making muscle wasting history

Ataluren from PTC in the USA is a drug that we all hoped would benefit boys with certain Duchenne gene variations. We all had real hopes for this drug and it comes as real bad news that there seems to be no benefit when those kids on high doses are compared to those tasking the placebo.

PTC spoke at our conference this year and I know that the company has been committed to Duchenne for 10 years now and this has equally come as a blow to them. Our clinical teams at Newcastle and Great Ormond Street have also worked very hard on this trial and they are also deeply disappointed.

So what has gone wrong? It really is hard to say at this stage. PTC do not have all the data to hand and it certainly has not been published. There is a lot we need to learn about the 6 minute walk test as an outcome measure for Duchenne trials. This is the first time it has been used for a Duchenne trial and it is a very complex measure with many variables.

We need to know how much dystrophin has been made by Ataluren reading through the stop codons present in these boys. This data is also vital for those trials coming up for exon skipping using antisense oligonucleotides.

We must all realise that this is a clinical trial and as such is an experiment. The aims of a trial are to collect data and gain understanding of how this drug might be working in humans. PTC are committed to analysing this information and getting us more answers.

Of course all of us hope that we find a drug that works brilliantly first time and even better might cure our kids. What has happened with Ataluren serves to remind me just how complex a disease like Duchenne really is and just how hard it is to find the answers we need.

If you have been on the trial then please post your comments. Make sure that you speak to your clinical teams and don't be afraid to ask questions and get information. We are all sharing your deep disapointment and if you would like to talk about the trial then please call me anytime on 07920723490.