The FA Center of Excellence (COE)

The FA Center of Excellence (COE) is a translational research and clinical care center devoted to Friedreich’s ataxia. The center’s goals include expediting basic science and drug discovery findings to new treatments and dedicating resources to clinical research and care to further understand the disease, inform drug development, and improve outcomes for individuals living with FA.

The FA Center of Excellence was established in March 2014, with a commitment to the Children’s Hospital of Philadelphia and University of Pennsylvania, presented by FARA in partnership with the Hamilton and Finneran families. FARA has maintained this funding partnership with the CureFA Foundation (established by the Hamilton and Finneran families) to advance research through the COE.

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Overview

The COE supports work within the core disciplines of neurology, cardiology, biomarker discovery and development, and drug discovery, fostering collaboration and synergy across these disciplines. The COE researchers have contributed to both the discovery of potential therapeutic targets and the study of new biomarkers to measure the disease and therapeutic impact, and have also participated in neurologic and cardiac clinical trials and studies. Funding provided by the COE supported the research findings that allowed these investigators to apply for and receive larger grants from the National Institutes of Health, the Department of Defense CDMRP program, and other foundations. In addition, the COE investigators have published over one hundred peer reviewed publications from their research.

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FA Center of Excellence Investigators

Portrait of Rob Wilson
CO-DIRECTOR, COE

Dr. Rob Wilson

Dr. Wilson applies his expertise in early drug discovery and screening to development of models for drug screening and to the identification of new therapeutic targets for FA.

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Portrait of Dr David Lynch
CO-DIRECTOR, COE

Dr. David Lynch

Dr. Lynch leads clinical and translational research teams focused on understanding the neurological aspects of FA and evaluating novel therapies. The clinical team is an active site for the UNIFAI natural history study, and they are active in other biomarker and treatment trials. The translational team focuses on studying how frataxin deficiency leads to cellular changes in neurons; the interaction of frataxin and other proteins; and testing drugs and treatment approaches in animal models.

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Portrait of Blair Mesaros
COE INVESTIGATORS

Dr. Ian Blair / Dr. Clementina Mesaros

The Blair lab has developed new and improved methods for the measurement of frataxin, the protein that is deficient in FA, for both pre-clinical and clinical development. In addition, this lab works on methods to identify and track changes in molecules that reflect changes in metabolism in FA.

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Portrait of Kim Berly Lin
COE INVESTIGATOR

Dr. Kimberly Lin

Dr. Lin is primarily focused on understanding the natural history of cardiac disease in FA, the discovery of biomarkers and endpoints that can be used in future clinical trials, novel approaches to treatment, and improved clinical outcomes.

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Portrait of Dr. Shana McCormack
COE INVESTIGATOR

Dr. Shana McCormack

Dr. McCormack's clinical practice and research efforts focus on growth and development, diabetes management, evaluation of muscle specific biomarkers, and studies of exercise and nutrition in FA.

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The Friedreich’s Ataxia Accelerator, Cambridge MA

The Friedreich’s Ataxia Accelerator (FAA) at the Broad Institute of MIT and Harvard was established in August 2020. Funded by FARA, in collaboration with the CureFA Foundation and EndFA, the accelerator is currently supporting the work of five world class scientists as they apply their expertise to the discovery of new approaches that could lead to treatments for FA.

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The FA Accelerator Investigators

Protrait Dr. Vamsi Mootha
FAA DIRECTOR

Dr. Vamsi Mootha

A Howard Hughes Medical Institute investigator, Dr. Mootha and his collaborators previously discovered that limited oxygen in cells, or hypoxia, rejuvenates cells that lack frataxin. They are now working to discover why low oxygen allows cells to bypass the need for frataxin and whether a therapy can be developed that mimics the effects of low oxygen.

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Portrait of Dr. Gary Ruvkun
FAA INVESTIGATOR

Dr. Gary Ruvkun

A professor of genetics at Harvard Medical School and Massachusetts General Hospital, Dr. Ruvkun is using simple model system to test different molecules for their ability to rescue frataxin-deficient cells.

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Portrait of Dr. David Liu
FAA INVESTIGATOR

Dr. David Liu

Dr. Liu, who is also a Howard Hughes Medical Institute investigator, is applying new gene editing technologies to directly correct the genetic causes of Friedreich’s ataxia.

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Portrait of Dr. Christine Seidman and Dr. Jonathan Seidman
FAA INVESTIGATORS

Dr. Christine Seidman / Dr. Jonathan Seidman

Jonathan G. Seidman, PhD, is the Henrietta B. and Frederick H. Bugher Foundation Professor of Genetics at Harvard Medical School. Christine E. Seidman, MD, is the Director of the Cardiovascular Genetics Program at Brigham and Women’s Hospital and the Thomas W. Smith Professor of Medicine at Harvard Medical School. She is also an Investigator of the Howard Hughes Medical Institute. The joint Seidman laboratory has done groundbreaking research, including the discovery of genetic etiologies and mechanisms for human heart disease.

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