NORTH BETHESDA, Md.--(BUSINESS WIRE)--The Foundation for the National Institutes of Health (FNIH) today announces study findings, published in Nature Medicine, demonstrating the effectiveness of noninvasive blood tests to diagnose nonalcoholic steatohepatitis (NASH).1 The results are a significant milestone on the path to regulatory approval by the U.S.

北贝塞斯达，医学博士（商业线）-美国国立卫生研究院（FNIH）基金会今天宣布研究结果，发表在Nature Medicine，展示无创血液检查诊断非酒精性脂肪性肝炎（NASH）的有效性.1结果是美国监管审批之路上的一个重要里程碑。

Food and Drug Administration (FDA). The findings came from the FNIH’s Biomarkers Consortium, which leads cross-sector efforts to validate and qualify biomarkers that accelerate the development of new therapeutics and health technologies..

食品药品监督管理局（FDA）。研究结果来自FNIH的生物标志物联盟，该联盟领导跨部门努力验证和鉴定生物标志物，加速新疗法和健康技术的发展。。

The research is part of the Biomarkers Consortium’s Noninvasive Biomarkers of Metabolic Liver Disease (NIMBLE) project, a public-private partnership bringing together the FDA, academic researchers, and industry partners to rigorously evaluate blood and imaging tests to measure the presence of liver disease.

该研究是Biomarkers联盟代谢性肝病非侵入性生物标志物（NIMBLE）项目的一部分，该项目是一个公私合作伙伴关系，将FDA，学术研究人员和行业合作伙伴聚集在一起，严格评估血液和影像学检查以测量肝脏疾病的存在。

Although several companies have developed such tests, none have met the burden of proof needed for FDA regulatory approval. This lack of approval limits the use of these tests for diagnosing NASH and recruiting patients into studies to develop therapies..

虽然有几家公司已经开发出这样的测试，但没有一家公司承担FDA监管部门批准所需的举证责任。这种缺乏批准限制了这些测试用于诊断NASH和招募患者进入研究以开发疗法。。

“This brings us a step closer toward the day when we can diagnose and treat patients with NASH without a potentially painful liver biopsy,” said Julie Gerberding, MD, MPH, President and CEO of the Foundation for the National Institutes of Health.

美国国立卫生研究院基金会主席兼首席执行官朱丽格伯丁医学博士，公共卫生硕士说：“这使我们更接近诊断和治疗NASH患者而没有潜在痛苦的肝活检的那一天。”。

The study measured the performance of five biomarkers that could be scaled up for widespread deployment if proven effective: ELF, NIS4, PROC3, Fibrometer-VCTE, and OWLiver. Each biomarker was tested for use in diagnosing NASH and advanced liver disease. Four of the five biomarkers performed better than current blood-based lab tests, as determined by an AUROC of 0.7 or higher, a standard performance threshold for biomarkers.

该研究测量了五种生物标志物的性能，如果证明有效，可以扩大广泛部署的性能：ELF，NIS4，PROC3，Fibrometer VCTE和OWLiver。测试每种生物标志物用于诊断NASH和晚期肝病。五种生物标志物中的四种表现优于目前基于血液的实验室测试，由生物标志物的标准性能阈值0.7或更高的AUROC确定。

The 1,073 study participants, representing the full spectrum of disease severity, were selected from the NASH Clinical Research Network, which is supported by the NIH’s National Institute of Diabetes and Digestive and Kidney Diseases..

代表疾病严重程度的1073名研究参与者选自NASH临床研究网络，该网络得到了NIH国家糖尿病，消化和肾脏疾病研究所的支持。。

NASH is a common cause of liver disease and a leading cause of liver-related morbidity and mortality. It affects up to 15 million people in the United States.2 People with NASH do not display symptoms until later stages of the condition, leaving many undiagnosed until they develop cirrhosis, which may require a liver transplant.

NASH是肝病的常见原因，也是肝脏相关发病率和死亡率的主要原因。它影响美国多达1500万人.2 NASH患者直到病情晚期才出现症状，许多人直到肝硬化才被诊断出来，这可能需要肝移植。

NASH also increases the risk of liver cancer, which can be fatal. Currently, the only method to diagnose NASH with certainty is a liver biopsy, a painful and expensive procedure that carries some risk of death for patients. By expanding the diagnostic options beyond liver biopsies, more healthcare facilities and healthcare professionals would be able to diagnose NASH with greater safety..

NASH还会增加肝癌的风险，这可能是致命的。目前，确定诊断NASH的唯一方法是肝活检，这是一种痛苦而昂贵的手术，给患者带来一定的死亡风险。通过将诊断选项扩展到肝活检之外，更多的医疗机构和医疗保健专业人员将能够更安全地诊断NASH。。

The need for a biopsy also hinders patient recruitment for clinical trials. These trials are necessary to approve new treatments for NASH, none of which are currently available to treat this growing population of patients. The use of noninvasive blood tests is anticipated to spark increased interest in new drug development, as these tests promise to facilitate enrollment in future studies and reduce the costs of running a trial..

活检的需要也阻碍了患者招募临床试验。这些试验对于批准NASH的新疗法是必要的，目前没有一种可用于治疗这种不断增长的患者群体。预计使用无创血液检查会引起人们对新药开发的兴趣增加，因为这些检查有望促进未来研究的登记并降低进行试验的成本。。

“Currently, to qualify for enrollment in clinical trials of NASH, patients must undergo liver biopsy, and many patients do not qualify. The FNIH NIMBLE project is assessing diagnostic biomarker tests to identify which patients may not qualify for enrollment without subjecting them to an invasive procedure,” said Jeffrey Siegel, MD, Office Director of the Office of Drug Evaluation Sciences, FDA.

“目前，为了有资格参加NASH临床试验，患者必须接受肝活检，许多患者不符合资格.FNIH NIMBLE项目正在评估诊断性生物标志物检测，以确定哪些患者在不使他们接受侵入性治疗的情况下可能不符合入组条件，”药物评估科学办公室办公室主任Jeffrey Siegel博士说，FDA。

“The FDA supports efforts to qualify non-invasive tests to meet this important goal.”.

“FDA支持努力使非侵入性测试符合这一重要目标。”。

“These promising results advance the process of fully qualifying these biomarkers for use in clinical studies for new NASH therapies,” said Tania Kamphaus, PhD, Director of Translational Science, Metabolic Disorders, and Director of Patient Engagement at the FNIH, and a co-author of the Nature Medicine article.

“这些有希望的结果推动了将这些生物标志物完全合格用于新NASH治疗的临床研究的过程，”FNIH转化科学，代谢紊乱主任和患者参与主任Tania Kamphaus博士说，自然医学文章的合着者。

“With regulatory approval, these noninvasive tests could become a new diagnostic standard of care for patients with liver disease.”.

“经监管部门批准，这些非侵入性检测可能成为肝病患者护理的新诊断标准。”。

Working alongside dedicated patient advocacy groups in the NASH space—including the Global Liver Institute, NASH Knowledge, and the Fatty Liver Foundation—the FNIH is contributing its expertise to enhance research and drive patient-centered initiatives to address this pressing health issue. For more information, visit Non-Invasive Biomarkers of Metabolic Liver Disease (NIMBLE) | FNIH..

与NASH领域的专门患者倡导团体（包括全球肝脏研究所，NASH知识和脂肪肝基金会）合作，FNIH正在贡献其专业知识，以加强研究并推动以患者为中心的举措，以解决这一紧迫的健康问题。欲了解更多信息，请访问代谢性肝病的非侵入性生物标志物（NIMBLE）| FNIH。。

About the Biomarkers Consortium: The Foundation for the National Institutes of Health’s Biomarkers Consortium (BC) leads cross-sector efforts to validate and qualify biomarkers that accelerate the development of new therapeutics and health technologies. The core operations of the BC are supported through its contributing membership program, which includes organizations representing private industry and not-for-profit organizations..

关于生物标志物联盟：美国国立卫生研究院生物标志物联盟（BC）基金会领导跨部门努力验证和鉴定生物标志物，加速新疗法和健康技术的发展。卑诗省的核心业务通过其贡献会员计划得到支持，该计划包括代表私营企业和非营利组织的组织。。

About the Foundation for the National Institutes of Health: The Foundation for the National Institutes of Health (FNIH) builds public-private partnerships that connect leading biomedical scientists at the National Institutes of Health (NIH), life sciences companies, foundations, academia, and regulatory agencies, including the Food and Drug Administration and European Medicines Agency.

关于美国国立卫生研究院基金会：美国国立卫生研究院基金会（FNIH）建立公私合作伙伴关系，将美国国立卫生研究院（NIH），生命科学公司，基金会，学术界的领先生物医学科学家联系起来。和监管机构，包括食品药品管理局和欧洲药品管理局。

Through team science, we solve complex health challenges and accelerate breakthroughs for patients, regardless of who they are or what health challenges they face. The FNIH accelerates new therapies, diagnostics, and potential cures; advances global health and equity in care; and celebrates and helps train the next generations of scientists.

通过团队科学，我们解决复杂的健康挑战，加速患者的突破，无论他们是谁，或者他们面临哪些健康挑战。FNIH加速新疗法，诊断和潜在治疗；推进全球卫生和公平护理；并庆祝并帮助培训下一代科学家。

Established by Congress in 1990 to support the mission of the NIH, the FNIH is a not-for-profit 501(c)(3) charitable organization. For more information about the FNIH, please visit fnih.org..

FNIH由国会于1990年成立，旨在支持NIH的使命，它是一个非营利性501（c）（3）慈善组织。有关FNIH的更多信息，请访问FNIH.org。。

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1 In June 2023, numerous global liver associations introduced a revised terminology. The condition known as nonalcoholic steatohepatitis (NASH) is now known as metabolic dysfunction-associated steatohepatitis (MASH). FNIH acknowledges this advancement and actively collaborates with the patient and expert liver disease sectors to appropriately integrate the change..

1 2023年6月，许多全球肝脏协会引入了修订后的术语。称为非酒精性脂肪性肝炎（NASH）的病症现在称为代谢功能障碍相关性脂肪性肝炎（MASH）。FNIH承认这一进步，并积极与患者和肝脏疾病专家部门合作，适当整合变革。。

2 Fatty Liver Disease – American Liver Foundation

2脂肪肝-美国肝脏基金会