Promising mouse study lays foundation for startup's autism drug program

Creatine transporter deficiency--an absence of creatine in the brain--causes an energy metabolism deficit that causes severe speech problems, developmental delay, seizures and profound mental retardation in boys who suffer from autism. Now investigators at the University of Cincinnati and the Cincinnati Children's Hospital Medical Center have found an old drug that promises a new way to treat the disease in rodents. And they are handing it over to a startup that is laying plans to test it in humans.

The compound is cyclocreatine, also dubbed CincY by the investigative team, a creatine analogue that was once tested as a possible oral cancer treatment adjunct. An Austin, TX-based startup, Lumos Pharma, has the license to pick up the preclinical work on the drug with a plan to see how it works in boys in about three years.

"CincY successfully entered the brain and reverses the mental retardation-like symptoms in mice, with benefits seen in 9 weeks of treatment," says Joe Clark, a professor at UC. "Treated mice exhibited a profound improvement in cognitive abilities, including recognition of novel objects, spatial learning and memory."

Lumos was selected as a project partner by the National Center for Advancing Translational Sciences' Therapeutics for Rare and Neglected Diseases program (TRND). Now Lumos will have TRND support for the next stage of development, which runs through the IND planned for 2015. 

- here's the press release

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