FDA Approves Landmark Psilocybin Trial for Treatment-Resistant Depression

The US Food and Drug Administration (FDA) has granted approval to COMPASS Pathways’ planned clinical trial to test psilocybin therapy in patients with treatment-resistant depression.

The phase 2b dose-ranging trial will involve 216 patients across 12-15 research sites in North America and Europe, beginning in the UK later this month. Federal regulatory agencies have already granted approval for such a trial in the UK, Canada, and the Netherlands.

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Psilocybin therapy—a combination therapy of psychoactive medicine and the active agreement found in psychedelic mushrooms, plus psychological support—has been a frequent, if not controversial, investigative treatment for psychiatric conditions since the 1950s.

Charles L. Raison, MD, a professor in the School of Human Ecology at the University of Wisconsin-Madison, said in an interview with MD Magazine® that the mushroom-based therapy was initially observed in patients suffering from alcoholism or cancer. In the early 2000s, privately-funded investigators began to notice a consistent positive effect of the drug on patients’ mood.

“It seems to have a long-term impact on people,” Raison said. “Even normal folks seem to feel better after, and they take better care of themselves afterwards.”

One study even suggested that patients’ personalities changed after being treated, Raison noted—more than a year after treatment, they were more expressive and less prone to depression. Another pair of randomized, controlled, crossover studies comparing a single psilocybin treatment versus placebo reported “amazing” results, Raison said.

“One treatment just completely reduced depression and anxiety scores, from people being very clinical to reaching remission,” Raison said. “No other treatment, and 6 months later, 60-70% people are in remission from a single treatment.”

COMPASS Pathways, a life sciences company founded in 2016, has focused its early efforts on late-stage clinical trial development for psilocybin in patients with treatment-resistant depression. They advocate for advances in neuroscience, psychotherapy, psychopharmacology, and technology.

George Goldsmith, chairman and co-founder, expressed excitement for the beginning of the landmark trial.

“Depression is the leading cause of ill-health and disability worldwide, and treatment-resistant depression affects more than 100 million people,” Goldsmith said in a statement. “It is a huge unmet need and the trial will teach us more about how this new approach might address it."