the scores of cognition tests. These positive effects were not however maintained through the second year of the study – the reasons why are not clear, but a more thorough analysis might reveal some answers. Read more (Abstract S23.003)
Whipworm eggs: The “hygiene hypothesis” proposes that the increased frequency of autoimmune diseases like MS in industrialized countries is due to a reduction in exposures to infectious bacteria, viruses and parasites. This idea gave rise to a small clinical trial reported by Dr. John Fleming (University of Wisconsin) and colleagues, funded by the National MS Society. Participants drank a sports drink containing the eggs of a parasite called porcine whipworm (Helminth) every two weeks for ten months. The number of active brain lesions detected at the end of the trial by MRI was moderately reduced compared to the number of lesions detected at the beginning of the trial, and they also found evidence that the treatment could promote the activity of disease-suppressing white blood cells. This was a small trial and needs to be repeated in a larger number of participants before definite conclusions can be made. (Abstract P3.149)
Ofatumumab in relapsing MS: Dr. Amit Bar-Or (Montreal Neurological Institute) presented results of a phase II trial testing different doses of ofatumumab in MS, funded by GlaxoSmithKline. This agent targets a type of white blood cell called a B cell, and is already approved for the treatment of B cell cancers. Investigators reported up to a 65% reduction in the number of active brain lesions detected by MRI in treated groups compared to the placebo groups. The most common adverse events were injection-related reactions. Five serious adverse events were reported in those who had received the high-dose regimen. These results add to previous studies with related agents and suggest that targeting B cells in MS is a promising approach. (Abstract I7-1.007)
Research Prize to Dr. Barry Arnason: The John Dystel Prize for MS Research was awarded to Professor Barry Arnason of the University of Chicago for discoveries that helped lay the groundwork for the development of immune-directed therapies in MS. Read more
Promising approach: Several presentations focused on early-stage molecules with potential to treat MS. One is IRX4204, a small molecule that selectively inhibits the activity of a receptor, or docking site, for a Vitamin A-like molecule called retinoic acid. Two studies suggested that IRX4204 may inhibit immune responses and promote the repair of myelin, the nerve fiber casing that’s damaged by MS immune attacks. If these early-stage lab studies hold up in further research, this has the potential to both stop immune responses that lead to nervous system damage and also repair what has been lost. (Abstracts S16.005, S16.006)
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