President Barack Obama, during his recent State of the Union Address, highlighted cystic fibrosis (CF) as the nation’s model for the White House’s upcoming strengthened strategy for “precision medicine,” or groundbreaking treatments that specifically target “chinks” in a disease’s “armor” — commonly found all the way down to a sub-molecular level.
Representing the CF community at the event was Bill Elder, Jr., a medical student and long-time CF advocate. He was first diagnosed with cystic fibrosis at the age of 8, and like many others, was expected to survive only until early adulthood. Thanks to generous support from the Cystic Fibrosis Foundation, and innovative precision medicine products such as Vertex Pharmaceuticals, Inc.’s Kalydeco, Elder has lived well beyond most people’s expectations.
The White House released an online statement on its website, saying, “Thanks to a unique collaboration between the Cystic Fibrosis Foundation, patients, researchers, and a pharmaceutical company, Bill, now 27, expects to live a long, full life.”
The CF community has much to celebrate with the President mentioning the disease in his address, as it recognizes the immeasurable dedication patients, philanthropists, politicians, and researchers have poured into making CF easier to live with, and the significant progress biomedical research has made in continuously finding better treatments. The State of the Union Address also foreshadows more years of bolstered support for advancements in precision medicine that not only cater to CF, but to a wide range of orphan diseases with largely unmet clinical needs.
“I want the country that eliminated polio and mapped the human genome to lead a new era of medicine: one that delivers the right treatment at the right time,” said the President. “In some patients with cystic fibrosis, this approach has reversed a disease once thought unstoppable.”
In other federal updates regarding CF, U.S. lawmakers recently introduced new legislation that could greatly benefit thousands of Americans suffering from cystic fibrosis (CF) and other rare diseases with largely unmet clinical needs by helping them gain access to and participate in clinical trials without having to worry about their health coverage. The new bill, called the “Ensuring Access to Clinical Trials Act of 2015,” was sponsored by a bipartisan group of senators and legislators.
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