IRSF awards $2M for research into a cure for Rett syndrome

Foundation announces new funding mechanisms to fast-track therapies

The International Rett Syndrome Foundation (IRSF) announced today that it is awarding grants totaling $2 million to support 18 innovative research projects in 2009. Each project will explore bold new ideas that have the potential to drive the field forward and speed the translation of research into treatments and a cure for Rett syndrome. With this announcement, the foundation has cumulatively funded over $20M in high quality, peer-reviewed grants that have contributed to the most significant advancements in the field to date.

New awards will be provided for the development of the first neuronal assays for drug screening derived from human-induced pluripotent stem cells (iPSCs). Additional grant awards will be provided to move forward 5 high impact translational research projects which focus on the development of therapeutics to treat or reverse Rett syndrome. Furthermore, a number of the projects chosen in this funding cycle may result in the identification of new drug targets that can be exploited for therapeutic intervention.

IRSF will shortly provide funding through the launch of two new grant mechanisms focused directly on translational research. The HELP ACCELERATE RETT THERAPEUTICS (HEART) grant awards will provide seed funding to foster early stage translational research programs. The ADVANCED NEUROTHERAPEUTIC GRANT OF EXCELLENCE (ANGEL) award mechanism will provide larger grant awards to more mature drug discovery and development programs that are further along the path to a clinical application. "The HEART and ANGEL awards will be provided to fast track research that is moving new therapies closer to the clinic" said Dr. Antony Horton, Chief Scientific Officer of IRSF.

Dr. John McCall, a drug discovery expert and member of the IRSF's Scientific Advisory Board commented: "A roadmap is now emerging that will lead to significant advances in therapeutic development, leading to treatments that will improve quality of life and could provide the path to an eventual cure for Rett syndrome." Dr. Horton added: "These new studies will generate fresh insights that will enable us to better understand the mechanisms underlying Rett Syndrome. While there are no quick fixes, we will continue to be aggressive in our search for new treatments for the eventual pharmacological reversal of Rett Syndrome."

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