People with CF, and their friends and families, were hopeful that this discovery would quickly lead to new drugs to fight this rare and fatal disease. But progress stalled. The challenge of taking scientific knowledge from the laboratory and transforming it into effective treatments for CF was a larger obstacle than anyone had anticipated.
By the late 1990s, two important CF drugs that helped battle some of the devastating symptoms of CF had been approved with the Foundation's support. However, there were no breakthrough treatments in development to address the underlying cause of the disease.
Did You Know?
The CF Foundation supported development of vital drugs to treat CF including lumacaftor/ivacaftor (Orkambi®), ivacaftor (Kalydeco®), aztreonam (Cayston®) and inhaled tobramycin (TOBI®).
At the time, pharmaceutical companies had little to no interest in rare diseases and were focused on developing the next big blockbuster drug -- one that would help tens of millions of people and result in large profits.
In 1998, the median predicted survival age of a person with CF was 32 years. People with CF and their families were desperate for faster progress. With a growing sense of impatience, Robert J. Beall, Ph.D., then president and CEO of the Cystic Fibrosis Foundation, landed on an idea that would break through the pharmaceutical industry's reluctance to get involved in cystic fibrosis research.
The CF Foundation pioneered venture philanthropy and led the movement of voluntary health organizations funding drug development with for-profit companies. This was a monumental shift as traditionally most disease nonprofits had focused their fundraising dollars on academic and medical research.
This unconventional strategy was considered a major gamble with no guarantees. Dr. Beall began working the phones to find a willing partner. Only two drug companies returned his calls. 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. It was unknown that such a compound existed. The Foundation chose to work with Aurora because the company specialized in high through-put screening, a unique technology that used robots to test the therapeutic properties of thousands of chemical compounds a day in cells in laboratory dishes.