Friedreich’s Ataxia Center of Excellence at CHOP Awarded $1.275 Million to Advance Medical Research

The Friedreich’s Ataxia Center of Excellence (COE) at Children’s Hospital of Philadelphia (CHOP) was awarded $1.275 million by the Friedreich’s Ataxia Research Alliance (FARA), along with the Hamilton and Finneran families and the CureFA Foundation, to support the development of breakthrough therapies to improve the quality of life for individuals with Friedreich’s ataxia.

Friedreich’s ataxia (FA) is a neurodegenerative condition that limits the production of frataxin, a protein in the mitochondria, the cells’ energy generators. The condition damages the nervous system and impairs muscle coordination, causing problems with movement and sensation that get progressively worse. Individuals may also experience cardiac dysfunction, scoliosis, diabetes, vision and hearing difficulties. About 1 in 50,000 individuals worldwide have FA, including 5,000 cases in the United States. Symptoms typically appear between age 5 and 15, though the condition can sometimes appear later in life as well.

“For the past two decades, we’ve collaborated with Penn Medicine and FARA to provide and push forward the care needed by Friedreich’s Ataxia patients,” said Brenda Banwell, MD, Chief of the Division of Neurology at CHOP. “Children have access to top-notch clinical care and cutting-edge research opportunities at our FA Center of Excellence, and we are uniquely equipped to provide the specialized care patients with this rare, complex disease need. Our FA Center of Excellence would not exist without the help of our donors, FARA and the Hamilton and Finneran families, whose generosity and shared vision enable us to accelerate progress towards promising new therapies and care.”

The Friedreich’s Ataxia COE at CHOP was established in March 2014 by CHOP, Penn Medicine and FARA, and was a result of a $3.25 million gift from FARA in partnership with the Hamilton and Finneran families. Since 2014, more than $8 million in research funding has been committed to the COE to create a multi-disciplinary, translational research and clinical care center devoted to FA. Its mission is to expedite basic science and drug discovery to treatments and dedicate resources to clinical research and care to improve outcomes for individuals living with FA.

“As part of the Friedreich’s Ataxia Center of Excellence and the largest Friedreich’s ataxia clinical program in the world, this generous grant enables us to continue important research efforts,” said David Lynch, MD, PhD, Friedreich’s Ataxia Program Co-Director at CHOP. “We are truly thankful for donors like FARA, the Hamilton and Finneran families and the CureFA Foundation. We could not develop pioneering treatments and provide high-quality family-centered care without their support and generosity.”

The Friedreich’s Ataxia Center of Excellence is co-directed by Dr. Lynch, and Robert B. Wilson, MD, PhD.

“The partnership between CHOP and FARA is the first-of-its-kind dedicated to improving Friedreich’s ataxia research and clinical care for children and adults from all over the world,” said Dr. Wilson. “We are fortunate to have access to this critical funding to make strides toward developing new drugs for FA patients and opening more clinical trials.”

“FARA and the CureFA Foundation are proud to continue funding and support for the COE at CHOP and Penn Medicine, as we believe the multi-disciplinary and collaborative approach to research accelerates the discovery of novel therapies and translation to clinical improvements for those living with FA,” said Jennifer Farmer, CEO of FARA. “We are especially excited to see how the COE has brought new investigators and clinicians, especially young investigators, to the FA research community, which expands our bandwidth across the research spectrum, fosters new synergies and collaborations and brings new ideas, innovation and energy to our mission, treatments and a cure for FA.”