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Monday, April 28, 2014

Guest Blog: A Cautionary Tale for Autism Drug Development

The Simons Foundation Autism Research Foundation

By Michael Ehlers
April 14, 2014

Steve Perrin provides a thoughtful critique of preclinical models for testing drug effects in human disease models and a powerful proposal for how to improve them in his 27 March comment in the journal Nature. Perrin argues that poorly designed animal drug studies lead to spurious conclusions that form the basis for costly, and ultimately negative, clinical trials. This may be even more true for autism research.

Perrin is the chief scientific officer at the
ALS Therapy Development Institute (ALSTDI) in Cambridge, Massachusetts, a scientific organization dedicated to identifying and advancing therapeutic development for amyotrophic lateral sclerosis (ALS).

That context is important, as the broader biomedical research community struggles with the swirling incentives of publication, funding, the maximization of shareholder value and bias toward positive results that collectively drive academic research, disease-oriented foundations and biopharmaceutical companies.

Experimental Noise

As Perrin documents, his institute could not reproduce study after study using specific ALS mouse models with a variety of drugs, all reporting impressive improvements.

The drugs all notably failed to demonstrate benefit in clinical trials. A skeptic might wonder — well, are those scientists at the institute missing key procedural details? Are they doing the studies correctly?

Perrin attributes the discrepancy, in general terms, to experimental ‘noise’ and lays out a set of recommendations on the design of animal studies to avoid spurious conclusions...

Read the rest of this article HERE.


Michael Ehlers is senior vice president and chief scientific officer for neuroscience at Pfizer in Cambridge, Massachusetts.

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