MDMA Treatment for PTSD Fails to Secure Federal Approval

The Food and Drug Administration (FDA) last week declined to approve the psychedelic MDMA, also known as ecstasy, to treat post-traumatic stress disorder (PTSD). The agency has asked for an additional Phase 3 clinical trial to test the safety and efficacy of the drug, according to a statement from Lykos Therapeutics, the company that had requested the approval.

The decision is “a huge blow” to efforts to get psychedelics approved for treating mental health conditions, Boris Heifets, an anesthesiologist studying psychedelics at Stanford University, tells NPR’s Will Stone. But some say it isn’t a nail in the coffin.

“I think it will be a temporary setback,” Holly Fernandez Lynch, a bioethicist researching FDA pharmaceutical policy at the University of Pennsylvania, says to NBC News’ Berkeley Lovelace Jr. “The advisory committee and FDA gave very clear indications of what they’re looking for in terms of study design and adverse event reporting, so Lykos and other companies should know pretty clearly how to proceed going forward if they want to get psychedelics approved.”

One day after the FDA’s decision, the journal Psychopharmacology retracted three papers on MDMA-assisted therapy that have researchers tied to Lykos as authors, per the New York Times’ Andrew Jacobs. The company says the research in the retracted papers was not submitted to the FDA as part of its appeal for approval.

The retractions came after the journal learned of unethical conduct by researchers associated with the project at one of the study sites, per the retraction notice. Lykos has previously acknowledged that a study participant in one trial was sexually abused by her therapists, according to Science’s Kai Kupferschmidt. Additionally, the journal wrote that some authors failed to disclose a potential conflict of interest.

In a statement, the drugmaker says “the ethical violations at issue were reported to the FDA, Health Canada and the Institutional Review Board (IRB)/Independent Ethics Committee (IEC), and a public statement was issued in May 2019,” per CNN’s Deidre McPhillips and Carma Hassan. “However, we did not disclose the violations to the journal itself, an additional step we should have taken and regret not doing.”

“Integrity matters in every arena of science, and psychedelic science is no exception,” Brian Barnett, a psychiatrist at Cleveland Clinic, tells CNN. “Hopefully, these retractions will help ensure high quality and transparency in the rapidly growing field of psychedelic science in years to come.”

MDMA is considered a Schedule 1 drug, which means it has a high potential for abuse and no currently accepted medical use. The FDA’s decision last week marked the first time the agency had considered any Schedule 1 psychedelic for a medical treatment. Researchers have studied how a number of psychedelics, including MDMA and psilocybin (the active compound in “magic mushrooms”) could be used to treat different mental health conditions.

Last September, a clinical trial found that when combined with therapy, MDMA reduced people’s PTSD symptoms. The research was funded by the Multidisciplinary Association for Psychedelic Studies, a corporation tied to Lykos.

Two months ago, an FDA advisory panel decided not to recommend that the agency approve MDMA’s use for treatment, citing ethical and safety issues, Healthline’s Shawn Radcliffe wrote at the time. Now, the FDA has requested further research on the drug.

“There are significant limitations to the data contained in the application that prevent the agency from concluding that this drug is safe and effective for the proposed indication,” an FDA spokesperson writes to NPR in an email.

In their statement, Lykos pushed back against the FDA’s ruling.

“While conducting another Phase 3 study would take several years, we still maintain that many of the requests that had been previously discussed with the FDA and raised at the Advisory Committee meeting can be addressed with existing data, post-approval requirements or through reference to the scientific literature,” Amy Emerson, CEO of Lykos, says in the statement. “The FDA request for another study is deeply disappointing, 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.”

PTSD affects around 6 percent of the U.S. population at some point in their lives, according to the Department of Veterans Affairs. Treatments for the disorder are currently limited to talk therapy and two antidepressant medications, per the National Institute for Mental Health. Had MDMA been approved, it would have become the first new treatment for PTSD in more than two decades.

“It’s frustrating to people who were hoping that this treatment would be found to be safe and effective and become available,” Michael Bogenschutz, a psychedelics researcher at NYU, tells CNN. “I think we just need to take a deep breath and be patient and let the data accumulate to show more definitively, one way or the other, whether, in this particular case, MDMA can become a bona fide medical treatment for PTSD.”

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Will Sullivan is a science writer based in Washington, D.C. His work has appeared in Inside Science and NOVA Next.