Autism

Autism, or autism spectrum disorder (ASD), is a neurological condition that can cause differences in socialization, communication, and behavior. Autism is the costliest developmental disorder in the USA and other countries. The latest data from CDC show a prevalence of one in 36 children. Currently, no drugs are approved to treat any core symptoms of autism. 

 

More than 50 % of autistic children have an altered Folate-metabolism. Children with ASD often show normal systemic folate levels with low cerebral folate. One of the reasons is the binding or blocking Folate Receptorα-Autoantibody (FRA). Several clinical studies have shown a positive effect on verbal communication with a high dose treatment containing Folinic Acid (5-Formyl-(6R,S)-THF, activity 50 %). The explanation is folinic acid allows uptake of Folate into the brain via an alternate route, specifically the Reduced Folate Carrier (RFC).

 

With pure Levo-form (5-Formyl-(6S)-THF, activity 100 %) it is possible to reduce the daily dose by half, from 2 mg per kg body weight to 1 mg per kg. The formulation as a solution filled in a device allows a precise, convenient dosing based on the body weight of the child. One drop contains exactly 1 mg of the drug, and this device is currently in use for preservative free eye drops. The drops can be diluted with water and provided to the children as a drink.

 

Three clinical studies (phase II) with a total of 280 children are ongoing in the USA with the above formulation. For the very first time in two studies children aged 2.5 to 5 years are treated. The goal is to use an early treatment to prevent irreperable harm of the developing brain caused by a local folate deficiency. Avoiding the exposure of the children to the unnatural D- or 6R-form and the precise, convenient dosing are the advantages of the used medication.