The Friedreich's Ataxia Biomarker Meeting will be held on September 28th, 2018 at the Center for Advance Medical Simulation and Learning in Tampa, FL. The purpose of the Biomarker meeting will be to review the state of biomarker and novel endpoint development for Friedreich's ataxia (FA). Our goal has been to support research to identify new biomarkers that might be appropriate for specific contexts of use (pharmacodynamics/response markers, prognostic and monitoring markers), and to prioritize which biomarkers should be moved into larger studies and for use in trials. The meeting will include sessions on systemic, CNS and cardiac biomarkers. FARA's first biomarker meeting in 2015 resulted in the initiation of three protocols to pilot new biomarkers, data from which will be presented this year. The goals of this 2018 biomarker meeting will be to assess our progress in biomarker development and, based on findings, prioritize specific areas of biomarker and endpoint work going forward.
The meeting will be held in conjunction with FARA's signature gala event- the FARA Energy Ball. This multi-day experience kicks off with an educational symposium the evening of Thursday, September 27 that includes research updates from the field's leading scientists as well as an interactive patient panel. The event series will culminate with a high energy gala on Saturday, September 29 complete with plated dinner, live entertainment, and a silent and live auction rich with exclusive items and experiences.
We hope you will be able to attend.
Questions? Please contact firstname.lastname@example.org
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.
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 http://www.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 and 2016.
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.