Major fundraising initiative launched for Rett syndrome research

International Rett Syndrome FoundationOct 26 2009

Entertainer Clint Black to chair campaign to accelerate treatments and a cure

Inspired by recent breakthroughs in research, today the International Rett Syndrome Foundation (IRSF) announced a major fundraising initiative entitled Research to Reality: Funding Progress. Funds raised by the campaign are earmarked to accelerate the progress of crucial research, and bring promise to reality. Country music sensation Clint Black, whose niece Courtney lost her battle with Rett syndrome in 2003 at age 16, will serve as Honorary Chair of the campaign. The foundation hopes to raise $3-5 million in proceeds, over and above its annual $2.5 million research budget, for scientists at major institutions who are committed to Rett syndrome research and other neurological disorders.

"At this pivotal time, I am honored to serve as chair of this important campaign to help accelerate treatments and a cure of Rett syndrome," said Mr. Black. "My family has experienced first-hand the devastation caused by this neurological disorder, and I am eager to support the exciting research on the horizon that can help reverse Rett syndrome."

Additionally, IRSF announced that it is the recipient of a lead one million dollar matching gift challenge donation from The Pioneer Fund, a private family foundation created by the late Helen M. McLoraine, to kick off the Research to Reality campaign. This gift came about as a result of a great friendship that developed between Mr. Black and Olympic Gold Medalist Scott Hamilton, who serves on the board of The Pioneer Fund, while the two were teammates on the 2009 Celebrity Apprentice.

"This generous gift will be a defining moment for our foundation and propel IRSF forward in our mission to provide care to families, while searching feverishly for a cure of Rett syndrome," said Kathryn Kissam, Chairman of the IRSF. "Breakthroughs in Rett research will help unlock the mysteries of many other conditions including autism, schizophrenia and mental retardation. We invite anyone with a passion for curing neurological disorders to join our campaign to reverse Rett syndrome."

The campaign, announced during Rett Syndrome Awareness Month, also coincides with the 10-year anniversary of the breakthrough discovery of the causative MECP2 gene, which was made by the lab of IRSF-funded researcher and key advisor, Dr. Huda Zoghbi at Baylor College of Medicine. "The last several years have been filled with exciting developments in Rett syndrome research, and major progress has occurred in the biomedical research arena," said Dr. Antony Horton, Chief Scientific Officer of IRSF. "IRSF has played a critical role in advancing the most important scientific breakthroughs in the last decade, including Dr. Adrian Bird's pivotal research demonstrating the reversibility of Rett syndrome symptoms in mice. This exciting proof-of-concept makes us very optimistic about the possibility of pharmacological reversal of Rett syndrome. This aggressive fundraising campaign will provide a much needed boost for drug discovery and development for this purpose, and various target compounds are being identified for testing in a clinical setting."

Around the country, at major research institutions, is a community of scientists committed to curing Rett syndrome and other neurological disorders. They are in position to benefit from funding allocations being finalized by the IRSF's Scientific Review Board and the National Institutes of Health (NIH). The NIH is the largest single source of medical research funds in the world, and has great influence on shaping scientific research agendas. Decision-makers there are looking to support collaborative research aimed at securing significant clinical results, research that represents a partnership between patient advocates, basic researchers and industry.

With this in mind, the IRSF scientific staff and expert advisors from academia and industry recently developed a comprehensive program for moving basic research projects closer to the clinic. The goal of the program is to support grants to facilitate drug discovery and development, and increase the likelihood of finding a successful treatment for Rett syndrome. A new funding mechanism, called the "ADVANCED NEURO-THERAPEUTIC GRANT OF EXCELLENCE" (ANGEL) award, will support high-impact projects to develop and test new therapies that could alleviate symptoms, restore function or possibly reverse Rett syndrome.

"IRSF-ANGEL awards will target several 'disease modifying' treatments already in pre-clinical development or poised for testing in pilot clinical trials," said Dr. Horton. "IRSF staff and scientific advisors have conducted careful due diligence and selected a number of the most promising therapeutic strategies now in development to treat Rett syndrome. "

Kissam adds, "With this Research to Reality campaign, IRSF is hoping to quickly double the size of its budget for research grants, while also growing its public-private partnerships and relationships with industry. Our hats are off to Mr. Black, The Pioneer Fund, and the countless other donors and families who will rise to the occasion of this exciting matching gift challenge and help us be successful in wiping out Rett syndrome once and for all."

Clint Black, along with his brother Kevin, will host a kick-off concert and golf tournament for the campaign in The Woodlands, Texas, on November 1-2, 2009. Additionally, local families will participate in a Strollathon October 31st.