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, the Duchenne Research Fund helped fund 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.
Vamorolone has been studied in a series of clinical trials in both adult healthy volunteers (Phase 1), and in Duchenne boys (Phase 2).
The Phase 1 studies were carried out in 2015 in 86 adult volunteers, who received a wide range of doses. The studies have been published and showed that Vamorolone was safe to the highest doses tested.
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, which enrolled 48 Duchenne boys aged 4-7.
In June 2018 ReveraGen shared results from its international clinical trial, which showed dose-related improvements of multiple function tests of strength and endurance. Click here to read the press release.
The large majority of the boys enrolled in the studies, their families and their physicians expressed a desire to continue treatment with Vamorolone after they had completed these initial clinical trials. A two-year long-term extension study was then implemented. Click here to read the press release about the completion of the long-term study in 2020.
The charities contributed the original 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.
What are the 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 has 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
Work ongoing 2014-21
Latest research news
The FDA has granted accelerated approval for the gene therapy treatment SRP-9001. This is the first time a gene therapy for Duchenne has been approved anywhere in the world …
On Rare Disease Day, watch Eli Crossley, son of our friend and Duchenne UK co-founder Emily, as he talks about – and demonstrates – the amazing technological developments that are being trialled for children with Duchenne muscular dystrophy. The ‘smart suit’ aims to help children who are losing upper body function in still continuing to use their arms.
We are delighted that the ground-breaking KineDMD activity monitoring study we funded has been published in the prestigious scientific journal Nature Medicine, and covered by the BBC …
The devastating impact of steroids
Click here to learn more about the effects that current steroid treatments have on boys with Duchenne.