Vertex Drug Could Be “Man on the Moon” for Cystic Fibrosis Therapy, Says Researcher Bonnie Ramsey
(Page 6 of 6)
probably less than five patients. So they have to go to many sites to get enough patients. But that wasn’t a problem, enrollment, in the Phase II trial.
X: Do you get many companies knocking on the CF Foundation door trying to get into the network?
BR: A lot of companies are interested. They have obviously heard the CF Foundation will support some companies. The foundation has had to get more selective, because we can’t support everybody. Companies know it’s a good system.
X: What about other disease foundations? Are they studying your model?
BR: I’ve talked to the autism group, the hydrocephalis group. Years ago I talked to a sickle-cell anemia group, pediatric nephrology. Over the years I’ve talked to lots of groups about it. Rare disease networks are now pretty commonplace.
X: But are there still people donating to the cause in a way that it enables enterprising biotech entrepreneurs to turn to groups like this, as a viable way of keeping their pipeline moving forward?
BR: Yes. The foundation is still supporting several biotech companies, and is still extremely active. Things are moving forward.
The NIH, as well, particularly now that Francis Collins is going to take it on, he’s been close to the CF community for years. As you may remember, he found the gene. He’s very interested in this model, the CF drug development model. I don’t know, I haven’t talked to him about this, but he may work on how can NIH better partner with industry to help with developing new therapies.
But it’s the federal system, so it’s got to be a rigid system, and it’s been hard for industry to partner for that reason.
X: So five, or 10 years out, what kind of prognosis or life expectancy will CF patients have?
BR: I hope in five or 10 years, one of the therapies is available and working, and we’ll see about it’s impact on lifespan and quality of life. I hope that both VX-770 and VX-809, or drugs with similar modes of action will be approved and available. I don’t like the word ‘cure,’ because I don’t know what that means. No matter what they get, they will have to be on treatment for the rest of their life. To me, a cure is like taking a vaccine and you’ll never get an illness. But these are treatments that will significantly alter the course of the illness.