Advocacy

Headquartered in the Washington, D.C. area, FARA's approach has been to work directly with the government agencies to build partnership, collaboration and cooperation. FARA has testified before the congressional appropriations committee about FA and FARA, but we have not approached members of congress to “earmark research dollars” for FA research. Rather, FARA has appealed to Congress to give the government agencies more money to conduct medical research. FARA believes in the philosophy that "a rising tide lifts all boats," and the FA boat will benefit and compete for the additional dollars on the exceptional merits of our scientists.

National Institutes of Health (NIH) (www.nih.gov)

The NIH is the premier US federal government entity responsible for biomedical research. The NIH conducts intramural research programs within its own institutes and funds extensive research at laboratories throughout the United States and around the world. NIH conducted phase I and phase II of the FA Idebenone trial at its own expense and has funded millions of dollars more in FA research. FARA also contributed to a successful application to the NIH’s Rapid Access to Interventional Development (RAID) program. Through the RAID program, the NIH provides resources for drug development.

The NIH consists of over twenty different institutes devoted to various types of disease. The NIH Institute of primary interest to the FA community is the National Institute of Neurological Disorders and Stroke (NINDS). However, because of the broad spectrum of FA symptoms and patient population, several other NIH institutes participate in FA research activities (for example, the Institutes of Child Health and Development; Heart, Lung and Blood; Diabetes and Digestive and Kidney Diseases; and the Institute on Aging.

National Institute of Neurological Disorders and Stroke (NINDS) (www.ninds.nih.gov)

The National Institute of Neurological Disorders and Stroke (NINDS) is the leading NIH institute supporting biomedical research on disorders of the nervous system. FARA's president serves on the NINDS Advisory Council and participates in the Council's quarterly meetings, where the Council advises the NINDS Director on matters of Institute policy and reviews all applications for NINDS research grants. FARA also works closely with the NINDS leadership to advance FRDA clinical trials and enhance the Institute's two-way communications with the patient and scientific communities.

Department of Defense (DOD)

Department of Defense (DOD) receives funding for medical research. Most of the DOD funds are directed toward diseases that the military medical personnel would be treating. One area is for military families and retirees (research for cancer, heart, etc.). Another area of research that DOD studies is more specific to war related/combat injuries (brain injury, gun shot wounds, and paralysis for example) that military hospitals and personnel would see more often than in the civilian medical setting.

Finally, DOD devotes some funding to medical research aimed at keeping healthy soldiers healthy or making them even healthier. FARA has found potential in these last two categories (combat injuries and healthy soldiers) and has been actively exploring that potential. Because the compounds FARA is advancing are showing strong signs of potentially improving mitochondrial function (increasing energy production and reducing oxidative damage) in FA patients, military researchers are interested in the compounds' potential for improving mitochondrial function in seriously wounded soldiers "right away" before they can be evacuated to intensive care. They are also interested in the potential of these compounds in making healthy soldiers even more capable.

Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) (www.hrsa.gov/heritabledisorderscommittee)

As therapies for treating FA are on the near horizon, FARA is committed to seeing such treatments given to patients at the earliest point so as to maximize benefit. At the suggestion of Dr. Story Landis, Director of the Neurological Institute of the NIH, FARA has begun to explore the newborn screening process and how this might be applied to FA. FARA has been working with a team of investigators at the Mayo Clinic in Minnesota led by Drs. Devin Oglesbee and Grazia Isaya to develop a newborn screening test. The ACHDNC’s purpose is to provide to the Secretary, US Department of Health and Human Services, evaluations and recommendations regarding proposed newborn screening tests as well as policies to support their implementation and counseling in State and local health agencies.

US Congress

As a patient advocacy group, FARA raises public awareness of Friedreich's ataxia through a variety of activities, including congressional testimony. FARA works regularly with congressional points of contact, tracking and encouraging progress in the development of the NIH budget. FARA also keeps abreast of the legal, moral, and ethical implications that arise in conducting research.

Center for Acadiana Genetics Established

"This is a unique opportunity to study the French Acadian (Cajun) population where Friedreich's ataxia occurs at a rate 2.5 times higher than the national average."
— Dr. Bronya Keats, Center Director

Spearheaded by then US Congressman Billy Tauzin (R- LA) and the Louisiana State University Health Sciences Center, the Center for Acadiana Genetics and Hereditary Health Care was established in New Orleans. The US Congress has appropriated about $4 million for the Center, which links a school of medicine, a biomedical research center, hospitals, rural clinics, and a strong telecommunications network to provide urgently needed health services and education regarding genetic diseases. It also conducts vital research into hereditary neurodegenerative disorders like Friedreich's ataxia.