Vertex Drug Could Be “Man on the Moon” for Cystic Fibrosis Therapy

Bonnie Ramsey remembers the dark days of treatment for cystic fibrosis. About 30 years ago, when she devoted her career to the research and treatment of this genetic lung disease, children who were diagnosed had a life expectancy of about 17 years. The outlook is much brighter for patients now, as quality of life has improved significantly, and median lifespans have doubled.

But part of what makes Ramsey more optimistic today is an experimental treatment from Vertex Pharmaceuticals (NASDAQ: VRTX), based in Cambridge, MA. This drug, VX-770, along with a related compound, VX-809, have the potential to be the first drugs ever to fix underlying genetic defects at the root of this condition that affects 30,000 people in the U.S., she says.

Ramsey’s opinion counts for a lot. She’s a clinical researcher affiliated with Seattle Children’s Hospital, and the University of Washington, and serves as executive director of the Cystic Fibrosis Foundation’s Therapeutic Development Network, in which she coordinates a 77-site network of clinical trial sites dedicated to helping companies develop new therapies for this rare disease.

The work for the CF Foundation is how she first got exposed to San Diego-based Aurora Biosciences in 2000, the company that began developing the new drug and was later acquired by Vertex. The CF Foundation has contributed more than $75 million to Vertex to keep the program going over the years, so this is a relationship that’s hugely important to both sides. The drug has now shown enough promise that it has advanced into the final stage of clinical trials needed to win FDA approval.

Ramsey spoke with me in depth about the changing standard of care in cystic fibrosis, why the Vertex programs matter, and how foundations are playing an increasingly important role in financing clinical trials. Here is an edited account of the conversation:

Xconomy: So you got started back in the dark days of treatment for cystic fibrosis, 30 years ago. What have been the major advances since then?

Bonnie Ramsey: There have been advances in terms of research, and those have been phenomenal. Things really turned around in the 1980s. Everyone is aware the gene was identified in 1989, but …Next Page »

Luke Timmerman is the National Biotech Editor of Xconomy, and the Editor of Xconomy Seattle. You can e-mail him at ltimmerman@xconomy.com, or follow him at twitter.com/ldtimmerman