A drug trialled by Leicestershire teenager Alex Hallam as part of a study into treatments for Duchenne Muscular Dystrophy, provides beneficial evidence of its ability to slowdown disease progression.
Latest data released by the drug sponsor Italfarmaco has revealed that Givinostat an anti-fibrotic drug shows a good tolerability profile and positive outcomes for the trial patients.
There is a tremendous unmet medical need for additional drugs to treat this debilitating rare disease and with these positive results, Italfarmaco intend to meet with regulatory agencies to share these findings and discuss a path forward to submit the complete dataset in a marketing application for potential approval.
Alex has been part of the clinical study in Newcastle upon Tyne hospital, for over three years, participating in the Phase 3 clinical trial for the past 18 months.
Duchenne is an aggressive form of muscular dystrophy affecting one in every 3,500 boys and there is currently no cure.
Alex’s mum Emma Hallam, who launched Alex’s Wish almost 10 years ago to raise money to fund medical research and ultimately eradicate Duchenne, said: “The latest data from this study is the news the Duchenne community has been waiting for. This provides new hope for boys living with Duchenne, their families, and the medical community. We know it’s not a cure, but we also know Alex is now on a medication that is buying him precious time while we wait for a cure to come along.
“Knowing that those dreadful stages we were told about when our son was diagnosed at age four, could now be delayed is the best news a parent could ever wish for. This is such a big step in the right direction.”
Alex is among just 179 boys on the trial. Patients were recruited globally, including in the UK. Alex’s Wish helps support The DMD Hub which was instrumental in recruiting patients onto this trial in the UK.
As a trial participant Alex has countless hospital visits, regular blood tests, physio tests, MRI scans and must take Givinostat – a liquid medicine which tastes ‘horrendous’ – twice a day. But this a small price to pay.
Emma added: “To know that Alex is helping in his own way to bring effective treatments to the Duchenne community melts my heart. We feel incredibly honoured and lucky to have the drug in our fridge.
We won’t know how long regulatory approval will take, but we must now fight to get access to this drug in the UK, so everyone has access to it. We must also continue to find a cure that will halt disease progression in boys living with Duchenne.
Alex’s Wish would like to thank all our wonderful supporters for being on this incredible journey with us. Without their amazing support, there would be no clinical trials, no new drugs, and no new medical research. The result of this trial clearly shows that investing in medical research does save lives.”