Trial Update

So we’ve reached that cliff-edge a little sooner than I thought.

Today, Shire (the pharmaceutical company that is running the trial) released this press release. I’ll give you a moment.

Read it? Confused? I’ll see if I can translate….

Now that all the boys have completed their first year on the drug or in the control group, Shire have looked at the data. Specifically they were looking at those dreaded cognitive assessments and seeing how they differed in the boys receiving the drug and those who weren’t. And these results haven’t shown enough of a difference to take the drug forward for approval.

To be honest, I’m not that surprised. Pudding hates the tests almost more than I do, and for the last two times at least has refused to do tasks that I know he is capable of. Why should he draw a circle for the man when he knows there is a football outside that he could be kicking down the corridor? These old-fashioned cognitive tests simply don’t work well with our MPS children. (And yes, clinicians and parents have already been telling them this.) Also, a year is such a short time to assess change in a long-term progressive disease.

So, the pharmaceutical company is going to now look in greater detail at the results from each individual to see if they can pick out useful trends and data. Many families – particularly those with boys who have been on it for years – can tell them of so many ways in which it is working for them. But I simply don’t know if in the end, that will be enough. In the meantime, Pudding and the other boys already enrolled on the trial will continue to receive their dose as usual.

So, it’s not the worst news, but it’s also certainly not the best. We’re teetering on the edge of that cliff and all we can do is wait. And for those families hoping for this treatment to be made more widely available, the wait is even longer.

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(This is my first blog post for a wee while as Hubby has been doing some behind-the-scenes work transferring the website to a new home. Hopefully I haven’t lost anyone in the move. If a few email subscribers could wave to say you’ve got this, that would be great!)

12 thoughts on “Trial Update

  1. Andrew

    Blooming murder trying to keep your feet on the ground when dealing with pharmaceuticals and stuff – have watched families struggle with it over the years with announcements and claims and counter arguments and blind alleys about treatments for kids with Duchenne muscular dystrophy. We feel for you guys v much so. We are still receiving the blog OK Take care for now Andrew and Helen xxxx

    Like

    1. huntersmum

      Yes indeed. I know the MD community has gone through a lot on this. At least we are in a period where so many more options are coming up – we just don’t know at the moment whether the right ones will get there in time!

      Like

  2. Kevin Willetts

    Rupert says Happy New Year to you all from Great Ormond Street after yet another GA operation. Adanoids and tonsils plus MLB. Here’s a wave from His granpa Kevin. x

    Like

  3. Pingback: The Good, The Bad, and The Ugly – A Hunter's Life

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