On the day following FARA's incorporation, the new organization submitted a grant application to the National Institutes of Health (NIH) for the first International Scientific Conference on Friedreich's Ataxia convened April 1999.
The National Ataxia Foundation (NAF), the Friedreich’s Ataxia Research Alliance (FARA), and Ataxia UK held the inaugural International Congress for Ataxia Research (ICAR) on November 1-4, 2022 in Addison TX.
The international scope of ataxia research was well represented by over 458 conference attendees from nineteen countries. We are grateful for such meaningful participation from all aspects of the research community including 177 attendees from academia, 111 attendees from industry, 156 junior investigators, and representatives from nine patient advocacy groups.
We were energized by the dynamic exchange of ideas during the oral and poster presentations as well as during the flash talks, mentoring sessions, and workshops. We hope attendees had the opportunity to foster collaborative relationships and left with a renewed sense of purpose in their work.
Click HERE to view the Abstract Book.
Click HERE to view the conference Program.
The FARA 2020 Biomarker & Clinical Endpoint Meeting was held virtually on September 24th and 25th, 2020.
The purpose of the meeting was to review the state of biomarker and novel endpoint development for Friedreich's ataxia. The focus was on pharmacodynamic and monitoring biomarkers both for translational and clinical applications, as well as clinical outcome assessments. FARA’s goal is to have a toolbox of biomarkers and outcome assessments that can be used in various settings and for diverse treatment approaches. Highly innovative biomarkers or outcome assessments at early stages of development were considered, as well as research in later or validation stages.
The meeting agenda can be viewed here
2019 International Ataxia Research Conference (IARC)
Nov 14th - 16th, 2019 Washington DC
The international scope of ataxia research was well represented by over 430 conference attendees from twenty-one countries! We are grateful for such meaningful participation from all aspects of the research community including 150 attendees from academia and regulatory, 140 attendees from industry, 100 junior investigators and representatives from seventeen patient advocacy groups.
We were energized by the dynamic exchange of ideas during the oral and poster presentations as well as during the flash talks and the mentoring session. We hope the attendees had the opportunity to foster collaborative relationships and left with a renewed sense of purpose.
Videos from the conference:
1 - Coverage of IARC 2019
2 - Interview with Jen Farmer
3 - Interview with Dave Lynch and Jane Larkindale
4 - Interview with Rob Wilson and Grazia Cotticelli
5 - Interview with Dave Corey and Merek Napierala
6 - Interview with Layne Rodden and Sanjay Bidichandani
7 - Interview with Michael Clark and Mark Payne
8 - Interview with Louise Corben and Adam Vogel
9 - Interview with Kathy Mathews and Antoine Duquette
10 - Interview with Dave Lynch
11 - Interview with Oliver Griso
12 - Interview with Helene Puccio and Massimo Pandolfo
13 - Interview with Ian Blair and Clementina Mesaros
14 - Before There Was a FARA
15 - Maybe This Won't Take Forever
16 - Get Us Together, It's Never Been Done Before
400+ scientists/ participants
15+ pharmaceutical companies and 7 advocacy groups
The conference was held in Pisa, Italy
2017 Conference Summary and Thanks to Partners and Sponsors
300+ scientists/ participants, 15 pharmaceutical companies and 7 advocacy groups. The conference was held in Windsor, England.
2015 Conference Photos and Thanks to Partners and Sponsors
200 scientists/participants from 15 countries, 7 pharmaceutical companies and 9 advocacy groups The conference was held in Strasbourg, France.
2011 Conference Photos and Thanks to Partners and Sponsors
150 scientists from 12 countries,
6 pharmaceutical companies and 6 advocacy groups
100 scientists from 12 countries,
4 pharmaceutical companies and 4 advocacy groups
80 scientists from around the world,
zero pharmaceutical companies and one advocacy group
Research Abstracts from meetings can be found in the right hand column.
In the interim time between its International Conferences on Friedreich’s Ataxia, FARA helped support the Ataxia Investigators Meeting held in 2006, 2008, 2010, 2012, and 2014.
FARA has also convened summit meetings to focus on significant areas of FA research such as endpoints, cardiology and mitochondrial function. Such meetings facilitate in-depth discussions among experts and advance a specific research need.
Patient Preferences in Drug Development
In June of 2017, FARA held an externally led patient focused drug development meeting. This meeting, co-organized by the Friedreich’s Ataxia Research Alliance, Muscular Dystrophy Association, the National Ataxia Foundation and the CureFA Foundation, marked the first time patients and families affected by FA had the opportunity to speak directly to the FDA and share their experiences in their own words. The Friedreich’s ataxia (FA) Patient Focused Drug Development (PFDD) meeting was set up with the goal of giving feedback to the U.S. Food and Drug Administration (FDA) and drug developers about challenges and burdens patients and families have experienced with FA, and what is most important to the community in evaluating potential new treatments for the disease. More information about the meeting, and the “Voice of the Patient” report from the meeting is at curefa.org/patient-focused-drug-development
In 2014, FARA partnered with the National Center for Advancing Translational Sciences (NCATS, part of the National Institutes of Health) to organize a meeting to determine what studies should be done to develop high quality biomarkers for FA. Approximately 50 people attended the meeting, including experts in FA, experts in biomarker development and companies with an interest in developing therapies for FA. The meeting was set up to encourage discussion, and there was lively debate between the participants. Many possibilities were suggested — some which had never been considered in FA previously, others where significant data exist ed. Discussion ranged from biochemistry to electrophysiology to imaging studies to gait analysis, while always remaining focused on what could be measured in important tissues to FA, and what those measurements would mean. Experts from outside of the FA world suggested technologies not previously studied in FA, while FA experts compared and contrasted with known FA data to determine which would have the highest probability of success.
The meeting has provided FARA with a priority list of potential biomarkers that might be relevant to FA. We now need to start both pilot studies to see if biomarkers not previously considered in FA might be feasible, and longer-term studies to see how markers with some evidence might change over time in patients. FARA will partner with companies working in these areas to fund and run these studies, and the resulting data will be available to everyone in the community. Development and validation of biomarkers should both accelerate progress in developing new therapies, and make the field even more attractive to drug developers.
Follow up biomarker meetings to update the field on progress and determine next steps were held in 2015, 2016 and 2018.
In 2012, 2013 and 2016 meetings organized by FARA brought FA scientists from around the world who are working on mouse models, induced pluripotent stem cell (iPS) models of FA, and gene therapy approaches. Researchers presented their latest data (not yet published or publically available) to help address gaps in the current models and share new discoveries and technologies. Cat Lutz from JAX labs reported on a significant breakthrough in a collaborative project, funded by FARA, to produce extensive characterization of several different FA mouse models. Several other investigators reported on ongoing efforts to continue to improve upon models. For example, the current mouse models are still relatively mild compared to humans (symptoms are mild and slow to appear) – if newer models had lower levels of frataxin or more cardiac symptoms, and showed symptoms earlier in life, our ability to screen drug candidates would be enhanced. As a result of these meetings, a Mouse Model Task Force was formed that meets regularly to advance this initiative.
The availability of well-characterized cellular models is highly beneficial for drug discovery and testing, as well as for developing gene and stem cell therapies. To facilitate communication, cooperation, and collaboration among researchers various types of cellular models, especially iPS-based neuronal and cardiac models, the Cellular Models and Cell Therapy meeting was held in Chicago in March, 2010. One of the key outcomes of the meeting was consensus on the need for reproducible, stable, well-characterized FA iPS cell lines that are appropriate for research (including drug screening), maintained in a reputable facility, and distributed among researchers.
More than 100 FA researchers and our advocacy partners from around the world gathered July 15-17, 2009 for the FA Therapeutics Symposium in Philadelphia, PA. Presentations and discussions highlighted:
- progress in the development of previously identified therapeutic candidates, such as HDACI and TAT-Frataxin results from clinical trials including the Phase I study of A0001 and Phase III of Idebenone
- recent discoveries that point to new therapies
- advancements in new cell models and drug discovery and development assays
- clinical research including biomarker studies and new clinical outcome measures
FARA co-sponsored a Mitochondrial Summit with the Muscular Dystrophy Association, on May 20-21, 2008, to share and discuss approaches, insights, and mechanisms that suggest new therapeutics for mitochondrial neurodegenerative diseases.
In 2007, a Cardiac Summit was convened of leading cardiologists and researchers gathered to discuss FA related cardiology issues. One of the primary outcomes of this meeting was the documentation of gaps in knowledge regarding cardiac disease in FA and the assignment of research teams to begin work in these areas. To further support these efforts, FARA and the American Heart Association formed a new partnership to co-fund grants. Proceedings of this meeting can be found here.
In a continued effort to bring more research and attention to cardiac involvement in FA, the 2nd FA Cardiac Summit was held on June 11, 2010. Twenty participants were invited, representing diverse areas of basic science, clinical research, and cardiology. One of the goals of the meeting was to identify clinical and basic science questions that are important to solve to better understand cardiac disease in FA, such as establishing hypotheses and explanations for how mitochondrial dysfunction in FA might lead to cardiac disease in FA. One outcome of the meeting was overwhelming consensus and support for achieving improvements in cardiac care for individuals with FA. This will be done through the development of standards of care and further development of the cardiac expertise at our Collaborative Clinical Research Network in FA sites and beyond.
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