Duchenne UK announces its DMD INSPIRE 2019 MAJOR GRANT CALL for projects

14th January 2019

Duchenne UK launch DMD INSPIRE 2019 Major Grant Call to fund the best research proposals into the treatment of Duchenne muscular dystrophy

Duchenne UK, a patient organisation based in the UK with global reach, is pleased to announce the launch of our 2019 Grant Call. We wish to invite research proposals for submission and review.

Specifically, we are interested in receiving proposals relating to two therapeutic approaches:

1. Treatment of the root cause of the disease through innovative gene therapies and/or more effective therapy delivery methods

2. Novel approaches designed to regenerate muscle fibres and/or remove/reduce fibrosis in muscles

Why have we chosen to focus on these two areas?

Over the last 5 years there has been remarkable progress in our ability to develop treatments which have the potential to address the root cause of Duchenne muscular dystrophy. Advances in gene therapy have led to clinical trials which indicate a real impact on the disease. But we are still a long way from optimal treatment for all DMD patients, irrespective of their specific mutation. More is needed to advance our understanding of the best constructs, the best targeting methods and the best ways we can safely, effectively and repeatedly deliver gene therapies to the muscles where they are needed.

But improving treatment of the disease itself is not enough. Genetic therapies will help very young DMD patients, and those who will be diagnosed in the coming few years, but there are thousands of patients who need a different sort of help in addition to treating the root cause. Muscles that have been damaged are currently difficult to repair. How can we attack the fatty deposits? How can we remove fibrosis? Then, crucially, how do we help the muscles regenerate and so halt progression of disability, or reverse it? We’d like to hear your novel ideas on this too.

This call has a broad remit in terms of drug development stage: Proposals that are in early preclinical stage, but which show great promise as alternative approaches are welcomed, as are drug development projects with the potential to be in clinical trials now or in 2–4 years and which have compelling pre-clinical data in appropriate animal models.

How to make your submission?

In the first instance send an email to Dr David Bull and Megan Mullany ([email protected] and [email protected]) indicating which of the two therapeutic approaches you are interested in, a brief (500 word) outline of the project and approximate costs. If you meet our criteria, we will then send you a detailed submission template which you should complete in full and return.

Full submissions, sent on the template referenced above, should be received at the latest by

Monday 18^th March 2019 SUBMISSIONS ARE NOW CLOSED

How will your submissions be reviewed?

All submissions will be carefully reviewed, initially by our joint CEOs and our Research Director/Research Manager. Subsequently we will have a thorough review of selected proposals at specially convened Scientific and Patient Advisory Board meetings. You will, of course, be advised of the progress of your submission.

The co-founders of Duchenne UK, Emily Crossley and Alex Johnson said:

Supporting and accelerating innovative research is at the heart of what we do at Duchenne UK and we are pleased to be offering these grants of up to £2million. Gene therapy is offering great promise, but challenges exist, especially in areas like antibody status. We hope that this call will encourage groups working in these areas to submit applications and engage with us on bringing new treatments to the clinic.

The Director of Research, Dr David Bull, said:

We know that research groups around the world are doing varied and exciting work aimed at defeating this disease. Some of those groups need help to achieve their goals and Duchenne UK wishes to partner with the best of these.

The 2016 Duchenne UK Innovate Grant Call

In 2016 Duchenne UK launched its first Innovate Grant call for £1million. The successful recipients were Prof Miranda Grounds and Dr Peter Arthur of University of Western Australia and their investigation into Taurine as a treatment for DMD.

What is Duchenne Muscular Dystrophy?

Duchenne Muscular Dystrophy 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. The charity has been formed by the coming together of Joining Jack and Duchenne Children's Trust, the two biggest funders of DMD research in the UK in the last three years. Its president is HRH The Duchess of Cornwall. Its patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell. 

For more information: visit www.duchenneuk.org or email Molly Hunt, Communications Manager: [email protected]

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 DUCH10 £10 to 70070