Vici Richardson's blog: Collective collaboration .. the road ahead

Vici Richardson, Trustee of Duchenne Now and member of Duchenne UK's Patient Advisory Board, has written this blog about thadult research workshop we hosted on Friday 19th of November in Newcastle with DMD Pathfinders.

At the workshop we discussed how we ensure adults can access treatments for DMD now and in the future.

You can read the blog below or on Vici's blog here:

Last Friday I attended a workshop organised by Duchenne UK, DMD Pathfinders and the Duchenne Hub. The workshop was to discuss how we can develop trials for adults in Duchenne.

Primarily I left encouraged.

  • Encouraged that we are now having conversations about trials in the adult population ( I think ) I’m not sure if everyone in the room embraced the word adult or if when the word adult is used they are actually meaning non ambulant. Currently there is only 1 trial that is for adult age the rest cut off at 16-18. That is not adult.
  • Encouraged because there are some real discussions taking place around outcomes and end points other than the 6 minute walk test. One of the slides was entitled ‘outcomes for grownups.’  The PUL (performance of upper limb) is now becoming a recognised standardised tool in clinical trials.  I’m sure there is further work to be done on tools to include adults and those with very limited upper body strength. There is definitely some work to be done still around measuring quality of life outcomes.
  • But most of all it was encouraging to see in one room representatives from health, research, pharma, patients and patient organisations

For the first time I felt there was a real desire for collaboration between industry, patient organisations,  health and research. That is testament to the hard work of Duchenne UK.

It’s been about two years since the emotionally charged meeting at the Centre for Life where patient organisations and researchers met in the midst of a crisis situation.

Clinical trials were being turned away as centres were at capacity. This situation had come about, I think, because of lack of foresight, collaboration and lack of funding.

Out of that meeting The DMD Hub was formed and Work began and now the situation is changing. In just two years we have more trial sites and more clinical trials taking place.

True Collaboration works!

Now we are faced with another challenge. Getting trials and treatments to the whole of the DMD population.

It’s a huge task! However we have the starting blocks.

  • We have standards of care that are now recognised for adults
  • We have conversations taking place
  • We have recognition from all sectors that this is needed.
  • And I think we have commitment … time will tell on that one.

Why is it so important ?

One day these children that we have invested in so heavily, in terms of research, care and trials they are going grow up. The majority will reach 18, 25 and possibly even 30!

They are going to be the next generation of adults. These children who can take advantage of trials now will need to be followed and monitored and we have to ensure we have the infrastructure to do that.

The drugs we have now are slowing the decline but they are not stopping it.

Better access to standards of care, better treatments , better heart and respiratory care isn’t going to ease the burden on our clinical infrastructure it’s going to increase it. The Duchenne adult population will increase as our children grow up and live longer. So more than ever we need to look at the bigger picture. Let’s not be so short sighted we create another crisis.

For example I was shocked to hear that one of the biggest hospitals in Scotland doesn’t have access to a hoist for dexa scans. We can start by getting the clinic infrastructure prepared.

I hope that yesterday was the start of something truly ground breaking. I am confident that Duchenne UK will hold every biotech and every researcher to account and we will further the opportunities for all with Duchenne.

As a side note : Catch 22

The first approved treatment for Duchenne took 18 years! I was listening to the representative from PTC who thoroughly depressed me with his presentation. He was proud of being the only biotech to have an approved treatment but as he went on to explain it took 18 years!

We don’t have 18 years to sort this for the adults with Duchenne.  I think we all know that by now.

When asked a question around ensuring access for adults on trials he said they are in a catch 22 situation. They have to design effective trials to meet endpoints to get approval or no one gets the drug. He said it’s about give and take. I still can not decide whether that was an insensitive or just a brutally honest answer.  However it was said to an adult patient and from what I understand there’s not been any giving or taking from the adult Duchenne population.  They don’t have the opportunities to give to research and they certainly haven’t been able to take from research.

We need industry and the regulators to really think outside their small 4-12 years old ambulant boxes.

We need to get ourselves out of this catch 22. Until we can find the magic bullet, which stops the disease in its tracks, we have got to remember that all these children will become adults and will need better access to treatment and care than there is currently.

We’ve got a long road ahead of us in this journey but finally it’s a journey that’s begun. 

Let’s not be afraid to make the paths where currently there are none, ensuring that those to come next can follow in these brave new steps.

Step by step let’s do everything we can collectively to ensure no one is left behind.

Duchenne UK is entirely reliant on donations to fund and accelerate the search for treatments for DMD
Please help us and donate today:
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  • Take part in one of our fundraising events – Events
  • Text DUCH10 £10 to 70070

Published on 22 October 2018

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