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Advancing Mechanism-driven Translational Research of Beneficial and Detrimental Effect of Psilocybin on Cancer and Other Health Conditions
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Topic Description
Post Date: April 10, 2026
Expiration Date: April 10, 2027
Purpose:
The purpose of this Topic is to address the critical scientific knowledge gaps in understanding the beneficial and detrimental effect of psilocybin on human health conditions, through rigorous human relevant and mechanism-driven translational research to inform clinical trial design and evidence-based real-world human use, as well as to develop improved therapeutics and to mitigate risks. The source of products can be chemically synthesized single compound, or mushroom extract, or whole product.
The priority areas include:
- Research to understand molecular targets and pathways, and to investigate the beneficial or detrimental effect on human health conditions, including areas of chronic use, microdosing, as well as potential interactions with standard of care, or investigational therapeutics.
- Investigate the effect of multiple factors, such as host genetics, epigenetics, sex, age, lifestyle, environmental factors, and/or health comorbidities that may impact safety and efficacy.
- Identify biomarkers to better inform patient stratification in clinical trial and human use.
- Develop innovative human relevant research models, computation methods and technologies.
Background:
Psilocybin is a naturally occurring alkaloid that is considered a psychedelic drug and is classified as a Schedule I controlled substance in the United States. Psilocybin is converted by the body to psilocin which can change peoples’ moods, thoughts, and perceptions, mainly through interaction with 5-HT2A receptor. Psilocybin is not yet approved by the U.S. Food and Drug Administration as a standard treatment for any disease. As an investigational drug, active clinical trials are ongoing, such as investigating its effect on improving mental health, pain and drug addiction. Preclinical and translational research also investigates neuronal mechanisms of action and molecular targets to develop better drug analogues. Rigorous scientific research exploring safety and efficacy is critically important to inform evidence-based for human use.
Participating ICOs
Psilocybin cancer research focuses on pain and mental health. Recent limited preclinical data suggests psilocybin’s effect on tumor and cancer treatment. Topics of interest include, but are not limited to:
- Investigate mechanism-driven research of its beneficial and detrimental effects on cancer therapeutics, interaction with standard care (e.g. chemotherapy, radiotherapy and immunotherapy), or develop improved therapeutics.
- Research the effect on neuron, immune and tumor behavior.
- Research systemic effect and the effect of confounding or multiple factors.
- Identify prognostic and predictive biomarkers for safety and efficacy.
- Develop innovative human relevant cancer research models, databases, AI tools and other technologies.
- Research safety and effect of long term and chronic use on cancer outcomes.
- Investigate the effects on mitigation or exacerbation of cancer-treatment related sequelae, e.g. pain and neurotoxicity.
- Investigate its use to mitigate cancer symptoms.
Dani Xi, Ph.D.
[email protected]
Rachel Altshuler, Ph.D.
[email protected]
NCCIH is interested in supporting research on psilocybin aimed at understanding the biological and psychological pathways relevant to non-cancer pain and pain comorbidities, integrating insights from traditional human use, neuroscience, and systems biology that are relevant to the Whole Person Health framework. Areas of interest include, but are not limited to:
Studies examining psilocybin multi-system mechanisms underlying effects on pain and symptom burden that integrate neurobiological, emotional and contextual factors (e.g., music, mindfulness, environment);
Characterization of optimal dosing and timing of psilocybin interventions in the context of pain and pain comorbidities; and
Investigating the broader metabolome of psilocybin-producing fungi (e.g., non-tryptamine metabolites) relevant to pain.
Patrick Still, PhD
[email protected]
Psilocybin has been shown to enhance neuroplasticity and induce long-lasting reductions in behaviors associated with substance use. NIDA is interested in supporting research to accelerate the translation and development of psilocybin or its analogs as an effective therapeutic intervention for substance use disorders (SUDs). Topics of interest include, but are not limited to:
- Molecular, cellular, and circuit-level mechanisms by which psilocybin/psilocin affect substance use and polysubstance use.
- Development of prodrugs or modified psilocybin/psilocin analogs to optimize therapeutic profiles.
- Preclinical and Phase I–II clinical studies assessing pharmacokinetics, safety, dose-ranging, and efficacy of psilocybin for SUDs treatment.
- Evaluation of psilocybin combined with medications, neuromodulation, digital therapeutics, or behavioral interventions for SUDs treatment.
- Studies on the effectiveness of psilocybin for treating individuals with polysubstance use disorder.
Jia Bei Wang, MD, PhD
[email protected]
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