ALS Advocacy

ALS Advocacy
Lou Gehrig's Disease - Motor Neuron Disease - Amyotrophic Lateral Sclerosis
Thought it had been cured by now? Still no known cause. Still no cure. Still quickly fatal. Still outrageous.

Friday, February 8, 2013

Was The Dexpramipexole Thud A Blessing In Disguise?

A month ago we heard a gigantic thud when the big Phase III clinical trial for dexpramipexole showed that it was a dud for ALS.

"The important thing we're learning about ALS is it's a spectrum of diseases," Lucie Bruijn, chief scientist at The ALS Association, said recently. "There are some who respond to treatment and some who don't; at the moment, we lump all the people together and that might be one of the reasons why the trials are failing."

Did dex fail because a subpopulation that it could have helped was not sufficiently represented in the big Phase III trial?

Today stakeholders are buzzing about finding new ways of identifying subpopulations and testing drug candidates for ALS.  It's a rather refreshing buzz and the gigantic dex disappointment has been credited as a big reason for the FDA Part 15 Hearing on ALS on February 25.

What if last month we had been told that dexpramipexole had shown even modest efficacy in that clinical trial?  There would be an ecstatic push to make it available for people with ALS, just as there was with riluzole in 1995.  There would be celebrations.  We would be breaking our arms patting ourselves on the back. Would we be challenging clinical trial design?  Would we be asking if the trial represented subpopulations sufficiently?  Would we be questioning the validity of what we do if it had generated the news we wanted to hear?


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