Rettsyndrome.Org awards $1.5M to advance translational research, support Neuro-Habilitation Program

The International Rett Syndrome Foundation now doing business as Rettsyndrome.org announces today that the Board of Trustees has awarded $1.5M to support 10 new grants to further translational research and launch of the neuro-habilitation therapeutic program, and fund clinical research.

Five of the grants fund the Translational Research Program which funds early and late stage translational research to treat and reverse Rett syndrome (RTT).

An additional five grants were awarded to the newly established Neuro-Habilitation Program. The goal of the program is to identify the best cognitive therapies, physical therapies, occupational therapies and speech therapies that will aid in establishing the networks critical for skill development in Rett syndrome. Today's new awards and the upcoming annual summer call for basic discovery research, training fellowships, and translational research in pharmacologic treatments will contribute to Rettsyndrome.org's program for bringing "Research to a Reality". Rettsyndrome.org has advanced our comprehensive strategy to accelerate discoveries in fundamental research toward clinical to improve the lives and treatment of those living with Rett syndrome. Our emphasis on translational and clinical research will ensure that new ideas are constantly flowing into the drug pipeline and neuro-habilitation platforms both aimed at accelerating the treatments and maximizing function in Rett syndrome.

Rettsyndrome.org's Chief Science Office, Steve Kaminsky, PhD comments on the research strategy, "With potential treatments on the horizon, we have coined the phrase that our pharmacologic treatments are designed to "correct Rett biology". However, as we proceed down this road, we have to identify new technologies and therapies that "reset the neurology" in Rett syndrome to maximize our girls' abilities. To this end, we have initiated a new program this year and it is called our Neuro-Habilitation Program. The time is right to begin looking at this area of research so that the fruits of this labor mature at the same time as our other research programs which identify pharmacologic treatments. My hope is that the work will converge on each other, and we can match the best practices in drug therapy to the best practices in neuro-habilitation therapy."

The mission of Rettsyndrome.org is to advance research towards new treatments for Rett syndrome by investing in both basic and translational research grants, which are vetted through a rigorous process of peer-review by members of Rettsyndrome.org's Scientific Review Board (SRB). Those applications that have the highest scientific and programmatic merit are recommended to the Board of Trustees for final funding decisions. New 2014 Rettsyndrome.org Awards

ANGEL

• Julian Paton, PhD, University of Bristol

"NLX-101 - a novel, highly selective and potent serotonergic 5-HT1A receptor agonist for the treatment of respiratory arrhythmias in Rett Syndrome: a pre-clinical proof-of-principle study in murine models"

HeART

• Michela Fagiolini, PhD, Boston Children's Hospital "Assessing NMDAr modulators to ameliorate cortical regression in Rett Syndrome" HeART Awards - Neuro-Habilitation
• Susan Bruce, PhD, Boston College

"Sensory Integration to Increase Functional Reaching in Children with Rett syndrome and related disorders"

• Pamela Diener, PhD, Marymount University and Jack Engsberg, PhD, Washington University of St. Louis

"Improving Upper Extremity Motor Skills in Children with RTT Using Selected Movements to Control Internet Virtual Reality Computer Games"

• David Koppenhaver, PhD, Appalachian State University

  "Investigating Visual Attention to Print in Children with Rett Syndrome"

• Charles Nelson, III, PhD, Boston Children's Hospital

  "New Methods to Assess Cognition and Affect in Girls with Rett Syndrome"

• Sarika Peters, PhD, Vanderbilt University Medical Center

  "Auditory processing, language, and learning in Rett and Rett-related disorders"

HeART Awards - Scout Program

• Bianca De Filippis, PhD, Istituto Superiore di Sanita, Italy

  "Preclinical evaluation of the bacterial protein CNF1 as a novel therapeutic approach for Rett syndrome"

• Maurizio Giustetto, PhD, University of Torino, Italy

  "Restoring the AKT/mTOR pathway to Treat Rett syndrome"

• David Katz, PhD, Case Western Reserve University

  "Preclinical Evaluation of LM22A-4"

Database Contracts

• John Christodoulou, AM, Sydney Children's Hospital Network, Australia

"RettBASE"

• Helen Leonard, MBChB MPH, The University of Western Australia

"InterRETT"