08.08.2019

  • Duchenne UK grant £73,500 to Leiden University to test whether Vitamin B3 (Nicotinamide Riboside) can improve regeneration of muscle and help retain muscle tissue
  • We will test a regular vitamin, B3, to see if it can help with the symptoms of DMD

The project is looking at how Vitamin B3 works with muscle stem cells to keep them functioning and encourage muscle regeneration.

The first part of the project will be looking at the effect of Vitamin B3 on DMD muscles in mouse models. Based on the results from this, the second part of the project will look at the effect of Vitamin B3 on muscle quality and function.

It is *just* a humble vitamin, BUT if B3 does show to help muscle cells to function and regenerate, it could be a very attractive potential treatment. We already have a good safety profile through other clinical trials taking place to test Vitamin B3 in several conditions, such as cardiovascular diseases and chronic kidney diseases. It is fairly cheap and widely available.

We are delighted to be working again with the researchers at Leiden University Medical Centre, who focus on research into genetic diseases, particularly DMD.  Duchenne UK is also funding their work on delivering exon skipping to DMD patients through antisense oligonucleotides.

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

“We know that some parents give their boys Vitamin B3 and this project will provide us with some hard data on the rationale and advantages of its use. We have worked with our colleagues at Leiden University Medical School on several projects and we look forward to seeing their data soon.”

Ingrid Verhaart – Senior postdoc, manager of the DMD validation lab, Department of Human Genetics, Leiden University Medical Center, Holland, said:

“We thank Duchenne UK for the opportunity to investigate the beneficial effects of vitamin B3 in DMD. We hope our research will contribute to the development of therapies to improve the quality of life of DMD patients.”

We would like to thank our partner charities and family funds for supporting this project: Joining Jack, Archie's March, William's Fund and Jacobi's Wish.

Q&A with Ingrid Verhaart – Senior postdoc, manager of the DMD validation lab, Department of Human Genetics, Leiden University Medical Center, the Netherlands

What is Nicotinamide Riboside?

Nicotinamide Riboside is the scientific name for vitamin B3.

Why is Vitamin B important to muscle regeneration?

Minor damage occurs in your muscle every time you use them. Precursor cells of the muscle, so called muscle stem cells, will develop into muscle cells that can replace the damaged muscle (muscle regeneration). Vitamin B3 helps to keep these muscle stem cells functional and thereby improves muscle regeneration.

How can an ordinary vitamin have an impact on a complex disease like DMD?

Since DMD is such a complex disease there are many aspects inside the body, which are disrupted. Vitamin B3 cannot counteract all these disturbances, but it can help to improve some parts and thereby help, next to other medicines, to alleviate some of the symptoms and/or prolong the maintenance of muscle function.

Should I start giving my son vitamin B now, and will it interfere with any other treatments?

These studies are being planned in mice first of all, to confirm that vitamin B3 does indeed help with the regeneration of dystrophic muscle. They will then be used to identify safe doses, as high levels of vitamin B3 can be toxic. It can also interfere with other medicines. We advise always speaking to your clinician before taking any vitamins or supplements.

If this project is successful, what will the next steps be?

This are early, preclinical experiments in a DMD mouse model to determine if vitamin B3 could indeed be helpful in the treatment of DMD. If positive effects are seen in mice, the next steps would be to test determined safe levels of vitamin B3 in a clinical trial in Duchenne patients.

If you have any further questions, do email [email protected]

This project is part of our DMD INSPIRE Major Grant Call. Find out more here:Duchenne UK invests £1,25 million into new research of treatments for DMD


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]