Hopes and Cures

The Cystic Fibrosis Foundation is the leading organization in the United States devoted to curing and controlling cystic fibrosis. We spoke with Mary Dwight about her organization and the important work they do.

 Can you tell us a bit about CFF’s purpose?  The Cystic Fibrosis Foundation is the leader in developing drugs to treat and ultimately cure CF — as well as ensuring that CF patients receive the best care nationwide through our network of accredited care centers. The centers provide patients and families with vital treatment and other CF resources.

 Who do you represent and how large is the scope of your work?  We represent about 30,000 people with CF, as well as their family, friends, and those who take care of them. At the same time, our unique drug development business model is being adapted widely by many other voluntary health organizations in the country.

 What are some of the successes in the treatment of this disease?  The biggest success is the gains made in life expectancy for people with cystic fibrosis.  In 1955, when CFF was founded, children who were diagnosed with CF usually did not live long enough to attend elementary school. In fact, it was considered solely a childhood disease, since no one survived to adulthood.  The lifespan has doubled in the last 25 years, and the median age of survival today is 37.  More than 44% of people with this disease are adults.    CFF has been the driving force behind much of this progress.

 What does the Foundation do to promote this kind of success?  We are a nonprofit and have 250,000 volunteers in 80 chapters and offices that raise money the old-fashioned way — dinner dances, golf outings, Great Strides walks and auctions. We invest a significant amount into drug research and development.  Ninety cents on the dollar goes to research, care and education programs.  In the past five years, $300 million went into drug discovery.

 You mention drug discovery.  Talk about your “drug pipeline”  There are more than 30 promising therapies in the CF pipeline. And, for the first time in the history of the disease, the pipeline has drugs under development that target the root cause of cystic fibrosis.  If successful, these therapies will add decades of life for people with CF. 

What role does SBIR play in CFF’s work?  The SBIR program is a valuable partner for us.  It provides the identical “spark” for early research.

PTC Therapeutics is one of the Cystic Fibrosis Foundation’s great partners in the effort to develop treatments for this disease.  The company has multiple promising CF therapies in development, including PTC-124, an innovative oral drug to treat the basic genetic defect of cystic fibrosis, and potentially 2400 other genetic disorders.  The company, like several of our partners, received an SBIR grant for the early discovery phase of the drug that ultimately became PTC-124.  The development of this ground-breaking therapy depended on the SBIR grant ,as it was considered too risky to be funded by private venture capital funding. 

They received a $150,000 SBIR grant to target the genetic defect, but because of the rule change to exclude companies with 51% ownership by venture capital firms, funding for phase II was denied.  This caused an 18 month delay in the development of the therapy.  In a disease where people have a short life expectancy caused by this disease, delays means a loss of life. 

 What can Congress do?  We ask that Congress reauthorize the Small Business Innovation Research (SBIR) program.  We support restoring funding to venture capital-backed firms.  We see VC investment in a company as a validator, as a sign that they are doing things the right way.  We also ask that Congress dedicate a portion of these grants for research for orphan and rare diseases, such as cystic fibrosis.