Scout Program: A Drug Discovery Screen in a Mouse Model of Rett Syndrome


Rettsyndrome.org is paving a new path for Rett syndrome research. Our mission is to move discoveries to the clinic. Since June of  2013, Rettsyndrome.org Board of Directors funded over $1.3M towards the Scout Program, a Drug Discovery Screen in a mouse model  of Rett syndrome.

We believe that the Scout Program will aggressively accelerate the testing of compounds, whether new or repurposed, through standardized preclinical studies in the Rett mouse model. This program will test many novel compounds and small molecules identified  in leading edge research over the next two years. The Scout Program is branched into two divisions, Academic and Pharma, as described below.

This program is in response to a workshop focused on the state of the art in animal studies of Rett syndrome that was convened by the  National Institute of Neurological Disorders and Stroke (NINDS), the Eunice Kennedy Shriver National Institute of Child Health and  Human Development (NICHD), Rettsyndrome.org and Rett Syndrome Research Trust. The participants recommended important  guidelines that the community of Rett researchers can implement to ensure more standardized study design and transparent. Much of the problems associated with preclinical research are discussed in this article written by Francis S. Collins and Lawrence A.  Tabak of  the National Institutes of Medicine.

Rettsyndrome.org developed the Scout Program-Academic Division to serve the many individuals that are performing preclinical research in academics laboratories and to move outstanding basic research to more clinical trials. It is critical to reproduce their efforts to aid in required regulatory review when they decide to take their compounds forward to human clinical trials. Rettsyndrome.org’s  strategy with this program is to independently test selected drugs using the guidelines set by the NINDS/NICHD workshop to provide the complementary high quality data required to move promising compounds along the drug development pipeline toward the clinic. Working together with academic labs, the Scout Program-Academic Division will therefore, ensure that rigorous and high-quality preclinical trial designs are employed to generate the reliable preclinical data required to shorten the time to effective treatments. 

An additional partner in developing drugs for treating Rett syndrome is the pharmaceutical industry. To address the need of partnering with the pharmaceutical industry for the development of drugs to change Rett biology, Rettsyndrome.org has also developed the Scout Program -Pharma Division. This branch of the Scout Program has been developed to provide a rigorous platform for the drug companies to use for either new or repurposed compounds that they believe might be useful in treating Rett syndrome. This aspect of the Scout Program provides an incentive to the drug company to make Rett syndrome a priority in their clinical research portfolio because Rettsyndrome.org provides the testing environment through our established partnership with a Contract Research Organization (CRO).

Rettsyndrome.org has executed a contract with the CRO PsychoGenics, Inc. in Tarrytown, NY. Dr. Taleen Hanania, Senior Vice President of Behavioral Pharmacology will be leading the testing team. As a contract research organization and drug discovery company, it offers a wide range of preclinical services and has developed partnerships with both disease-oriented nonprofit organizations as well as other corporate institutions. 

A group of scientists has served as the initial Scout Advisory Committee for developing and prioritizing the biological pathways to target  and the compounds to be tested in the Scout Program. This following group and others have aided in building the Scout Program:

Dr. John McCall, PharMac, LLC
Dr. Garry Smith, Fox Chase Chemical Diversity Center, Inc.
Dr. Stephen Ginsberg, Nathan Kline Institute, New York University
Dr. Aleksandra Djukic, Montefiore Medical Center, Albert Einstein College of Medicine
Dr. Raj Ratan, Burke Medical Research Institute, Cornell University
Dr. Marcie Glicksman, Brigham and Women’s Hospital, Harvard University

 


   

At the 2015 Society for Neuroscience Annual meeting, the PsychoGenics team presented the poster “Assessing the Mecp2 (Bird) Model of Rett Syndrome Across Species, Sex, and Age”, which describes the battery of tests performed in the Rettsyndrome.org Scout Program. Click the image above to read more from the poster.


Press Releases

December 6, 2016 - Revive Therapeutics Ltd. Announces Positive Study Results in Rett Syndrome

May 20, 2016 – Anavex Announces US FDA Orphan Drug Designation to Anavex 2-73 for the Treatment of Rett Syndrome

February 25, 2016 - Positive Rett Syndrome Data from Anavex Life Science Corps
April 28, 2015 -  Revive Therapeutics Announces Positive Study Results

Publications

October 14, 2016 - A CASE STUDY OF RETTSYNDROME.ORG’S SCOUT PROGRAM

Rettsyndrome.org HeART Awardee Bianca DeFillippis, PhD
Modulation of Rho GTPases rescues brain mitochondrial dysfunction, cognitive deficits and aberrant synaptic plasticity in female mice modeling Rett syndrome
De Filippis B, Valenti D, Chiodi V, Ferrante A, de Bari L, Fiorentini C, Domenici MR, Ricceri L, Vacca RA, Fabbri A, Laviola G.
Eur Neuropsychopharmacol. 2015 Jun;25