The Friedreich's Ataxia Research Alliance (FARA) is a national, public, 501(c)(3), non-profit, tax-exempt organization dedicated to curing Friedrech’s ataxia (FA) through research. FARA grants and activities provide support for basic and translational FA research, pharmaceutical/ biotech drug development, clinical trials, and scientific conferences. FARA also serves as a catalyst, between the public and scientific community, to create worldwide exchanges of information that drive medical advances.
FARA was founded in September 1998 by a group of patient families and three of the world’s leading FA scientists — Drs. Rob Wilson, Bronya Keats, and Massimo Pandolfo. It was staffed and managed as an all volunteer organization until late 2005. FARA's Leadership and numerous patient families and friends have raised critical funds over the years to support FA research. Thanks to the brilliant and committed efforts of many FA scientists, we now understand the cause of FA and specific mechanisms leading to damage in FA patients. These understandings allow for more targeted approaches to treatment which are currently being developed for clinical trials.
FARA’s Decade of Progress
In only ten years, because of the focused efforts and dedication of so many individuals, families, organizations and institutions, FARA and the FA research community’s accomplishments include:
Assembled and grew the FA scientific community (details) |
- First-of-kind International Scientific Conferences on FA.
- 1999 (80 scientists, 1 other advocacy group, 0 drug companies)
- 2003 (100 scientists, 4 other advocacy groups, 4 drug companies)
- 2006 (150 scientists of 180 applicants, 6 advocacy groups, 6 drug companies)
- Organized issue focused summit meetings engaging additional scientific disciplines and disease groups.
- Biomarker Summit 2006
- Cardiac Summit 2007
- Mitochondrial Summit 2008
- Tripled the number of scientists working on FA.
- Grant proposals received per year grew from 1-2 to more than 30; Current grant funding average $1.5 million/ year.
Supported basic, translational and clinical research with increasing emphasis on moving to clinic (details) |
Achieved clinical trial status within 7 years of FA gene discovery. In 2008, three promising FA clinical trials underway with several others in late stages of preparation (details) |
- 2001 Phase I Idebenone- Complete
- 2005 Phase II Idebenone- Complete
- 2008 Phase III Idebenone- Enrolling Now in US and Europe
- 2008 Phase I/II Deferiprone- Enrolling Now in Europe
- 2008 Phase I A0001- Enrolling Now in healthy subjects in US
Established essential clinical research infrastructure to support clinical trials (details) |
- Patient Registry
- Collaborative Clinical Research Network in FA
- Natural history studies
- Cell and DNA repositories
- Clinical outcome measures (scales)
Established, nurtured and grew powerful public-private partnerships, becoming a recognized model in the field of collaborative research (details) |
- NIH: Rapid Access to Intervention Development (RAID) program supported development of FA drug; variety of NIH grants awarded to FA research; NIH supported FA scientific conferences; FARA collaborated with NIH-sponsored FA cell repository
- U.S. Congress: FARA testified before congressional appropriators encouraging redoubled funding for NIH medical research
- Pharmaceutical Industry: FARA collaborated with 6 drug companies in developing drugs; initiated “joint-venture philanthropy” supporting and investing in pharmaceutical research
- Other Non-profit Organizations: FARA collaborates and co-funds research with a growing number of non-profit partners including the Muscular Dystrophy Association, National Ataxia Foundation, American Heart Association, Friedreich’s Ataxia Research Association, Ataxia UK, Go-Friedreich’s Ataxia Research, and The Spanish Federation of Ataxia (FEDEAS)