FDA experts assess potential MDMA treatment for PTSD
The category of the drug MDMA is known as a Schedule I substance according to the Controlled Substances Act, and its approval for medical use would be a significant change of perception. This is the first time the Food and Drug Administration (FDA) has considered a psychedelic drug for therapeutic purposes. MDMA, known as an entactogen, can induce feelings of emotional connection, relatedness, and emotional openness.
PTSD is a disorder affecting around 5% of adults in the United States, or approximately 13 million people, according to the National Center for PTSD. However, current treatment options such as antidepressants and cognitive behavioral therapy have limitations, and alternative therapies are scarce for those who do not effectively receive them.
Lykos Therapeutics has recently reported positive results from two late-stage clinical trials that utilized MDMA in combination with therapy to treat PTSD. These trials witnessed the participation of about two hundred individuals, in a treatment program involving three eight-hour sessions with received the MDMA in presence of two therapists. These sessions were scheduled about a month apart, accompanied by more therapy sessions.
In a briefing document about the studies, the FDA commented that participants reported "rapid, clinically significant, and long-lasting improvements in their PTSD symptoms." However, there are several uncertainties concerning the trials' interpretability and potential risks which the Psychopharmacologic Drugs Advisory Committee members need to consider.
This committee discusses potential drug approvals and generally follows the FDA's decisions but is not bound to do so.
The trials were designed to be double-blind, meaning neither the participants nor the researchers were aware of who received the MDMA treatment and who didn't. However, MDMA's hallucinogenic effects have a significant impact on the state of being, making it challenging to maintain blindness. The Institute for Clinical and Economic Review, which conducts independent studies on treatments, tests, and procedures, raised concerns regarding the validity of the results due to the unblinding of the trials and other influencing factors.
There are concerns around potential safety issues associated with MDMA as well. The drug's potential for abuse has been recognized, but the research did not adequately address this issue in the context of the treatment program. Reports from recreational use of MDMA indicate possible damage to heart and liver health. The phase III clinical trials indicated "significant increases in blood pressure and pulse" in participants, but some related assessments were incomplete, and liver health had been evaluated only in smaller groups from earlier phases.
Lykos has been in talks with the FDA during their research and considers this advisory committee meeting a crucial step in the process. The project's goal is to provide alternative treatment options for the significant number of patients requiring new options, while safety is their utmost priority.
The growing interest in psychedelics as potential therapies extends to other drugs, such as ketamine and psilocybin mushrooms, which are being studied for treating a variety of mental health conditions. "Depression, PTSD, eating disorders - they're all different," emphasized Amy Emerson, CEO of Lykos Therapeutics. "You need to study it, look at the safety and efficacy profiles of each, and we have to be careful not to treat these things as panaceas or magic bullets."
Regardless of the outcome, the way the FDA and advisory committee approach the use of this drug will play a critical role in future precedents. Dr. Joshua Gordon, director of the National Institute of Mental Health, acknowledges the exciting potential while recognizing the need for further research: "It's an early but exciting moment. The promise is there, the potential is there, and we just have to find out through careful study and time how impactful these medications will be."
Further clarifications are needed on the conditions necessary to effectively utilize this treatment and the degree to which the FDA's Advisory Board might support it. Still, despite the limitations, even the most optimistic experts suggest a cautious approach to understanding the possible benefits of the Lykos treatment.
Many unanswered questions remain regarding the ideal circumstances and precise conditions to experience effective outcomes with this treatment. As the debate continues, the FDA's decision will likely set crucial precedents that will play a significant role in determining the future application and understanding of the drug.
"I'm hoping the drug gets approved, but in a way that enables it to be accessible and supervised responsibly," shared Dr. Rachel Yehuda, director of the Center for Psychedelic Psychotherapy and Trauma Research at Mount Sinai's Icahn School of Medicine, and not a part of the FDA advisory committee. "My worry is that we might lose our curiosity, cease asking the pertinent questions, and give up trying to figure out the treatment's mechanisms or methods of improvement."
Psychedelics are sensitive to the blurring lines between strictly regulated medical therapies given by skilled practitioners in closely monitored contexts and recreational usage, according to Dr. Jon Alpert, head of the American Psychiatric Association's research council and the department of psychiatry and behavioral sciences at the Albert Einstein College of Medicine. "The worst case scenario is having a treatment with immense potential for a terrible ailment becoming too unsupervised and unstructured."
It's paramount to safeguard those who could profit from this treatment, as specialists explain.
"There's an acknowledgement that psychedelics hold a unique susceptibility to the merging of strictly enforced medical treatments provided by experienced therapists in careful conditions and casual usage," Dr. Alpert commented. "There's an intense drive to ensure this is done correctly."
It's essential to collect more data on how the treatment impacts delicate populations, such as adolescents and those who suffer from concomitant issues like schizophrenia or bipolar illness, he added.
"An FDA endorsement of MDMA must be partnered with thorough laws, stringent prescriptive and distribution regulations, comprehensive patient education, and sustained monitoring and survey systems," the American Psychiatric Association stated in a letter to the FDA's advisory board.
Besides possibly establishing a precedent in mental health treatment, the program Lykos has investigated is time-consuming and resource-intensive, as she explained. It necessitates substantial investments in both logistics and emotion from those utilizing the treatment and their providers.
"We've never witnessed a case where something deemed detrimental by the government becomes a sanctioned treatment for a predicament," Yehuda noted. "The subsequent question is, 'Can we use this pivotal moment to improve the psychological well-being of our population?'"
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The FDA's Psychopharmacologic Drugs Advisory Committee needs to consider the uncertainties surrounding the interpretability and potential risks of the MDMA trials for PTSD treatment.
If approved, stringent laws, prescriptive and distribution regulations, comprehensive patient education, and sustained monitoring and survey systems should be implemented for MDMA use as a treatment for PTSD.
