In 1997, a year after the gene that causes Friedreich's ataxia was identified, Ron and Raychel Bartek received the diagnosis of their son, Keith, and discovered that there was no treatment for the disease and no group dedicated to finding one. So they started Friedreich's Ataxia Research Alliance (FARA). Since then, FARA alone has funded more than $20 million in research on the disease and helped secure a similar amount from co-funding organizations. That has helped lead to multiple clinical trials encompassing a range of treatment approaches.
FARA President Ron Bartek remembers when he and his wife decided to start a nonprofit dedicated to finding a treatment for Friedreich's ataxia.
"On the night we received Keith's diagnosis, we saw online there was no treatment, very little research into the disease and no organization devoted entirely to supporting such research. We were living in the DC suburbs, we were 30 minutes from the [National Institutes of Health], and we both worked on Capitol Hill," he recalled. "Maybe we're supposed to do this."
Things moved quickly after that: On the day FARA was approved for incorporation, Bartek drove to the NIH to apply for a workshop grant. A friend helped them quickly attain 501(c)(3) status; with that secured, the NIH approved the workshop grant. Two months later, FARA co-hosted with the National Institute of Neurological Disorders and Stroke the world's first Friedreich's ataxia workshop. Sixty-five scientists from around the world joined 15 NINDS researchers for that first conference.
Though a treatment remains elusive, that's about the only thing that hasn't changed. The last conference on Friedreich's ataxia was held in Strasbourg, France; 200 people attended, and many others were turned away due to space limitations. Pharmaceutical companies and advocacy organizations were well-represented. The Barteks' initial goal was being accomplished.
"That was our first objective: to grow the field," Bartek said.
Most importantly, an approved treatment may be near at handâwith others following along and providing a "cocktail therapy" to treat the disease effectively and eliminate it in subsequent generations.
Research!America's obvious effectiveness at advocacy is a critical reason why FARA is a member.
"Research!America is a key advocacy organization in support of medical and health research across the front. They're so effective at the highest levels and in collaborating with other advocates," Bartek said. "With all of us pulling together, we're such a powerful voice."