Program areas at FARA
Education, awareness & outreach programsfriedreich Ataxia (fa) is a rare disease, affecting 1 in 50,000 individuals. Fara is dedicated to advocacy and raising awareness for fa. Fara has utilized both traditional and social media strategies to bring greater awareness to fa in the general public and to engage and educate the fa community. For example, fara conducted a social media campaign that encouraged community participation leading up to rare disease day and fa awareness day. Fara and the national Ataxia foundation (naf) also partnered on several advocacy initiatives relevant to the Ataxia community. Mostly notably, the efforts of the two organizations resulted in fa being added to the congressionally directed medical Research program (cdmrp) at the department of defense in fiscal year (fy) 22, resulting in six fa researchers being recommended for funding totaling over $14 million. Fara and naf were also successful in getting a resolution passed declaring september 25, 2022 national Ataxia awareness day (s.res 850) and hosting the fourth united against Ataxia hill day. In 2022, 78 congressional meetings were held with 88 fara and naf advocates participating from 31 states and the district of columbia. The primary ask was to broaden the definition on the cdmrp from "friedreich's Ataxia" to "hereditary Ataxia", which would provide additional funding for Ataxia researchers in fy23. President biden signed the fiscal year (fy) 23 congressional budget on december 29 which listed "hereditary Ataxia" under the cdmrp's peer reviewed medical Research program. The fara ambassador program, which was launched in 2011 with >20 participants had a year of continued growth and activity in 2022. The program now includes >80 participants. The mission of the fara ambassadors is to be positive, supportive, peer representatives for the fa community; actively raising awareness and funds for fara. The fara ambassador program members organize service project teams including:1. A blog team that facilitates weekly meet the community interviews with people living with fa and a monthly meet the researcher interview. 2. A card writing team that writes personalized cards to send to the various stakeholders and community members throughout the year to say thank you or to offer encouragement. 3. A monthly peer zoom hangout groups for the greater fa community to attend and connect with one another online (one is geared to adults, and one is for teens with fa). 4. A social media team that generates engaging content in support of fara initiatives and campaigns. 5. A speaking team where participants practice telling their story living with fa on behalf of the organization.ambassadors and other community representatives connected with many of fara's academic and industry partners as well as regulatory agencies like the food and drug administration by serving on patient panels at 12 events. Ambassadors also participated in education programs targeting medical professionals, e.g., speaking to trainees in genetic counseling programs and medical students. For more information visit curefa.org/ambassadors.group fara hosted several education and engagement initiatives in 2022. For example. Fara continued a video series to educate fa families and supporters on the fara grant program. Each episode explains a type of Research funded by fara in a 5-minute animated video, narrated by an fa community member. Fara released six minutes of science videos in 2022. Fara also offered the flash talks series in may which allowed young investigators to explain their Research in layman terms and to answer questions in a virtual format. In october, fara published a guide for clinical trials participants. This 12-page guide contains information on the different phases of clinical trials, terms to know, clinical trial etiquette, safety monitoring, and a guide to informed consent. It is intended to help people understand the drug development process so that they can make an informed decision on participating in clinical trials.
Clinical Research infrastructurein addition to Research grants, fara funds the ongoing development of domain resources in the form of vital clinical Research infrastructure. Clinical Research infrastructure refers to the resources needed to facilitate any type of Research, including clinical trials that involve patients. These resources include programs like: Friedreich's Ataxia global patient registry: the Friedreich's Ataxia global patient registry (fagpr) is the only worldwide registry of Friedreich's Ataxia patients. The goals of the fagpr are to collect information on all fa patients in one registry, to develop the registry into a powerful resource for Research, and to engage the fa community in studies aimed at advancing our knowledge of fa and the treatments being developed. Fara partners with international patient advocacy organizations through a governance board to ensure multi-stakeholder engagement and oversight of the fagpr. More than 1000 individuals with fa are enrolled and fagpr was used to recruit for several clinical trials and many clinical Research studies. To learn more, visit curefa.net/registry. Collaborative clinical Research network in fa (ccrn in fa): the ccrn is an international network of 14 clinical Research centers (united states, canada, australia, new zealand, and india) that work together to advance treatments and clinical care for individuals with Friedreich's Ataxia. Having such a network means that there are trained physicians and Research coordinators ready to do clinical Research studies and trials. Also, this network is backed by a data coordination center that facilitates all aspects of data collection, database management, and statistical analysis of study data. To learn more and review a list of Research publications, visit curefa.org/network.htmlthe ccrn leads a natural history and clinical outcome measure study, called facoms. Facoms has more than 1400 individuals with fa enrolled and the longitudinal data has led to a better understanding of symptoms and progression of disease. Furthermore, the ccrn and facoms have validated clinical outcome measures and the fars scale, studied speech, vision and hearing, launched biomarker studies, established dna and rna repositories, and provided many blood samples to researchers around the world. The ccrn in fa investigators have been involved in >12 clinical trials including a few that were designed and conducted solely through network sites. One clinical trial conducted through ccrn centers has led to the first approved medication or treatment of fa, and the natural history study data was also critical in designing the trial and understanding the meaningfulness of the trial outcomes.the facoms study and the efacts natural history study (european union countries) have been conducted in parallel, for over a decade, with many similarities. In 2022, fara brought together investigators of both facoms and efacts to combine efforts into a single global consortium with a unified natural history and clinical Research infrastructure. The new fa global clinical consortium and a global natural history study protocol, the unifai study, are being established to continue to improve the panel of outcome measures that can be used in fa clinical trials, with the goals to better organize and harmonize the natural history ecosystem and allow for a complete view of disease progression of fa. Additional information about fara's programs in 2022 can be accessed via the annual report at: curefa.org/financials
Workshops, symposia, & conferencesfara hosted a hybrid in-person and virtual single-day educational symposium for >200 patients and caregivers in king of prussia, pa (september 25, 2022). This symposium provided an opportunity to educate the patient/family community on Research advances, progress on clinical trials and was a unique forum for patients and researchers to engage and learn from each other's experiences and perspectives. International congress for Ataxia researchin partnership with the national Ataxia foundation and Ataxia uk, fara also organized and sponsored the international congress for Ataxia Research in november 2022. Over 450 people attended, including those from academia, industry, and patient advocacy groups. More than one third of the participants were junior investigators which supports the goal of bringing new talent to the field and growing the next generation of Ataxia researchers. In addition to the opportunity to view nearly 300 presentations, most attendees reported making new contacts, which stimulates collaboration and partnerships. Key takeaways from the meeting included new insights into the disease mechanisms in fa, potential new biomarkers, and the use of digital assessments of ataxia.gene therapy in fa meetingin 2022, fara organized a meeting with the food and drug administration (fda) and over a dozen companies working on gene therapy and gene editing programs for fa. The meeting resulted in alignment with the regulatory agency on several aspects of these innovative approaches.