JULY 2019

As part of its commitment to accelerate the search for treatments, Duchenne UK is investing in the development of a new anti-oxidant drug that could potentially help protect muscle, with a grant to the University of Reading of £63,618. 

Many of the symptoms of DMD – including muscle fibre death, inflammation and fibrosis - are caused by harmful molecules called Reactive Oxygen Species (ROS) acting on cells in the body. In this project, scientists will examine whether the new anti-oxidant drug TEMPOL can reduce ROS, protect cells and limit muscle damage.

TEMPOL is an attractive compound for Duchenne UK to look at, because it’s already being trialled in humans for a number of other conditions including diabetes and cancer. This means that if it does show promise, it could take much less time than new drugs to go get approval.

This potential new treatment would be effective on ALL mutations of DMD.

This study will be in a new model of DMD mouse (D2-mdx) which it’s hoped will better mimic effects in humans. This is a 14 month study.

Duchenne UK has invested more than £11 million so far into research into DMD. 

Dr Ketan Patel, Head of Molecular and Cellular Medicine, University of Reading said:

We are pleased to be working with Duchenne UK on this important project to test the efficacy of TEMPOL to slow the disease progression of Duchenne muscular dystrophy.

Dr David Bull, Director of Research, Duchenne UK said:

Duchenne UK is absolutely committed to bringing treatments to patients as robustly and quickly as possible. TEMPOL has the potential to quickly advance to clinical trials if the data from this study shows promise. We are pleased to be working with Dr Patel on this study.

Duchenne UK would like to thank our family funds Jacobi’s Wish and Help Harry for supporting this project.

Q&A with Dr Ketan Patel, Head of Molecular and Cellular Medicine, University of Reading

Dr Ketan Patel, how did you meet Duchenne UK and how did this project come about?

My awareness of Duchenne UK cones through a number of avenues. Firstly they have supported research projects that yielded highly influential scientific papers related to DMD. Additionally I have reviewed papers originating from studies funded by Duchenne UK. Having worked on DMD for the past 25 years, I am also aware of all the bodies, including Duchenne UK who promote research into this disease. Lastly, I met Dr David Bull at a World Muscle Society conference where we had a number of stimulating discussions related to DMD and the number of treatments currently being developed.

 

What is Tempol?

It is small organic molecule that acts as an antioxidant by two mechanisms. It catalyses the breakdown of many dangerous reactive oxygen species into harmless molecules. Secondly it is also thought to work by increasing the levels of enzymes, in particular one called super oxide dismutase which itself promotes the breakdown of reactive oxygen species. A number of studies have demonstrated that it is one of the most powerful, yet biologically safe anti-oxidants thus far investigated.

 

What will you be using Duchenne UK's grant to do?

A number of mouse base studies using models for DMD have shown that it is possible to slow down disease progression by addressing some of the events initiated by the lack of dystrophin without having to address the primary insult. One of the mechanisms that we and others have examined that leads to DMD pathology is that related to elevated levels of harmful reactive oxygen species. This grant funds research that examines the efficacy of TEMPOL to slow disease progression in a mouse model of DMD. We are excited not only by the opportunity to use a powerful anti-oxidant but also a relatively new mouse model of DMD; one that better models human disease compared to the conventional mdx model.

 

Would Tempol need to go through the standard clinical trial process of a phase 1,2,3?

TEMPOL will have to be assessed through clinical trials. However, some stages should be accelerated since TEMPOL has been used in a number of previous trials without having issues related to safety of the compound. So it would take less time to get this treatment to patients, compared to a brand new drug.

 


NOTES FOR EDITORS

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 and interview requests:

Visit www.duchenneuk.org

Molly Hunt – Head of Communications, Duchenne UK E: [email protected]