Playing Catch Up, Cystic Fibrosis Research Gets a Big Boost

In the U.S., pulmonary disorders like cystic fibrosis don’t necessarily get as much funding attention as other genetic diseases like ALS. At least not lately. This is one of the many reasons why grants from the Cystic Fibrosis Foundation are key for researchers racing to find a cure.

The Cystic Fibrosis Foundation recently awarded over $23 million in grants out of its Research Development Program toward accelerating the discovery of new cystic fibrosis treatments. A few recent grantees include:

The Colorado Research Development Program, which received a four-year, $2.1 million grant in support of its research related to nontuberculous mycobacteria (NTM) infections. These infections are chronically difficult to diagnose and treat and often times, can accelerate the decline of lung function.

Emory University, Children's Healthcare of Atlanta and Georgia Tech will receive $1.8 million over the next four years to establish a CF research and development program in Atlanta. Once established, researchers will study common coexisting conditions related to CF.

The remaining nine grants were awarded as renewal grants to programs that are already a part of the Cystic Fibrosis Foundation’s Research Development program network.

Cystic fibrosis is a debilitating genetic disorder that causes a thick buildup of mucus in the sufferers’ lungs and pancreas. The mucus that gets trapped in the lungs also traps bacteria with it. This then leads to infections that can and often do, result in serious lung damage. In the pancreas, the mucus buildup prevents the organ for releasing digestive enzymes necessary for the body to break down food and absorb nutrients. This mucus build up isn’t limited to the lungs and pancreas as it can spread to other organs of the body. The average life expectancy for CF patients is around 37 years.

Unfortunately, researchers are playing a bit of catch-up to improve the quality of life and life expectancy of CF patients. Back in 2011, the global funding shortfall for CF was so severe, that the U.K. based CF Gene Therapy Consortium, which spent 10 years working on an innovative way to treat the disease, had to put its planned trials on hold. It reportedly took an additional year for the consortium to receive enough funding to move forward with its clinical trial, which began in 2012.

Much of the global funding shortfall for cystic fibrosis was attributed to the global economic downturn. Then again, the CF Foundation didn’t help matters when it came under fire earlier this year. Around 2000, the foundation invested some $150 million in Vertex Pharmaceuticals to support the biotech company’s research efforts toward finding a cure.

The investment led to the development of the drug Kalydeco, which was purported to be one of the first drugs to treat the underlying cause of CF. Public outcry ensued when Vertex put a $300,000 per year price tag on the treatment. The response from the CF foundation is that it couldn’t control the price of Kalydeco. The foundation eventually sold its royalty rights to the drug for a reported $3.3 billion and directed those profits into the foundation to help further its mission.

Hopefully, the CF Foundation’s reputation has bounced back and the funding to find a cure for CF will continue to flow not only from the foundation, but other like-minded funders as well.