August 08, 2017
BENEFITING: Cystic Fibrosis Foundation
ORGANIZER: Cystic Fibrosis Foundation
The constant source of comedic relief for our tight knit group of friends, Bobby has never let his battle with CF interfere with having a great time. A daily inspiration for me, I will be running the 2017 NYC Marathon to raise money for Bobby and the Cystic Fibrosis Foundation.
Consider this an early wedding present Bob... and hopefully I’ll be fully recovered by the time we board the plane for Punta Cana to see you and Ali get hitched. Four months should be justtt enough time.
Below is an excerpt from Forbes coming out in Forbes in September 5, 2017. Fundraising and the work CFF is doing is making an impact:
Technically, Mark Sleeper doesn't know for sure he didn't get the placebo, but nothing in his 27 years of fighting the deadly lung disease cystic fibrosis has made him feel as good as the pill he recently took in a clinical trial. "It was kind of mind-blowing," he says. The energy he usually spent coughing and simply breathing was suddenly available for use in daily living. At first, he stayed up for two days. After 15 days on the treatment, his lung capacity, measured by the amount of air he can breathe out in a second, went from 70% of normal for a man his age to normal. "With what I experienced, I want the drug to be approved, because it was pretty radical," he says. "It was pretty cool to just be on it for 15 days. It's amazing to think of what it would be like to take it for a month."
Study results released in July for three different but similar drug combinations, all developed by Boston's Vertex Pharmaceuticals, back up Sleeper's experience, showing the amount of air patients could exhale in a second increased by ten percentage points or more--an unheard-of amount. There's only one precedent for results like this in CF: another Vertex drug, Kalydeco, approved for use in 6% of the 30,000 Americans with the disorder. The new combinations could bring a similar benefit to almost all cystic fibrosis patients. "There's going to be a paradigm shift in the way we think about treating this disease," says Jeffrey Leiden, Vertex's chief executive. "It's not going to be four different medicines for different subsets of patients, or incremental improvements. It's going to jump to the end. And it's going to be Kalydeco-like efficacy or better, and 90% of the patients getting a single regimen."Longer studies will be needed, but Leiden isn't alone in his excitement. "This is what we dreamed would someday happen," says Francis Collins, who led the team that discovered the CF gene 28 years ago and is now the director of the National Institutes of Health.
The mission of the Cystic Fibrosis Foundation is to cure cystic fibrosis and to provide all people with the disease the opportunity to lead full, productive lives by funding research and drug development, promoting individualized treatment, and ensuring access to high-quality, specialized care. The Cystic Fibrosis Foundation is the world’s leader in the search for a cure for cystic fibrosis.