Muscarinic Agonists for the Treatment of Autism Spectrum Disorder

Project ID:  D2011-54 Background Autism Spectrum Disorder (ASD) describes a group of developmental disorders that is often characterized by persistent repetitive and restrictive behaviors and deficits in social communication that, overall, affect the ability of the individual to functional normally in a social setting.  Although early treatment and care can help with the difficulties there is no best treatment for ASD. This is mostly due to the variety of difficulties exhibited on the spectrum. It is thus important to continue to develop therapies that address the deficits and behaviors seen in ASD.   Invention DescriptionResearchers at The University of Toledo have designed muscarinic agonists that result in the selective activation of M1 muscarinic receptors. The lead compound has exhibited positive effects in an ASD mouse model and may be useful in the treatment of restrictive and repetitive behaviors in ASD patients.    Applications•       The muscarinic agonist compound may be administered to patients suffering from ASD•       The compound may also be used in patients with Attention Deficit Hyperactivity Disorder (ADHD)•       The compound may be used to treat executive function deficit in schizophrenia•       The compound may be used to treat other cognitive and neurological disorders  Advantages•       The mechanism of action of the lead compound is known•       In vitro and in vivo preclinical toxicity data is available•       An IND has been filed and Phase I clinical trials have been completed•       The compound exhibits CNS penetration and activity•       The lead compound improves behavioral flexibility in normal rats IP Status:       US Patent Issued (9,549,928) Publication:   Ragozzino ME, et al., The selective M1 muscarinic cholinergic agonist CDD-0102A enhances working memory and cognitive flexibility. Journal of Pharmacology & Experimental Therapeutics, 2012, 588-594

USA