Study Will Measure Serotonin, Gut Microbes in ASDs

Nova Mentis has launched an observational study examining the interconnected roles that gut microbes, serotonin, and the immune system play in the gut and brain development of people with autism spectrum disorders (ASDs), including fragile X syndrome.

The study (NCT04869930) aims to develop a set of tools — a “Diagnostic and Therapeutic Index” — capable of identifying distinct ASD subtypes to help physicians make better diagnoses and more accurately predict treatment responses.

“We are developing this set of multidimensional biomarkers to help differentiate subsets of symptomatic, as well as pre-symptomatic, ASD and FXS patients — an approach that currently does not exist in clinical practice,” Julia V. Perederiy, PhD, NovaMentis’ lead scientist and the study’s principal investigator, said in a press release.

“Furthermore,” she continued, “we will use the index to subsegment participants in upcoming Phase 1 and Phase 2 clinical trials of [Nova Mentis’] proprietary psilocybin formulation — an essential step for study design and development of hard endpoints [goals] to corroborate clinical results.”

Psilocybin is a psychedelic compound that has shown some potential as a behavioral therapy for ASD conditions and fragile X — the most common genetic cause of autism — by affecting patients’ serotonin levels. The chemical comes from various mushroom species, known colloquially as “magic mushrooms.”

Enrollment is currently open for people ages 6 months to 21 years, in the United States and Canada. Nova Mentis seeks at least 200 people with ASD or fragile X, and at least 100 neurotypical people to serve as controls. An Institutional Review Board has approved the study in the U.S. and the company expects to expand into Canada soon.

Participants will be asked to provide a variety of tissue and fluid samples for analysis. These will come from cheek cells, urine, blood (taken via finger or toe pricks), and feces.

Investigators will analyze these samples for their amount of serotonin, and for the types and quantities of bacterial species found within them.

People with ASD tend to have higher-than-average levels of serotonin, a hormone that plays key roles in several brain processes, making it an ASD biomarker. Gut bacteria populations differ from person to person and scientists believe these may influence how effective therapies are for a given individual.

Information obtained from patient samples will contribute to an ASD databank maintained by Nova Mentis. The company plans to use this data to identify associations that might cause ASD, and to design a psilocybin-based dosing trial planned to take place in the U.S. later this year.

The root cause of ASD remains unknown and the spectrum of disorders are currently diagnosed based on behavioral patterns. Identifying biological processes underlying these conditions could be key to finding more targeted — and potentially personalized — treatments.