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FARAFARA Cure FA

Research funding is a precious resource

Last week FARA was notified of an opportunity to participate in a roundtable discussion about the US budget sequestration with Senator Bob Casey. Senator Casey has made it one of his top priorities to advocate for maximum National Institutes of Health (NIH) funding for medical research so that NIH funding does not suffer (as much) from the sequestration process.

Elected officials are approached on a daily basis with statistics and pretty graphs that illustrate trends supporting all kinds of issues. However we were told today that it is the stories from individuals that people remember and work wonders in Washington. To that end, Senator Casey and his staff held this roundtable discussion to collect stories of the individuals who will be affected by NIH budget cuts so he can use the stories when he is fighting for these issues in Washington.

The event was held at the American Cancer Society in Philadelphia. There were inspiring stories of survival from the cancer community, a woman who just recently completed an Ironman after a hit and run on her bike causing a life threatening Traumatic Brain Injury (TBI), and two sisters who are fighting for research for their son/nephew who has Fibrodysplasia Ossifans Progressiva (FOP) - a debilitating disease affecting only 800 people worldwide. Each person had a story of how they benefited from research, whether it was in treatments they received, clinical trials they participated in, or hope that was created in the search for a cure.

I talked about my own story of being diagnosed with Friedreich's ataxia (FA) and my family feeling incredibly alone and helpless - no treatment, no cure, and only 1 in 50,000 people affected. I spoke about how my family decided to take action on our bikes and how that action produced some research funding for a young researcher named Dr. Marek Napierala. Though the grant funded by FARA and NAF was small, Marek was able to produce some great results which allowed him to apply and receive NIH funding to take his results to the next level.

I also talked about how NIH provides not only funding but infrastructure. This point was illustrated by the phase 1 study of Idebinone for which I stayed a week at a NIH hospital in Bethesda, MD to participate in the trial while I watched lots of Sports Center and ate plenty of ice cream.

Additionally I mentioned the current Phase 2b study of EPI-743 sponsored by Edison Pharmaceuticals. In the infancy of the molecule that became EPI-743 Edison was a tiny company with only 6 employees. Therefore all of the preclinical work took place at the NIH where Edison had access to an incredible amount of infrastructure, expertise and experience.

Our research simply would not be where it is without the NIH.

Lastly I told the Senator that the research is our hope. Any decrease to the NIH research budget is a decrease in hope.

The FA community applauds Senator Casey's continued fight for NIH research funding. The FA community applauds Senator Casey's continued fight for NIH research funding.

Kyle

About the Author

The FARA Ambassador Program

The FARA Ambassador Program

The FARA Ambassadors are a united team of patient volunteers living with FA who are committed to supporting FARA in the search for a treatment and cure.
 
Together we seek to know more about FA, and FARA so we can be prepared to represent the community when the opportunity arises; speaking at events, to volunteers, potential donors, scientific groups, pharma partners, media interviews and other awareness and fundraising opportunities. We believe support is key to continued success toward our ultimate goal of treatment and a cure. Participants in the FARA Ambassador Program are passionate about building and upholding relationships within the FA community.
 
The FARA Ambassadors are positive, supportive, peer representatives for the FA community, actively raising awareness and funds for FARA. To learn more about the FARA Ambassador Program or to have a FARA Ambassador speak at your event, please contact: info@cureFA.org.

 

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