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v2.2.0

Investigational Treatments

Clinical trial treatments including psilocybin, MDMA, and other breakthrough therapies currently under research.

24 Investigational Treatments
Evidence-Based

5-MeO-DMT Therapy

5-MeO-DMT therapy involves the carefully screened, clinically supervised administration of 5-methoxy-N,N-dimethyltryptamine. The compound produces a rapid-onset, ultra-short psychedelic experience often characterized by ego dissolution and non-dual awareness. Early research and observational reports suggest potential benefits for depression, anxiety, trauma-related symptoms, and existential distress. Because of the experience’s intensity, rigorous preparation, risk screening, medical safeguards, and structured integration are essential.

Investigational

Trial Phase

Phase I–II (varies by protocol)

Status

Investigational / Controlled Substance (jurisdiction-dependent)

Session Time

Primary effects 10–30 minutes; total visit 2–4 hours including monitoring

Course Length

Single-session

AMPA Modulators (Ampakines)

AMPA modulators—often called 'ampakines' or AMPA positive allosteric modulators (PAMs)—increase the activity of AMPA-type glutamate receptors without directly activating them. By slowing receptor desensitization and/or deactivation, these agents can strengthen excitatory neurotransmission, promote long-term potentiation (LTP), and upregulate neurotrophic factors such as BDNF. A growing pipeline of AMPA PAMs is being investigated for major depressive disorder, cognitive impairment, schizophrenia (negative/cognitive symptoms), attention disorders, and other neuropsychiatric conditions.

Investigational

Trial Phase

Phase I–II (varies by compound)

Status

Investigational

Session Time

Daily dosing or study-defined titration windows

Course Length

Short-term

Ayahuasca Therapy

Ayahuasca is a psychoactive brew prepared from the Banisteriopsis caapi vine and the Psychotria viridis (or Diplopterys cabrerana) leaf. It combines β-carboline alkaloids (harmine, harmaline, tetrahydroharmine) that act as MAO inhibitors with the psychedelic tryptamine DMT. In ceremonial and clinical contexts, ayahuasca induces intense visionary experiences, emotional catharsis, and altered states of consciousness. Early research and growing clinical interest suggest potential efficacy for treatment-resistant depression, PTSD, anxiety, and substance use disorders when combined with structured preparation and integration support.

Investigational

Trial Phase

Phase I–II (varies by study)

Status

Controlled in many jurisdictions; authorized in some religious/spiritual contexts

Session Time

4–6 hours

Course Length

Single ceremony

Brain-Computer Interfaces (BCIs)

Brain-computer interfaces (BCIs) are systems that establish a direct pathway between brain activity and external devices such as computers, prosthetics, or stimulation systems. They may be invasive (implanted electrodes) or non-invasive (EEG, MEG, fNIRS). BCIs are being explored for restoring communication in paralyzed patients, enhancing motor recovery after stroke, controlling neuroprosthetics, and modulating brain activity in psychiatric conditions. Research is ongoing to determine safety, reliability, and therapeutic efficacy across diverse conditions.

Investigational

Trial Phase

Phase I–II

Status

Investigational; FDA Breakthrough Device Designations in some cases

Session Time

30 minutes – several hours

Course Length

Single sessions

Chemogenetics

Chemogenetics involves genetically engineering neurons to express Designer Receptors Exclusively Activated by Designer Drugs (DREADDs). These engineered receptors respond only to specific synthetic ligands, such as clozapine-N-oxide (CNO), which are otherwise biologically inert. By selectively activating or silencing neuronal populations, researchers can precisely probe brain circuits underlying psychiatric and neurological disorders. While primarily a research tool, chemogenetics is being investigated for therapeutic applications in epilepsy, Parkinson’s disease, depression, addiction, and chronic pain. Translation to humans faces significant hurdles, including safe gene delivery, receptor stability, and ethical considerations.

Investigational

Trial Phase

Preclinical, early translational

Status

Investigational (no approved therapeutic use)

Session Time

Ligand administration typically short (minutes–hours), but genetic modification is permanent

Course Length

L

Closed-Loop Neurostimulation

Unlike open-loop neuromodulation, which delivers stimulation continuously or in pre-set patterns, closed-loop neurostimulation dynamically adjusts its output based on recorded neural or physiological signals. Devices use sensors to detect abnormal brain or nervous system activity, and algorithms trigger or modulate stimulation accordingly. This responsive system aims to improve efficacy, minimize side effects, and extend device lifespan. Current clinical applications include the NeuroPace RNS® System for drug-resistant epilepsy and investigational systems for depression, Parkinson’s disease, and chronic pain.

Investigational

Trial Phase

FDA-approved for epilepsy; investigational for other conditions

Status

Partially approved (RNS for epilepsy); investigational for depression, Parkinson’s, chronic pain

Session Time

Continuous, device-driven

Course Length

C

CRISPR-Based Therapies for Mental Health

CRISPR-based therapeutics use programmable nucleases and effectors to edit DNA or regulate gene expression. While CRISPR has advanced rapidly in monogenic and systemic diseases, psychiatric applications remain investigational due to polygenic risk, complex brain circuitry, and delivery barriers to the CNS. Approaches under study include in vivo CRISPR gene editing, CRISPR interference/activation (CRISPRi/a) for transcriptional tuning, base and prime editing for precise changes, and RNA-targeting systems. Targets of interest include stress-response genes (e.g., FKBP5), serotonergic and glutamatergic pathways (e.g., SLC6A4, GRIN genes), neuroplasticity regulators (e.g., BDNF), and epigenetic marks linked to trauma. Translation requires safe, cell-type–specific delivery (AAV, LNP, engineered capsids), rigorous off-target profiling, and ethical safeguards.

Investigational

Trial Phase

Preclinical; early human feasibility in non-psychiatric CNS diseases

Status

Investigational; no clinical approval for psychiatric use

Session Time

One-time or limited dosing; biological effects can be durable

Course Length

Long-term expression (AAV)

DMT-Assisted Therapy

DMT is a potent, fast-acting serotonergic psychedelic naturally occurring in plants and endogenous human metabolism. In therapeutic contexts, DMT is administered via intravenous or intramuscular routes, producing an intense but brief psychedelic state (5–30 minutes). Early-phase clinical studies and preclinical work suggest rapid effects on mood and neural plasticity, potentially offering a novel treatment for mental health disorders. Investigational protocols pair DMT with psychotherapy, preparation, and integration. Its extremely short duration differentiates it from psilocybin or LSD, raising interest in its potential as a more clinic-manageable psychedelic intervention.

Investigational

Trial Phase

Phase 1–2 clinical trials

Status

Schedule I (US); investigational only

Session Time

5–30 minutes (psychedelic experience), with 1–2 hours preparation and integration

Course Length

S

Experimental Gene Therapy (Mental Health Applications)

Experimental gene therapy for mental health aims to alter gene expression or introduce corrective genetic material in specific neural circuits. Approaches include AAV- or lentiviral-based gene delivery, RNA interference (siRNA/shRNA), antisense oligonucleotides (ASOs), and programmable systems for transcriptional tuning (e.g., CRISPRi/a) delivered via adeno-associated virus or lipid nanoparticles. Targets span stress-response signaling, monoaminergic transporters and receptors, glutamatergic plasticity genes, neurotrophic factors, and synaptic scaffolding proteins. While gene therapy is established in some non-psychiatric conditions, psychiatric applications face unique challenges: polygenic risk architecture, cell-type specificity, durable yet controllable expression, blood–brain barrier delivery, and ethical safeguards.

Investigational

Trial Phase

Preclinical to early human feasibility (primarily non-psychiatric CNS to date)

Status

Investigational; no approved psychiatric indications

Session Time

Dose administration typically brief; expression may persist months–years (vector/platform dependent)

Course Length

One-time dosing (AAV/viral)

Experimental Glutamate Modulators

Glutamate is the brain’s primary excitatory neurotransmitter, central to synaptic plasticity, learning, memory, and mood regulation. Dysregulation of glutamatergic signaling has been implicated in depression, anxiety, OCD, PTSD, and schizophrenia. Experimental glutamate modulators explore a wide range of mechanisms—beyond ketamine and esketamine (already FDA-approved)—such as NMDA receptor subunit–selective antagonists (e.g., NR2B-selective), glycine-site modulators, AMPA receptor positive allosteric modulators (AMPAR PAMs), mGluR2/3 agonists or antagonists, and triple reuptake inhibitors with glutamatergic activity. The aim is to achieve rapid, durable antidepressant and anxiolytic effects without dissociation or abuse liability.

Investigational

Trial Phase

Phase I–II (compound-specific)

Status

Investigational; none approved for psychiatric use beyond ketamine/esketamine

Session Time

Minutes to hours (depending on administration)

Course Length

Single-dose studies

Fecal Microbiota Transplantation (FMT)

Fecal microbiota transplantation (FMT) involves transferring processed stool from a healthy donor into the gastrointestinal tract of a recipient. This therapy aims to reestablish microbial diversity and function, which may be disrupted in various diseases. While already established for recurrent Clostridioides difficile infection, FMT is being investigated in psychiatry and neurology for conditions such as depression, autism spectrum disorder, and Parkinson’s disease, based on evidence linking gut microbiota to brain function via the gut–brain axis.

Investigational

Trial Phase

N/A

Status

N/A

Session Time

Procedure duration varies (30–60 minutes for colonoscopy; oral capsules taken over days)

Course Length

Single administration or repeated doses over weeks

Focused Ultrasound (FUS) Therapy

Focused Ultrasound (FUS) is an emerging neuromodulation modality that delivers acoustic energy through the skull to precise brain targets without open surgery. Depending on intensity and frequency, FUS can be used for noninvasive neuromodulation (low-intensity FUS, or LIFU) or permanent lesioning (high-intensity focused ultrasound, HIFU). MR-guided FUS allows real-time targeting and monitoring. Research applications in psychiatry include modulation of the anterior cingulate cortex, amygdala, and striatum to reduce symptoms of depression, OCD, and anxiety, as well as permanent lesioning of circuits implicated in severe treatment-resistant cases.

Investigational

Trial Phase

Phase I–II (psychiatric use)

Status

FDA-approved for essential tremor and tremor-dominant Parkinson’s disease; investigational for psychiatric disorders

Session Time

1–3 hours (including MRI setup and procedure)

Course Length

Single-session for lesioning

GLP-1 Receptor Agonists

Glucagon-like peptide-1 (GLP-1) receptor agonists enhance glucose-dependent insulin secretion, slow gastric emptying, and reduce appetite. Beyond metabolic effects, GLP-1 receptors are expressed in brain regions involved in reward, motivation, and mood (e.g., VTA, nucleus accumbens, prefrontal cortex). Early clinical and preclinical research suggests potential benefits for major depressive disorder, anhedonia, cognitive impairment, and substance use disorders by modulating dopamine signaling, reducing neuroinflammation, and improving metabolic health. Psychiatric applications remain investigational.

Investigational

Trial Phase

N/A

Status

N/A

Session Time

N/A

Course Length

Short-term

Ibogaine Therapy

Ibogaine is a naturally occurring indole alkaloid found in the root bark of the African shrub *Tabernanthe iboga*. Traditionally used in Bwiti spiritual practices, it has gained attention for its purported ability to rapidly reduce withdrawal symptoms and cravings in opioid and stimulant addiction. Its complex pharmacology includes interactions with NMDA, opioid, sigma, and serotonergic receptors, as well as modulation of neurotrophic factors. Despite anecdotal and early clinical evidence, ibogaine remains investigational due to serious safety concerns, especially cardiotoxicity and risk of fatal arrhythmias. Research continues into safer analogs such as noribogaine.

Investigational

Trial Phase

Phase I–II (early human trials, observational studies)

Status

Schedule I (U.S.); available in some countries (Mexico, Brazil, New Zealand) in specialized clinics; investigational globally

Session Time

12–24 hours acute psychedelic phase, followed by integration

Course Length

Typically single session, may be followed by booster or integration therapy

Lysergic Acid Diethylamide (LSD) Therapy

LSD (lysergic acid diethylamide) is a potent serotonergic psychedelic that profoundly alters perception, cognition, and sense of self. When administered in a controlled therapeutic setting, LSD is paired with psychotherapy to facilitate emotional release, enhanced self-reflection, and potential restructuring of maladaptive cognitive and behavioral patterns. Current research focuses on LSD-assisted therapy for anxiety associated with life-threatening illness, treatment-resistant depression, cluster headaches, and substance use disorders. While promising, LSD therapy remains investigational and highly regulated.

Investigational

Trial Phase

Phase II (psychiatric research)

Status

Schedule I controlled substance in most jurisdictions; investigational use only

Session Time

6–10 hours (including preparation and integration)

Course Length

Single or multiple sessions spaced weeks apart

MDMA-Assisted Psychotherapy

3,4-Methylenedioxymethamphetamine (MDMA) is a psychoactive compound known for its empathogenic and prosocial effects. In clinical settings, MDMA is paired with psychotherapy to facilitate trauma processing, emotional openness, and reduction of fear responses. Research, particularly through MAPS-sponsored trials, has demonstrated robust efficacy for severe, treatment-resistant PTSD. MDMA-assisted psychotherapy is being investigated for depression, social anxiety, and substance use disorders. It is currently in late-stage clinical development and under FDA review.

Investigational

Trial Phase

Phase III (for PTSD)

Status

Schedule I controlled substance in most jurisdictions; FDA Breakthrough Therapy designation for PTSD

Session Time

6–8 hours per dosing session

Course Length

Typically 2–3 MDMA sessions with preparatory and integration therapy

Mescaline-Assisted Therapy

Mescaline is a classic psychedelic alkaloid found in peyote, San Pedro, and Peruvian torch cacti. Historically used in Native American religious and healing practices, it produces profound alterations in perception, cognition, and emotional experience. Emerging modern research suggests mescaline may offer therapeutic benefits for depression, anxiety, and addiction when combined with structured psychotherapy. Scientific exploration is still at an early stage compared to psilocybin and MDMA, but growing interest in mescaline's long duration and spiritual quality of experiences is fueling investigational studies.

Investigational

Trial Phase

Early-stage clinical and observational research

Status

Schedule I controlled substance in most jurisdictions; not FDA-approved

Session Time

8–12 hours per dosing session

Course Length

1–3 mescaline sessions with preparatory and integration therapy

Multimodal Antidepressants

Unlike traditional SSRIs and SNRIs, multimodal antidepressants act on multiple neurotransmitter systems simultaneously. For example, vortioxetine and vilazodone not only inhibit serotonin reuptake but also directly modulate serotonin receptor subtypes. This approach aims to enhance antidepressant efficacy, improve cognitive symptoms, and reduce side effects like sexual dysfunction. Research continues to evaluate the comparative benefits of these newer medications over conventional antidepressants.

Investigational

Trial Phase

Approved in some countries; ongoing comparative and expansion studies

Status

FDA-approved (vortioxetine, vilazodone) for major depressive disorder

Session Time

Daily oral dosing

Course Length

Chronic, ongoing treatment

NMDA Antagonists

NMDA antagonists act on the N-methyl-D-aspartate (NMDA) receptor, a subtype of glutamate receptor critical in excitatory neurotransmission and synaptic plasticity. By modulating glutamate signaling, these drugs can induce rapid antidepressant effects, sometimes within hours. Ketamine, delivered intravenously, and intranasal esketamine are the leading examples, with FDA approval for treatment-resistant depression and depressive symptoms in major depressive disorder with acute suicidal ideation. Other NMDA antagonists, including investigational agents like rapastinel and apimostinel, are being studied to achieve similar efficacy with improved safety and tolerability.

Investigational

Trial Phase

Phase II–III for novel agents; ketamine/esketamine FDA-approved for TRD

Status

FDA-approved for esketamine in TRD and depressive symptoms with suicidality; ketamine off-label use in psychiatry

Session Time

40 minutes (IV ketamine) to 2 hours (including monitoring)

Course Length

Acute and maintenance protocols, often weeks to months

Novel Antidepressants

Novel antidepressants represent a new wave of pharmacological strategies for mood disorders, particularly treatment-resistant depression (TRD). Unlike conventional antidepressants, which primarily act on monoamine neurotransmitters (serotonin, norepinephrine, dopamine), these therapies focus on alternative systems, including NMDA receptor modulation, AMPA potentiation, kappa-opioid receptor antagonism, and neurotrophic pathways such as BDNF signaling. Examples include esketamine (NMDA antagonist, FDA-approved), zuranolone (neurosteroid modulator), and investigational compounds like rapastinel, an NMDA partial agonist, and SAGE-217. They hold promise for rapid-acting effects, better safety profiles, and improved outcomes in TRD and comorbid psychiatric conditions.

Investigational

Trial Phase

Phase II–III for most investigational agents; some FDA-approved

Status

Esketamine and zuranolone FDA-approved; other agents investigational

Session Time

30–120 minutes (depending on administration)

Course Length

Acute courses with possible maintenance protocols

Showing 20 of 24 items

Important Medical Disclaimer

The information provided here is for educational purposes only and should not replace professional medical advice. Always consult with a qualified healthcare provider before starting any new treatment.