Dr. Clancy is one of the top CF researchers in the country, and also a practicing pulmonologist at Cincinnati Children's CF Clinic. He's been a part some of the biggest new drug studies, including the one for the drug VX-770 which is currently at the FDA awaiting approval for use in patients. If you hadn't seen my post on huge this drug is to the CF world, click here. It essentially corrects the basic defect of CF in patients carrying a certain CF mutation. Its a remarkable medical breakthrough for the entire genetic disease community, not just CF.
So back to Dr. Clancy. He had arranged with the CF Foundation a tour of the Clinic and inpatient care facilities, as well as the CF research center. It was open to anyone - patients and families, large donors, people who were just interested in learning more about CF. Today was the tour and I had the honor of attending. It started with about an hour long chat with Dr. Clancy telling us all about the great stuff that's currently going on in the research world, including current studies going on and drugs that are coming down the pipeline. Never has anyone involved with any genetic disease ever been so close to finding the cure as the CF researchers are to curing Cystic Fibrosis. Dr. Clancy talked a lot about the different drugs that help any number of the 1800 different mutations. With so many different mutations, they are grouped into 6 classes based on their defect and how they affect the person. The drug that the FDA currently has, VX-770 or Kalydeco, was created and essentially corrects the problem called by 1 specific mutation called G551D. I asked Dr. Clancy today if that drug was designed to just help that specific mutation, or if it would be beneficial to anyone who had a mutation in the same class as the mutation that G551D falls into. He said that there has been some testing done recently on the other mutations within Class 3 (that's the class that G551D falls into) and the drug seemed to have the same positive affect on the other mutations tested. And one of Drew's mutation, R560T is a class 3 mutation! We won't know for sure until the drug is available to try it if it will help him. The tricky thing with CF is that with two mutations, both falling into different classes, its hard to say if fixing just one of them will correct your problem. In some people it certainly seems to, but in others they improve but not enough to be symptom free. The good news is that the drug that would correct Drew's other mutation, F508del, is in the works (VX809 - you can read about it by clicking on VX809 and then finding it in the pipeline (near the top) and clicking on the hyperlink). Hopefully within the next 3-5 years, this drug will also improve and make it to the FDA for approval and then use in patients. Taking these two drugs together have the potenial to "cure" CF for Drew and thousands of other people. Obviously I'm hoping that the 770 does the trick because that will be available, with any luck, in the next year. The longer we go without a cure, the more risk of irreversible lung damage we have. Of course we will take a cure whenever we can get one, but the sooner the better.
So the tour and the talk today were fabulous and left me feeling so good and hopeful. I could listen to Dr. Clancy talk for hours on end. If you ever get the chance to hear him speak I highly recommend taking it. He's a brilliant man who has his hand in so many different things, and yet he can still relate to you on a very easy to understand level. He's a really nice guy and I think he's a big part of the cure.
I know this post seemed to ramble on and on. I couldn't contain my excitement and now anticipation of the approval of this new drug. I know I am constantly harping on how important funding is, but without donations and support of our wonderful family and friends, these drugs wouldn't be what they are today. In this season of giving, the CF Foundation would so greatly appreciate your support through a donation. And so would I. We're going to find a cure for this disease and soon, I just know it!