On February 2nd, 2009 an article was published that many in the Duchenne community, especially those following the PTC124 trials and information may find confusing. The publication in PNAS (Proceedings of the National Academy of Sciences) by Auld and colleagues focuses on the difficulties of relying on a single high throughput screen (HTS) assay for the discovery of potential drugs. High throughput screens are used in the early stages of drug development to identify potential molecules that show promising therapeutic activity. The authors caution against using a single HTS assay in the identification of potential therapies.
PTC has been very thorough throughout the ten-year development of ataluren (PTC124), and has conducted extensive testing in the laboratory, in animal models and in clinical trials. As a result, there is substantial evidence, much of it published in internationally recognized peer-reviewed journals, indicating that ataluren selectively reads through nonsense mutations inducing the production of dystrophin in animal models and in Duchenne patients.
I think it is important for all of us to understand that the information being presented in the PNAS article reminds us how important and critical the entire drug development and clinical trial process is. As always, we at PPMD will keep on top of the trial results and outcomes and make sure to pass along any and all information.