Dear friends,
Thanks to your incredible generosity, we are on track to fund over $8M in research this year. This work spans across our grant program, the Collaborative Clinical Research Network, FARA directed research like the TRACK-FA study, and institutional support of the FA Center of Excellence at Children's Hospital of Philadelphia and the FA Accelerator Program at the Broad Institute. These are projects that will provide new insights into FA and bring new therapies to the treatment pipeline. You brought the resources to fund these critical projects by cycling at rideATAXIA, hosting your own grassroots events, attending the Energy Ball, and giving to our most recent #GiftofResearch campaign (which has thus far earned over $195,000 of the $200,000 matching pledge!). I am so honored to be a part of a community that rallies together to make research progress a reality. Wishing you a happy holiday season and a new year with continued progress towards treatments and a cure for FA.
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Sincerely,
Jen
Jen Farmer,
Chief Executive Officer
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Design Therapeutics is developing gene targeted chimeras (GeneTACs™), which are designed to target the underlying cause of inherited nucleotide repeat expansion diseases to potentially modify the course of disease. The FA program is expected to begin a Phase 1 clinical trial in the first half of 2022 for people affected with Friedreich’s ataxia, age >18 years old.
You can watch the entire webinar by clicking on the video to the right.
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Reata Pharmaceuticals Receives Fast Track Designation From the FDA for Omaveloxolone for the Treatment of Friedreich’s Ataxia.
The Fast Track program is designed to accelerate the development and review of products such as omaveloxolone, which are intended to treat serious diseases and for which there is an unmet medical need. Fast Track Designation enables more frequent communication with the FDA and eligibility for FDA programs such as priority review and rolling review, if relevant criteria are met.
Warren Huff, Reata’s President and Chief Executive Officer said, “We remain committed to submitting our New Drug Application during the first quarter of 2022 and continue working with the FDA to secure regulatory approval as quickly as possible.” To read the full press release click here.
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Kyle Bryant Translational Research Award
Therapeutic activity of a hematopoietic stem cell delivered tissue penetrating peptide in a Friedreich's ataxia mouse model
Arturo Sala, PhD – Brunel University, London
The main goal of this study is to validate a new therapeutic approach using a mouse model of FA. The idea is to isolate hematopoietic stem cells from a patient's blood and modify them to carry a virus that produces a version of frataxin that is secreted and easily penetrates other cells. The frataxin-producing stem cells can then be reinfused into patients where they will be retained in the bone marrow and can migrate to damaged tissues and deliver frataxin.
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Broad Institute Friedreich's Ataxia Accelerator |
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FARA is funding a dedicated research effort at The Broad Institute called the Friedreich’s Ataxia Accelerator (FAA). This highly collaborative effort leverages multiple Institute platforms and capabilities to illuminate the biological underpinnings of FA, with the goal of producing more therapeutic possibilities.
The FAA team at the Broad Institute is simultaneously pursuing fundamental and applied research in the following three therapeutic areas: oxygen-inspired therapy, small molecule therapy, and genome editing.
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The Broad Institute FAA team recently hosted an event to learn more about the clinical and patient perspectives in FA featuring Dr. David Lynch and FA Advocates Alex Fielding, Annie Hamilton, and Nick Carbone. Click the video above to watch. |
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FARA Ambassador, Shandra Trantham featured in People Magazine |
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FARA Ambassador, Shandra Trantham was featured in People Magazine! You can read the feature piece by clicking on the image to the right.
Please join us in congratulating Shandra and thanking her for bringing such incredible awareness of FA.
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The latest Minutes of Science video is live!
Narrator Kate Walker explains the Friedreich’s Ataxia Clinical Outcome Measures Study (FACOMS) and its critical role in advancing FA research. Thank you to everyone who participates in this study and the staff at the CCRN sites who carry it out. Click the video to the left to watch.
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Please consider enrolling in this important study. Participants needed at all study sites, especially at the University of Minnesota.
TRACK-FA is a natural history study to investigate brain and spinal cord changes in individuals with Friedreich ataxia. If imaging differences in the central nervous system are correlated with progression of FA, these differences could be used in future clinical trials to monitor treatment outcome.
Eligibility criteria include:
- Age >5 years with genetically confirmed diagnosis of FA.
- GAA repeat expansion >55 in length on both alleles of the FXN gene or GAA repeat expansion on one allele and another mutation type on the other allele (e.g., point mutation, gene deletion).
- Age of disease onset less than 25 years.
- Disease duration less than 25 years.
- Able to undergo MRI
- Participants can be involved in an investigational drug trial and still be eligible for the TRACK-FA study.
There are 3 study visits, each approximately 12 months apart. At each study visit, participants will be asked to complete neurologic and functional testing, blood draw and brain and spinal cord MRI scan
Sites are open for enrollment at University of Minnesota (Minneapolis), University of Florida (Gainesville), Children’s Hospital of Philadelphia (Pennsylvania), RWTH Aachen University (Aachen, Germany), University of Campinas (Brazil), and Monash University (Victoria, Australia). To contact a study coordinator, please click here.
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*This study has new expanded inclusion criteria*
27 FA participants and 17 unaffected siblings are needed to fill this study by March 2022. The outcomes of this study are critical for allowing remote data collection during future clinical trials.
Participants are needed at all clinic sites in Baltimore (Johns Hopkins), Boston (MGH), Chicago (University of Chicago), Los Angeles (UCLA) or Portland (Oregon Health & Science University).
For additional eligibility criteria, please contact study coordinator Hannah Casey (hannahcasey@uchicago.edu) or (773) 702-4610. Please specify your city of interest for enrollment - Baltimore, Boston, Chicago, Los Angeles or Portland (OR).
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If you are selected to participate in this study, you would complete 2 in-person study visits, each lasting 2 days, over a 12-week study period at CHOP and Penn. During the study period, you may complete blood testing, exercise testing, administration of a dietary supplement, MRI scans, oral glucose tolerance testing (with a non-FDA approved stable isotope product) and optional muscle biopsies.
Participants will be compensated for time in the study, and participants will be provided with a recumbent bike, which they can keep after study completion.
To learn more about the study and determine your eligibility, contact the study coordinator, Anna DeDio, by email dedioa@email.chop.edu or by phone
(267) 425-1998.
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Anyone considering participating in a clinical trial should discuss the matter with his or her physician. FARA does not endorse or recommend any particular study. |
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The goals of the FA Global Patient Registry (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.
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The Pathway
You can help fund research progress all year by joining our monthly giving program, The Pathway. When you join The Pathway with a monthly donation of $10 or more, you are joining a community invested in setting a path to treatments and a cure for FA through research. Click the image to give today.
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