Vertex Drug Could Be “Man on the Moon” for Cystic Fibrosis Therapy, Says Researcher Bonnie Ramsey

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got bought out by Vertex. The idea was to do this high-throughput screening. It was really challenging, because they didn’t have the right kind of assays. You’ve got to have an assay to do your screening. To find an assay looking for changes in chloride transport—that was something they had to develop. The CF Foundation, and CF researchers around the country, really worked with Aurora to develop these assays and then develop a drug screening process. They got some hits. Then Aurora, and subsequently Vertex, really did a phenomenal job, with a lot of support from the CF Foundation. They then used combinatorial chemistry to get an initial hit, they’d make changes in the compound, go back and test it again, and all the while developing assays to see if anything was getting better.

I was fortunate enough to be on one of the advisory boards. It was an amazing process to watch. They’d say, ‘Well, we’ve hit another barrier, I’m not sure we can make it any better.’ Then, sure enough, you’d come back six months later, and they figured out a way to make it 100-fold, or 1,000-fold more potent. So it was an amazing process. Then fortunately, as you develop a medicinal product, you can have a very effective chemical, which unfortunately can’t be a drug. It might be toxic, or not properly absorbed, or metabolized too quickly. There’s a lot of things that can go wrong. They really lucked out. These small-molecules were really good drugs. But one thing they couldn’t do was find one drug that could take care of the whole process. So, in patients with the most common type of CF defect, delta F508, the protein is not properly transported to the surface of the cell, and then it doesn’t properly open. It looked like there were different kinds of small molecules needed for the trafficking, and for the opening. They basically came up with two drugs, rather than one miracle drug that could do it all. I think initially, they were hoping it could all be done by one, but so far it looks like two drugs. That’s VX-770, and VX-809.

X: So the CF Foundation has put a lot of money, and a lot of time, into this. Did the foundation have internal debates, or face critics who said, ‘You’re putting too many eggs in this one basket,’ or, ‘Why are you putting all this money in, and letting somebody else make all the money from it?’

BR: You’d have to ask (CF Foundation president) Bob Beall those questions. I must say, first of all, the CF Foundation board was very supportive of the concept. Bob Beall is truly an amazing human being. He’s visionary, and focused. I remember early on, he said, ‘This is going to work.’ You know, I’ve been involved in CF research for 30 years, and I can remember thinking that there was no way this is going to work. But I will say, that once I realized the quality of the team that had been put together, both on the CF Foundation side, and on the Vertex side, if anybody was going to be able to pull this off, they would.

Oftentimes, you’ll hear people going off on these fishing expeditions where they just don’t have the talent to pull it off. But they really, really had a phenomenal team. And a very careful team. This was very methodical, all along the way, and in getting the right people in there to do it. And there’s luck. There’s no question there’s luck. You could do every step right, and you could still end up with a drug that was too toxic.

X: But it hasn’t gone all the way through trials yet. What gives you so much confidence?

BR: That’s absolutely right. We’re in a Phase III study now, which will tell us whether it really works or not. The reason I’m feeling more confident is that it’s gone into humans in Phase I and II. There haven’t been any showstoppers. Often if you are going to see acute toxicity, you’ll see it there. They are able to get good drug levels in the body. It’s an oral tablet, it’s easy to take, easy to tolerate. That’s all very encouraging. That’s been the case for both Vertex drugs. VX-809 is one phase behind VX-770. There hasn’t been a showstopper yet for 809 either. The difference there is we don’t have data yet on how effective it is.

With VX-809, we may not be able to truly tell how effective it is until the time comes when it can be combined with VX-770. Remember, VX-809 does the first step, it gets the protein up to the surface of the cell, and then VX-770 opens it up. So if you just get it to the surface, without anything to open the protein, you may not see a lot. Except that you got it to the surface.

With VX-770, there was a natural selection process that helped them. There’s a genetic defect called G551D, which does the trafficking to the surface itself. So all you have to do is open the protein. If you choose those patients—and that’s why they chose an ultra-small patient population—if you choose G551D patients, their proteins can be opened up.

X: That’s maybe 10 percent of all CF patients, right?

BR: Right. The downside is there aren’t a lot of these patients out there. They are going to test it … Next Page »

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16 responses to “Vertex Drug Could Be “Man on the Moon” for Cystic Fibrosis Therapy, Says Researcher Bonnie Ramsey”

  1. Marla says:

    Praise God! Keep the research going, my two girls need it!

  2. Donna says:

    My daughter is going to be 38 years old in January. I’ve been involved with CF for a long time! The research and trials are exciting, but in the mean time we have to keep our children as healthy as possible. I am involved with a product called Juice Plus+. Nutrition is so important for our health. The studies show that Juice Plus+ can give you the fruits and vegetables that you are missing in your diet. JP+ increases blood antioxidant levels in 28 days, it strengthens the immune system, repairs damaged DNA and protects DNA from free radical damage. We need to get our CF patients on this whole food based nutrition. One of my colleagues daughter’s pulmonary function went from 25% to 40% after being on JP+ orchard/garden and Vineyard blend. Look at my web site and see all the research and science that have been peer reviewed and published in medical journals. See what the experts are saying. For more information please contact me at or call me at 847/680-4860. Good health to all!

  3. Kevin Chow says:

    Nice article. It would be worthwhile pointing out that there is another potential therapeutic for aiding mucus clearance in CF patients being developed by Gilead’s pulmonary R&D group based right here in Seattle. It’s a collaborative effort with Parion Sciences of Durham, NC and currently it is in Phase 1 clinical studies.

  4. cecilia says:

    yo tengo tres hijos con fibrosis quistica, y mi duda es , si ya estamos cerca de algo mas prometedor para los homocigotos deltaf508.gracias

  5. BRANDY HARPER says:


  6. Andrew Kolodziej says:

    This is truly walking on the moon. The CF foundation has been the NASA for this endeavor–funding research to improve antibiotic treatment, creating the development network to test therapies and spread best practices has had a huge impact, bringing all CF families together to share experience and knowledge, and funding companies like Vertex, Aurora, Inspire and EPIX to find cures to the underlying problem. There is good reason to hope that there will be a cure or at least a significant ameliorative soon.

  7. Brenda Friend says:

    I want to thank Dr. Ramsey, the CF Foundation and everyone involved in this drug research. Dr. Ramsey was my daughter’s physician 26 years ago. It’s great to have her working diligently on saving our children/adults, and being so encouraging to the rest of us! Bonnie, your Kodiak baby girl says hello and is doing well. She’s teaching 2nd grade and trying to stay healthy. We appreciate all of your hard work and know you’ll get this kicked for us!

  8. Luis Carrillo says:

    My son has CF, he is 8 years old, at the begining, we did not know what this disease was, its been very hard for us to accept that his life is threaten. Its been a new life since two months ago, we hope for the cure or more medical treatments soon, I’m sure doctors are working hard to find it. And, not only 30,000 patients in the US are waiting but all of their families as well. Thank very much.

  9. Alfred arayza says:

    i am a kid with cf.I am 15 and hearing about the cure makes me very happy cuz i want to be normal not haveing to take pills all the time I HOPE THEY FIND A CURE.

  10. jill marchione says:

    My son has the G551D Mutation, we are currently waiting to start the trial at CHOP.I cant tell you how excited we are. for years we have watched promising treatments turn out to be nothing. But we have real hope with VX770!

  11. Brandon B says:

    I have CF. I am a 25 yr old college senior, kinda old to still be a college student, but trips to the hospital makes it hard to continue the course. I live in Houston is there research being done around here? I am all for donating my time to help the others who have CF, because I know we are all sick and tired of taking all those pills and breathing treatments!!!
    Note to all the CF fishermen: Good way to tell if your fishing spot has fish that are feeding, take a good couple of deep coughs spit the mucus in the lake. If the mucus is gone before you can cough up more, throw in, you are going to catch alot of fish!

  12. I am a CF parnet, unfortunetly we lost our daughter at the age of 2 who also was battling CF. I am still very active in fundraising and staying up on all of the accomplishments that have been made. Vertex, I know this is a great leap in the CF community and I comand all that this will work for and I suppoort all and everyone that is out there fighting. I am behind all of you and best of luck to all. Hope and thoughts are going out to all.

    God Bless !

  13. I am a CF parent and unfortunetly we lost our daughter to CF at the age of 2. I am still very active in the fundraising and stay up to date on all of the accomplishments that have been made. I hope and I know deep down that Vertex will be the CURE. I command all of you that are affected by this diesase and all of the parents, freinds, and families that stand behind and supprt all of this research.

    God Bless to all !

  14. Jan Johnson says:

    My niece has CF and after a lung transplant is not doing well. She is 22 years old and in ICU on a breathing machine. Depending on which mutation she has is there any way she could benefit from the clinical trials? This is a last resort so please let me know if there is any hope for her.

  15. SM says:

    I’m 43 and I have the delta F508 mutation. My lung function has declined a LOT in the past 10 years. These drugs are very promising and I am hopeful that it will improve my quality of life. Keep up the good work!