字号·· | 护眼
路透社

简讯:基于诺贝尔奖研究的失明疗法展现出前景NEWSLETTER: Blindness treatment based on Nobel-winning research shows promise

点「原文对照」整页切到原文,或双击某段只看那段的原文。

10月9日——眼下正值诺贝尔奖颁奖季,今天我们为大家介绍一种治疗失明的新技术所取得的初步成果。本周,三位科学家因这项技术荣获诺贝尔奖。此外,我们还将报道在美国放射肿瘤学会年会上发布的两项关于不同治疗方案对比的研究。获诺贝尔奖的技术在失明患者治疗中初显成效在一项小规模临床试验中,因视网膜色素变性而失明的患者在接受基于2026年诺贝尔生理学或医学奖获奖研究成果的治疗后,其识别物体的能力得到了恢复,这充分展现了该新疗法的潜力。

Oct 9 - It's Nobel Prize season and today we feature promising early results of a novel technique for treating blindness that made Nobel laureates of three scientists this week. We also report on two studies comparing treatment options presented at the American Society for Radiation Oncology meeting. NOBEL-WINNING THERAPY SHOWS EARLY PROMISE IN BLIND PATIENTS Patients with blindness due to retinitis pigmentosa who received a treatment based on research that won the 2026 Nobel Prize in Physiology or Medicine had their ability to detect objects restored in a small trial that demonstrated the potential of the novel approach.

这种所谓的光遗传学疗法利用光线来控制脑细胞。在这项研究中,研究人员先将能使存活的视网膜神经节细胞对光产生反应的基因疗法与光线刺激相结合,从而激活这些细胞。

So-called optogenetic therapy uses light to control brain cells. In the study, researchers combined gene therapy that made surviving retinal ganglion cells sensitive to light with delivery of light to stimulate these cells.

参与试验的10名患者各自接受了一针注射,其中含有来自GenSight Biologics公司(股票代码:SIGHT.PA)的名为ChrimsonR的光敏蛋白的基因指令。随后,患者们佩戴特制护目镜:该护目镜能捕捉视觉信息并将其转化为特定波长的光信号,从而激活视网膜细胞中经改造后的ChrimsonR蛋白,再将这些光信号传回眼睛。

The 10 trial participants each received an injection into one eye carrying genetic instructions for ChrimsonR, a light-sensitive protein from GenSight Biologics (SIGHT.PA). Patients then wore goggles that captured visual information, converted it into patterns of light at wavelengths that activated ChrimsonR in the modified retinal cells, and sent the patterns of light back to the eye.

结果显示,7名患者对光线变得更加敏感,其中6人的病情出现了具有临床意义的改善——他们能够察觉到物体的存在、判断其位置并准确伸手去触碰。

Seven patients became more sensitive to light, with six achieving clinically meaningful improvements, such as being able to detect when an object was present, determine where it was located and reach toward it accurately.

研究人员在《新英格兰医学杂志》上报告称,注射后即刻出现了一例严重不良反应,但该症状几分钟内便自行消退。

One severe adverse event occurred immediately after injection and resolved within minutes, researchers reported in The New England Journal of Medicine.

研究人员指出,虽然还需要开展更大规模的试验才能最终证实ChrimsonR蛋白与护目镜的实际疗效,但此次研究结果证明:即便在失明程度已相当严重的情况下,那些依然存活的视网膜神经节细胞仍可被诱导对光产生反应,进而将视觉信息传递给大脑。

While larger studies are needed to definitively prove the usefulness of ChrimsonR and the goggles, the findings show that even at a very advanced stage of blindness, surviving retinal ganglion cells can be made responsive to light and transmit visual information to the brain, the researchers said.

“即便对于视力严重受损的患者,其视觉系统依然具备处理新信息的强大能力,”匹兹堡大学的研究负责人何塞-阿兰·萨赫尔博士在一份声明中说道。

"Even in people with profound vision loss, the visual system retains a remarkable capacity to process new information," study leader Dr. José-Alain Sahel of the University of Pittsburgh said in a statement.

放射治疗治疗局部前列腺癌的效果与手术相当 研究人员在近期于波士顿举行的美国放射肿瘤学会年会上报告称,放射治疗在控制局部前列腺癌方面的效果与手术不相上下,且副作用更少。

RADIATION EFFECTIVE AS SURGERY FOR LOCALIZED PROSTATE CANCER Radiotherapy kept localized prostate cancer under control just as well as surgery, with fewer side effects, researchers reported at the recent American Society for Radiation Oncology, or ASTRO, meeting in Boston.

在平均8年的随访后,两组患者的无复发存活率并无统计学差异。在接受为期两周、共5次的立体定向体部放射治疗的患者中,91%的人未出现癌症复发;而接受手术的患者中这一比例则为84%。

After a median of eight years of following patients, survival without recurrence was statistically similar. Of those who received five sessions of stereotactic body radiation therapy delivered over two weeks, 91% were alive without cancer recurrence versus 84% after surgery.

这种立体定向体部放射治疗技术能将放射线精准作用于前列腺,同时最大限度减少对周围健康组织的损伤。

SBRT delivers the radiation directly to the prostate while limiting exposure to nearby healthy tissue.

该试验共纳入123名患者,其中多数患有低危或中危前列腺癌。研究人员对他们进行了肿瘤是否复发以及前列腺特异性抗原水平变化情况的监测——后者是前列腺癌的重要潜在指标。

The trial involved 123 patients, most with low- or intermediate-risk cancers who were monitored for reappearance of a tumor or a rise in prostate-specific antigen levels, a potential indicator of prostate cancer.

该试验最初的目的是比较两种治疗方式对患者生活质量的影响。但由于新冠疫情的影响,原本计划招募两倍人数的计划未能实现,最终参与试验的患者数量不足以回答这一问题。

The trial’s original goal was to compare quality of life after the interventions. Efforts to enroll nearly twice as many participants were thwarted by the COVID pandemic, leaving the study with too few patients to answer that question.

由于该试验的设计目的并非证明两种治疗在癌症控制效果上具有非劣效性,因此也无法证实二者在这一指标上完全相当。

Because it was not designed to establish noninferiority for cancer control, the trial cannot prove the treatments are equivalent for this outcome.

“目前我们可以确定的是……已有充分数据表明,仅进行5次放射治疗即可取得极佳的癌症治疗效果,”英国皇家马斯登 NHS 基金会信托基金的尼古拉斯·范阿斯博士在声明中说道。

“What we can say is that... we have mature data showing excellent cancer outcomes with five-treatment radiotherapy,” study leader Dr. Nicholas van As of The Royal Marsden NHS Foundation Trust in the UK said in a statement.

在ASTRO会议上公布的另一项分析结果显示:两组患者的性功能评分均出现下降,但接受放射治疗后,其评分仍显著优于术前水平。在满分25分的评分体系中,得分越高代表性功能越好;五年后,接受立体定向放射治疗的患者平均得分为19分,而接受前列腺切除术的患者平均得分仅为5分。

A separate analysis presented at ASTRO found that patient-reported sexual-function scores declined in both groups but remained significantly better after the radiation therapy. Median erectile-function scores on a 25-point scale, where higher scores indicate better function, were 19 with SBRT and 5 for prostatectomy after five years.

同样在五年后,接受立体定向放射治疗的患者中仅有8.3%需要使用尿垫来控制漏尿情况,而接受手术的患者中这一比例高达48%。

Also at five years, 8.3% of SBRT patients were using urinary pads for leaks, compared with 48% of surgery patients.

罕见肺癌患者可避免全脑放疗根据ASTRO会议上公布的一项研究数据,患有一种罕见且侵袭性较强的肺癌类型并出现脑转移的患者,可受益于一种对常见肺癌肿瘤而言已是标准疗法的放射治疗技术——这种技术安全性更高。

AVOIDING WHOLE-BRAIN RADIATION IN RARE LUNG CANCER Patients with an uncommon, aggressive form of lung cancer who develop brain metastases can benefit from a safer radiotherapy technique that is already standard for common lung tumors, according to data from a study reported at ASTRO.

立体定向放射外科治疗通过高剂量放射束来摧毁脑肿瘤,无需手术切口,且对周围脑组织的损伤极小。目前,该疗法已是大多数其他类型肿瘤所致脑转移的首选治疗方案。不过对于小细胞肺癌而言,由于担心仅治疗可见肿瘤可能导致残留癌细胞,该疗法一直未被广泛采用。

Stereotactic radiosurgery uses high-dose radiation beams to destroy brain tumors without surgical incisions and with minimal damage to nearby brain tissues. It is already the preferred treatment for brain metastases from most other tumors. Small cell lung cancer has been an exception for fear that treating only visible tumors could leave cancer cells behind.

正因如此,这类患者往往需接受全脑放疗,但这会显著增加其出现记忆力减退及其他认知功能下降的风险。

As a result, patients undergo whole-brain radiation, increasing their risks for memory loss and other cognitive declines.

在这项试验中,151名患者分别接受了立体定向放射外科治疗或新型全脑放疗。结果显示:接受立体定向放射外科治疗的患者中位生存期达17个月,而接受全脑放疗的患者中位生存期仅为9个月。

In the trial, 151 patients received either stereotactic radiosurgery or a modern form of whole-brain radiation. Patients lived a median of 17 months after focused stereotactic radiosurgery versus nine months following whole-brain radiotherapy.

该研究旨在比较不同疗法对患者认知功能的影响。研究人员指出,两种疗法在认知功能下降方面并无显著差异,这或许得益于新型全脑放疗技术所提供的保护作用。不过由于接受全脑放疗的患者中存活至后期随访的人数较少,认知功能方面的对比分析可能受到了一定干扰。

The trial was designed to assess the length of time before patients experienced cognitive decline from each treatment method. The researchers found no significant difference, possibly because of protection afforded by the newer form of whole-brain radiation, they said. Comparisons of cognitive outcomes may also have been complicated by fewer whole-brain radiation patients alive for later follow-up.

虽然延长生存期并非这项试验的主要目的,也无法仅凭本研究予以证实,但研究结果充分体现了这两种治疗方法的显著进步,伊利诺伊州沃伦维尔市西北医学西部地区及质子治疗中心的首席研究员维奈·贡迪博士在一份声明中说道。

While prolonged survival was not the trial's primary goal and cannot be proved by this study, the results show how far both approaches have come, principal investigator Dr. Vinai Gondi of the Northwestern Medicine West Region and Proton Center in Warrenville, Illinois, said in a statement.

“对于小细胞肺癌患者而言,如今我们有了两种先进的放射治疗方案可供选择,同时也有随机对照试验的数据来帮助我们确定最佳治疗方案,”贡迪补充道。

“For patients with small cell lung cancer, we now have two modern radiation therapy strategies to consider and randomized evidence to help us understand how best to use them,” Gondi said.

本文节选自《Health Rounds》,该刊物荣获2026年出版商通讯奖之最佳科学类通讯奖项。我们每周二和周四会在此发布最新的医学研究成果。如需免费订阅完整版通讯,请立即注册。

This is an excerpt of Health Rounds, winner of the 2026 Publishers Newsletter Award for Best Science Newsletter, where we present latest medical studies on Tuesdays and Thursdays. To receive the full newsletter in your inbox for free sign up