Dear friends,
This Saturday, May 21st is FA Awareness Day, and we invite you to participate in the #LendUsSomeMuscle Global Challenge! We're challenging everyone to #FlexForFA with 50 bicep curls each day of May, share your #Bicep50Challenge or your best muscle pose on social using the hashtags, and/ or donate $15 in honor of the 15,000 individuals living with FA.
Click here for more information or to donate to help us reach our goal of $15,000.
Sincerely,
Jen
Jen Farmer,
FARA Chief Executive Officer
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FARA is proud to present: The third annual FA Research Flash Talks series - featuring Young Investigators from FARA funded laboratories around the world. This four-part series covers key aspects of FARA funded research from gene silencing and disease mechanisms to therapeutic avenues and clinical insights. Each session includes Flash Talks from four to five Young Investigators (ie, postdocs and graduate students) moderated by an FA Community member. Each Flash Talk is limited to a maximum of five minutes and a single PowerPoint slide and is suitable for a lay audience. View the recordings from the first two sessions here. Register for the last two sessions here.
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Reata Pharmaceuticals has announced the company's first quarter financial results and provided an update on their clinical development programs. Updates on their FA program include:
- They completed submission of a New Drug Application (NDA) to the U.S Food and Drug Administration (FDA) for Omaveloxolone for treatment for patients with Friedreich's Ataxia in March. Reata CEO, Warren Huff states, “. . .we are looking forward to working with the FDA on its review of our NDA throughout this year. If approved, we are preparing to be in a position to launch this important drug by early 2023.”
- This program has received Fast Track Designation, Orphan Drug Designation, and Rare Pediatric Disease Designation.
- They secured an agreement on their Pediatric Investigation Plan with the European Medicines Agency (“EMA”) Pediatric Committee.
- They are continuing to complete the regulatory procedures and submissions required prior to filing a Marketing Authorization Application (“MAA”) in Europe for approval of omaveloxolone for the treatment of patients with Friedreich’s ataxia. They plan to submit an MAA to the EMA in the fourth quarter of 2022.
To read the full press release click here.
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FARA Newly Funded Research |
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The FARA Grant Program is pleased to announce funding for a new research grant. Drs. R. Mark Payne and Thomas O'Connell, Ph.D. from the Indiana University School of Medicine will be working on a project that seeks diagnostic and mechanistic validation of a metabolic biomarker panel to guide therapeutic interventions in Friedreich's Ataxia.
A key roadblock for all therapies has been a lack of biomarkers which can rapidly report on the efficacy and/or toxicity of a therapeutic intervention, and the biochemistry of the disease. In Friedreich Ataxia, affected tissues, such as heart or brain, cannot be assayed to quantify frataxin levels in response to therapies. The goal of this project is to conduct pre-clinical studies on a new set of biomarkers that Dr. Payne and Dr. O’Connell have discovered that reveals a distinct and highly specific metabolic profile in FA patients. Dr. Payne and Dr. O’Connell will test the hypothesis that a metabolic panel can be used as biomarkers for loss or recovery of FXN expression.
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You can watch Episode 12: Patient Reported Outcome now. Narrated by Morgan Swaney, episode 12 talks about Patient Reported Outcomes - Health information reported directly from the patient without interpretation by a clinician or anyone else.
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Save the Date! FARA Energy Ball |
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Join us for a fun and high energy evening in support of FA research! The FARA Energy Ball gala will take place in Tampa, FL on October 15th!
Tickets and sponsorship now available.
For more information, click HERE.
The commitment deadline for Groundbreaker and above sponsors to appear in the invitation is June 8. To have your sponsorship recognized in the invitation, please email ava.faraenergyball@curefa.org by June 8.
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Researchers at Vanderbilt University Medical Center in Nashville, TN want to learn more about changes in glucose control and cardiac features in children with Friedreich's Ataxia. They are recruiting participants (ages 7-17 years) with FA for a study to determine if there is a relationship between blood sugar abnormalities (risk of diabetes) and cardiac changes.
If your child is selected to participate in this study, your child may complete blood testing, oral glucose tolerance testing (with a non-FDA approved stable isotope product), placement of a continuous glucose monitor, echocardiogram (ultrasound of the heart), and dual-energy X-Ray absorptiometry (DXA, x-ray scan) during a 1-2-day study visit. You and your child will be compensated for your time. Click here for more information about this study.
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This is a first-in-human, Phase 1 study of DT-216 which means that trial will begin with single dose cohorts to assess safety. Enrollment will be in cohorts (typically 5-8 people). The first cohort will be enrolled and tested at the first dose, then there will be a review of the safety, then typically several weeks later the next cohort will be enrolled to test the next dose. After the single dose study, there will be a multiple dose study, which is also a safety study. Multiple dose studies typically assess safety over a period of several weeks to a month. More information on the multiple dose study will be available once the single dose study is further along.
Inclusion criteria:
- Individuals with FA ages 18 to 55 years old (inclusive) at screening
- Genetically confirmed diagnosis of FA with homozygous GAA repeat expansions
- Weight between 90 and 200lbs (approximately)
- Ability to sit upright with thighs together and arms crossed without requiring support on more than two sides
- Ability to perform basic daily care such as feeding yourself and basic personal hygiene with minimal assistance
- Must have completed full COVID-19 vaccination at least 4 weeks before treatment
This study is being conducted at Clinilabs and will require about a week long stay at the study site. Compensation will be provided for qualified participants and caregivers. Individuals and caregivers who are not local to the research facility will receive no-cost transportation and accommodations. Click here for more information and to read the study flyer.
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Please consider enrolling in this important study. Participants needed at all study sites.
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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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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