Autism and Parents: The Potential of the N-of-1 Case Study
by Lisa DiFalco
The factors affecting children with autism show that what may work for one child may not work for another. There is no “silver bullet” that will address the needs of all children with autism. While ABA therapies are one tool to attempt to reduce undesired behaviors, there is simply so much that is not known about ways to effectively treat children. Parents are often the ones to research and stumble upon noteworthy effects that improve their children’s health and verbalization. Richard Frye, pediatric neurologist at the University of Arkansas in Little Rock and involved with an expanded autism study which began with initial N-of-1 findings said this of the potential of the N-of-1 trial:
“You can’t do double-blind clinical trials on everything. N-of-1 trials provide an important jumping-off point. They can generate better data about what might be causing a disease and what might work to treat it.”
These initial findings become part of the growing body of single-subject trials or No-of-1 case studies that provoke interest in novel concepts in autism treatment and trigger larger studies. Health professionals, case managers, mental health professionals, educators and families should be aware of this growing body of autism research that may change treatment for those affected with autism.
Thanksgiving With the Rodakis Family
Rather than enjoying the typical Thanksgiving, the festive holiday saw John Rodakis’ two children come down with a strep infection. Both children, one a four-year-old with autism, were given a course of amoxicillin as prescribed from the doctor. Before the amoxicillin, the child rarely spoke and when he did, it was in single words. In addition, he made little eye contact and had difficulty socializing with other children. The antibiotics began to trigger some unexpected changes. In a few days after starting the antibiotics, Rodakis’ son was able to:
- Begin making eye contact; and
- Speak in short sentences.
“Each day, he seemed to get better; I had no idea what was going on.”
Rodakis found little research linking antibiotics with children with autism but he found corroborating stories with many parents in internet groups. He shared:
“So many parents had seen a similar pattern, but there wasn’t much in the media or scientific literature. It was very frustrating. I had no idea why there wasn’t any follow-up.”
During the initial experience, Rodakis had the habit of logging the behaviors and their severity on the Autism Tracker iPhone app. In addition, he continued to log behaviors after the treatment with antibiotics and noted the return of autism-related behaviors. Like all children, his young son came down with future illnesses requiring antibiotics and Rodakis observed his son’s improvement as it related to autistic behaviors with amoxicillin but not with the combination of amoxicillin and co-trimoxazole in cotrimoxazole. Rodakis brought his observations to the notice of autism researchers, and they believed he should publish the details in the form of an ‘N-of-1’ case study.
There are autism researchers that are using the initial N-of-1 trials from Rodakis and other parents of those with autism to explore new treatment options. Frye said:
“Parents want to find answers, and we have to listen to them and be skeptical at the same time. N-of-1 trials can be a messy way of looking at things, but they provide important clues about what’s going on. I think they are really under-utilized in medicine.”
Opening Minds for New Autism Treatment
Rodakis is one of many parents who have experienced frustration at doctors refusing to acknowledge his observations about his son. He is a parent with a venture capital background and launched N of One: Autism Research Foundation, a 501(c)(3) non-profit. The objective of this foundation is to provide funding for pilot studies that use data from small groups of people, specifically as it relates to autism and the microbiome. He, fellow parents and researchers put forth their mission to:
“Facilitate, Fund, and Communicate Breakthroughs in Autism Research.”
Researchers with a history of excellence are encouraged to participate, and an interdisciplinary approach is welcome, as autism is seen as a “multi-system disorder.” The non-profit wants to encourage communication across medical specialties to further the understanding of autism.
Where to Find More Studies
Those interested in researching the latest studies and archived materials on a range of health conditions, including autism, ASD and Asperger’s may want to investigate:
- ClinicalTrials.gov– a website that is easily searchable by keywords, topic or map, in addition to providing guidance on how to read study records. The National Library of Medicine (NLM) located at the National Institutes of Health (NIH) maintains the site. Not all studies are required to be registered and therefore the site does not encompass all clinical studies currently conducted in the US.
- Autismspeaks.org– a website that provides news on studies, information on research grants and fellowships, ways to participate in research and other resources and programs.
- Spectrumnews.org– a website that provides the latest news on autism findings and studies. A recent article, The Builders: How Parents Shaped Autism Research, provides empowering hope for parents that want to come together and work with researchers to make new discoveries in the field of autism.
Parents are often careful observers of their child’s health and the expression of autism-related symptoms. When parents arm themselves with research that may be useful in providing insight to treatment, they need the support of the healthcare community, including their family physicians and pediatricians. A big red flag for parents should be the brushing off of potential research by a healthcare professional in cases where the study seems to correlate with an affected individual’s symptoms and behaviors and well as parents’ observations. Parents need to know they are not alone and their observations are worth noting. Remember, families always have the choice of choosing an open-minded practitioner who will consider their perspective.