VamoroloneAlternative to existing steroids for Duchenne patients
The only current standard of care for boys with Duchenne is early treatment with long-term steroids, which gives some protection to muscles but with significant side effects. In July 2014, alongside three other Duchenne charities we funded a $2.1 million Phase 1 clinical trial of Vamorolone – an alternative anti-inflammatory drug that aims to maximise the benefit and minimise or remove side effects.
We were delighted to hear that in March 2016 the project received a further £4.6 million of funding from an EU grant known as Horizon 20/20, enabling the clinical trial to progress to Phase 2.
The charities contributed the funds under a co-ordinated venture philanthropy model, providing return on investment based on future drug sales. Vamorolone has been shown to be effective in animal models of Duchenne muscular dystrophy, and clinical trials in humans are now under way – in part, thanks to funds we and our partner charities have donated.
In June 2018 ReveraGen shared results from its international clinical trial in 48 boys aged 4-7, which showed dose-related improvements of multiple function tests of strength and endurance. Click here to read the press release.
What are the potential benefits?
In studies, the drug reduced inflammation and stabilised cell membranes, both of which are believed to be relevant to treating Duchenne, a disease in which muscle fibre membranes are unstable. The drug is thought to have similar properties to steroids such as prednisolone, which up until now have been the cornerstone of treatment for boys with Duchenne. The benefit of Vamorolone, however, is that it is thought to have significantly fewer side effects than existing steroids, which can be life changing in themselves. Side effects include diabetes, high blood pressure, osteoporosis, short stature and weight gain.
Funded in 2014
News from the Hospital for Sick Kids, Toronto
Solid Biosciences announces preliminary gene therapy clinical trial results and intention to dose escalate
Solid Biosciences has announced preliminary data from its gene therapy clinical trial, IGNITE DMD …
Five years ago we identified gene therapy as a viable treatment for Duchenne. Today we are reflecting on the exciting and inspiring events of the past week in the gene therapy arena which are now bringing hope and cautious optimism to the Duchenne community. We have...
Message from Solid Biosciences: "We’re pleased to share with you that the FDA has lifted the clinical hold on IGNITE DMD, our clinical trial investigating SGT-001 as a potential treatment for Duchenne muscular dystrophy (DMD). SGT-001 is a microdystrophin gene...
Solid Biosciences recently reported that it has raised up to $50 million which will enable the biotech to continue to advance its programs for Duchenne muscular dystrophy, including development of a microdystrophin gene. We are forever grateful to our incredible...
We are thrilled that the money raised through the Duchenne Research Fund’s 2013 dinner and donations since then have enabled us to seed fund the incredible Solid GT project, which has today received industry investment (read the press release below). While gene therapy is an exciting piece of the puzzle for beating Duchenne for all boys, it is now more critical than ever that we continue to raise vital funds to push Duchenne therapies into clinical trial as quickly and safely as possible and to get them to our boys NOW.
The Duchenne Research Fund is incredibly proud of Dr Dongsheng Duan – an important Scientific Advisory Board member of Solid GT, a gene therapy programme pioneered by Solid Biosciences and that we helped to fund. Dr Duan’s latest gene therapy work on muscular dystrophy dogs is set to significantly impact 100% of the Duchenne population.
He also recently received a five-year, $3 million grant from the NIH (National Institutes of Health) …
The devastating impact of steroids
Click here to learn more about the effects that current steroid treatments have on boys with Duchenne.