Wednesday, September 24, 2014

More bad news...but then some good news for fragile X research

Let's get the bad news out of the way first---Roche announced that the clinical trial of its lead mGluR5 antagonist for fragile X had failed to show any superiority on any of the outcome measures used (either primary or secondary.) They have also announced that they are cancelling their fragile X program. There aren't many details available at this point, though Roche has pledged to present and publish the data...eventually. I'm heading to the international mGluR conference in Sicily in a couple of days, and this is sure to be the hot topic; perhaps some new tidbits will be forthcoming. In any case, this was hardly a surprise. The Novartis results strongly suggested that the Roche compound would follow the same path; if anything, reports from the families participating in the Roche trial were even less promising than in the Novartis trial. If we are thinking that tolerance is a major problem, then the more potent and longer acting Roche drug may have even more problems with tolerance.

Now for some good news: the awards for the new fragile X research centers have been announced by the NIH. Three "Centers for Collaborative Research in Fragile X" will receive $35 million in funding over the next 5 years, and we couldn't be happier with the choices. These are all research groups that have been heavily supported by FRAXA over the years, and we think they will make a real difference, especially now that they have the resources to get things done!