Isis Myotonic Drug Considered for Autism Accelerated Trials

The fast fail initiative is considering many potential autism drugs including Isis Pharmaceutical’s pending drug to treat myotonic dystrophy. ThisĀ  “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.

Myotonic dystrophy is one of the single gene causes of autism in children and Juveniles with over 50% of children reported to have some form of Autism spectrum disorder. The Autism that Kids with Myotonic dystrophy get do not involve the violent or disruptive forms of the disease. The current theory on protein disruption causing autism leads strongly to the MRNA in the myotonic cell clogging up the cellular works and restricting critical proteins from reaching the nerve synapses. By careful studying of Myotonic dystrophy patients the proteins involved in the violent or disruptive behavior may be able to be isolated.

Isis drug has already proven itself in animal models to be an exceptional candidate and it shows an unusual ability to cure the MRNA clogging that occurs in myotonic dystrophy. This also allows the release of proteins into the body vital for many functions. Scientific evidence is now growing about the local protein synthesis and Autism. As is well known myotonic dystrophy blocks the release of some vital proteins from the cells, and this may be the cause for ASD in the childhood forms of myotonic dystrophy.

Some Science background Emerging evidence of autism as a disorder affecting
synaptic connections has resynaptic connections has recently been presented (Garber, 2007). Inefficiently organised synaptic connections influence the formation of functional connectivity between different cortical regions. Synapse-specific local protein synthesis is thought to be crucial for neurode velopment and plasticity and involves neuronal RNA-binding proteins. As described earlier, it would be reasonable to assume that both
sliceopathy of Tau, NMDAR1and APP, as well as impaired transcription of DMWD, will have an impact on synaptogenesis and accordingly development of autistic symtoms in DM1 Click here for full study ==>.Neurodevelopment pathways in Autism
It is likely that this Isis Drug may be the first drug to reverse the course and essential cure autism in the individuals with myotonic dystrophy. It will also point to and help pinpoint a more general treatment approach for the autism community as a whole. We will wait and see the outcome of the Committees decision on what drugs they will pursue.
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