Fecal transplants for autism deliver success in clinical trials

粪便移植治疗自闭症在临床试验中取得成功

Scientific research continues to uncover interesting connections between the gut microbiome and human health, including everything from depression to PTSD to autoimmune disease. Another example of this are emerging ties between gut health and autism. Exciting new research, now moving to Phase 3 human trials, has found boosting microbial diversity via fecal transplants can dramatically reduce autism symptoms in the long term.

科学研究不断揭示肠道微生物群与人类健康之间有趣的联系，涵盖了从抑郁症、创伤后应激障碍（PTSD）到自身免疫性疾病等方方面面。肠道健康与自闭症之间日益显现的关联便是另一个例证。目前正进入三期临床试验的一项令人振奋的新研究发现，通过粪便移植增加微生物多样性，可以长期显著减轻自闭症症状。

One in every 59 children born in the US is diagnosed with autism, according to the Centers for Disease Control and Prevention, and unfortunately for many of them, chronic gastrointestinal issues are a harsh reality of their condition. According to scientists at Arizona State University (ASU), who conducted the current study, around 30 to 50% of people with autism experience serious gut problems like constipation, diarrhea and stomach pain.

根据美国疾病控制与预防中心（CDC）的数据，美国每59名儿童中就有一人被诊断出患有自闭症。不幸的是，对于其中许多人来说，慢性胃肠道问题是他们病症中残酷的现实。据进行这项研究的亚利桑那州立大学（ASU）科学家称，约30%至50%的自闭症患者会经历严重的肠道问题，如便秘、腹泻和胃痛。

“Many kids with autism have gastrointestinal problems, and some studies, including ours, have found that those children also have worse autism-related symptoms,” ASU’s Rosa Krajmalnik-Brown said back in 2019 during the early stages of the work. “In many cases, when you are able to treat those gastrointestinal problems, their behavior improves.”

“许多自闭症儿童都有胃肠道问题，包括我们在内的一些研究发现，这些儿童的自闭症相关症状也更严重，”亚利桑那州立大学的Rosa Krajmalnik-Brown在2019年该研究的早期阶段曾表示，“在许多情况下，当你能够治疗这些胃肠道问题时，他们的行为就会有所改善。”

A key study in 2019 built on earlier research from 2017 that found introducing new bacteria via fecal transplants in 18 autistic children brought about marked improvements in their behavior, as measured through questionnaires assessing their social skills, hyperactivity, communication and other factors. These improvements held for eight weeks, an impressive outcome to be sure. But the Arizona State University researchers then set out to investigate the enduring effects of the treatment, which involved a bowel cleanse and daily transplants of fecal microbiota over a period of seven to eight weeks.

2019年的一项关键研究建立在2017年的早期研究基础之上。该研究发现，通过粪便移植为18名自闭症儿童引入新细菌，在评估其社交技能、多动症、沟通能力及其他因素的问卷调查中，他们的行为表现有了显著改善。这些改善持续了八周，这无疑是一个令人印象深刻的结果。随后，亚利桑那州立大学的研究人员着手调查该疗法的持久效果，该疗法包括肠道清洁以及为期七到八周的每日粪便微生物群移植。

Two years after the treatment, the researchers found that not only did the benefits persist, they seemed to improve. Doctors’ observations at the eight-week mark found that psychological autism symptoms of the patients had decreased by 24%. But two years later those symptoms had almost been cut in half, with a professional evaluator finding a decrease of 45% in autism symptoms compared to baseline.

治疗两年后，研究人员发现，这些益处不仅持续存在，似乎还有所改善。医生在八周时的观察发现，患者的心理自闭症症状减轻了24%。但两年后，这些症状几乎减少了一半，专业评估人员发现，与基线相比，自闭症症状减轻了45%。

“We are finding a very strong connection between the microbes that live in our intestines and signals that travel to the brain,” Krajmalnik-Brown said in 2019. “Two years later, the children are doing even better, which is amazing.”

“我们发现生活在我们肠道中的微生物与传向大脑的信号之间存在着非常紧密的联系，”Krajmalnik-Brown在2019年说道，“两年后，这些孩子的情况甚至变得更好，这太令人惊叹了。”

The next steps were larger placebo-controlled clinical trials designed to verify their results, with a view to gaining FDA approval for the therapy. In early 2022, Krajmalnik-Brown and colleagues patented a specific bacterial formulation and spun-off a commercial company called Gut-Brain Axis Therapeutics. The treatment, dubbed Microbiota Transplant Therapy (MTT), moved through a Phase 2 human placebo-controlled trial over the following years and the initial data has been incredibly promising.

接下来的步骤是进行更大规模的安慰剂对照临床试验，旨在验证研究结果，并争取获得美国食品药品监督管理局（FDA）对该疗法的批准。2022年初，Krajmalnik-Brown及其同事为一种特定的细菌配方申请了专利，并分拆成立了一家名为“肠脑轴疗法”（Gut-Brain Axis Therapeutics）的商业公司。这种被称为“微生物群移植疗法”（MTT）的治疗方法在随后的几年里完成了二期人体安慰剂对照试验，初步数据非常令人振奋。

Now, the team is looking to raise funds to move through the large-scale Phase 3 trials necessary for final FDA approval.

目前，该团队正在寻求资金，以推进获得FDA最终批准所需的大规模三期临床试验。