Each year, only five percent of the estimated 1.5 million children with an autism spectrum disorder in the US participate in clinical research studies. This is in stark contrast to pediatric cancer studies, which have a nearly 90 percent enrollment rate - a rate that has helped advance treatment and outcomes for childhood cancers substantially in the last decade.
In an effort to accelerate similar progress within the autism community, a group of academic medical centers has collaborated to launch an Autism Spectrum Disorder (ASD) sub-registry within ResearchMatch, a disease-neutral national database connecting patients with ongoing research. The new ASD sub-registry helps act as a matchmaker, linking families with autism researchers around the nation. Registration on ResearchMatch takes about 5 minutes and is open to volunteers of all ages and conditions including volunteers without health conditions.
"If we could raise the autism research participation level to that of the pediatric cancer community, we think we could realize similar gains in new knowledge, treatments and outcomes," said Rose Hallarn, Program Director for Clinical Trials Recruitment at the Center for Clinical and Translational Science (CCTS) at The Ohio State University and Institutional Liaison for ResearchMatch.
Working with researchers at Vanderbilt University, Hallarn's team interviewed families of autistic children, autism advocates and researchers to come up with five additional questions that have been added to the registration process for those who have identified themselves (or their dependents) with ASD during registration. The questions help identify behaviors and medications that could make children eligible for certain studies. The registry allows for a range of participation levels from volunteers - some studies involve going to a lab or taking medication, other studies are just looking for volunteers to take online health information surveys.
"Research studies can be stalled or prematurely closed if they are unable to enroll enough study participants. We're hopeful that the simplicity of this registry will encourage people to join," said Hallarn.
Ohio State autism researcher, Julia Pinsonneault knows all too well about the difficulty of finding eligible patients. In 2010, she was awarded a CCTS pilot to study genetic factors impacting attention deficit hyperactivity disorder (ADHD) in children with ASD, and how those factors might influence the efficacy of ADHD medication.
"About 40% of autistic children also exhibit ADHD or hyperactivity symptoms. My hypothesis is that the same candidate genes that may be contributing to ADHD in typically-developing children might also be functional in autistic children with ADHD symptoms. To test this hypothesis, we've been looking for kids with ADHD and ASD, and kids with just ASD," explained Pinsonneault, a research scientist with Ohio State's department of pharmacology who teamed up with Ohio State's Nisonger Center to conduct her study.
For her study, Pinsonneault had no difficulty finding participants with ASD and ADHD, but filling the other arm has been challenging. She's been able to move forward with some pieces of the study - such as identifying a combination of genetic variations that may contribute to a malfunction in the chemical messengers of the brain - but more participants could help inform and broaden the implications of her findings.
"There are probably genetic reasons why certain medications work in some people and don't work in others. If we can link specific genetic variations for children diagnosed with ASD with actual behaviors, we would not only be able to select a potentially more efficacious treatment, but it may also provide clues to the larger population of kids that have ADHD without ASD."