Norring Sarah A, Spigarelli Michael G
PsyBio Therapeutics, Inc., Sunrise, FL, USA.
Drug Des Devel Ther. 2024 Apr 10;18:1143-1151. doi: 10.2147/DDDT.S443177. eCollection 2024.
Psilocybin, a tryptamine psychedelic, has been touted in the media both historically and recently as a potential game-changing mental health therapeutic. ClinicalTrials.gov has over one hundred and thirty psilocybin clinical trials listed covering the last twenty years. The single most important aspect of any therapeutic is to gain approval for marketing and thus enter the real-world phase of development. A typical new chemical entity progresses from inception to US Food and Drug Administration (FDA) approval in approximately 12 years and seeks approval for a single indication.
An observational study was conducted with the available information on the ClinicalTrials.gov site to observe the extent of progress made demonstrating the clinical utility of psilocybin.
The results showed 134 psilocybin trials typically unblinded studies of 10-20 participants, recruited over years at a single site. Additionally, there have been only three advanced trials (1 Phase 2/3 and 2 Phase 3) submitted, and only in the last two years.
The hundreds of psilocybin clinical trials initiated over the past twenty years comprising a myriad of potential indications may actually be slowing this potential game-changing mental health therapeutic agent's approval and is costing excessive amounts of capital. To fully evaluate the actual potential of psilocybin, purposeful clinical trials need to be designed well, executed efficiently, and analyzed utilizing sequential and statistically valid processes for each potential indication. This will require a change from the current exploratory forays to defined, well-funded, sequential pharmaceutical development practices, including adequate and appropriate blinding of studies, statistical design to determine the number of participants and more importantly, professional expertise in conducting multicenter trials. Unfortunately, these results demonstrate little real progress towards FDA approval of psilocybin and a field with no clear direction forward.
裸盖菇素是一种色胺类致幻剂，无论在历史上还是近期，都在媒体上被吹捧为一种可能改变游戏规则的心理健康治疗药物。ClinicalTrials.gov网站列出了过去二十年来的一百三十多项裸盖菇素临床试验。任何治疗方法最重要的一个方面就是获得上市批准，从而进入实际的开发阶段。一个典型的新化学实体从研发到获得美国食品药品监督管理局（FDA）的批准大约需要12年时间，并且只为单一适应症寻求批准。
利用ClinicalTrials.gov网站上的现有信息进行了一项观察性研究，以观察在证明裸盖菇素临床效用方面所取得的进展程度。
结果显示，有134项裸盖菇素试验，通常是针对10至20名参与者的非盲研究，在单个地点进行了多年招募。此外，仅提交了三项进展较为深入的试验（1项2/3期试验和2项3期试验），而且都是在过去两年才提交的。
在过去二十年里启动的数百项裸盖菇素临床试验涵盖了众多潜在适应症，但这实际上可能正在延缓这种可能改变游戏规则的心理健康治疗药物的批准进程，并且耗费了大量资金。为了全面评估裸盖菇素的实际潜力，需要精心设计有针对性的临床试验，高效执行，并针对每个潜在适应症采用连续且统计有效的流程进行分析。这将需要从当前的探索性尝试转变为明确的、资金充足的、连续的药物开发实践，包括对研究进行充分且恰当的设盲、进行统计设计以确定参与者数量，更重要的是，需要具备开展多中心试验的专业知识。不幸的是，这些结果表明在裸盖菇素获得FDA批准方面几乎没有取得实际进展，而且该领域也没有明确的前进方向。
