7th October 2020

Simvastatin, a drug used to treat high cholesterol, had shown promise as a treatment for Duchenne. But a scientific paper published this week – which describes some work funded by Duchenne UK - showed that the drug had no impact on DMD progression.

We are disappointed to share this news with the community. But it was an important study to give us a definitive answer at this early stage, so no further money or time is spent.

In 2016, scientists at the University of Washington published promising data about the effect of simvastatin in treating some of the symptoms of DMD. They showed that Simvastatin reduced cardiac and skeletal damage, and enhanced muscle function in an animal model. Their research caught our eye because Duchenne UK is interested in repurposing drugs - testing existing medicines and developing them as treatments for DMD. Repurposing is attractive because it can dramatically reduce the time it can take to get medicines in to clinical trials on patients, because these medicines are already approved as safe for use in humans. This also reduces the cost of the medicine.

Before investing more in Simvastatin, we wanted to make sure we could replicate these results in a different laboratory. Replication work is common in scientific research and seen as good practice to confirm positive findings in an independent laboratory.

Unfortunately Prof Aartsma Rus and her teams results did not replicate the data from the original study. They could not get simvastatin into the mice plasma at the expected level and it showed no effect.

Whilst these findings are disappointing, we are at least relieved that we funded this independent work, rather than committing millions of pounds to run a clinical trial to test simvastatin as a treatment for DMD. And we remain focussed and committed to developing treatments for this disease.

As a community, we are painfully aware of the challenges of drug development. Only a small amount of medicines that show promise in a laboratory actually result in approved new medicines. Often this is because what can look promising early on, ends up not working in the very complex human body. This is why it’s very important to cross check early positive results to make sure that you are embarking on a drug development pathway with the most promising and tested compounds.

If you have any questions, please email [email protected] 

 


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 has one clear aim – to end Duchenne, a devastating muscle-wasting disease. As the leading Duchenne charity in the UK, we connect the best researchers with industry, the NHS and families to challenge every stage of drug development to make the incurable, curable. Together, we will find treatments and cures for this generation of patients with Duchenne.

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