NASH: A new approach brings new hope

写的:

 

Sudha Shankar

Global Clinical Head, NASH BioPharmaceuticals R&D

 

丹尼尔·林登

Director/Senior Principal Scientist Bioscience Metabolism, BioPharmaceuticals R&D

肝脏可以被看作是新陈代谢管弦乐队的指挥,那么澳门第一赌城在线娱乐如何才能解开科学之谜,为患者改变曲调呢?

6月10日国际非酒精性脂肪性肝炎日强调全球对非酒精性脂肪性肝炎日益增长的未满足需求. Where virally-driven liver disease used to be predominant, NASH is now the growing cause of liver disease around the world. 这在一定程度上是由于病毒性肝病的高效治疗方法的发展, 但更重要的是,这是全球肥胖和糖尿病水平上升的直接结果.   

The NASH story so far

NASH是一种相对较新的疾病,直到20世纪90年代才作为一个概念存在. 虽然现在已经知道NASH有遗传和环境因素, we are still building a picture of what NASH entails and the mechanisms at play. 诊断往往很晚,因为它是一种临床沉默的疾病,患者很少有症状,直到疾病的晚期. The rate of disease progression may be slow and asymptomatic, 但NASH可导致严重的疾病,包括肝细胞癌和突发性代偿失调——几乎总是需要肝移植来防止死亡. 目前还没有批准的NASH治疗方法,这种疾病主要通过改变饮食和生活方式来控制.

 

NASH的下一章:澳门在线赌城娱乐如何以不同的方式思考,为患者提供新的可能性

First and foremost, 澳门第一赌城在线娱乐认为,发现疾病的关键驱动因素对于创造第一代治疗方法至关重要. 接下来是进一步阐明其他新途径,从而开发下一代治疗方法. 澳门第一赌城在线娱乐的方法 in NASH is no different. We recognise that NASH is a pathophysiologically evolving, multi-modal disease with a major metabolic component. 像这样, it is becoming evident that by targeting the different disease drivers, such as reversal of steatosis, fibrosis and inflammation, we can reasonably expect to reduce the totality of disease burden, 从而最终改善NASH患者的整体临床结果.

第二项重要努力是澳门第一赌城在线娱乐在开发新诊断工具方面的投资, both to aid clinical trials and to improve the patient experience. Currently, diagnosis is invasive and requires a liver biopsy. As part of our current studies, 澳门第一赌城在线娱乐正在研究非侵入性和侵入性诊断程序的系统比较. Our data set continues to grow gradually, 通过持续的投资,澳门第一赌城在线娱乐希望这将减少对患者进行侵入性手术的数量,并推动NASH的早期诊断.

第三个关键因素来自于澳门第一赌城在线娱乐开发创新治疗模式的能力,这些治疗模式是针对以前被认为无法治疗的靶点的. 目前的任务是分离出NASH的最佳治疗靶点,并确定哪种治疗方式将带来预期的效果. Coupled with precision medicine, we are able to segment the broad NASH patient population, 要么通过特定的遗传成分,要么通过特定疾病标记的流行, will enable us to optimise the response of tailored treatment interventions. Not only does this mean we can run smaller, more efficient clinical trials, 但澳门第一赌城在线娱乐也可以增加澳门第一赌城在线娱乐澳门第一赌城在线娱乐正在研发的分子的信心.

And finally, we recognise that all this progress cannot happen in isolation. We value our involvement in pre-competitive, public-private partnerships and collaborations, -如欧盟/英国肝脏调查:脂肪性肝炎标志物测试(LITMUS)联盟和美国代谢性肝病非侵入性生物标志物(NIMBLE)联盟-以更好地定义患者群体并发现潜在的非侵入性生物标志物,以帮助和加速新疗法的开发. Support from global relationships allows us access to different cohorts, data and samples – all of which help us define the natural history of disease, characterise disease progression, 确定那些有进展风险的患者,并确定干预的关键时机. 很简单, the more we understand the science, the closer we get to developing an effective treatment.

为肝脏和更广泛的代谢系统提供精确的药理解决方案

肝脏可以被看作是新陈代谢管弦乐队的指挥,也是澳门第一赌城在线娱乐关注的焦点, 因此, starts at the liver. Given its pivotal influence over other organs and processes, we need to be cognisant of the broader impact of drugging liver targets. At the same time, 这是一个考虑可能带来的肝外益处的机会.

凭借在心血管领域的丰富经验,澳门第一赌城在线娱乐有能力探索这一思维领域, renal and metabolic disease, 因此, 澳门第一赌城在线娱乐目前的NASH项目正在发现相关的心血管和肾脏益处. 解决互补生物学的潜在联合治疗方法可能会为这些经常有许多合并症的患者带来更广泛的益处.

Leveraging our expertise in genetic medicine, we are leading the way with precision medicine approaches. 这包括  treating NASH patients carrying a variant in the PNPLA3 gene 导致肝酶功能失调,不仅不能消化脂肪本身, but impedes another type of enzyme from doing it too.

Our vision for NASH patients

澳门第一赌城在线娱乐相信,真正改善患者的疾病体验是通过提高疾病识别能力的结合, simple non-invasive diagnostic, and effective multi-modal holistic therapeutic options. We are striving to arrest disease progression, reverse existing disease, 改善合并症,减少总体发病率负担,使NASH患者生活得更好, healthier lives.



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Veeva ID: Z4-49018
Date of preparation: October 2022