We partner with the best, the brightest, and the most open-minded.
N of One seeks to partner with the very best researchers in the world at leading institutions to sponsor and catalyze breakthrough research into the fundamentals of autism. We seek out researchers with a history of research excellence and and an open-minded approach to their research. We believe this mindset is important because, if the basis for autism were simple, we would have already found it by now.
We follow leads.
The history of science is filled with serendipitous observations. Alexander Fleming discovered penicillin by observing that bacteria did not grow near moldy bread. As early as the 18th century babies with cystic fibrosis were said by mid-wives to “taste salty” an observation that preceded the discovery in 1988 that cystic fibrosis arises from gene defects in chloride channels. For a seemingly intractable condition, autism is filled with a surprising number of examples of parents observing that their child’s autism temporarily improves in response to a variety of things such as: fever, antibiotics, or any number of diets. While some parents use these as therapies, these observations form promising leads for researchers to leverage in order to reveal the fundamental mechanisms of autism.
We foster an interdisciplinary approach.
Autism is very complicated. At the moment, there is no medical sub-specialty for autism. Any number of medical specialists may see or conduct research in autism, but we know that autism is a multi-system disorder and no single medical specialty receives training in all the fields that autism touches. Further complicating the research picture, many of the specialties required to understand autism have historically had little reason to interact. To make major advances in autism research, an interdisciplinary approach is required. N of One emphasizes interdisciplinary, collaborative research looking at how various systems interact to develop a more complete picture of autism.
Our approach to research
At N of One our aim is to sponsor research that can reveal the fundamental biological processes that lead to autism. In medicine this is often called the “mechanism” of the disorder. In all likelihood, there will not be one single mechanism for all cases of autism, but rather several. However, history has shown that when the mechanism of a disease is discovered, rapid progress is made for treatments and prevention strategies. In choosing which projects to pursue, we follow a few basic principles:
No drug is yet approved to treat the core symptoms of autism spectrum disorder (ASD). Low‐dose suramin was effective in the maternal immune activation and Fragile X mouse models of ASD. The Suramin Autism Treatment‐1 (SAT‐1) trial was a double‐blind, placebo‐controlled, translational pilot study to examine the safety and activity of low‐dose suramin in children with ASD.
Sulforaphane treatment of autism spectrum disorder (ASD)
October 13, 2014
Autism spectrum disorder (ASD), encompassing impaired communication and social interaction, and repetitive stereotypic behavior and language, affects 1–2% of predominantly male individuals and is an enormous medical and economic problem for which there is no documented, mechanism-based treatment. In a placebo-controlled, randomized, double-blind clinical trial, daily oral administration for 18 wk of the phytochemical sulforaphane (derived from broccoli sprouts) to 29 young men with ASD substantially (and reversibly) improved behavior compared with 15 placebo recipients. Behavior was quantified by both parents/caregivers and physicians by three widely accepted measures. Sulforaphane, which showed negligible toxicity, was selected because it upregulates genes that protect aerobic cells against oxidative stress, inflammation, and DNA-damage, all of which are prominent and possibly mechanistic characteristics of ASD.
An n=1 case report of a child with autism improving on antibiotics and a father's quest to understand what it may mean
The author, a parent of a child with autism, describes an n=1 case in which his child's autism symptoms dramatically and rapidly improved following administration of a common antibiotic. The author asserts that this finding is not unusual in the autism population and that, when combined with prior recent medical research, suggests that a link between autism and the microbiome in some children is not just plausible, but in fact likely for some meaningful percentage of cases. The author argues for increased funding for a more thorough examination of links between autism and the microbiome and poses a series of questions to be further examined in future research.
There is a growing body of scientific evidence that the health of the microbiome (the trillions of microbes that inhabit the human host) plays an important role in maintaining the health of the host and that disruptions in the microbiome may play a role in certain disease processes. An increasing number of research studies have provided evidence that the composition of the gut (enteric) microbiome (GM) in at least a subset of individuals with autism spectrum disorder (ASD) deviates from what is usually observed in typically developing individuals. There are several lines of research that suggest that specific changes in the GM could be causative or highly associated with driving core and associated ASD symptoms, pathology, and comorbidities which include gastrointestinal symptoms, although it is also a possibility that these changes, in whole or in part, could be a consequence of underlying pathophysiological features associated with ASD. However, if the GM truly plays a causative role in ASD, then the manipulation of the GM could potentially be leveraged as a therapeutic approach to improve ASD symptoms and/or comorbidities, including gastrointestinal symptoms.