蒙大拿州近期扩展了"右试"法律，允许非绝症患者在药物完成I期临床试验后申请获得未获批的实验性药物[1]。蒙大拿州卫生部门已于上周正式确定了相关诊所规则，并建立了实验治疗审查委员会来审查此类申请[1]。
患者Kris DeVault的儿子Brody患有罕见的肌酸转运蛋白缺陷症[1]。法国生物技术公司Ceres正在开发一种鼻喷药物治疗该病，该药物已在48名健康成人中完成I期临床试验，但尚未在患者或儿童中测试[1]。DeVault希望通过蒙大拿州新成立的审查委员会为其儿子获得这一实验性治疗[1]。然而，Ceres公司首席执行官担心此举可能触怒美国食品药品监督管理局[1]。
DeVault对这项法律寄予厚望，表示"我看到这个，就像，这是Brody唯一的机会"[1]。但他也表示担忧，即使药物最终获批，到达美国市场也需要至少数年时间，届时Brody将错过其神经发育的关键窗口期[1]。
Montana has broadened its "right to try" legislation to permit patients with non-terminal conditions to access experimental drugs that have completed Phase I clinical trials [1]. The state's health department formally established clinic rules last week and created an Experimental Treatment Review Board (ETRB) to evaluate patient applications for unapproved medications [1].
For families like that of Brody DeVault, who suffers from a rare creatine transporter protein deficiency, the new pathway offers a potential lifeline. The boy's father, Kris DeVault, is seeking to obtain an experimental nasal spray treatment being developed by French biotechnology company Ceres through Montana's newly established review process [1]. The drug has completed Phase I trials in 48 healthy adults but has not yet been tested in patients with the disease or in children [1]. DeVault expressed his determination, stating: "I look at this, and I'm like, that is my one shot for Brody" [1].
However, significant hurdles remain. The company's CEO has expressed concern about potentially antagonizing the FDA through participation in the program [1]. Additionally, DeVault fears that even if the drug eventually receives approval, it may take several years to reach the U.S. market—a timeline that could be too late for his son, who may pass what DeVault describes as his "plasticity window" before treatment becomes available [1].
