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

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in the more common patient type, the delta F508s, but they decided to start with this group because they figured the odds would be best.

They did a Phase 2 test in G551Ds, which showed a dramatic improvement in the sweat test. This was incredibly exciting, because there is no treatment—ever—that has been able to change the sweat test. The sweat test is a measurement of the underlying protein abnormality. Also, it showed an improvement in lung function. And it showed a change in the nasal potential difference, which is another measure of the channel, the ion channel. We measure voltage transfer across nasal passages. So the sweat test, and the nasal potential difference, are both measures of the protein function. And both of those changed with VX-770. That, and the fact that the drug is safe and easily absorbed and so forth, is the reason that it’s encouraging.

X: So you saw an alignment between fixing the underlying biological problem, and the clinical outcome? It’s rational.

BR: Absolutely. The whole thing makes sense. The other time I felt like this, way back over a decade ago, was when we developed the inhaled tobramycin [treatment]. It all made sense. The drug killed the bugs. The lung function got better. It was a similar situation. We had seen in Phase II what we thought we’d see. Fortunately we saw the same thing in Phase III and the drug got approved. So we’re hoping the same thing will happen here. If they can just show again what they showed in Phase II, that will be a strong message.

X: We hear a lot from biotech companies, in the downturn, increasingly turning to disease foundations like the CF Foundation and others for help in development. Why does this model make sense, and what makes for a good partnership?

BR: The reality is that while CF is one of the more common rare diseases, it’s still a rare disease. In the past—and I will say this is changing, because somebody like Vertex could potentially be very profitable here—traditionally, big companies like Pfizer or GSK didn’t want to touch rare diseases. There’s just not a big enough market. With something like this, where else are you going to use it besides with CF? If you’re developing a blockbuster, you want to be able to use it for multiple indications, or across millions of people. The only way rare diseases could really attract a company to work in this area would be to go to them and say, ‘We’re going to reduce every possible barrier you might have.’ So we take the financial risk early on. We let you get a profit later. As long as we get the drug. And we’ll have a system set up so that you can get access to the patients.

There are two big risks that companies have. There’s the financial risk upfront. But second of all, they need to get patients enrolled in studies so they can get their drug approved. There are many barriers to doing trials in a pediatric population, because they’re a vulnerable population. But I think that with rare diseases, the families are more motivated to get treatments. They sign up for studies.

But that’s largely because the CF Foundation has done a huge job of putting together this clinical trial network called the Therapeutics Development Network which we run. There are 77 sites now around the country. That means the doctors, and nurses, and researchers are all very well trained in how you conduct trials. The other thing the foundation has done is set up a huge national education effort for families. It’s set up to explain to them why they need to participate in research. How do you tell whether a research study is a good study, or one that’s worthless? The program is called “I am the key.” They have all this education material to get families to enroll.

X: How has that played out in the Phase III trial for Vertex?

BR: Everybody knows within the network who the patients are. They are all being followed in the clinic. It’s not like a hypertension trial, where you go out and put ads in the newspapers and hope people start coming in. This is a very captured audience. But, it’s a tiny audience. So each center will only have … 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!