美国神经科学家Bryan Roth在美国国立卫生研究院BRAIN计划会议上披露,中国正在进行至少七项化学遗传疗法的人体临床试验[1]。这些试验采用了Roth二十年前开发的DREADD(设计受体激活的设计药物)技术[1],标志着该技术首次被应用于临床治疗。
该系列试验涵盖顽固性癫痫、帕金森病和神经病理性疼痛的治疗[1]。其中三项试验使用腺相关病毒作为载体[1],试验中采用了氯丙嗪这一抗精神病药物[1]。
Roth指出,"如果该试验成功,那么基本上为几乎所有神经精神疾病的基于回路的治疗方法打开了道路"[1]。神经科学家Jacques Carolan在社交媒体X平台上发表评论称,"如果我们需要更多证据证明中国在神经科学领域领先,这就是了"[1]。
American neuroscientist Bryan Roth revealed at a National Institutes of Health BRAIN Initiative conference that China is conducting at least seven clinical trials of chemogenetic therapies based on DREADD technology, which Roth developed two decades earlier [1]. The trials represent the first clinical applications of this designer protein approach and target treatment-resistant epilepsy, Parkinson's disease, and neuropathic pain [1].
The chemogenetic trials employ various approaches, including the use of chlorpromazine, an antipsychotic drug, as well as adeno-associated viruses as delivery vectors in at least three of the studies [1]. Roth expressed optimism about the potential impact, stating that "if these trials succeed, it basically opens the door to circuit-based therapeutic approaches for almost all neuropsychiatric diseases" [1]. Researcher Jacques Carolan posted on X platform that "if we needed more evidence that China is ahead in neuroscience, this is it" [1].