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Autism Speaks Launches Preclinical Autism Consortium for Therapeutics

New initiative will create best-possible tools and standards for early testing of promising medicines for autism’s core symptoms
October 03, 2013


Autism Speaks has launched the Preclinical Autism Consortium for Therapeutics (PACT) in collaboration with leading autism researchers at the University of California-Davis MIND Institute, Boston Children’s Hospital and Tennessee's Vanderbilt Univeristy.

“This unprecedented consortium is dedicated to advancing the discovery and development of treatments for individuals with autism” says Daniel Smith, Autism Speaks senior director of discovery neuroscience. The unmet medical needs of individuals with autism are profound, he adds. Yet currently there are no FDA-approved medicines for treating the disorder’s core symptoms.

In recent years, a few promising autism medications have moved into early clinical trials. However, the process is costly and carries a high failure rate.

“We need more shots-on-goal to succeed in improving the quality of life for individuals with autism,” Dr. Smith says.

Autism Speaks created PACT with the goal of improving the assessment of potential autism medicines in the early phases of the discovery and development process. More specifically, the consortium will work to increase the reliability of the early testing tools and animal models used during these early stages of research. This greater reliability is needed to attract the large investments required to move the most promising treatments into clinical trials, Dr. Smith explains.

The PACT team has already begun developing a platform of preclinical tests to improve the evaluation of experimental medicines in the best mouse and rat models of autism. In particular, this platform will assess a medicine’s effects on social and repetitive behaviors, sensory issues, learning, anxiety and brain activity in these animal models.

“By emphasizing a translational research approach, we hope to speed the delivery of safe and effective products that can transform lives,” Dr. Smith says.

During an initial two-year phase, all PACT testing procedures and results will be widely shared with the autism research community. Once the PACT platform is complete, Autism Speaks will make it available to institutions committed to developing new autism medicines.

PACT Brief Overview