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FARA Funded Research

Your generous support has funded all the research listed below.


For more information on FARA-funded research & scientists, please visit FARA Supported Research, Active Clinical Trials and the Featured Scientist.

Global, multi-stakeholder, consortium launched to study neuroimaging biomarkers for Friedreich Ataxia

A Natural History Study to TRACK Brain and Spinal Cord Changes in Individuals with Friedreich Ataxia (TRACK-FA)

Downingtown, PA (September 24, 2020) - The Friedreich’s Ataxia Research Alliance (FARA) and partner organizations proudly announce an international consortium of academic, industry, and patient advocacy partners to launch a natural history study to TRACK brain and spinal cord changes in individuals with Friedreich’s ataxia (FA). Friedreich’s ataxia is a rare debilitating, life-shortening, degenerative neuro-muscular disorder. About 5,000 people in the United States and 15,000 worldwide live with FA.

The TRACK-FA study is the most extensive worldwide longitudinal, multi-center neuroimaging study in FA with 200 children and adults (and ~100 matched controls) and three assessments (baseline, 12-month, and 24-month follow-up). The TRACK-FA study aims to improve understanding of the natural disease history of FA (specifically, related to changes in the brain and spinal cord), validate neuroimaging measurements in FA to deliver a set of trial-ready biomarkers, and develop a comprehensive database to facilitate ongoing community research and discovery. The study is a collaboration between six international sites, including, Monash University (Australia), University of Minnesota (USA), Aachen University (Germany), University of Campinas (Brazil), University of Florida (USA), and the Children’s Hospital of Philadelphia (USA). FARA (USA) and several industry partners will provide input on study design, endpoints, and monitoring. The goal is to begin enrolling before the end of 2020, as individual sites are able to return to clinical research activities. Updates on opening enrollment will be shared through each of the study sites, FARA and clinicaltrials.gov, clinicaltrials.gov/ct2/show/NCT04349514

FARA CEO, Jennifer Farmer said, “TRACK-FA is a great example of public-private partnership and research advancement in the pre-competitive space. As we all need better tools to understand and measure what is happening in the FA brain and spinal cord, FARA is proud to support this international consortium. The goal of TRACK-FA is to deliver such tools for future clinical trials.”

Professor Georgiou-Karistianis from Monash University states, “We are very excited that this international collaboration brings together significant expertise in FA from across the globe. For the first time, TRACK-FA will validate neuroimaging biomarkers so that they’re ready to be pushed through the drug development pipeline. TRACK-FA provides real promise to accelerate the effort for new treatments in this rare disease.”

The study has been registered with ClinicalTrials.gov, clinicaltrials.gov/ct2/show/NCT04349514 and provides more information about TRACK-FA and a list of sites with contact information.

Emerging therapies in Friedreich's Ataxia

This review covers past and emerging therapies for Friedreich's Ataxia (FRDA), including antioxidants and mitochondrial-related agents, nuclear factor erythroid-derived 2-related factor 2 (Nrf2) activators, deuterated polyunsaturated fatty acids, iron chelators, histone deacetylase (HDAC) inhibitors, trans-activator of transcription (TAT)-frataxin, interferon gamma (IFNγ), erythropoietin, resveratrol, gene therapy, and anti-sense oligonucleotides (ASOs), among others. While drug discovery has been challenging, new and exciting prospective treatments for FRDA are currently on the horizon, including pharmaceutical agents and gene therapy. Agents that enhance mitochondrial function, such as Nrf2 activators, dPUFAs and catalytic antioxidants, as well as novel methods of frataxin augmentation and genetic modulation will hopefully provide treatment for this devastating disease.

Read the Entire Article Here

The Friedreich’s Ataxia Accelerator will apply genomics tools to promote discovery of new treatments

A new research and drug discovery effort at the Broad Institute of MIT and Harvard, The Friedreich's Ataxia Accelerator, will help build a community of researchers at Broad focused on learning more about the molecular mechanisms underlying FA with the ultimate goal of developing therapeutic strategies for the disorder.

FARA is excited to have the Broad Institute investigators on our team applying novel ideas and technologies and bringing new expertise to the FA research community. We believe that building a collaborative community of partners and investing in research are essential for the discovery of treatments for FA.

Read the Entire Article Here

Ataxia: Hope starts with measurement

FARA and AFAF (our advocacy partner in France) awarded a research grant, from rideATAXIA raised funds, to Dr. Corben and colleagues to develop and test a digital spoon as a way to measure upper limb function in FA. With that original grant award the team demonstrated that this device was able to measure function reliability and accurately in individuals with mild and severe symptoms of ataxia. With this new funding they can take this device and other devices for measuring ataxia to the clinic so that we have improved tools for monitoring outcomes and treatment responses in our natural history study and clinical trials.

Read the Entire Article Here

Frataxin gene editing rescues Friedreich's ataxia pathology in dorsal root ganglia organoid-derived sensory neurons

In this study, the authors generate dorsal root ganglia organoids (DRG organoids) by in vitro differentiation of human iPSCs. Bulk and single-cell RNA sequencing show that DRG organoids present a transcriptional signature similar to native DRGs and display the main peripheral sensory neuronal and glial cell subtypes. Furthermore, when co-cultured with human intrafusal muscle fibers, DRG organoid sensory neurons contact their peripheral targets and reconstitute the muscle spindle proprioceptive receptors. FRDA DRG organoids model some molecular and cellular deficits of the disease that are rescued when the entire FXN intron 1 is removed, and not with the excision of the expanded GAA tract. These results strongly suggest that removal of the repressed chromatin flanking the GAA tract might contribute to rescue FXN total expression and fully revert the pathological hallmarks of FRDA DRG neurons.

Read the Entire Article Here

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