Center of Excellence Awarded Nearly $1.3M to Advance Research

The Friedreich’s Ataxia Center of Excellence of the Children’s Hospital of Philadelphia (CHOP) has been awarded a total of $1.275 million to advance research and clinical care of people with Friedreich’s ataxia (FA).

The grant came from the Friedreich’s Ataxia Research Alliance (FARA), the Hamilton and Finneran families, and the CureFA Foundation. Of note, members of the Hamilton and Finneran families have founded and/or are part of the board of directors of CureFA Foundation.

“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,” David Lynch, MD, PhD, co-director of the FA Center of Excellence and CHOP’s FA program, said in a press release.

“We could not develop pioneering treatments and provide high-quality family-centered care without [these donors’] support and generosity,” he added.

The FA Center of Excellence was created in March 2014 by three longtime allies — CHOP, Penn Medicine, and FARA — with the help of a $3.25 million donation from FARA, and the Hamilton and Finneran families.

Karen and Tom Hamilton have dedicated their lives to finding a cure for FA, after their daughter Annie was diagnosed with the condition in 2013. They have been supporting FA research ever since, together with Karen’s parents, Patty and Bill Finneran.

“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 CHOP’s neurology division.

“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,” she said.

Over the years, the center has been granted more than $8 million to create a multi-disciplinary, bench-to-bedside research, and clinical care center devoted to FA. Its mission is to identify new biomarkers and therapeutic targets, explore new therapeutic approaches, and promote a quick translation from preclinical data to human studies.

Other goals include opening more clinical trials, developing and validating outcome measures, and improving the knowledge and care of heart problems associated with FA.

FARA and the CureFA Foundation “are proud” to continue funding and supporting the center, as they 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, FARA’s CEO.

“We are especially excited to see how the [center] has brought new investigators and clinicians, especially young investigators, to the FA research community,” she added.