Assembled and grew the FA scientific community
When FARA was founded there were few scientists working on FA and there was no organization or entity behind bringing new researchers to FA or assembling the FA research community to promote growth and collaboration in the field.
- First-of-kind International Scientific Conferences on FA.*
- 1999 (80 scientists, 1 other advocacy group, 0 pharma companies)
- 2003 (100 scientists, 4 other advocacy groups, 4 pharma companies)
- 2006 (150 scientists of 180 applicants, 6 advocacy groups, 6 pharma companies)
- 2011 (200 scientists from 15 countries and 25 post-doctoral scholarships awarded, 10 advocacy groups and 13 pharma and biotech companies participating or sponsoring)
- Organized issue focused summit meetings or task forces engaging additional scientific disciplines and disease groups to advance FA science in specific areas.
- Biomarker Summit 2006
- Cardiac Summit 2007 and 2010
- Mitochondrial Summit 2008 Cell Model Task Force 2010-2011
- Animal Model Task Force 2010-2011
- Cellular & Mouse Model Meeting 2012
- Gene Therapy Meeting 2013
- Biomarker Meeting 2014
* For additional information about these conferences - http://www.curefa.org/conference
Supported basic, translational and clinical research
FARA has established a competitive, responsive, and highly regarded grant program for funding FA research worldwide. The sun never sets on FA research. In the early years many of the research projects FARA funded focused on establishing the basic understanding of FA at the genetic, protein and cellular level. In recent years FARA’s grant program has grown and expanded to include development of animal and cellular models, drug discovery and development and clinical trials. In 2014, FARA funded >30 FA research projects; $4 Million in grant award commitments. (More about FARA Grants)
Achieved clinical trial status within 7 years of FA gene discovery
In 1996, the gene and gene mutation that causes FA was identified. This was a huge milestone to understanding the cause of the disease. FARA’s founders viewed this discovery as a critical component to advancing research to development treatments for FA. In 1998, when FARA was founded there were no treatments or drugs on the horizon.
Clinical trials are an important milestone in the advancement of novel therapies because they are the study of the drug in humans. It is important to remember that clinical trials are still research experiments and while we are excited when a therapeutic candidate reaches the clinical trial stage of development (phases 1-3) it does not mean that it will be beneficial or successful as a treatment. Clinical trials can fail to meet our anticipated outcomes for many, many reasons however every study contributes significant knowledge and experience to our efforts to bring treatments to FA. Below is a partial list of trials completed and ongoing. For more information about these therapeutic candidates and outcomes of studies visit our Research Pipeline.
|Clinical Trial Status|
Established essential translational research tools
FARA has established essential translational research tools and clinical research infrastructure to accelerate and inform the drug development and clinical trials.
- Drug Development Tools - FARA has funded discovery scientists working on animal and cellular models as well as development and validation of high-throughput assays for drug discovery. We are grateful to the discovery scientists who have worked hard to bring us these important assets and continue to give to the community by sharing their results, knowledge, expertise and resources.
- Patient Registry – FARA’s patient registry is the largest FA registry in the world. It is considered a model for other rare disease advocacy registries.
- Collaborative Clinical Research Network in FA - The Collaborative Clinical Research Network in Friedreich's Ataxia (CCRN in FA) is an international network of clinical research centers that work together to advance treatments and clinical care for individuals with Friedreich's ataxia. The network collaborates with pharmaceutical companies, government agencies and other research centers and the patient community to facilitate clinical research and trials needed to identify new therapies. The CCRN in FA has also collected natural history data on a large cohort of FA patients, developed and validated the Friedreich’s Ataxia Rating Scales (FARS) and the functional measures being used in FA clinical trials and is testing and evaluating a variety of FA biomarkers.
Established, nurtured and grew powerful public-private partnerships
FARA has 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
- Institute of Medicine: FARA representatives provided testimony and served on the working committee for the IOM’s consensus report on Rare Diseases and Orphan Products: Accelerating Research and Development.
- Pharmaceutical Industry: FARA collaborated with 18 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)