The psychedelic medicine revolution just took a big loss

Advisers to the federal agency that regulates drugs said Tuesday the psychedelic ecstasy, when combined with therapy, is not an effective treatment for post-traumatic stress disorder and its use puts patients at risk of serious side effects.

An expert advisory committee voted 9-2 that Lykos Therapeutics’ combo regimen of talk therapy and MDMA, the technical name for the drug, is not an effective PTSD treatment and 10-1 that the therapy's risks outweigh its benefits.

"Premature introduction of a treatment can actually stifle development and stifle innovation," said Dr. Paul Holtzheimer, a member of the committee and deputy director for research in the executive division of the Department of Veterans Affairs’ National Center for PTSD.

If the FDA follows its advisers, as it typically does, it could upend a burgeoning industry already banking on using a variety of mind-altering drugs to treat disorders ranging from depression to anxiety.

Why it matters: There's a profound need for better PTSD treatments. Current options are notoriously ineffective, with many people with PTSD left white-knuckling through daily life.

But the advisers found that Lykos’ trials most likely suffered from expectation bias since patients were able to figure out they received the drug, rather than a placebo, and that might have made them more likely to say it was beneficial. Members of the committee also cited concerns about missing safety data, allegations of trial manipulation and the potential risk for misconduct among therapy practitioners.

Lykos pointed to its trial data finding sixty-seven percent of participants with severe PTSD who received MDMA combined with therapy no longer met the diagnostic criteria for the disorder, compared with 32 percent in the group that received therapy and a placebo.

In its 2023 trial, Lykos said 71 percent of participants who received the combo treatment no longer met the criteria for PTSD.

But Lykos has come under fire for allegedly designing its studies to make the case for approval. The head of the National Institute on Drug Abuse, Nora Volkow, last month compared the hype for psychedelics as a cure for mental illness to belief in “fairy tales,” in Senate testimony.

In March, an independent, nonpartisan group that evaluates new drug applications found Lykos’ trials suspect.

"We havesubstantial concerns about the validity of the results," the Institute for Clinical and Economic Review wrote in a draft report. The institute cited safety, ethical and cost concerns and projected difficulty in widely implementing the treatment.

The institute also critiqued the psychedelic community, which it said treats psychedelic drugs "more like a religious movement than like pharmaceutical products," feelings the report said "were sometimes inculcated in patients participating in the trials."

And in April, a citizen petition from psychedelic researchers critical of Lykos called on the FDA to hold an extended open public hearing so that the advisory committee could hear the public's concerns.

The advisers’ decision also may dash hopes in Congress and at the Department of Veterans Affairs that Lykos’ protocol could help treat the mental illnesses that now afflict more than a quarter of Americans, including hundreds of thousands of people who served in the military.

The VA cites research finding 7 percent of veterans will suffer from PTSD at some point in their lives. It can cause flashbacks, nightmares and anxiety brought on by traumatic events — like combat. Nearly one in four vets who seek care at a VA facility have the condition, had it or will get it.

Last month, the VA’s undersecretary for health, Dr. Shereef Elnahal, spoke enthusiastically about psychedelic medicine at a conference in New York put on by the Horizons Center Public Benefit Corp., which promotes use of the drugs for mental health conditions.

In March, President Joe Biden signed legislation that included an amendment by Reps. Lou Correa (D-Calif) and Jack Bergman (R-Mich.) encouraging the VA to use federal funding to study psychedelics as mental health treatments for veterans.

The FDA has previously designated MDMA and psilocybin, another psychedelic drug, as breakthrough therapies, a label meant to expedite their path toward approval.

What’s next: The FDA said it expects to make a decision by mid-August.