At N of One, we don't just donate to hospitals and hope for the best. We work closely with the world's top researchers to formulate and adhere to a plan that gets the best answers, economically. We get involved and help drive relevant answers for ASD families.
Studies Underway
Auto-Antibodies & Autism:
Is auto-immunity a common feature of autism? (New Study)
N of One has partnered with Dr. Judy Van de Water at the UC Davis MIND Institute, one of the foremost experts in the immune system in autism, to investigate whether auto-immunity is a commonly found feature in autism.
The study is utilizing a newly developed technology out of Oxford that has never been used in autism. This technology allows for the screening of thousands of types of auto-antibodies at a time which has never been possible before.
In the study we are analyzing blood samples from children with and without autism to determine if there is a pattern of autoimmunity in children with autism and if certain auto-antibodies may be responsible for some of the features seen in autism.
The pilot study phase is complete and, based on the intriguing results, the study is advancing to the next phase. The potential results of this trial could allow us to develop an inexpensive test to identify children with ASD with a particular auto-immune profile allowing for more tailored treatment strategies.
Naviaux Newborn Screening Study
Can a blood test at birth predict which children are at risk of developing autism? (New Study)
Newborns receive a heel prick at birth to determine the presence of rare diseases. Could that drop of blood also determine which ones are at risk of developing autism? Dr. Robert Naviaux at UCSD, who we partnered with on the suramin study, aims to find out by examining the blood spot records of children.
Years ago researchers identified that children with ASD appear to have a different blood chemistry pattern than those without. Does this profile or a related one exist at birth? Dr. Naviaux and his team are investigating this possibility using the latest metabolomic and machine learning technologies.
This study is currently recruiting children who are between 3-10 (with and without autism) and were born in California. If interested in learning more:
Baylor College of Medicine to investigate why some children's autism symptoms change when taking antibiotics
For years parents and clinicians have reported that some children with autism experience significant changes in their symptoms - typically improvements, though sometimes declines, while taking antibiotics.
While many theories have surfaced about this "antibiotic effect," and most suspect microbiome involvement, there are no definitive answers. N of One views this phenomenon as a clue about the underlying biology of autism and has worked with researchers at Baylor College of Medicine to design a clinical trial to document and study it. Baylor College of Medicine is a leader in the field of the microbiome and autism and the trial is led by Dr. Ruth Ann Luna, a scientific advisor to N of One.
The trial is now underway and will hopefully provide some answers about the underlying mechanism of this phenomenon that may then lead to treatments.
Parents who are interested in participating in the trial can visit the trial website:
https://www.bcm.edu/research/clinical-trials/h-39137 or contact Dr Luna at raluna@bcm.edu
Results from N of One Sponsored Studies:
Low-dose suramin in autism spectrum disorder: a small, phase I/II, randomized clinical trial
Results from an N of One: Autism Research Foundation supported clinical trial out of University of California San Diego School of Medicine show that a single dose of suramin given to boys with autism resulted in statistically significant improvements in the core symptoms of autism.
The double-blind, placebo controlled trial's success is not only notable for producing the first potential treatment for the core symptoms of autism - a condition that can be debilitating for many people - but also lends substantial support to the theory put forth by Dr. Robert Naviaux suggesting that underneath the outward symptoms of autism lies a biochemical process called the Cell Danger Response which is amenable to treatment.
The next step in this research, which is currently being planned, is to follow up this trial with a larger trial that will test if multiple doses of suramin continue to be safe and provide benefits.
You can learn more about this landmark trial here
Sulforaphane treatment of autism spectrum disorder (ASD)
N of One Scientific Advisor Dr. Andrew Zimmerman and his collaborators have announced results from their study on the effects of a compound called sulforaphane which is found in broccoli (especially the sprouts) as well as Brussels sprouts and cauliflower.
Sulforaphane has been known for years to impact various elements of the immune system including molecules in our bodies called "heat-shock proteins." Dr. Zimmerman was the pioneer physician researcher who first documented that many children with autism had improvements in their autism when they had a fever. Theorizing that "heat-shock proteins," which react to heat, may play a role he began looking for safe compounds that that may activate them which sulforaphane was known to do.
In this study 40 young men (ages 13-27) were given sulforaphane or a placebo for 18 weeks. Those that received the sulforaphane had substantial improvements in a number of standard autism behavioral tests and showed improvements social interaction, abnormal behavior, and verbal communication.
While the next step is to replicate the results, this study opens up yet another potential treatment for those with autism and importantly may illuminate some of the mysteries of the workings of autism
Past Research Initiatives
June 26, 2014 Conference on Autism and the Microbiome - Just two months after its launch, N of One in close collaboration with Childrens Hospital, Little Rock, Arkansas, organized and sponsored the first of conference focusing on the scientific links between autism and the microbiome. The full day conference featured nine speakers from around the world. To see archived presentations from speakers as well as hear John Rodakis talk about the mission of N of One please click here: www.microbiome-autism.com
If you would like to support research like this:
If you see the potential and want to support this research,
please consider dedicating a portion of your charitable donations to
N of One: Autism Research Foundation through a one time or an annual gift.