Transforming clinical development in NASH

今年的国际非酒精性脂肪性肝炎日是一项全球努力,旨在提高非酒精性脂肪性肝炎(NASH)的可见度和紧迫性,并重点关注人们如何采取行动,应对这种肝脏疾病危及生命的后果. We share the same mission, taking action to develop groundbreaking R&D approaches to slow or stop disease progression.


Driving NASH diagnosis earlier in disease to improve patient outcomes

Despite its growing prevalence, NASH is frequently underdiagnosed, and patients may not be identified until they develop symptoms from late-stage complications.1 这种疾病的发病是逐渐的,需要几年到几十年的时间,早期阶段通常在临床上是沉默的,被患者和临床医生忽视.This, combined with a lack of precise non-invasive diagnostics, often leads to late, or even missed diagnoses.3,4

As we progress our NASH therapies into late-stage clinical development, there is a fast-growing need for large-scale recruitment of patients with NASH. Yet, due to the difficulties in diagnosing, identifying and engaging patients with NASH, there is a lack of candidates eligible for clinical trials. In precision medicine research, these trials target specific genetically defined populations, 因此有必要对更多的患者进行筛查,以确定哪些患者具有相关特征. Here, 澳门葡京赌博游戏正在改变通过基因检测参与者识别NASH最高风险患者的方式,以支持澳门葡京赌博游戏的精准医疗项目. In addition, we are developing ways to simplify how our clinical trials operate, making participation easier for clinicians and patients with NASH.

澳门葡京赌博游戏还确保医生获得他们推荐合适的试验参与者所需的所有信息. Educating healthcare professionals, 特别是那些接触到糖尿病和肥胖症等高危人群的人, is essential to identifying suitable participants for clinical trials.

Our goal is to bring life-changing NASH treatments to patients sooner. 这意味着通过在整个药物开发过程中进行创新,了解和解决诊断和疾病意识方面的高度未满足需求, including the critical stages of clinical trial recruitment. Today, recruitment rates remain low, 目前的系统不能充分支持正在进行的130多个NASH临床试验.5 澳门葡京赌博游戏需要借此机会调整澳门葡京赌博游戏的方法以适应传统的临床试验,并尽快将治疗方法带给患者.

 

从Arun Sanyal博士那里了解更多关于NASH及其与澳门葡京赌博游戏整体代谢健康的关系, Professor of Medicine, Virginia Commonwealth University


在接下来的章节中,澳门葡京赌博游戏将讨论澳门葡京赌博游戏正在采取的一些关键行动,以改变NASH患者的临床发展.




Accelerate clinical trial recruitment

We are at the forefront of clinical trial innovation, 高度专注于改善和简化招聘流程,并为医生和患者带来好处,包括:

  • 让更多NASH患者有机会在多个合适的介入NASH临床试验中进行选择, so they can take part and personally select those trials most relevant or convenient to them.
  • 将基因预筛选站点网络与澳门葡京赌博游戏的干预性试验集合紧密联系起来, enabling rapid assessment of trial eligibility based on the characteristics of their illness, including genetic drivers and disease stage.
  • Providing a single point of care for education, screening, 试验干预措施,有可能根据遗传洞察力和风险概况获得更有针对性的治疗.
  • The opportunity for trial sites to participate in multiple interventional trials, where they can be more active partners. Sites will also benefit from a model tailored to their specific needs and processes.
  • Improving workflows between sites and our teams, 帮助在旅程开始时建立协作关系,并使全球团队能够利用当地的见解来调整试验流程和协议. 

这种试验交付的方法是伟大的,因为它使澳门葡京赌博游戏能够在许多NASH患者进展到晚期疾病之前为他们做更多的事情.

Participating hepatologist

Looking beyond this initiative, 开发一种更容易获得和负担得起的检测方法,作为肝活检的一种侵入性更小的替代方法,将使更多的高危患者得到筛查. To that end, we have partnered with research consortia, such as LITMUS in Europe and NIMBLE in the US, 寻找潜在的非侵入性NASH生物标志物,这些标志物可以发展成新的诊断测试.


Target key disease drivers

NASH的科学研究帮助澳门葡京赌博游戏找到了针对不同人群潜在病因的新方法. By integrating genetic testing into our screening process, 澳门葡京赌博游戏也许能够识别出最有可能从特定的精准医学治疗中受益的NASH患者群体. 这种方法将有助于确保根据患者的疾病特征招募合适的患者进行合适的试验.

Tackling another important step in the development process, we’re developing sophisticated liver organoids to mimic NASH progression. 澳门葡京赌博游戏的目标是确定新的靶点,以此筛选潜在的药物,并开发适合肝脏组织再生的细胞系.




Draw on our expertise across CVRM to combat NASH

NASH的负担将随着肥胖率的增加而显著增加, and contributions from metabolic disease including diabetes and insulin resistance.1 At advanced stages, patients with NASH have an increased risk of developing hepatocellular carcinoma (HCC), the most common type of liver cancer.6 nash引起的肝硬化的发病率预计在未来七年内将翻一番,7 and the disease is now the fastest growing indication for liver transplant worldwide.8

However, the treatment of NASH has implications beyond the liver, and effective treatments could also address related co-morbidities. The interconnection of CVRM diseases is well established,9-11 and any new treatment for NASH may have a positive impact on the health of other organs, such as the heart and kidneys. 澳门葡京赌博游戏为每天为改善数百万患有复杂慢性粒细胞白血病的患者的预后而采取的行动感到自豪.12


Topics:



You may also like


References:

1.    Rinella M, Cryer DR, Articolo A, Fisher T, Schneider J, Nadolsky K. Nonalcoholic steatohepatitis medical patient journey from the perspective of hepatologists, gastroenterologists and patients: a cross-sectional survey. BMC Gastroenterol. 2022 Jul 10;22(1):335. doi: 10.1186/s12876-022-02410-x. PMID: 35811319; PMCID: PMC9272554.

2.    National Institute of Diabetes and Digestive and Kidney Diseases. Symptoms & Causes of NAFLD & NASH. 2021. Available from URL: http://www.niddk.nih.gov/health-information/liver-disease/nafld-nash/symptoms-causes [Accessed 16 May 2023].

3.    National Institute of Diabetes and Digestive and Kidney Diseases. Diagnosis of NAFLD & NASH. 2021. Available from URL: http://www.niddk.nih.gov/health-information/liver-disease/nafld-nash/diagnosis [Accessed 16 May 2023].

4.    Cleveland ER, Ning H, Vos MB, Lewis CE, Rinella ME, Carr JJ, Lloyd-Jones DM, VanWagner LB. 在以人群为基础的队列样本中,对非酒精性脂肪性肝病的低认知度:CARDIA研究. J Gen Intern Med. 2019 Dec;34(12):2772-2778. doi: 10.1007/s11606-019-05340-9. Epub 2019 Oct 8. PMID: 31595464; PMCID: PMC6854130.

5.    Yoo JJ, Kim W, Kim MY, Jun DW, Kim SG, Yeon JE, Lee JW, Cho YK, Park SH, Sohn JH; the Korean Association for the Study of the Liver (KASL)-Korea Nonalcoholic fatty liver Study Group (KNSG). Recent research trends and updates on nonalcoholic fatty liver disease. Clin Mol Hepatol. 2019 Mar;25(1):1-11. doi: 10.3350/cmh.2018.0037. Epub 2018 Aug 8. PMID: 30086613; PMCID: PMC6435971.

6.    Llovet JM, Kelley RK, Villanueva A, Singal AG, Pikarsky E, Roayaie S, Lencioni R, Koike K, Zucman-Rossi J, Finn RS. Hepatocellular carcinoma. Nat Rev Dis Primers. 2021 Jan 21;7(1):6. doi: 10.1038/s41572-020-00240-3. PMID: 33479224.

7.    Younossi Z, Tacke F, Arrese M, Chander Sharma B, Mostafa I, Bugianesi E, Wai-Sun Wong V, Yilmaz Y, George J, Fan J, Vos MB. Global Perspectives on Nonalcoholic Fatty Liver Disease and Nonalcoholic Steatohepatitis. Hepatology. 2019 Jun;69(6):2672-2682. doi: 10.1002/hep.30251. PMID: 30179269.

8.    Taneja S, Roy A. Nonalcoholic steatohepatitis recurrence after liver transplant. Transl Gastroenterol Hepatol. 2020 Apr 5;5:24. doi: 10.21037/tgh.2019.10.12. PMID: 32258528; PMCID: PMC7063497.

9.    Sarnak MJ, Amann K, Bangalore S, Cavalcante JL, Charytan DM, Craig JC, Gill JS, Hlatky MA, Jardine AG, Landmesser U, Newby LK, Herzog CA, Cheung M, Wheeler DC, Winkelmayer WC, Marwick TH; Conference Participants. Chronic Kidney Disease and Coronary Artery Disease: JACC State-of-the-Art Review. J Am Coll Cardiol. 2019 Oct 8;74(14):1823-1838. doi: 10.1016/j.jacc.2019.08.1017. PMID: 31582143.

10.  Tourki B, Halade GV. 保留射血分数的心力衰竭综合征是肥胖患者非消解性炎症的代谢簇. Front Cardiovasc Med. 2021 Aug 2;8:695952. doi: 10.3389/fcvm.2021.695952. PMID: 34409075; PMCID: PMC8367012.

11.  Rangaswami J, Bhalla V, Blair JEA, Chang TI, Costa S, Lentine KL, Lerma EV, Mezue K, Molitch M, Mullens W, Ronco C, Tang WHW, McCullough PA; American Heart Association Council on the Kidney in Cardiovascular Disease and Council on Clinical Cardiology. Cardiorenal Syndrome: Classification, Pathophysiology, Diagnosis, and Treatment Strategies: A Scientific Statement From the American Heart Association. Circulation. 2019 Apr 16;139(16):e840-e878. doi: 10.1161/CIR.0000000000000664. PMID: 30852913.

12.  GBD 2019 Diseases and Injuries Collaborators. Global burden of 369 diseases and injuries in 204 countries and territories, 1990-2019: a systematic analysis for the Global Burden of Disease Study 2019. Lancet. 2020 Oct 17;396(10258):1204-1222. doi: 10.1016/S0140-6736(20)30925-9. Erratum in: Lancet. 2020 Nov 14;396(10262):1562. PMID: 33069326; PMCID: PMC7567026.


Veeva ID: Z4-55414
Date of preparation: June 2023