Cystic Fibrosis Foundation leaders and cystic fibrosis (CF) community advocates met at the White House with President Barack Obama, officials from the National Institutes of Health (NIH) and the U.S. Food and Drug Administration (FDA), and other CF stakeholders last month for the Precision Medicine Initiative (PMI) Summit.
The summit was an opportunity to learn about PMI and of progress being made in the field of precision medicine. Participants were also given the chance to discuss strategies in these efforts at a round table, and hear of the Obama administration’s continued commitment to advancing specialized care for CF patients and others needing it, according to a press release.
PMI was launched in 2015 by President Obama, who also in that year’s State of the Union address called the progress being made in CF an example of the potential of precision medicine to treat all diseases. He specifically mentioned Bill Elder Jr., a CF patient whose condition markedly improved after genetically targeted treatments, saying:
“One day Bill will be able to tell his grandchildren about how he used the miracle of his own life to not only serve as an example but also an inspiration. And that’s the spirit of hope and resilience and community that has always carried America forward.”
Francis S. Collins, director of NIH, used the event to announce his organization’s latest efforts to implement the PMI Cohort Program, an effort to collect and assess health and genetic data on over 1 million Americans so as to better understand and treat diseases. Researchers expect to enroll nearly 79,000 volunteers by the end of 2016.
Preston W. Campbell, MD, president and chief executive officer of the CF Foundation, was also among the guests invited to the PMI summit, along with Foundation community advocate Katrina Young and her 17-year-old son, Sean, who has CF.
Scientists are looking to precision medicine, or personalized treatment, as a way for healthcare providers to tailor treatment and prevention strategies to an individual based on that person’s unique characteristics — like their genome sequence, microbiome, health history, lifestyle, and diet — in the near future.
“In the coming years, we hope that doctors will have an arsenal of new therapies at their disposal to help provide the best care and treatment options for individuals with CF so that they can live healthy and fulfilling lives. In this new era, doctors will be able to use information about a person, including his or her specific CF mutations, lung function and bacterial infections, to select from a number of therapies to determine the best treatment options,” John P. Clancy, MD, said at the 2015 North American CF Conference, where he outlined a history of personalized medicine.
“It is encouraging to see how the progress of drugs that target the underlying cause of CF has changed the lives of so many living with the disease” he continued. “Walking out of this plenary, I am even more excited to think about the potential that these therapies and others in development have for improving the lives of individuals with CF in the near future. Personalized medicine at its finest.”
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