My mission is to invest in efforts, organizations or people that are dedicated to the development of treatments and a cure for Cystic Fibrosis.
February 18, 2004 was the day we learned that our youngest son, Jeremy, has Cystic Fibrosis. CF is a rare progressive genetic disease that causes persistent lung infections, limits the ability to breathe and threatens to shorten his life. He was 17 at the time and the average life expectancy was 32. We immediately began research and learned about the Cystic Fibrosis Foundation and their mission to ensure patients access to high-quality, specialized care and to find a cure for CF by funding research and drug development. We learned the challenge in finding a cure and discovering new treatments for CF was because of the small population of CF patients. With only 30,000 people in the United States affected by CF, the pharmaceutical companies had little to no interest in investing resources in rare diseases but were focused on developing the next big blockbuster drug that would treat millions of people and result in large profits.
To overcome that obstacle, the CF Foundation pioneered “venture philanthropy” and led the movement of voluntary health organizations funding drug development with for-profit companies. In 2000, the Foundation made its first large investment: $40 million with Aurora Biosciences (now Vertex Pharmaceuticals) to discover compounds that might correct the core genetic defect in people with CF. This was a monumental shift as traditionally most disease nonprofits had focused their fundraising dollars on academic and medical research. In total, the Foundation has invested $425 million as part of its venture philanthropy model to accelerate drug development for cystic fibrosis.
The investment paid off January 31, 2012, a historic day for the CF community. The U.S. Food and Drug Administration (FDA) approved the first drug to address the underlying cause of cystic fibrosis (Kalydeco®).
Back in 2004, in support of the CF Foundation and their mission, our family joined forces with other families and friends hosted annual fund-raising events — Galas, Tailgating events and more recently a Brewer’s Ball. From 2004 to 2017 these events in Columbia have raised over $713,000 to support the Cystic Fibrosis Foundation. But there is so much more needed.
The recent drugs that have been developed target and potentially correct the more common of the genetic defects in CF patients. But with over 1,700 possible mutations, CF researchers are trying to increase the number of mutations that these drugs are approved to treat. Many CF patients could benefit from these drugs but since the FDA labeling does not include their specific mutation, insurance will not pay for the treatment costing over $300,000 per year.
Cystic fibrosis researchers are at the forefront of a growing field of personalized medicine in which doctors will increasingly be able to tailor therapies to patients based on their genetic makeup. More research funding is needed for these kinds of trials benefitting patients with rare mutations.
Based on the collected data, the median age at death was 30.7 years for the 380 people with CF who died in 2017. About 15 percent of those deaths occurred before 20 years of age. Many scientists and researchers believe that a cure for Cystic Fibrosis could be discovered in the next 15-20 years. Please consider joining me to support a cause that will one day change the meaning of CF to Cure Found!