MONDAY, Aug. 12, 2024 (HealthDay News) -- Advocates for the use of the psychedelic drug MDMA (midomafetamine) to treat mental health conditions were delivered a setback on Friday after the U.S. Food and Drug Administration declined to approve it as treatment for post-traumatic stress disorder (PTSD).

There is insufficient evidence that MDMA -- currently illegal and also known as Ecstasy or molly -- can ease PTSD, the agency told Lykos Therapeutics, a company that's been seeking to gain approval for the drug's legitimate use.

The FDA asked that Lykos conduct another clinical trial to determine whether MDMA is safe and effective against PTSD -- a demand that could mean many years and millions of dollars before another chance at approval would arise.

"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, who have not seen any new treatment options in over two decades," Lykos Therapeutics CEO Amy Emerson said in a statement.

"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 [agency's] advisory committee meeting can be addressed with existing data, post-approval requirements or through reference to the scientific literature," Emerson added.

Many had assumed that FDA approval was assured, given that Lykos' trials had been designed in consultation with the agency.

The company trial involved 100 people who had been diagnosed with PTSD for an average of 15 years.

However, the decision was not a complete surprise: An FDA advisory board that met in June decided against recommending MDMA as treatment.

If it had been approved, the drug would have been the first psychedelic drug regulated by federal authorities.

“This is an earthquake for those in the field who thought FDA approval would be a cinch,” Michael Pollan, co-founder of the UC Berkeley Center for the Science of Psychedelics, told the New York Times. He's also the author of the best-selling “How to Change Your Mind,” a book that's helped alter public attitudes towards psychedelics in general.

Key to the FDA's rejection of MDMA is the fact that Lykos' trials did not address the level of cardiac risk tied to the drug's use.

Some benefit did seem to be there: According to company data, more than two-thirds of patients with PTSD at the beginning of the clinical trial no longer qualified for such a diagnosis just six months after beginning treatment.

Overall, 86 percent of participants who received MDMA combined with psychotherapy saw symptoms subside, and 71 percent no longer met criteria for PTSD by the study's end. That's compared to 48% of people who took a placebo alongside their talk therapy.

For years, people battling the flashbacks and anxiety of PTSD have been going abroad to seek out the controversial treatment.

The FDA has not released the letter it sent to Lykos announcing its decision.

But the Times said that, in a statement, an FDA spokesman said “the agency recognizes that there is great need for additional treatment options for mental health conditions such as PTSD. We will continue to encourage research and drug development that will further innovation for psychedelic treatments and other therapies to address these medical needs.”

According to the U.S. Drug Enforcement Agency, MDMA stands alongside heroin and marijuana as Schedule I illegal drugs with “no currently accepted medical use and a high potential for abuse."

Still, the FDA granted MDMA "breakthrough status" several years ago, essentially giving it a fast-track path through the review process.

But there were unconventional elements to Lykos' work with MDMA.

One key sticking point, the company said, is that the drug was given alongside psychotherapy. The FDA does not regulate psychotherapy, and its addition might have been a roadblock.

Another issue may lie in the fact that many patients involved in the clinical trial were not naive to the treatment, and had tried MDMA before.

According to Lykos, the FDA also appeared to be uncertain over how long any benefit from MDMA might last, and the level of benefit credited to the psychotherapy used by patients.

"The advisory committee panelists also raised psychotherapy as a concern, with some recommending to further characterize the extent to which psychotherapy contributes to treatment benefit and if it is even necessary," the company said. "Lykos acknowledges that midomafetamine-assisted therapy represents a novel combination of drug and therapy that raises unique research questions and will continue to engage the FDA as appropriate on these challenges. Lykos remains committed to continuing development of this integrated approach."

New approaches to easing PTSD are needed. The FDA has not approved any new PTSD therapy for more than 20 years, and the most common meds used, SSRI antidepressants, only help less than a third of patients, the Times noted.

“We are frustrated that the FDA has kicked the can down the road in the face of such urgent need and clear demand from the veteran community for more effective PTSD treatments,” Lt. General Martin Steele, a former Marine Corps commander and a co-founder of Reason for Hope, an organization that promotes psychedelic therapy for veterans with tough-to-treat psychiatric problems, told the Times. “Delay will only lead to more unnecessary suffering and lives lost.”

Still, other companies have psychedelics in the pipeline. One such company is Sunstone Therapies, which is conducting five clinical trials on psychedelic compounds.

Sunstone CEO Dr. Manish Agrawal told the Times that the FDA's decision should be viewed as a lesson for trials going forward.

“While disappointing for the people at Lykos who have been working on this for decades, in the big picture, it’s not a huge setback,” he said. “At the end of the day, some trials might get refined and more finely tuned, but the fact that we are even having this conversation and the FDA is considering this medication and asking hard questions is a good thing.”

More information

There's more on PTSD at the National Institute of Mental Health.

SOURCES: Lykos Therapeutics, statement, Aug. 9, 2024; New York Times

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