Informal reports continue to give good vibes on the drug that Isis pharmaceuticals is developing for myotonic dystrophy (DM). The drug is an antisense approach one that will break up the clogs that are causing the DM disease. Very little information is being released by the company, however.
The fast fail initiative is considering many potential autism drugs including Isis Pharmaceutical’s pending drug to treat myotonic dystrophy. This “FAST FAIL” initiative funded by NIH looks at promising drugs to treat Autism and several other conditions. It enables the drug to go to human trials earlier and see if the drug will pan out. The Isis drug by far is the most probable drug to treat and reverse the course of the Autism disease.
Race for the Cure for Myotonic Dystrophy
Myotonic Dystrophy may be the first of the muscular Dystrophy to have a definitive cure established. In the last few years researchers have isolated the issue they believe causes myotonic dystrophy. In the last couple of years therapies that have targeted this problem have reversed the disease in cell and animal models. This has set up a race now for researchers to refine the therapeutic compounds and begin with human trials to see if the animal to human therapeutic transfer works as it is believed to. Thus, this would lead to the first potential cure for a type of muscular dystrophy.
The Myotonic Dystrophy Foundation (MDF) in Menlo Park, CA has taken a lead in assisting researchers with moving forward with this disease elimination process with funding and assistance in establishing a patient registry. The MDF was the early funder of a new Research Collaborative effort lead by Charles Thorton out of the University of Rochester. The foundation has also been instrumental in educating patients and physicians in this complex and slowly progressing disease that results in premature death. A patient conference is planned for early November of 2013 in Houston, TX
Good news today! Another company has launched a potential drug for myotonic dystrophy and by extension this may also treat the childhood forms of myotonic dystrophy. The childhood forms of myotonic dystrophy are highly associated with autism spectrum disorder, so it is hopeful that this new drug will have some effects on this as well as the cognitive effects in the adult forms of the disease. For the general autism population reversing the effect in the childhood forms of myotonic dystrophy. May help narrow the mechanism of action and suggest certain treatments in the future. The childhood form of myotonic dystrophy is one of the few single gene causes of autism. Because the drug will work through an RNA mechanism, it is unlikely this drug will have a direct effect on the general autism population.
Here is a copy of Dr. Eckstrom Thesis on Childhood Myotonic Dystrophy. Its pretty comprehensive and a bit technical. Click below to open a PDF Copy. The thesis is almost 90 pages long and full of a lot of good information. Dr. Eckstrom is one of the leaders in the knowledge of childhood myotonic dystrophy and Autism
EckStrom Doctoral Thesis Childhood Myotonic Dystrophy
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