Pepsi Refresh grant helps in launching Rett syndrome drug development program

IRSF to launch the 'Selected Molecular Agents for Rett Therapeutics' (SMART) Initiative -- a new Rett syndrome-specific drug development program

The International Rett Syndrome Foundation (IRSF) believes that accelerating the pace of meritorious research, supporting families, and raising awareness are the minimum effort necessary to successfully search for treatments and a cure for one of the most devastating neurological diseases to affect young girls. On October 1, IRSF became the recipient of a $250,000 grant from the Pepsi Refresh contest that was officially confirmed later in the month. The contest was a highly-publicized and competitive online grant program to benefit non-profit organizations. In March 2010, IRSF entered the challenge when Donna Wright contacted IRSF's Director of Family Support, Paige Nues, to discuss the competition on behalf of her granddaughter, Naomi, who suffers from Rett syndrome.

The Pepsi Challenge funds will be put to immediate use in part, to launch the "Selected Molecular Agents for Rett Therapeutics" (SMART) Initiative - a new Rett syndrome-specific drug development program. The SMART Initiative was the outcome of an intensive two day meeting convened in March, where IRSF brought together a blue ribbon panel of advisors to discuss the development of new medicines for reversing the symptoms of Rett syndrome. The panel drew on the expertise of leading clinicians and researchers working on Rett syndrome, experts drawn from the biotech and pharma industries together with advisors from the FDA and the NIH.

The SMART Initiative will be directed by two leading medicinal chemists: Professor Alan Kozikowski and Dr. Irina Gaisina at the University of Illinois, Chicago, in consultation with external advisors Drs. John McCall and Clark Eid--key participants at the March meeting. The new consortium's first task will be to assemble a collection of brain-specific drugs which target select biological mechanisms important in Rett syndrome. The research team in Chicago will select compounds on the basis of their mechanism of action, and their drug-like qualities. They will create an electronic database of all of the drugs that are procured or synthesized, and keep records ensuring their purity. The database will also obtain information on all drugs that are assayed. Rett researchers from around the globe will have access to the compound collection upon written request to facilitate studies of these selected agents.

Commenting on the SMART Initiative, Dr. Kozikowski said, "In the first phase of this program, the consortium will be highly selective in choosing the most appropriate drugs for testing. As the identification of effective therapeutics requires a strong marriage between chemistry and biology, we believe that this initiative will help advance the development of new drugs for Rett syndrome." Dr. Kozikowski added, "The chemistry group in Chicago is extremely grateful to the grass roots effort that played a key role in obtaining the Pepsi Challenge funding on behalf of IRSF."

Dr. McCall, said, "Drug discovery is often driven by access to compounds that target specific mechanisms. The consortium will provide valuable tools allowing researchers to efficiently explore new approaches to treatment." "We often talk about looking for keys under the street lamp - we intend to provide the street lamp," he added.

To win the Pepsi Refresh competition, IRSF launched an intensive grassroots effort, mobilizing thousands of volunteers who voted every day for four months until the Foundation was chosen as the winner on the eve of "Rett Syndrome Awareness Month." Ms. Nues, whose daughter Katie has Rett syndrome, commented, "The true win goes to our girls and women who wait patiently while our researchers work diligently for new treatments and an eventual cure for this devastating disease."