“Excited and beyond belief” is how Ron Bartek, co-founder and president of the Friedreich’s Ataxia Research Alliance (FARA), describes current prospects for finding treatments for the incurable genetic disease that claimed his son, Keith, at age 24.
Bartek spoke to Friedreich’s Ataxia News on the sidelines of the recent 2019 World Orphan Drug Congress USA, in Oxon Hill, Maryland. Some of the 30 to 35 companies working on potential ataxia treatments attended the event, as did a number of nonprofit groups such as FARA that advocate for rare-disease patients and their families.
“At this conference, the majority of presentations centered on the tremendous importance of patient advocacy groups working with our industry and government partners,” Bartek said. “We knew from the beginning there was absolutely no way we would accomplish anything by ourselves. So we started working with anybody and everybody we could. Eventually that led to industry partners coming to our side.”
Friedreich’s Ataxia Awareness Day — celebrated the third Saturday in May — is May 18 this year. FARA aims to raise $250,000 through the worldwide “Lend Us Some Muscle” campaign, which was established two years ago in Australia to help find a cure for FA.
“We are just excited beyond belief about this year,” Bartek said. “This is the year regenerative medicines are coming to our doorstep. That includes enzyme replacement therapies and gene therapy. We’re expecting that this year, we’ll have at least two, if not three, clinical trials commencing in those kinds of regenerative medicines.”
What thrills Bartek the most, he said, is that in 2019, “we will actually begin clinical trials in regenerative medicines — or approaches that have the prospect of addressing the underlying cause of FA, which is low levels of frataxin protein.”