Why We Fundraise

How Donating to Charites that Focus on Efficiency and Results can Make a Difference to CF Medicine

Big Pharma ~ the pharmaceutical industry. No doubt it creates drugs that can improve the lives of millions of people, but it definitely is also a money-making machine that thrives on churning out drugs with a high demand. This means a huge focus on drugs for conditions that affect a large number of people, like cancer or diabetes.

So what about the little guys?

Cystic fibrosis afflicts only 35,000 people in the US. A huge milestone in CF research was the discovery of the CFTR gene that causes the disease in 1989. At the time, CF families became very hopeful for the development of new therapies that could stem from this new knowledge. However, with such small market potential, the big drug companies did not take on the challenge.

That’s where the Cystic Fibrosis Foundation came in. Founded in 1955, the organization was dedicated to raising money to fund research and support patients. But like other organizations devoted to specific diseases, the donations went to university research that focused more on understanding the disease than on drug development. Obviously this research is very important, and it lead to major breakthroughs for CF: the gene discovery in 1989 came from research funded by the CFF. The problem came from application of the knowledge. If no one was willing to develop treatments based on the scientific knowledge of the disease, that knowledge wouldn’t be very helpful for patients.

Ten years ago the president of the CFF, Dr. Robert Beall, decided to take a risk by contacting private drug companies and asking if they’d be willing to get involved with drug development for CF, with the offer of funding the research. This was highly unusual for a foundation of this type, and the response was somewhat lacking. But one company agreed - a company called Aurora Biosciences in San Diego, which later was acquired by Vertex. The result: the big news of a drug with the potential to target the genetic defect that causes cystic fibrosis.

The company creates test that can be used to screen thousands of chemicals to look for drug candidates. The CFF funded the initial $2 million to create the test, which consisted of a cell with a defective chloride channel and a method to measure chloride flow across the membrane. The next step was to test potential drugs. This step was funded by both the CFF and the Bill & Melinda Gates Foundation, who gave the single largest donation the CFF had ever received. It seems that this testing has paid off: one drug was able to improve flow across a defective CFTR channel, as described in this article. Though the majority of CF patients have CFTR defects that prevent the channel from ever making it to the cell membrane, there is a new potential drug on the horizon that may free up the defective protein so that it can make it to the membrane. In any case, these results are exciting.

The CFF has shown an inspirational dedication to developing new treatments for cystic fibrosis since its inception. The foundation places a great importance on efficient use of donations, with 90% of all donations going to fund research. This dedication has earned them a four star rating from the Charity Navigator, the largest evaluator of charities in the US. Here at Breathe Hope, we really like efficiency and dedication to results. The CFF is a great example of an organization that employs both these qualities to creating advancements for treatment of cystic fibrosis. And it is paying off, as the Gates Foundation cited similar reasons for making such a large donation in the first place, in this statement by William H. Gates Sr, the co-chair of the Bill & Melinda Gates Foundation at the time of the grant:

“One of the missions at the Bill & Melinda Gates Foundation is to support organizations that are not only doing something important to improve global health, but are also efficient at what they do. The CF Foundation is a proven leader because of their vision and determination to identify the resources and people needed to cure this disease. We are pleased to provide this grant and hope that others will be encouraged to follow our lead.”

By donating and fundraising for charities like these, we can feel confident that we can, in fact, play a part in improving the lives of those affected by cystic fibrosis.

For further reading:
the CFF was recently highlighted on Business Week’s Website. Read more
The Bill & Melinda Gates Foundation
The Cystic Fibrosis Foundation