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Your Dollars@Work: Accelerating Development of New Autism Medicines

By developmental pediatrician Daniel Coury, medical director of the Autism Speaks Autism Treatment Network. Dr. Coury is also the chief of developmental-behavioral pediatrics at Nationwide Children’s Hospital, in Columbus, Ohio.

 Our families have long been frustrated by the slow pace of scientific discovery and the development of new options for helping those with autism live healthier, happier lives. Our scientific community shares this frustration. I’m happy to report that I’m seeing a new emphasis on more-rapid development of promising new therapies. And I see it coming from several different fronts, some of which are interacting and developing an exciting new synergy.

My optimism comes, in part, from what I saw and heard at this year’s International Meeting for Autism Research (IMFAR). (See all of Autism Speaks’ IMFAR 2014 coverage here.)

Autism Speaks and EU-AIMS
IMFAR keynote speaker Declan Murphy, of Kings College, London, reported on the activities of the European Autism Interventions Multicenter Study for Developing Autism Medications (EU-AIMs).  Autism Speaks is playing a unique and active role in this endeavor by adding a critical advocacy voice and ensuring alignment with its research programs, including our Autism Treatment Network and Autism Genetic Resource Exchange

By fostering collaboration among autism researchers and physicians across Europe, EU-AIMS promises to speed the large treatment trials needed to test the safety and efficacy of promising new medicines.

Autism Speaks’ PACT
At the same time, Autism Speaks’ Preclinical Autism Consortium for Therapeutics (PACT) is improving the assessment of potential autism medicines in the earlier phases of the discovery process. It’s doing so by increasing the usefulness and reliability of the animal models and evaluation tests used in early evaluation of promising medicines. This greater reliability is needed to attract the large investments required to move the most promising treatments into the more-expensive clinical trials.

The PACT team includes leading autism researchers at the University of California-Davis MIND Institute, Boston Children’s Hospital and Vanderbilt University Medical Center. They’ve already begun developing a platform of preclinical tests that improves the evaluation of experimental medicines in the best mouse and rat models of autism. This platform assesses a medicine’s effects on social and repetitive behaviors, sensory issues, learning, anxiety and brain activity in animal models.

These behaviors truly represent the core symptoms of autism. It’s so important to develop new medicines that address them, medicines that go beyond current medical treatments directed at secondary problems.

During its initial two-year phase, all PACT testing procedures and results are being 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.

As always, our aim is to speed the delivery of safe and effective treatments and supports that can transform lives.

Fast-Fail to Fast-Forward
On yet another front, researchers around the world are beginning to embrace a new “fast-fail” approach to medicines testing. This approach goes in an opposite but complementary direction to standard clinical trials. Usually, clinical trials start with a drug that appears promising and enroll modest numbers of participants to test safety and efficacy. Only then does the treatment go into more-comprehensive trials enrolling hundreds of subjects.

The “fast fail” process starts with a test of a treatment’s feasibility. In other words, if a treatment is given as a nasal spray that must be taken as three sprays in each nostril twice a day, the goal of the initial trial is to prove that this is practical and acceptable to patients. At the same time, the researchers look for evidence of benefits.

If a medication passes this stage, then and only then does the treatment go into studies of safety and efficacy. This approach permits researchers to “fast fail” impractical treatments much earlier in the pipeline. As a result far less time and money are wasted.

Biomarkers for targeted treatments
Autism Speaks is also committed to the discovery and effective use of biological markers (i.e. biomarkers) to support new drug development, preventive medicine and medical diagnostics. Biomarkers are measurable biologic findings like those we get from an objective test. Real world examples include the PSA test for prostate cancer or a screen for the BRCA gene to determine breast cancer risk. Effective biomarkers for autism would help with earlier identification of autism and may point to the underlying cause of an individual’s autism. That, in turn, promises to lead to more effective, individualized treatments.

I see a collaborative spirit binding all of these initiatives together. It’s wonderful thing to see, and I’m excited to see what develops from these activities in the next two years.

Explore all the research and family-service projects that Autism Speaks is funding using this website’s grant search. These projects are made possible by the passion and generosity of our families, donors and volunteers.

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The Autism Speaks blog features opinions from people throughout the autism community. Each blog represents the point of view of the author and does not necessarily reflect Autism Speaks' beliefs or point of view.