C11387: Hydroxyurea for the Treatment of AutismNovelty:
This technology employs hydroxyurea (HU), a common treatment for sickle cell disease, to reduce core symptoms, like social responsiveness, in patients of all ages with autism.
Value Proposition:
Currently, the only available drugs for the treatment of autism are symptomatic. These drugs aim to modulate behavior, but do not target the underlying cause. This invention uses HU or similar compounds to simulate the effects of a fever, which has been shown to alleviate behavioral symptoms in children with autism. This treatment of autism could increase cognitive function at the cellular level and decrease the need for symptomatic treatment. Advantages of this invention include:
• Possibility to change the course of the disorder in young patients
• HU is able to penetrate the blood brain barrier for easy drug delivery
• HU is already on the market for treatment of sickle cell disease
Technical Details:
A Johns Hopkins researcher has developed a novel drug treatment for autism based on a previous study showing a decrease of behavioral symptoms in autistic children during a fever. It is hypothesized that the increased cell stress during a fever creates a condition in the brain that increases cognitive function. This effect has also been observed in children being treated with HU for sickle cell disease. On this basis, HU and similar compounds have the potential to treat autism at every stage of development.
Looking for Partners:
To develop and commercialize a therapeutic drug for the treatment of autism.
Stage of Development:
Phase II
Data Availability:
Under CDA/NDA
Publications/Associated Cases:
Not available at this time.
