Nova’s clinical research program is designed to fill an unmet medical need in accurate diagnosis and treatment of autism spectrum disorder (ASD) and fragile X syndrome (FXS).
We’re conducting a large-scale observational study to assess the gut microbiome, immune response and serotonin activity, as all these systems are likely involved in producing the variety of behavioural symptoms observed in ASD and FXS.
Unravelling the Mysteries of ASD
A true spectrum, autism is a collection of developmental disorders currently diagnosed mostly on the basis of neurobehavioural patterning – an approach that (1) cannot distinguish subtle differences between ASD subtypes, (2) is subject to observer bias, and, therefore, (3) introduces uncertainty regarding root causes and response to treatments.
The molecular underpinnings of ASD are largely unknown, particularly with regard to the dynamic interaction between co-existing biological systems that drive development of the brain and gut: serotonergic signaling, microbiota and the immune response. Investigating these systems, their interaction and pharmacological modulation will shed light on the pathophysiology of ‘autisms’ and identify biomarkers to be used to subtype ASD and to guide development of new therapies.
Both genetic and environmental factors are involved in the pathogenesis of ASD, with FXS being the leading genetic cause of ASD. In ASD and FXS, the development of the brain and gut appears to be disrupted by altered serotonergic signaling, chronic inflammation and shifts in the gut microbial population.
Serotonin has a key role in normal brain development. Humans undergo a period of high brain serotonin synthesis during childhood – reaching a peak between three and five years of age – and this developmental process is disrupted in autistic children. Serotonergic insufficiency during childhood may have a compounding effect on brain patterning in ASD and FSX, manifesting as behavioural and emotional symptoms. As such, therapeutics that stimulate serotonergic signaling such as psilocybin and other tryptamine derivatives offer promise as potential early-childhood interventions for at-risk children.
It is reported that autistic children are more likely to have gastrointestinal issues, but the specific involvement of gut microbiota in this phenomenon is yet unclear due to small sample sizes and inconsistencies in methods/findings across various studies. What is clear, however, is the need to examine dynamic relationships between serotonin, immune system and microbiota in order to discover diagnostics that differentiate between subtypes and guide development of innovative treatments.
Our Three-Pronged Approach
Our research plan includes three clinical studies:
- an observational study to establish an ASD Diagnostic and Therapeutic Index (ASD-DTI), which simultaneously measures systemic inflammation, gut microbiome taxa and serotonergic activity in an effort to differentiate between subsets of ASD-related neurodevelopmental disorders and guide drug treatment in subsequent dosing trials.
- a Phase 1 clinical trial to assess safety of our proprietary psilocybin formulation.
- a Phase 2 clinical trial to assess efficacy of psilocybin in ASD and FXS.
We’re aiming to recruit at least 300 participants across the U.S. and Canada for our observational study to establish the ASD-DTI:
- 200+ with diagnosed ASD and/or FXS.
- 100+ neurotypical controls, including children under age five who may be showing symptoms suggestive of ASD but have not yet received a formal diagnosis.
The study will involve collecting samples from eligible participants, including urine, feces, finger/toe-prick blood, and cheek cells and the measurements will be compared with those from age-matched neurotypical controls. We expect to begin the process of collecting samples by fall of 2021. We will distribute test kits in the mail and participants will be encouraged to submit at least two of the four sample types.
These data will be analyzed with the help of advanced computational methods and used to guide design of the upcoming clinical trials.
Express your Interest in Joining the Study:
- Sign up here to express your interest in participating in our observational study and we will keep you up to date on our plans.
- Please email us or call 1-888-505-NOVA if you have questions about the research or how to participate.
We look forward to hearing from you!