CF Foundation Congressional Briefing Speaks to the Role of Precision Medicine in Treating CF and Other Diseases

On December 8, The Cystic Fibrosis Foundation, in collaboration with the Congressional Cystic Fibrosis Caucus and its co-chairs, U.S. Reps. James McGovern (D-Mass.) and Tom Marino (R-Pa.), presented a congressional briefing on cystic fibrosis and precision medicine.

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Dr. Skach and Rep. McGovern at Congressional briefing.
Representative James McGovern (D-MA) (left) and William Skach, M.D., (right) opened the briefing by speaking about the Congressional Cystic Fibrosis Caucus and the need for more members of Congress to join.

 

William R. Skach, M.D., senior vice president for research affairs at the CF Foundation, served as the moderator for the event, Precision Medicine & Cystic Fibrosis: Using Genetics to Treat Disease, which educated members of Congress and their staff on the role of precision medicine and genetically-targeted treatments and therapies.

The panelists included Eric D. Green, M.D., Ph.D., director of the National Human Genome Research Institute (NHGRI) at the National Institutes of Health (NIH); Noreen Roth Henig, M.D., chief development officer of ProQR Therapeutics NV; and Mara Cray, a 19-year-old woman with CF.

During welcoming remarks, Representative McGovern shared how he became involved in the cause after learning that a friend's son had cystic fibrosis. He highlighted the role of precision medicine as an important approach to finding treatments for CF and encouraged the congressional staff members in attendance to ask their members to join the CF Caucus. Representative John Fleming (R-La.) joined him to reiterate the need for funding for CF research and shared his own personal connection to the disease.

Attendees ask questions during Congressional briefing.
The briefing’s panelists each described their unique perspective and approach to personalized medicine.

Dr. Skach highlighted the story of cystic fibrosis and the great strides made by the CF Foundation to combat the disease. He cited the development of ivacaftor (Kalydeco®), as transformational and a model for the type of treatments that are possible through precision medicine. He called for continued and increased funding of the NIH to maintain a well-educated biomedical workforce to advance the work of this technology and new frontier.

“Without the NIH, we simply would not have made progress,” explained Skach.

Dr. Green informed the audience about the role that genomics has played in CF research and how the ability to look at an individual's genetic “blueprint” has helped tailor treatments. He described CF research as being on the “leading edge” of precision medicine.

Mara Cray speaks during Congressional briefing.
Mara Cray, an adult with CF and longtime CF Foundation supporter, spoke candidly about the daily struggles people with CF face and how she has benefited from taking Orkambi.

 

Nineteen-year-old Mara Cray outlined the daily life and struggles that a person born with CF faces. She relayed her own experiences of being too sick to continue high school when her health started to rapidly decline. But her life changed dramatically after she started taking lumacaftor/ivacaftor (Orkambi™), during a clinical trial.

“It stopped my health decline in its tracks,” Cray said. “I was able to start planning my life and my future again.”

Dr. Henig outlined the work ProQR Therapeutics is doing in emerging therapeutics and its commitment to fighting cystic fibrosis. She highlighted CF as a top priority for the company.

The panelists emphasized the need to continue funding for basic research, which serves as the bedrock for precision medicine. The key takeaway was that knowledge builds on top of itself and that everyone will eventually benefit from basic science research.

Audience chat amongst themselves during Congressional briefing.
The audience, which included many congressional staff members, listened closely to the panelists as they outlined the past, present and future of personalized medicine.

 

To learn more about the advocacy efforts of the CF Foundation, please visit our advocacy page to find out how you can get involved.

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