making muscle wasting history

Like all parents and young men living with Duchenne this announcement leaves me deeply disappointed. In truth I'm gutted. But I would like to share with everyone some information from Biomarin and some thoughts on where we go from here.

Biomarin inform me that there was no difference between those taking a placebo and those taking the drug in terms of utrophin upregulation in their phase 1 trial. There are other factors too but simply the drug does not work and there is no point continuing the trial. We have to understand that 90% of drugs fail at clinical trial stages 1 and 2 and this failure certainly puts those figures into some perspective.

The development of this drug has been a hard road. We have been working with Kay Davies, Vastox, Summit and Biomarin funding and supporting this effort for several years. No drug company was interested in Kays work or Duchenne for that matter and so with Jon Tinsley and others she had to set up Summit to try to get the drug trialled. Biomarin finally agreed a partnership and has worked hard to get this well organised double blind trial underway.
Unfortunately we have not got the result we wanted.
Biomarin remain committed to supporting Duchenne drug development and we will have to wait and see if they develop more candidates. They have agreed to come to our conference in November in London to discuss the out comes of this trial and their future work.

We must understand the need for working with companies like Biomarin who can organise the sort of trials that can give us answers. Otherwise we continue to work on drugs or medicines in the lab and never get to conclusive human trials.

Action Duchenne has teamed up with Charley's fund and others in the USA to establish Pilot Trials Now - this will be a programme of pilot clinical trials to see if known drugs for other diseases could benefit children with Duchenne. The first trial is underway testing Viagra in the US. Action Duchenne has committed $250,000 already to further trials of other drugs. We want to do more.

Exon skipping drugs have passed through initial trials. Action Duchenne has funded and supported research in AO drug development. AVI, Prosensa, GSK are making rapid progress in this field. Action Duchenne and Duchenne Ireland are currently funding a multi skipping project at Oxford to see if we can use exon skipping for rarer deletions and duplications.

In the near future we hope to develop some of this research in China.

Other drugs and research is in the pipeline and some of these research groups and companies will present at our Conference in November.

When we established Action Duchenne in 2001 there were no human clinical trials taking place. That's zero trials anywhere in the world! Steroids were not being used by many clinicians.

We now have major pharmaceutical companies taking an interest and running trials. But it is not enough. Its not fast enough and we don't have enough money for all the research that needs to be done.

Cure. No compromise. I'm still here. Lets keep up the fight to cure Duchenne for every boy and girl. To do that we need to raise money and work together and work with other Duchenne charities and trusts as we are doing with Pilot Trials Now.