Ataluren Update

A recent study found that Ataluren failed to enhance read through past stop codons (they tested all the possible stop codon combinations in different situations), instead they suggested that Ataluren interacted with the drug screening system, the luciferase enzyme. I've summarised the findings here: http://sixtyfiverosesblog.wordpress....aluren-update/ “Where does this leave us with PTC124 and its potential to tackle the 10% of cases of genetic disease that are thought to result from nonsense mutations? We should remember at this point that McElroy and colleagues only test cells (not intact animals), that they only look at the read-through activity of this drug, and that there are several publications that do suggest clinical efficacy (particularly for cystic fibrosis). The conclusion remains, however, that if PTC124 does indeed have beneficial effects on some genetic diseases, it’s more likely that this is down to serendipity than the purported mechanism of translational read-through. Numerous groups are still working on developing effective drugs that can override premature stop codons—in case you wondered, G418 is sadly too toxic for clinical use. PTC124 aside, this manuscript also raises interesting issues for the design and interpretation of high-throughput drug screening assays, and also potentially for the consequences of an initially strong positive screen result on subsequent evaluation of drug efficacy.”...
Source: Cystic Fibrosis CFTR Modulation and Gene Therapy Forum - Category: Respiratory Medicine Authors: Tags: CFTR Modulation and Gene Therapy Talk Source Type: forums