Results are positive for drug backed by charity

TRIALS SUGGEST A POTENTIAL NEW TREATMENT FOR SYMPTOMS

A CLINICAL trial supported by a county charity has come back with positive results for people living with Duchenne muscular dystrophy.

Alex’s Wish, set up by Emma Hallam amd named after her son, who has the genetic disease, invested £47,000 to help fund clinical trials of the drug Vamorolone.

The results suggest Vamorolone was better than taking the placebo and had advantages over prednisolone, one of the drugs commonly prescribed to Duchenne muscular dystrophy patients.

Duchenne causes progressive muscular degeneration and weakness. It primarily affects boys, who can often need wheelchairs by the age of 12.

Corticosteroid treatments, such as prednisolone, which Alex started on when he was four, come with sideeffects, such as stunting of growth, weight gain and poor bone health.

Emma said: “Alex’s Wish concentrates on raising money to invest into clinical research, clinical trials and increasing clinical trial capacity to help delay symptoms by several years, effectively buying time for a cure to be found.

“We wanted to support a project to help bring a new drug to market that will hopefully, one day, replace traditional corticosteroids.

“Something that has the same antiinflammatory benefits with less sideeffects that can have a serious impact on patients’ quality of life.

“We invested £47,000 into Vamorolone with Eric Hoffman, chief executive at ReverGen BioPharma, in 2015.

“This funding has helped support the clinical trials necessary to prove the drug’s effectiveness.

“The results following a phase two clinical trial showed that boys on Vamorolone did better than those taking the placebo.

“As a charity, we’re thrilled about the positive results of this study as it represents a culmination of over a decade of scientific research and a tremendous milestone for the Duchenne community.

“We are incredibly grateful to have been able to contribute towards this important work.

“We can only do that with our supporters’ help so I’d like to say a huge thank you to everyone who has and continues to support Alex’s Wish.

“Their support is making a profound difference to those who live with Duchenne muscular dystrophy.”

Vamorolone has Promising Innovate Medicine status from the UK Medical and Healthcare products Regulatory Agency.

This may make it eligible for the Early Access to Medicines Scheme and would mean patients could access it before it is formally approved for use.