making muscle wasting history

Personalised medicines to treat Duchenne seemed to be a distant dream for many of our families. The results from recent trials (PTC, Prosensa and AVI) have looked promising but many families have been worried that they will not ever see their sons treated especially if they had a rarer deletion or duplication. The meeting at the European Medicines Agency (EMEA) on September 25th in London that I and Action Duchenne trustee Divyesh Popat attended was a vital step towards developing new personalised medicines for Duchenne.

Elizabeth Vroom a parent from Holland put our joint case to the meeting. Elizabeth spoke about the cruel progressive muscle wasting in Duchenne. Medicines are judged by the risk of doing harm.

Elizabeth told the regulators that by doing nothing, in the case of Duchenne, leads to the highest risk of all - early death.

So what is a personalised medicine? Duchenne is caused by a fault in the bodies largest gene, Dystrophin, and every boy is affected in a different way. Early trials with drugs called antisense oligonucleotides have shown promising results in splicing the RNA to make a functional protein that could slow or even halt the muscle wasting. But each gene variation could well need a designer drug to get the exon skipping working. This might seem impossible but in reality each drug could have the same backbone but be designed to fit any boy.

The problem has been so far to convince the regulatory authorities to see these medicines as a class of drugs. In normal drug development every new chemical compound is treated as a different medicine and has to undergo expensive and time consuming animal and human studies.

All 98 clinicians, researchers and patient group members at the EMEA meeting were asking the regulators to allow us to develop a wide range of personalized drugs for Duchenne that would not compromise safety but would not require such long and expensive testing for each one.

You can see in the documents (go to your dashboard and visit the document library) and press releases on our website that the regulators are now working closely with us to try to agree a way forward.

This is very exciting progress and gives me great hope for us to be able to roll out drugs for the majority of our sons.

You can meet with biotech companies at our conference this year by registering via the homepage conference 2009 button.