DMD research

Update on Duchenne UK’s JAK Inhibitors project with AGADA Biosciences

10th November 2020

We are disappointed to share the news that our JAK Inhibitors project with AGADA Biosciences, funded through Duchenne UK’s INSPIRE 2019 Major Grant Call, is being terminated early due to lack of efficacy

Duchenne UK made a grant to AGADA Biosciences to test the impact of two approved drugs on protecting muscle in DMD.  The two drugs, Tofacitinib and Ruxolitinib, are understood to work by blocking the Janus Kinases (JAK) pathways which communicate information to cells, and contribute to the production of collagens, causing a build-up of fibrosis in the muscles.

Unfortunately, the data generated by Dr Nagaraju’s group at AGADA Biosciences in Canada did not demonstrate any clear benefit.

The team at AGADA used a systematic and thorough process to see if these two drugs could reduce inflammation and fibrosis, and improve muscle regeneration, in a similar way as they do for other diseases they are approved for.

The two drugs were tested very carefully in a range of experimental procedures known to be capable of demonstrating a positive drug effect in DMD.

Despite trying a range of doses in appropriate strength and endurance tests the data did not show efficacy with either drug. After a comprehensive online presentation/discussion with Dr Nagaraju and key members of his team, we all decided that there was little reason to continue with further planned work.

It is disappointing we did not get the results we hoped for. But it is important to share all scientific discoveries – whether they give us the result we wanted or not. We hope that by sharing this information, the data will help inform other groups who may be working in this area.

Duchenne UK carefully funds projects in staged payments. In this case, the remaining sum of £83,750 can be reallocated to other work that Duchenne UK supports.

We would like to thank our partner charities and family funds for supporting this project: Joining Jack, Archie's March, Muscles for Mitchell, Cure4George and Jacobi's wish.


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

Published on 10 November 2020

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