Vertex Drug Could Be “Man on the Moon” for Cystic Fibrosis Therapy, Says Researcher Bonnie Ramsey
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and get vitamin deficiencies. That nutritional aspect, and management of it, has had a major impact.
You could say, well, why didn’t you do that 30 years ago? I think a lot of it has to do with the mindset. It’s interesting. A lot of it is a matter of saying, ‘Yes, we can change this.’ This is also what happened with leukemia in the 1960s. People have said, ‘We can do something about it.’ Then you get much more aggressive. I do think that a secondary impact of discovering the gene and the protein in 1989 was that people said, ‘You know, what? There’s a chance we can cure this disease. Therefore, we had better make sure these patients are doing well.’ Hope has a huge impact on the way people approach disease management.
X: Even with the gene, people turned to gene therapy, and it didn’t work.
BR: That’s right, but, psychologically, it changed the way we approached patients.
X: So we’re still benefitting from the fundamental understanding of biology. But what kind of potential does this really offer to patients if lifespan has doubled?
BR: First of all, lifespan doubled, but that means you’re getting into [the mid-30s] or so. And remember, we’re talking about median survival, so there’s a bell-shaped curve. You’re still going to have patients who are getting very sick when they are teenagers. Then other people get sick when they are 30 and 40. Even a doubled life span is not a normal American life span. You still are decades away from the average lifespan, so we need to improve that.
Second of all, although quality of life has dramatically improved, these patients still have to go through a very time-intensive regimen. They have to spend hours just managing all their medications, their airway clearance. They are more susceptible to infections. Most of them will have a couple of major illnesses a year where they have absenteeism from school or work. It’s not a normal life. So it’s intensive in terms of the time a patient spends on it, and it’s intensive in terms of health care.
X: Why are you excited about the Vertex treatment?
BR: This is the first time that a therapy—a small-molecule, not gene therapy—is actually directed at trying to correct the protein. We’ve had other therapies that tried to improve ion transport by going around and using alternative channels. That’s the approach that [Durham, NC-based] Inspire Pharmaceuticals has with denufosol. But this [Vertex's VX-770] is actually trying to correct the protein. So if it works, we have shown this concept works. You actually can change a dysfunctional protein into a functional channel. Whether it turns out that Vertex is 100 percent successful or not, this is such a giant step forward, it’s like a man walking on the moon.
Now, the CF Foundation has had a long partnership with Vertex. The CF Foundation decided it had to try to go out and find a small-molecule. So they set up a partnership with Aurora, which then … Next Page »