Preventing Mutism in Autism
There are no drugs available to prevent 20,000 US children who develop autism from becoming non-verbal and requiring life-long assisted living.
Comparing the genetics of people with non-verbal vs verbal autism (nature.com/articles/tp2013124) we have identified specific ion channels as drug targets to prevent disruption of active language development in children at about 12 months of age. Fenamates, a particular class of NSAIDs, are known to activate these ion channels.
Among them, mefenamic acid has been used to treat chronic pain in UK children with juvenile arthritis from 6 months of age for over 50 years. No major safety concerns have been identified. We will use a market exclusive controlled release form of MFA to further reduce risks from accidental overdose and intestinal irritation. As an ester does not create a New Chemical Entity (FDA), we can use the 505(b)(2) regulatory pathway to bring the breakthrough drug to the market after a single phase 2/3 trial.