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About FARA
FARA will continue to:
FARA is exempt from federal income tax under section 501(a) of the Internal Revenue Code as an organization described in section 501(c)(3). How FARA Got Started The first to come to the research alliance, to form its Board of Directors, were a group of patient families and three of the world’s leading FRDA scientists — Drs. Rob Wilson, Bronya Keats, and Massimo Pandolfo. On the day following FARA’s incorporation, the new organization submitted to the National Institutes of Health (NIH) its application for a grant to hold the first International Scientific Conference on Friedreich’s Ataxia. That three-day conference was convened in April 1999, gathering about 80 scientists from around the world. The alliance has continued to gain momentum. When FARA again collaborated with NIH to conduct the second International Conference on Freidreich’s Ataxia in February 2003, 100 scientists from 12 countries gathered for five days to share their exciting insights and chart the path to treatments and a cure. FARA continues to build the research alliance and provide the support these scientists need to get us all across the finish line. As a result of the generosity of its donors, the tireless dedication of the families involved in fundraising activities, and the cooperation of Seek A Miracle / MDA, FARA provided, in its first six years, more than $2 million directly to the support of FRDA scientific research around the world. About half of that total, in fact, was provided in 2004, and the pace and quality of the research, along with the rate of FARA funding for it, continue to increase steadily. The Road Ahead — FARA’s Vision for Progress The foundation for progress in all three mission areas, of course, is laid by the advances we are enjoying in basic science. We are deeply indebted to the scientists that have identified the FRDA gene, deciphered its mutations, analyzed the composition and function of its protein, and sorted out the mechanism of the damage being done. None of the progress being made in slowing, stopping or reversing this disease would be possible without the marvelous advances in such basic understanding. More needs to be done in this basic science arena but, based on the giant steps taken to date, promising research is underway in all three mission areas. For the most part, the research being conducted on antioxidant therapies (Idebenone, CoQ10, MitoQ, etc.) we can consider in the first mission area — slowing the disease progression and providing our scientists and our patients more time to make advances in the other two mission areas. We are supporting a wide spectrum of research aimed at stopping the disease. The common ground shared in most of these investigations is the attempt to increase the availability of the frataxin protein. Some scientists are pursuing that goal with gene-based approaches to delete, repair, or stabilize the mutation so that more of the protein is produced. Others are working to synthesize the protein and deliver it to the mitochondria directly. In whatever way it is accomplished, making more of the frataxin protein available in the right place holds the promise of stopping the disease in its tracks. In both of these first two mission areas, we support a variety of promising approaches and monitor progress closely, looking for the optimum approach or combination of approaches that will take us to treatments. The third mission — reversing the damage, regenerating lost capabilities — seems to present considerable challenges. We do anticipate that a small amount of reversal will be achieved when we are able to slow and stop the disease, because some “sick” cells will be rescued in the process. However, scientists do not yet know how to rescue the cells that are already dead, so more significant reversal will await a different approach. Currently, the approach that seems most promising in that regard is stem-cell research. FARA will leave no stone unturned in its commitment to accomplish all three missions. Much progress has been made and continues to be made in the projects we are all supporting around the world. Especially in slowing the disease, antioxidants and other drugs are moving from drug screening to animal models and the large human trials needed to obtain approved therapies. We need to continue accelerating that process by working closely with the NIH, the other scientists preparing the drug screenings, animal experiments and clinical trials, and with the pharmaceutical companies interested in supporting clinical trials and developing additional drugs. As that “slowing” process advances, we need to take the “stopping” and “reversing” research to the next level. To go to the next level in the gene therapy and protein therapy, for example, needed to stop the disease and the stem-cell research needed to reverse it, our strategy is to get excellent scientists, who develop such therapeutic technologies and approaches for a living, together with our excellent scientists who know enough about FRDA to determine how to apply those technologies and approaches to FRDA. We need to continue to encourage the best minds to think about FRDA and to do research on FRDA, because no single scientist or group of scientists has a monopoly on good ideas and no single group of scientists can “do it all.” We have therefore established fruitful collaborations with key institutions accomplished in the types of scientific endeavor needed to stop and reverse FRDA. Another key area of collaboration is with elements of the pharmaceutical community. As FRDA scientists continue their promising progress toward therapeutic discovery, we need to put in place the support structure we will need to take such discoveries through subsequent steps. For example, when our scientists discover a drug that is beneficial, we need to accelerate the drug through pre-clinical research and clinical trials, drug development, and make it available to patients. FARA has helped make giant strides in establishing the required clinical structure. FARA has worked closely with NIH on the Idebenone trial and with the pharmaceutical companies involved in producing Idebenone. FARA is also supporting the seven centers across the United States that are refining the ataxia scales required to measure therapeutic progress in FRDA clinical trials. FARA is also consulting with experienced experts in drug discovery, drug development, clinical trials, and drug marketing to ensure that the appropriate mechanisms are in place to take beneficial therapies to patients when the time comes. This comprehensive research strategy is ambitious, but we need to be ambitious to slow, stop, and reverse this disease. With your continued, generous support, this strategy will be successful. FRDA scientists are increasingly certain they will conquer Friedreich’s ataxia. They are convinced it is no longer a question of “if” but, rather, a question of “when.” They tell us, too, that what we have all done together already has taken years off the road to “when.” We need to continue to build our momentum and drive this research across the finish line of treatments and a cure. FARA Officers and Board of Directors Ronald J. Bartek, President / Director / Co-Founder Marilyn E. Downing, Secretary / Director Terrence M. Downing, Treasurer / Director Paul Avery, Director John Cubbin, Director Thomas A. DeCotiis, Director Nicholas A. Johnson, Director Laura Kalick, Director Paul Marcotte, Director Edward Ramsey, Director Nancy Schneid, Director Dr. Earl Giller, Scientific Director Dr. Bronya J. B. Keats, Scientific Director Dr. Bernard Ravina, Scientific Director Mary Beth Kozmicki, Communication Advisor Mary Caruso, Development Advisor William Harnett, IT & Development Advisor William Krutzer, Development Advisor Sandy Lane, Development Advisor Samantha Litke, Development Advisor Marianne Wilcox, IT Advisor |
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