Some answers from an expert on cystic fibrosis

Published 11:02 pm Saturday, March 7, 2009

Dr. Bonnie Ramsey, director of the Center for Clinical and Translational Research, Seattle Children’s Research Institute, is a leading researcher in cystic fibrosis.

A graduate of Stanford University and Harvard Medical School, she first began her research into the disease when she came to Seattle in 1978.

In 2005, the Bonnie W. Ramsey endowed professorship in cystic fibrosis was created in her honor at the University of Washington.

Q: Why did you choose to specialize in the research of cystic fibrosis?

A: I came out here to be a cancer specialist. Then … they needed somebody to take over the cystic fibrosis program. I said I’ll be glad to do that at least short term. But at that time there was no hope in the illness. We didn’t know what caused the illness. The lifespan was to the early teens and the quality of life was different than today. They had to spend the majority of their lives in the hospital.

Q: How did research into the disease progress?

A: In the ’80s, they found the protein, what the mutations were, what the mutations did. … Then when you get an interesting scientific story you have people flocking to the area. They can get funding and get answers. Now the cystic fibrosis (research) community has moved into small molecules.

At the same time we’ve done a huge amount in treating of the secondary complications, lung infections and mucus buildup.

Q: What eventually causes patients to die of cystic fibrosis?

A: They have these recurrent lung infections. It’s almost impossible to clear them.

Over time each infection slowly takes a toll. It cause scar tissue in the airway of the lung and scar tissue over time (results) in lung failure.

In this stage … (people) die of lung failure or get lung transplants.

I don’t think lung transplants are what we want as a good, long-term solution.

Q: What cystic fibrosis research is currently under way at Seattle Children’s Research Institute?

A: Children’s is involved in several clinical trials to develop new therapies to treat cystic fibrosis, in particular to treat related lung infections.

Q: What new treatments are now being developed, either in Seattle or elsewhere?

A: There are several really exciting new treatments in development. For the first time we have treatments which may correct the abnormal protein in cystic fibrosis called “CFTR.”