NIH grant and generous family make new research approach possible
A new study by the UC Davis MIND Institute’s David Hessl will test whether a smartphone-based app may help improve the accuracy of data in some clinical trials involving individuals with intellectual disability.
“It’s a really smart use of a ubiquitous technology and it could make a huge difference in the accuracy of measurement in clinical trials,” said Hessl, a professor in the Department of Psychiatry and Behavioral Sciences.
Hessl was awarded a two-year, $431,000 grant from the National Institutes of Health (NIH) for the project. It’s part of the NIH’s INCLUDE initiative, which is centered on investigating conditions that co-occur with Down syndrome across the lifespan, including attention deficit/hyperactivity disorder (ADHD).
Hessl’s study will focus on children and young adults with intellectual disability. The most common genetic causes of intellectual disability are Down syndrome and fragile X syndrome. The goal is to track symptoms related to executive function, often associated with ADHD, using the new smartphone app, called iBehavior.
It’s a really smart use of a ubiquitous technology and it could make a huge difference in the accuracy of measurement in clinical trials.
A quest for more accurate data
“The majority of clinical trials focused on people with intellectual disability have relied on parent or caregiver questionnaires, much like traditional paper and pencil rating scales,” explained Hessl.
That means families have to remember over a fairly long period of time – sometimes weeks – what their child’s behavior was like.
“The app uses what we call an ecological momentary assessment, where a parent or teacher observes the child or young adult and provides ratings of behavior much closer to the time the behavior occurred,” said Hessl. “People obviously have varying degrees of accuracy with their memory, and we are all subject to different kinds of bias, which can negatively impact studies. So, we’re hoping with the more immediate recording of the information, we’ll get a more sensitive and accurate representation of whether patients are improving or not.”
Families will be trained in how to use iBehavior and what behavior to look for. The app will send a text when it’s time for them to start observing their child or young adult, they’ll observe for two hours, then they’ll get another text that it’s time to record what they observed. They’ll record information about aggression and irritability, hyperactivity and impulsivity, attention problems, anxiety-related behavior and avoidance and more, likely daily. Classroom teachers may also be asked to take part.
The new app was Hessl’s idea, and Michael Bass of Northwestern University programmed iBehavior with guidance from Hessl’s team and a group of consultants.
Building on existing research
Researchers will evaluate the tool with families who are taking part in another NIH-funded long-term study called “A Cognitive Test Battery for Intellectual Disabilities.” Hessl is also the Principal Investigator of this study. “We are really working to leverage all of what we’ve learned in this parent study by pairing up with the iBehavior app method,” he said.
As part of the parent project, Hessl and his colleagues recently validated the NIH Toolbox Cognition Battery, a series of tests delivered on a tablet or iPad to assess cognitive function, for use in individuals with intellectual disability.
“We’ll invite families to provide data on the app, as well as take part in the traditional assessments and that way we’ll be able to cross-compare. For example, we’d expect that if a parent reports a high level of executive function difficulties in the child’s daily life, for example impulsivity or inattention, that the Toolbox, which measures cognition in the lab, would show those same kinds of difficulties,” Hessl explained.
This is a game-changer for our son’s future and for all families living with fragile X syndrome and other neurodevelopmental conditions.
A family’s gift enables new approach to research
Before Hessl could apply for NIH funding, his idea had to be developed.
That’s where Brian and Shari Silver come in. Their son, Justin, has fragile X syndrome and has received treatment and taken part in research studies at the MIND Institute. They believed in iBehavior and made donations, both personally and through their non-profit organization, the Fly With Me Fund, that were critical to the development of the app.
“When the opportunity was presented to support Dr. Hessl in getting a new project off the ground, there was no doubt we would do what we could,” said Shari Silver. “Having participated in research, and also having had to evaluate medications as they were introduced to our son, we recognized that if we had a tool like iBehavior, capturing our observations daily with the battery of questions in the app would have helped us to provide the most accurate feedback to clinicians.”
There is currently no treatment for fragile X syndrome; there are only treatments for symptoms. Silver noted that they are not always effective, and she’s hopeful that this app will improve the assessment of a child’s behavior before, during and after treatment as well improve the assessment of potential targeted treatments.
“This is a game-changer for our son’s future and for all families living with fragile X syndrome and other neurodevelopmental conditions,” she said.
The Silvers have supported other MIND Institute research as well, including a new blood processing machine for laboratory work, travel grants for patients with fragile X syndrome to visit the MIND Institute and telehealth services. They’ve also tested the iBehavior app and Shari is “sure of its viability.”
What’s next for iBehavior
Starting in 2023, the study will also include a clinical trial of a stimulant medication, a common treatment for ADHD.
Hessl notes that a number of clinical trials have been “negative” – or showed medications to be ineffective. There is debate about whether some treatments are truly ineffective, or whether the previously used outcome measures were adequately sensitive to patient behavioral change.
“For example, if a trial fails, we assume it’s because the medication was not effective, but there’s a possibility that there was some efficacy but the tools used weren’t sharp enough, not accurate enough to pick up the improvement.”
The hope is to validate the use of the app as an outcome measure for clinical trials.
The UC Davis MIND Institute in Sacramento, Calif. was founded in 1998 as a unique interdisciplinary research center where families, community leaders, researchers, clinicians and volunteers work together toward a common goal: researching causes, treatments and potential prevention of challenges associated with neurodevelopmental disabilities. The institute has major research efforts in autism, fragile X syndrome, chromosome 22q11.2 deletion syndrome, attention-deficit/hyperactivity disorder (ADHD) and Down syndrome. More information about the institute and its Distinguished Lecturer Series, including previous presentations in this series, is available on the Web at mindinstitute.ucdavis.edu.