Advocates say the push for psychedelic medicine isn't over, post-FDA rejection

Editor's Note: A version of this post was first published in WBUR's weekly health newsletter, CommonHealth. If you like what you read and want it in your inbox, sign up here. This post has been updated with additional information about Lykos and Rick Doblin.

Proponents of psychedelic medicine had a setback this month. The federal Food and Drug Administration declined to approve a combination of talk therapy and MDMA, also known as ecstasy and “Molly,” as a treatment for post-traumatic stress disorder.

The application had been hailed as a potential milestone in the treatment of PTSD — and the broader field of psychedelic medicine. But the FDA said it needed more research.

This frustrated many patients and the drugmaker, Lykos Therapeutics, which worked with the FDA to design the trials.

“The FDA request for another study is deeply disappointing,” said Amy Emerson, Lykos’ chief executive, in a written statement, “not just for all those who dedicated their lives to this pioneering effort, but principally for the millions of Americans with PTSD, along with their loved ones, who have not seen any new treatment options in over two decades.”

She estimated it would take several years to conduct the additional clinical trial.

The FDA’s response wasn’t surprising. An advisory group had already given Lykos a thumbs down after concerns were raised about its research. Critics alleged the data failed to reflect some participants’ negative experiences; a pair of therapists was removed from the trials following serious allegations of sexual misconduct. (This recently contributed to the retractions of several research papers.)

Members of the advisory panel also pointed out a more fundamental problem: Many participants who received MDMA — and their therapists — had a pretty good idea they were not part of the control group. Some had even taken MDMA before. (This was allowed in the clinical trials, but appeared to be among the concerns raised during the FDA’s review.) This meant their desire to derive some benefit from the treatment may have clouded the results. In other words, it’s pretty tough to blind a clinical trial when the effects of the medication under review are, well, trippy.

There are other concerns, too, including how the government would regulate a novel combination of psychotherapy and a drug, and the way MDMA and other psychedelics can put patients in vulnerable states.

Despite the decision and the concerns, many scientists in the field of psychedelic medicine expect it’s a matter of when, not if, regulatory approvals come — for PTSD and a range of other mental health issues. Lykos had hoped its application for MDMA would be the first to pry open the door.

Lykos is a spinoff of the Multidisciplinary Association for Psychedelic Studies, or MAPS, the driving force behind recent efforts to legalize psychedelic medicine. MAPS started with MDMA, in part, because of personal experiences described by its founder, Rick Doblin.

In a 2021 interview, Doblin told then-WBUR reporter Angus Chen that he first tried MDMA with a girlfriend when he was in his 20s.

“We were able to have this discussion in a way that was really smooth and loving and open. So, that’s where I felt like, ‘God, I wish I could be this clear and defenseless and listening all the time,’ ” Doblin explained.

Later, he recalled helping a friend take MDMA as she grappled with a traumatic experience. To him, it seemed like the drug helped her open up and gain confidence in her ability to move forward.

“That was incredible,” he said. These experiences contributed to his belief that MDMA had therapeutic potential.

“You can take the lessons from the MDMA state and apply them into your daily life, and practice being that way without being on MDMA,” he said. “I think that’s why it’s so powerful as a therapy drug.”

After the FDA’s request for more proof that the benefits of MDMA-assisted therapy outweigh the risks, Doblin told WBUR it was “one in a series of bumps in the road.”

“It means that we need to put out more efforts,” he said. “But we will continue to do whatever is necessary because we believe that this can be a potentially very helpful addition to the treatment of PTSD.”

Days later, Doblin left Lykos' board. He told the publication STAT he expects another company, Compass Pathways, to win the race for FDA approval with a psilocybin treatment. He also indicated Lykos is considering decoupling MDMA from therapy to garner a green light from the FDA. Doblin sounded cool to that approach, calling it "not what’s best for patients."

"The therapy component is so important," he said.

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