080210xf's Blog

L'X fragile sera vaincu | Fragile X will be conquered

X fragile : du nouveau à propos des tests cliniques à venir au Canada (AFQ056) | Treatment update Novartis plans New Phase 2 Fragile X Clinical Trial in Canada

Fondation canadienne de recherche sur l’X fragile / Fragile X researche foundation of Canada |

November 2010

In April of this year, the Swiss pharmaceutical company Novartis announced that their experimental drug known as AFQ056 had succeeded in a small European clinical trial in showing improvements in several behavioural symptoms that are troublesome for people with fragile X syndrome and for their families.  These exciting comments were disclosed in an interview with Novartis, and appeared in the April 29, 2010 issue of the New York Times Newspaper in the U.S.

While these are early results and further testing will be required to clearly demonstrate the safety and efficacy of the drug, the Fragile X Research Foundation of Canada is very excited to announce that Novartis will test their lead mGluR5 blocking drug, (known as AFQ056) to treat Fragile X in adult patients at several North American centres, including the Surrey Place Centre Fragile X Clinic in Toronto. The clinic is run by Dr. Carlo Paribello, President and Medical Director of the Fragile X Research Foundation of Canada.  Recruitment of participants for this trial is expected to start in January 2011 and more details regarding this study will become available in the near future in our newsletters and on the clinicaltrials.gov website.

Longtime FXRFC supporters will recall that the Foundation has been talking about the potential of mGluR5 drugs to treat Fragile X for several years now. Ever since the original finding in Mark Bear’s lab at Brown University in 2000, researchers have been working continuously to conduct the pivotal preclinical research which has validated these drugs as possible treatments for Fragile X.

Researchers in university labs around the world have worked to confirm Mark Bear’s mGluR Theory of Fragile X since 2001 so that they could demonstrate to pharmaceutical companies that mGluR5 antagonists have specific therapeutic effects for Fragile X. This strategy has exceeded expectations, as three pharmaceutical companies are now in active clinical trials using mGluR5 antagonists for Fragile X. The FXRFC believes that it is essential to facilitate multiple research programs in order to bring new Fragile X treatments to patients, because any one drug faces many hurdles in the process of approval by Health Canada. The fact that many companies are also developing drugs in this class for other indications (like Parkinson’s disease) makes it even more likely that at least one mGluR5 antagonist will be available in the near future.

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