Opinions expressed by Entrepreneur contributors are their own.
Much like cannabis, psychedelics are catching the eye of investors for their potential to break out of their classification of scheduled substances and move closer to becoming valuable tools in the treatment of mental health issues.
Researchers and public companies are currently gathering substantial evidence in phase 2 and phase 3 clinical trials for the therapeutic potential of psychedelic molecules in indications like PTSD, depression and substance addiction.
For now, autism spectrum disorders have, for the most part, remained outside the realm of psychedelic medicine. However, as research into psychedelics advances, some companies in the space are aiming to create psychedelics-based solutions targeting ailments in this category.
Psilocybin for autism and fragile X syndrome
Autism spectrum disorders can have severe consequences in the lives of patients and their families. With a prevalence of approximately 1 in 54 children in the U.S., the estimated cost of autism for the average family reaches $60,000 a year through the patient’s childhood.
Disorders in this spectrum are some of the most common indications lacking a known cure or overall satisfactory solution. According to the nonprofit Autism Speaks, the economic cost of caring for Americans with autism reached $268 billion in 2015 and would rise to $461 billion by 2025 in the absence of more effective interventions and support across the patient’s life span.
Canadian biotech Nova Mentis Life Science Corp. recently announced a partnership with Mycrodose Therapeutics to develop patented psilocybin products to treat neuroinflammatory disorders, with autism in mind.
Psilocybin is also known as the active compound in so-called “magic mushrooms.”
The partnership intends to explore the use of psilocybin-based therapeutics in disorders such as fragile X syndrome and autism spectrum disorder, by using Mycrodose’s patented drug delivery systems with Nova Mentis’s proprietary version of psilocybin.
Dr. Marvin S. Hausman, chairman of Nova’s Scientific Advisory Board, said that psilocybin has shown promising results in preclinical models of fragile X syndrome and autism spectrum disorder in sub-hallucinogenic doses tested in four preclinical trials.
The company added, in an email, that based on preclinical research, psilocybin could improve symptoms such as anxiety and cognition problems which are typical in ASD patients.
Researchers hope that psilocybin could have even deeper effects than merely treating autism spectrum symptoms in fragile X syndrome.
Possible treatments would involve low doses of psilocybin (colloquially referred to as “microdoses”), allowing for a broadened patient base and possible pediatric applications.
Fragile X syndrome, a genetic condition that causes a variety of developmental problems including learning disabilities and cognitive impairment, is associated with autism as one in three individuals with fragile X syndrome presents features related to autism spectrum disorder.
“The results are exciting and have shown that psilocybin corrects the cognitive deficits displayed by a rat model of [fragile X syndrome] and mitigates the anxiety-like traits observed in an environmental model of autism based on prenatal exposure to valproic acid,” Hausman said.
The companies recently announced having received orphan drug designation from the European Medicines Agency (EMA).
Orphan drug is a designation granted to certain compounds that have the ability to treat rare and genetic conditions with unmet solutions, affecting relatively small populations. The designation helps pharmaceutical companies waive fees, receive tax credits and fast-track the development of the compound into an available drug.
The companies have also applied for the same designation from the FDA in the U.S., and have formed a new company meant to take the treatment through clinical trials in the near future.
Using MDMA for autism and social anxiety
Mind Medicine (MindMed) Inc., a unicorn biotech company developing a range of psychedelic molecules for mental health, announced on Tuesday a new program meant to deploy MDMA for the treatment of social anxiety and functioning in diagnoses that include autism spectrum disorder.
MDMA is currently undergoing phase 3 clinical trials for the treatment of post-traumatic stress disorder by the nonprofit MAPS. The drug is expected to become the first FDA-approved psychedelic treatment to come online in late 2022 or early 2023.
Known by its street name “ecstasy” or “molly,” MDMA also offers a wide range of benefits for a number of diverse patient populations. MindMed is currently recruiting subjects for a study combining MDMA with LSD.
Social anxiety and impairments in social functioning are hallmarks of ASD, which occurs in approximately 2% of individuals in the U.S.
“At present, there are no approved therapies for the core symptoms of ASD and there remains a significant unmet need for novel therapies to support people with ASD,” noted MindMed in a press release.
Beyond autism spectrum disorder, approximately 12% of the U.S. general population experience social anxiety disorder at some point in their lives, according to the National Institute of Mental Health.
A MAPS-sponsored phase 2 pilot safety study of MDMA-assisted therapy for social anxiety in autistic adults showed strong and statistically significant improvements in social anxiety and functioning from short-term treatment with MDMA.
Modifying MDMA for autism and other indications
MDMA exists as a combination of two enantiomers: mirrored compounds with the same atoms arranged in exactly opposite ways.
MindMed poses that these two enantiomers could each have unique pharmacological activities. “Preclinical data suggest that the R(-) enantiomer maintains the acute pro-social and empathogenic benefits of racemic MDMA,” according to the company.
This leads the biotech to conclude that this half of MDMA could have therapeutic applications beyond those of “whole” MDMA, including the potential for more accessible delivery models and repeated dosing.
MindMed plans to advance its R(-)-MDMA development program targeting US and EU registration and expects to initiate its first clinical trials in 2022.
The company, in association with long-time partner Liechti Lab at University Hospital Basel, plans to initiate a comparative clinical trial of both MDMA enantiomers by 2022 in a double-blind, placebo-controlled crossover study.
“The compelling clinical efficacy of MDMA coupled with the unique pharmacological benefits of its R(-) enantiomer suggest that there is an enormous opportunity to bring this second-generation psychedelic program to market with the potential for new clinical applications, novel treatment paradigms and enhanced accessibility,” said MindMed CEO Robert Barrow.