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 mechanism of action for myotonic dystrophy is now well know. The clogging of cells by MRNA according to may of the researchers is unusually sensitive to the antisense ISIS approach. Tests in mouse models have reversed the disease and the therapy is long lasting. One series of injections lasts for months. IF replicated in human trials the results would be astounding reversing the course of this insidious disease
The drug we believe is scheduled from last reports to be on track for first human clinical trials in 2014/2015. This may accelerate if the drug is selected for the fast fail program that the NIH has proposed for Autsim drugs. Rumor has it that this is a drug that in under consideration by the committee. Autism has an incidence of 1/88 while myotonic dystrophy is 1/8000 and the congenital form is 1/47,000.
The childhood forms of DM has an autism rate of greater than 50%. This form does not have the violent or disturbed behavior of some forms of autism. The mechanism that has been postulated by Dr. Eckstrom related to the proteins that interact at the neural synapses. This approach is being increasingly identified by researchers in the autism field as a strong candidate for the Mechanism of action for the disease.
Thus the DM drug that Isis is developing would very possibly reverse the course of the disease in patients with DM. While it would not cure other forms of DM it would point to very clear and replicable pathways for treatment of DM and also identify the proteins that are responsible for more disturbing aspects of autism.
Isis has the lead on this drug and may be the first to market. If the results are as pronounced as we think they area. It is highly probably that this drug will be fast tracked by FDA for approval and will contribute to ISIS bottom line in the very near future.