Psychedelics Have ‘Promising Medical Applications,’ Congressional Watchdog Agency Says, But Research Challenges Remain

A federal agency has published a short report on the medical use of psychedelics, finding that their ability to “change a person’s perceptions and sense of self” can make for “promising medical applications.”

The paper, from the Government Accountability Office (GAO), says there’s a need for further research, but it also notes that because psychedelics such as LSD, MDMA and psilocybin remain Schedule I controlled substances, securing permission to carry out that research can be a challenge.

“To conduct research on these drugs, scientists need to follow several steps,” the report from GAO, which is often referred to as Congress’s “watchdog,” states in a section about ongoing challenges. “These include obtaining permission from the U.S. Drug Enforcement Administration, finding clinical grade drugs to test, and identifying appropriate spaces in which to test and store these drugs.”

“Difficulties associated with conducting large, blind trials of psychedelics have limited researchers’ ability to determine the safety and effectiveness of these drugs,” GAO continues, “which is required for them to gain approval from the Food and Drug Administration (FDA). FDA approval is generally required before prescription drugs can be marketed for sale in the U.S.”

Despite the obstacles, the paper acknowledges that hundreds of clinical trials have investigated psychedelics as a potential treatment for post-traumatic stress disorder (PTSD) and depression.

“Between 2015 and early 2025, over 340 trials on psychedelics began or were completed,” it says. “For example, one study found that psilocybin reduced depression symptoms more than escitalopram, an SSRI.”

Science & Tech Spotlight: Medical Use of Psychedelics https://t.co/xD6DHLwNcT

— U.S. GAO (@USGAO) March 6, 2025

Other opportunities identified by GAO include mental health and pain management.

“Research has shown that psychedelics, such as psilocybin and MDMA, decrease fear and anxiety, with the potential to positively affect behavior in combination with therapy,” the report says, adding that psychedelics also “appear to show promise for patients with certain headache disorders and cancer pain,” apparently by reducing inflammation and altering pain perception through interactions with the brain’s serotonin receptors.

The agency also noted the relatively minor risks associated with psychedelics compared to many other pharmaceutical treatments:

“Psychedelics may have non-life-threatening side effects, such as headaches or vomiting. In some cases, the use of these drugs can lead to safety risks due to impairment, or illicit psychedelics may be contaminated with dangerous substances such as fentanyl. While some research suggests that the use of psychedelics does not typically lead to physical addiction, the adverse effects of these drugs have not been fully studied.”

Beyond policy-related obstacles to research, the GAO report also points to methodological challenges. For example, in a typical blinded trial, participants aren’t supposed to know whether they received the experimental compound or a placebo. “Because of the distinct effects of psychedelics on recipients,” the new paper says, “it is difficult to prevent trial participants from knowing whether they received the treatment.”

Government Accountability Office

“This may complicate efforts to design such trials and interpret their findings. Clinical trials have tried to work around this by using an active placebo, such as a low dose of a psychedelic,” authors wrote. “Alternatively, researchers might compare the treatment with a more typical one, such as an SSRI.”

As the GAO report notes, the past decade has seen a flurry of new study into psychedelics.

Late last year, for example, researchers for the first time ever administered LSD to patients in a Phase 3 clinical trial into whether the psychedelic can be used to effectively treat generalized anxiety disorder (GAD).

The study is eventually expected to enroll about 200 people in the U.S. and will compare the effects of a proprietary LSD product to a placebo.

In March of last year, the Food and Drug Administration (FDA) granted the LSD product “breakthrough therapy” status as a treatment for GAD.

Last summer, meanwhile, a scientific review into MDMA found that five out of six studies looking at the substances’ potential to treat for post-traumatic stress disorder (PTSD) “provide evidence for the apparent safety and efficacy” of the therapy.

Authors called the findings “encouraging” but said more robust research is needed before MDMA-assisted therapy sees widespread adoption over currently available forms of treatment.

That review came on the heels of an FDA advisory panel rejecting an application to authorize MDMA-assisted therapy. A group of bipartisan lawmakers and veterans advocates, however, almost immediately pushed back on that decision.

“What we’re asking for the FDA to recognize is the science,” said Rep. Morgan Luttrell (R-TX), who in a June op-ed for Marijuana Moment criticized the FDA Psychopharmacologic Drugs Advisory Committee for recommending against against MDMA-assisted therapy.

Psilocybin has also been the subject of considerable research, including as a mental health aid and a treatment for substance use disorders.

Earlier this year, for example, a study on the use of psilocybin-assisted therapy to treat methamphetamine use disorder found that the treatment “was feasible to implement in an outpatient setting, did not appear to generate safety concerns, and demonstrated signals of effectiveness warranting further investigation.”

That report, which was not peer reviewed, was published by The Lancet as a preprint in January. It found that among a small group of people in a stimulant treatment program, “Methamphetamine craving decreased while quality of life, depression, anxiety, and stress improved from baseline to day 28 and 90 follow-up.”

The National Institutes of Health last year announced that it would put $2.4 million toward funding studies on the use of psychedelics to treat methamphetamine use disorders—funding that came as federal health officials noted sharp increases in deaths from methamphetamine and other psychostimulants in recent years, with fatal overdoses involving the substances rising nearly fivefold between 2015 and 2022.

In 2023, meanwhile, the National Institute on Drug Abuse (NIDA) announced a $1.5 million funding round to further study psychedelics and addiction.

Psilocybin has already been given federal breakthrough therapy status for the treatment of certain types of depression.

Last year the National Center for Complementary and Integrative Health (NCCIH), which is part of the National Institutes of Health, also published an informational web page about psilocybin, acknowledging the substance as a possible treatment for alcohol use disorder, anxiety and depression.

The page, “Psilocybin for Mental Health and Addiction: What You Need To Know,” also highlighted psilocybin research being funded by the federal government into the drug’s effects on pain, migraines, psychiatric disorders and various other conditions.

Findings of another recent study suggests that the use of full-spectrum psychedelic mushroom extract has a more powerful effect than chemically synthesized psilocybin alone, which could have implications for psychedelic-assisted therapy. The findings imply that the experience of entheogenic mushrooms may involve a so-called “entourage effect” similar to what’s observed with cannabis and its many components.

A separate study recently published by the American Medical Associatio (AMA) found that single-dose psilocybin use was “not associated with risk of paranoia,” while other adverse effects such as headaches are generally “tolerable and resolved within 48 hours.”

That study, published in JAMA Psychiatry, involved a meta-analysis of double-blind clinical trials where psilocybin was used to treat anxiety and depression from 1966 to last year.

AMA published another recent study that similarly contradicted commonly held beliefs about the potential risks of psychedelics use, finding the substances “may be associated with lower rates of psychotic symptoms among adolescents.”

Also, results of a clinical trial published by AMA in late 2023 “suggest efficacy and safety” of psilocybin-assisted psychotherapy for treatment of bipolar II disorder, a mental health condition often associated with debilitating and difficult-to-treat depressive episodes.

The association also published research earlier that year finding that people with major depression experienced “clinically significant sustained reduction” in their symptoms after just one dose of psilocybin.

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Photo courtesy of Wikimedia/Workman.

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