14th November 2019

Today we are hosting a gene therapy meeting in Newcastle. The meeting, organised through the DMD Hub, brings together 100 partners from hospitals around the UK and the pharmaceutical industry to discuss bringing gene therapy trials to the UK. 

Here is Emily’s speech welcoming everyone and updating on the progress of the DMD Hub so far.

Emily Crossley, CEO & Co-founder, Duchenne UK, said:

So why are we here today?

I am the mother of a 12 year old boy with Duchenne. His name is Eli. And my co-founder Alex is mum to Jack – also 12. And what both these boys want, and what Alex and I want, is for us to have the option to enrol them onto a gene therapy trial in the UK. So that’s why I’m here.

Why are you all here?

Because – quite simply – you are the people who can make that happen. 

And I know that TOGETHER we can be a very powerful force. 

The reason I think – or rather I KNOW - it can work is simple. It was four years ago in 2015 that I stood here to address a similar audience. And back then we were addressing a similarly challenging issue. Why WAS the UK – with its wealth of knowledge, expertise, experience and leadership TURNING AWAY clinical trials – RIGHT at the time when more and more companies were entering this space and running trials.

The problem was a lack of a capacity. And we came up with the solution.

So this was the picture in 2015. Two centres – running trials – but at capacity and turning them away.

Other sites – in yellow – were taking part in trials but were constrained by capacity issues to do more.

So we launched the DMD Hub – run by Emma Heslop here in Newcastle – a partnership between Duchenne UK, GOSH and Newcastle.

And this is the picture today in 2019.

The UK is no longer turning away clinical trials. We are open for business.

Instead of TWO trials centres – we now have nine! All running clinical trials today that otherwise wouldn’t be running.

And people told us it couldn’t be done – they told us: Charities don’t fund NHS posts …

Well it’s a good job we did!

Duchenne UK has invested £2.7million – funding 30 posts.

We’ve got 250 more patients on clinical trials than we did in 2015. 

And we’ve successfully used a pump priming model, where Duchenne UK funds the initial 100% of the costs of the post – but which tapers down once money has come in from industry to the sites. The idea is that this money then makes the posts self-sustaining. And this is already happening at two of our Hub sites, Alder Hey & Leeds.

And the Hub is more than that. It’s the interface between supporting hospitals, helping patients and working with industry.

So how do we do that? 

We launched our clinical trial finder – it provides up to the minute detailed information on EVERY trial in the UK. 

It tells you what each trial is doing. What the exclusion/inclusion criteria are.

It gives contact information for each trial site. And tells patients which sites are recruiting and which are not.

Emma worked closely with Catabasis to help them recruit. They did in in under 3 months in the UK. 

Their CMO Joanne Donovan said: 

“I believe the DMD Hub Clinical Trial Finder has significantly facilitated the recruitment for our POLARIS trial in the UK.” 

And I want to finish with another quote – this time from Prof Dirk Fisher who is the PI on the Tamoxifen for DMD Phase III study. 

“The DMD Hub has supported the TAMDMD study in a very positive manner by supporting and accelerating all regulatory issues in the UK in a professional way. Furthermore, additional study sites have been set up to enhance patient recruitment, a crucial aspect for the success of the study. I would also like to emphasise the efficient and straightforward management of the DMD Hub - Emma is doing an excellent job!”

So 4 years ago – we identified a problem and we fixed it! We went from 2 sites – at capacity – to 9 sites running clinical trials – with 250 more boys on trials than otherwise would have been. And the NHS and NIHR are now funding those posts that are delivering trials and brining money to sites. 

So – let’s do it all again and focus our minds today on the following 3 points:

  1. We want gene therapy trials in the UK.
  2. What are the barriers stopping us from doing this?
  3. How can we address, target and overcome these barriers?

For more information visit DMDHUB.org


What is Duchenne Muscular Dystrophy?

Duchenne muscular dystrophy (DMD) is the most common fatal genetic disease diagnosed in childhood. Children born with DMD cannot produce the protein dystrophin which is vital for muscle strength and function. Muscle weakness starts in early childhood. Many use a wheelchair by around the age of 12. As deterioration continues it leads to paralysis and early death, often in their 20s. It almost exclusively affects boys. There is no treatment or cure. In the UK there are around 2,500 boys affected and around 300, 000 worldwide. It is classified as a rare disease.

Who are Duchenne UK?

Duchenne UK is a lean, ambitious and highly focused charity with a clear vision: to fund and accelerate treatments and a cure for Duchenne muscular dystrophy.

We are investing millions of pounds in research right now to bring treatments and a cure to help this generation. Duchenne UK is the largest funder of DMD research in the UK. We are also committed to accelerating the pace of research. 90p in every £1 raised is committed to research.

Our president is HRH The Duchess of Cornwall. Our patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell. 

We need your help, because we need to keep funding promising new research.

How to donate?

Duchenne UK is entirely reliant on donations to fund research for treatments and a cure to DMD. This can be done via:

  • Direct Debit – Duchenne Direct
  • Individual Donation – Donate
  • If you are a family or friend affected by DMD you can set up your own fund with Duchenne UK – Family and Friend Funds
  • Take part in one of our fundraising events – Events
  • Text DUCHENNE to 70085 to donate £5. This costs £5 plus a std rate msg.

For more information visit www.duchenneuk.org