FDA staff questions safety of ecstasy for PTSD
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A Food and Drug Administration staff report questions the safety of using the drug known as ecstasy to treat PTSD, citing the potential for abuse and possible harmful side effects to the heart and liver.
Why it matters: The findings cast new doubts on what could become the first psychedelic-based treatment approved in the U.S. ahead of a June 4 meeting during which FDA advisers will review a new drug application from Lykos Therapeutics.
What they're saying: Clinical trials using MDMA to treat PTSD made it difficult to assess safety and effectiveness, FDA staff wrote.
- They said the studies lacked sufficient data on how it induces euphoria or elated mood to make recommendations for monitoring potential abuse by patients.
- The small sample sizes in Phase 1 and 2 studies also made it difficult to confirm the treatment would not cause liver damage observed in earlier studies of illicit use of the drug, the report states.
- It's also not clear how much of the benefits came from the drug or the psychotherapy that was paired with it, FDA staff wrote.
The FDA staff additionally raised the prospect of bias in the trial. Patients weren't told if they received a drug or a placebo, but because of the drug's notable effects, patients who got MDMA would know it — and that could raise their expectations of benefitting from the treatment.
Context: MDMA, ecstasy's chemical name, targets multiple neurotransmitters in the brain, potentially reducing fear responses and helping ease the way for therapy.
- The FDA report notes Lykos' application includes evidence patients who received the drug experienced "statistically significant and clinically meaningful improvement" in their PTSD symptoms, and that improvement appears to last for at least several months.
- But staff noted that beyond the way the drug affects the mind, it's been observed to significantly elevate blood pressure and pulse, which can trigger cardiovascular events.
The influential Institute for Clinical and Economic Review earlier this month concluded publicly available evidence for MDMA-assisted therapy is insufficient and that the clinical trials may have been tainted by investigator biases and understated possible harmful effects.
Lykos CEO Amy Emerson said in a statement Friday that the company stands by its research and development program and would discuss it in more detail at the June 4 meeting.
- "If the FDA approves MDMA-assisted therapy, we will be planning for a responsible introduction of this new treatment into the healthcare system with careful consideration of its potential benefits and risks," Emerson said.
- The FDA, which has fast-tracked review of Lykos' treatment, is expected to make a decision by Aug. 11.
Editor's note: The story has been updated with the Lykos CEO's comment.
