The Friedreich’s Ataxia Research Alliance (FARA)
is a national, public, 501(c)(3), non-profit, tax-exempt organization dedicated to the pursuit of scientific research leading to treatments and a cure for Friedreich’s ataxia.
FARA’s Mission is to marshal and focus the resources and relationships needed to cure FA by raising funds for research, promoting public awareness, and aligning scientists, patients, clinicians, government agencies, pharmaceutical companies and other organizations dedicated to curing FA and related diseases.
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:
•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.
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:
•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:
•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:
•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)
FARA focuses on grant making for FA research and building collaborations with organizations dedicated to advancing treatments for FA. Due to the progressive nature of the disease and the promise of treatments in development, there is urgency to our efforts. Directing attention and resources to FA research and partnering with others that share this commitment, FARA believes it can help bring forward effective treatments and a cure for FA.
“With their grassroots fundraising efforts on behalf of FARA, FA families and the communities that support them are powering FA research forward. We are in the treatment era for FA research not only because of our dedicated scientists but because of FA families who ask themselves- ‘What can I do with my two hands and my voice to make this situation better?’ and then answer that inquiry with action. To everyone who has hosted or participated in a fundraising event- athletic, dinner/ dance, backyard party, or holiday boutique, thank you for being a part of the united effort moving us that much closer to treatment that much faster.” -Felicia DeRosa, MPA, Program Director/FARA
- Raise funds for advancing scientific research in FA.
- Facilitate a competitive and highly regarded grant making program that supports greater than 1 million dollars in research annually and focuses on translational and clinical research (moving discoveries through development to clinical improvements for patients).
- Manage an FA patient registry and support the development of a collaborative clinical network for FA clinical trials.
- Foster public-private partnerships between academic research investigators and pharmaceutical companies.
- Promote the collaborative exchange of information within the scientific community through conferences and networks.
- Work cooperatively with government entities and the other organizations that support scientific research aimed at treatments for this disorder.
- Rally patients, patient families, scientific investigators, healthcare providers, and others to be supporters and advocates for scientific advancements that will lead to treatments and a cure.
FARA has funded nearly $11 million in research since 2005. To learn more about the efforts of and advancements in research, please visit the Stephanie’s Hope research page.
“Acting alone, there is very little any of us can accomplish. Acting together, there is very little we will NOT accomplish!” -Ron Bartek, FARA President and Founder