Autism Speaks announces pilot sites for groundbreaking project linking discovery science and clinical research

May 21, 2021

With the selection of six sites across North America to pilot a bold new research program, Autism Speaks and Autism Speaks Canada announce the launch of PATH to Discovery (Precision Autism Treatments for Health), a research program to build a roadmap to personalized medicine for people across the autism spectrum.  The program is co-led by Dean Hartley, senior director of genomic discovery, and Donna Murray, vice president of clinical programs.

“PATH is a groundbreaking partnership that links our discovery sciences and clinical network resources to realize new paths forward in autism,” said Thomas W. Frazier, Ph.D., chief science officer at Autism Speaks. “This initial study will help build the discovery platform that connects clinical care and biological information, which can scale up to deliver a road map to personalized medicine and healthcare for individuals with autism.”

PATH has benefitted from early, generous investments totaling $6.15M including a leading gift of $3.5M from the Wise Family Foundation of Toronto, Canada. These generous commitments support the $12 million expansion of PATH, the next phase of MSSNG, to include not only whole genome sequence data but also innovative biological and clinical measures from autistic individuals and their families.

PATH is excited to partner with the Autism Care Network to select six network centers to pilot Phase 1 of the PATH program:

  • Children's Hospital of Philadelphia, Philadelphia, PA
  • Cincinnati Children’s Hospital Medical Center, Cincinnati, OH
  • Holland Bloorview Kids Rehabilitation Hospital, Toronto Ontario, Canada
  • Massachusetts General Hospital, Boston, MA
  • Nationwide Children's Hospital, Columbus, OH
  • University of California, Irvine, Irvine, CA

In this pilot phase, the sites will enroll 100 families to collect broader and deeper information not only on their behaviors, but also collect information on their biology to teach us how autism begins, develops and is responsible for spectrum of autistic traits.  

Families will share this biological information through saliva or blood samples, which is then connected to behavioral and genetic findings, as well as health issues monitored by their healthcare providers.

By pairing biological data with deeper behavioral information, researchers can discover specific biological changes that influence health and potential new treatments, while tracking clinical care and how those issues are treated. This critical link helps to understand which of those treatments improve health of people who have similar subtypes of autism.

Taking advantage of the expanding Autism Care Network footprint allows more individuals and families to enroll, thus expanding the scope and scale of the data and findings. These larger datasets increase the ability for researchers to find patterns and connections in groups of very different people.

Due to the broad and variable ways each person is affected by autism, when testing interventions, researchers will see a “washout effect,” where the treatment only works for a small number of participants, while for others, benefits are not observed, or “washed out.” PATH will ultimately test treatments within distinct groups, helping to personalize treatments for their specific needs.

Long-term, PATH will also help address specific needs for people across the spectrum.

“What we know in the field about distinct autism subtypes is primarily around genetic syndromes that cause more significant challenges,” said Frazier. “PATH will help extend our understanding of what treatments are helpful for which concerns, which group of people they work for, and ultimately help providers in their work to improve health and quality of life for autistic people.”

About Autism Care Network

A first of its kind learning health system for autism, the Autism Care Network, with support from Autism Speaks is built on more than a decade of work in improving autism care and a family-centered approach to drive improvements with speed and at scale. Autism Speaks is a founding partner in the Autism Care Network, which connects patients, families, researchers, and healthcare teams to continuously improve whole-person and whole-family autism care.

About Autism Speaks Science program

For nearly 15 years, Autism Speaks has invested over $230 million in scientific research that has shaped the field’s understanding of autism and provided data-driven, long-term solutions to help people with autism and their families. The MSSNG, Autism Care Network and PATH to Discovery initiatives are strategic investments to complement and advance the global autism research field.