Name: Dr. Robert Wilson
Where do you work? Children’s Hospital of Philadelphia
How long have you been working on FA and who was the first fellow FA researcher you met? I’ve been working on FA since 1996. Lucky for me, the first fellow FA researcher I met was Dave Lynch, who is an expert neurologist and a great scientist and colleague.
What got you interested in FA research? I read the seminal paper identifying the disease gene and was struck by the fact that the gene is conserved in lower organisms, suggesting a fundamental cellular function. I knocked out the yeast frataxin homolog and observed a decrease in mitochondrial function. I was able to substitute the human gene for the yeast gene, suggesting that studying the yeast gene might yield valuable insights into FA. I called up Dave, who was seeing individuals with the disorder, and we’ve been working on FA since.
What question or challenge were you setting out to address when you started this work? I wanted to elucidate the function of the gene, and its encoded protein, frataxin. I figured that the more we understood about what frataxin does, the more therapeutic possibilities would suggest themselves.
What research topics or questions are you currently focused on? I am currently focused on developing and testing therapeutics. These include compounds discovered in my own lab, as well as compounds being developed by various pharmaceutical companies.
What do you hope to achieve or what excites you in FA research? I hope to achieve effective treatments. What excites me is that we have so many possibilities. Drug development is very difficult, and most drugs fail, so having many possibilities is critical for success. I am confident that one or more will make it to the finish line as a safe and effective treatment.
If you have met someone living with FA, please tell us about that interaction. Did it have an impact on your work? I’ve met many individuals with FA, and their families. They have been an important source of motivation and inspiration that keeps me going on days when nothing is going right. And meeting Kyle Bryant has turned me into a pretty serious cyclist!
You serve voluntarily on FARA’s Scientific Advisory Board. Please tell us what you see as FARA’s key role in the research process. FARA’s role in the research process is essential. FA is a rare disease, so the kind of funding necessary to make significant advances can be hard to come by without the help of foundation grants. Also, research to find effective treatments takes a village, and FARA has successfully organized initiatives on all the fronts necessary to maximize the chances of success.
Tell us more about yourself and/ or your journey with FA research. In the late 1990s I got a phone call from Ron Bartek saying that he was starting a foundation and asking if I would be the first Scientific Director. Ron mapped out a strategy involving multiple entities working together: the new foundation, academics like me, the NIH, pharmaceutical companies, and the patient community. Ron was persuasive, so I signed on. We worked together to hold the first three international FA conferences and Ron has worked tirelessly ever since to make his vision a reality. FARA is now a model foundation. The people who work for, and on behalf of, FARA are remarkable. I’m lucky to know them.