Wednesday 25 June 2014

What the Vertex Phase III Results Mean

Following yesterday's announcement from Vertex Pharmaceuticals of the results of the Phase III clinical trial of the ivacaftor and lumacaftor combination therapy, our Director of Care & Research Dr Janet Allen explains the key results.

Vertex announced the Phase III
combination clinical trial results for people with cystic fibrosis carrying two copies of F508del (which accounts for around 50% of the UK cystic fibrosis population). Now the dust has settled from the flurry of excitement, I’ve had a chance to look through some of the details in the text beyond the headline data on FEV1 lung function. What I find striking is the effect of the combination treatment on pulmonary exacerbations, which is remarkable, especially alongside such modest effects on FEV1. This finding on pulmonary exacerbations translates through to significant reductions in hospitalisations (39-61%) and IV antibiotics (45-56%). As we all know, pulmonary exacerbations contribute to continuing decline in lung function for people with cystic fibrosis. Together with the need for hospitalisations and IV antibiotics, this has a demoralising effect on individuals and their quality of life. The data here implies that these events could be halved by the combination treatment.  We have just come off the phone with Vertex where we discussed this exciting data and the team commented that many of its expert panel have also remarked on the beneficial effect on pulmonary exacerbations and how disproportionate it is to the effect on FEV1. Clearly more analysis needs to be completed and we need to have a better understanding of this effect but it is remarkable and shows that FEV1 is not necessarily the gold standard marker for measuring the effect of a drug in cystic fibrosis.

The other point that should not be missed is that this is a landmark scientific achievement. The F508del mutation in CFTR causes this protein to be fold incorrectly, which is a really difficult problem to fix. Many other conditions are caused by misfolded proteins and many programmes have tried unsuccessfully to restore the folding pathways in people. Here, a small molecule has been discovered that affects the complex folding of the protein, CFTR. This is an amazing achievement and shows the real value of linking high quality science and drug discovery with the determination to make a difference. 

The Trust is looking for people who took part in the trial to share their experiences – please email James Barrow, Head of Public Affairs, at james.barrow@cysticfibrosis.org.uk.

10 comments:

  1. This is amazing news, however I have only one mutation of F508del and the other Q220X can this help those with only one mutation?

    ReplyDelete
  2. that is great news, My daughter only has only one mutation of f508del i was wondering too if any hope for only mutation ? =)

    ReplyDelete
  3. My 11 year old son has one F508del and the other one unidentified. I also want to know if the heterozygous state could benefit from the treatment.

    ReplyDelete
  4. Could Dr Allen comment on the following questions please:

    1) Is there a scientific reason why the benefit would not be observed in CF-sufferers with only one version of the f508del mutation or it is simply that the study was limited to those sufferers with 2 copies?

    2) Is there a reason why benefits would not be seen in sufferers below age 12 or is this just the limit of the study due to the people involved in the trials?

    Your comments / feedback would be appreciated.

    Thank you

    ReplyDelete
  5. Dear Tom, thank you for the explanation and comments.

    ReplyDelete
  6. Problem: HP Printer not connecting to my laptop.
    I had an issue while connecting my 2 year old HP printer to my brother's laptop that I had borrowed for starting my own business. I used a quick google search to fix the problem but that did not help me. I then decided to get professional help to solve my problem. After having received many quotations from various companies, i decided to go ahead with Online Tech Repair (www.onlinetechrepairs.com).
    Reasons I chose them over the others:
    1) They were extremely friendly and patient with me during my initial discussions and responded promptly to my request.
    2) Their prices were extremely reasonable.
    3) They were ready and willing to walk me through the entire process step by step and were on call with me till i got it fixed.
    How did they do it
    1) They first asked me to state my problem clearly and asked me a few questions. This was done to detect any physical connectivity issues with the printer.
    2) After having answered this, they confirmed that the printer and the laptop were functioning correctly.
    3) They then, asked me if they could access my laptop remotely to troubleshoot the problem and fix it. I agreed.
    4) One of the tech support executives accessed my laptop and started troubleshooting.
    5) I sat back and watched as the tech support executive was navigating my laptop to spot the issue. The issue was fixed.
    6) I was told that it was due to an older version of the driver that had been installed.
    My Experience I loved the entire friendly conversation that took place with them. They understood my needs clearly and acted upon the solution immediately. Being a technical noob, i sometimes find it difficult to communicate with tech support teams. It was a very
    different experience with the guys at Online Tech Repairs. You can check out their website www.onlinetechrepairs.com or call them on 1-914-613-3786.
    Would definitely recommend this service to anyone who needs help fixing their computers.
    Thanks a ton guys. Great Job....

    ReplyDelete
  7. Is Your VIRUS REMOVAL
    Computer Sluggish or Plagued With a Virus? – If So you Need Online Tech Repairs
    As a leader in online computer repair, Online Tech Repairs Inc has the experience to deliver professional system optimization and virus removal.Headquartered in Great Neck, New York our certified technicians have been providing online computer repair and virus removal for customers around the world since 2004.
    Our three step system is easy to use; and provides you a safe, unobtrusive, and cost effective alternative to your computer service needs. By using state-of-the-art technology our computer experts can diagnose, and repair your computer system through the internet, no matter where you are.
    Our technician will guide you through the installation of Online Tech Repair Inc secure software. This software allows your dedicated computer expert to see and operate your computer just as if he was in the room with you. That means you don't have to unplug everything and bring it to our shop, or have a stranger tramping through your home.
    From our remote location the Online Tech Repairs.com expert can handle any computer issue you want addressed, like:
    • - System Optimization
    • - How it works Software Installations or Upgrades
    • - How it works Virus Removal
    • - How it works Home Network Set-ups
    Just to name a few.
    If you are unsure of what the problem may be, that is okay. We can run a complete diagnostic on your system and fix the problems we encounter. When we are done our software is removed; leaving you with a safe, secure and properly functioning system. The whole process usually takes less than an hour. You probably couldn't even get your computer to your local repair shop that fast!
    Call us now for a FREE COMPUTER DIAGONISTIC using DISCOUNT CODE (otr214425@gmail.com) on +1-914-613-3786 or chat with us on www.onlinetechrepairs.com.

    ReplyDelete
  8. Software Development Company offers on & offshore Software Development Services. Mag Web Enterprise IT services deliver excellence to new age IT businesses. Explore new possibilities of enterprise automation.

    ReplyDelete
  9. Hello

    It is really very good for service like Trust accounting software, Self Directed IRA Accounting, Religion foundation trust accounting software etc. and we also providing those finest services for our cilents. for more information visit on us :-http://www.hwainternational.com/

    ReplyDelete