Precision medicine
What is precision medicine?
Precision medicine is an innovative approach for tailoring disease treatment and prevention. 这使得医生和研究人员能够更准确地预测哪种治疗方法对病人更有效, taking into account individual genetic and molecular make-up, environment, and lifestyle.
By unravelling the complex underlying biology of many diseases, 开拓和应用先进技术, we are leading the way in the application of precision medicine. Our work, 以及澳门葡京赌博游戏与整个行业的合作伙伴的合作, biotech and academia, are driving for better treatments for patients as well as a more sustainable future for healthcare systems.
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通过澳门葡京赌博游戏的投资组合塑造医学的未来
澳门葡京赌博游戏在整个R应用精准医疗方法&D组合加深了澳门葡京赌博游戏对疾病生物学的理解, developing novel therapies and supporting the development of innovative diagnostic tests to improve clinical trial success and help target the right medicines to the right people across oncology, chronic and rare diseases.
Precision medicine in oncology
澳门葡京赌博游戏正在推动肿瘤科学的发展, aiming to provide precision medicines matched to the patients who can benefit most from them. 澳门葡京赌博游戏致力于澳门葡京赌博游戏富有远见的管道, focused on the development of precision medicine advances across multiple hard to treat tumour types that can help shape the cancer environment by changing both clinical and medical practice.
澳门葡京赌博游戏在六个科学平台上的进步帮助改善了患者的体验，同时在表观遗传学和跨细胞方面取得了进展, gene and combination therapy hold the promise of new possibilities.
慢性病的精准医疗
慢性疾病，如哮喘, 慢性阻塞性肺病(COPD), heart failure, and chronic kidney disease (CKD) – affect billions of people worldwide.
它们是复杂和异构的, 这意味着它们可以以多种方式表现出来, 哪些因素会给准确诊断带来挑战. 这种复杂性反映了广泛的潜在生物学原因，每个原因都需要不同的治疗方法. It is for these reasons that COPD and CKD have seen little innovation in decades and metabolic dysfunction-associated steatohepatitis (MASH) still has no approved treatment.
Using a precision medicine approach could help to detect disease earlier and identify the causes of disease, which has the potential to inform and improve treatment decisions.
罕见疾病的精准医疗
While a single rare disease may affect a small number of people, collectively the more than 7,000 known rare diseases affect an estimated 400 million people globally.
Studying rare diseases poses unique challenges as patient populations are often small and dispersed. The underlying biology of many of these conditions is not well understood and few diagnostic tools exist, leading to a lack of awareness among scientific and medical communities. As a result, the majority of rare conditions still do not have an approved treatment.
Precision medicine is creating opportunities to understand the fundamental biology of rare diseases, 确定新的分子靶点，开辟新的研究途径，有可能加快发现新的治疗方案的步伐.
Our scientists are working hard to deliver the benefits of precision medicine. Using multi-omics, novel technologies, imaging, artificial intelligence, and machine learning they are digging deep into the biological processes that cause and drive disease. Our ambition is to develop and validate new treatments that make it possible to diagnose and intervene earlier, halt disease progression, achieve remission, and enable patients to have better outcomes and healthier futures across the world.
澳门葡京赌博游戏的精准医疗方法
Our pioneering research into precision medicine harnesses huge networks of scientific and patient data to uncover new knowledge and important disease insights that will enable us to:
- Identify novel drug targets that are expected to have a higher probability of success.
- Identify biomarkers that can help characterise patients into subgroups most likely to benefit from treatment.
- Support the design of better clinical trials, where the right patients are recruited to participate.
- Develop diagnostic tests that can help guide treatment in the real world.
We’re applying a precision medicine approach across 90% of our R&D portfolio. 澳门葡京赌博游戏把病人放在医学研究的中心, confident that by matching the right treatment to the right patient at the right time, 在病情恶化之前进行干预, we can change the course of disease and allow patients to live better, healthier lives.
Asthma
Asthma
In respiratory diseases, like asthma, there is intense interest in precisely targeting molecules that stimulate key inflammatory pathways that could lead to the development of novel precision medicines in the future.
Asthma can be a devastating disease with 176 million attacks each year and debilitating symptoms, 尤其是那些患有更严重疾病的人.1
By targeting the inflammatory drivers that play an important pathological role in asthma we can break whole patient populations into subgroups depending on their individual disease biomarkers.
One example is our work to identify specific leukotrienes that drive inflammation, 疾病中支气管收缩和粘液的产生. Our precision medicine research is exploring these molecules as urinary biomarkers to help classify patients’ disease and match them with targeted treatments they are more likely to respond to.
Chronic kidney disease
Chronic kidney disease (CKD)
Chronic Kidney Disease (CKD) encompasses various primary disorders and stages of progression, 患者群体是高度异质性的. The current symptom-based approach ignores the different underlying mechanisms. Our aim is to close this gap, 通过发现疾病的潜在遗传和分子驱动因素，为合适的病人找到合适的治疗方法.
Using unique datasets, 该团队应用机器学习和人工智能算法将患者分为亚类. For the first time, 澳门葡京赌博游戏的研究表明，这些基于分子数据的疾病分类与以往CKD的临床分类不同. 澳门葡京赌博游戏现在正在寻找尿液生物标记物，可以用来无创地识别患者的分子疾病类别, allowing us greater precision when aligning the right patients to the right trials. In the future, our ambition is to provide tailored treatments based on individual disease, or patient, 由分子疾病驱动因素决定的类别.
慢性阻塞性肺疾病
慢性阻塞性肺病(COPD)
在慢性阻塞性肺疾病(COPD)方面，尽管它是全球第三大死亡原因，但几十年来澳门葡京赌博游戏几乎没有看到什么创新.2 To address this, we are dissecting the 500+ abnormally expressed genes and defining their role in disease. This precision medicine approach will help us identify potential novel targets for life-changing medicines.
通过靶向COPD的疾病驱动因素, such as oxidative stress, cell senescence, 慢性炎症和纤维化, 澳门葡京赌博游戏的目标是开发专注于疾病改变的精准药物，以减缓和阻止疾病的进展. 为这些新疗法找到合适的病人, we need to understand more about COPD in different people and find new ways to assess the effects the disease. Access to data from cohorts of people with COPD is providing new insights into the molecular mechanisms of COPD and has helped us to develop non-invasive imaging methods that could be used in future to help stratify patients or monitor treatment response. Incorporation of precision medicine approaches like this early in the clinical development pipeline will support the identification of the right patients for subsequent clinical trials.
Heart failure
Heart failure
In heart failure (HF), 患者可能有多种合并症, any of which may affect their outcome and response to heart failure therapies. To deliver our precision medicine approach we need to understand the molecular and genetic landscape, including the impact of comorbidities such as chronic kidney disease or obesity.
Targeting key mechanisms of HF including widespread inflammation, fibrosis, hypertrophy and microvascular dysfunction is a major priority.
许多心衰病例都有遗传成分，通过鉴定基因组中的变异，澳门葡京赌博游戏可以获得新的遗传见解. Among the genetic drivers in dilated cardiomyopathy is a mutation in the gene for phospholamban, which is linked to impaired heart muscle contraction and relaxation. 这种理解使得探索这种基因变异以开发潜在的新治疗靶点成为可能.
代谢紊乱相关的脂肪性肝炎
代谢功能障碍相关的脂肪性肝炎
Our precision medicine approach in metabolic-disorder associated steatohepatitis (MASH) is two-fold; firstly, we aim to identify the right patients with the presence of disease-causing gene variants, then we selectively disrupt this protein expression with novel antisense oligonucleotide (ASO) therapy.
MASH is a multi-component disease with high unmet needs and severe patient outcomes. 处于精准医学研究的前沿, 澳门葡京赌博游戏的目标是与MASH相关的基因突变, which are responsible for an approximately four-fold increase in risk of the disease.
For example, a single nucleotide substitution in the PNPLA3 gene severely impairs normal fat breakdown in liver cells. We are investigating ways to downregulate PNPLA3 and so potentially restore lipid metabolism.
Systemic lupus erythematosus
系统性红斑狼疮(SLE)
澳门葡京赌博游戏的研究正在开启免疫系统科学，以解决系统性红斑狼疮(SLE)的重大未满足需求。. This complex disease is driven by multiple cell types and mediators, and we are exploring these with our precision medicine approach.
SLE是一种慢性免疫驱动疾病，机体免疫系统攻击身体任何部位的健康组织.
澳门葡京赌博游戏的精准医学方法的前沿是干扰素(IFN)途径，它在SLE中起着核心炎症作用. Approximately three quarters of patients with SLE have an elevated IFN gene signature and clinical trials have shown that over expression correlates with a positive treatment response.
利用这种精确的指导研究可以将这种方法扩展到其他条件下, even though symptoms may differ, 潜在的1型IFN基因标记是保守的.
应用最先进的技术
澳门葡京赌博游戏的技术驱动方法正在加速澳门葡京赌博游戏设计和开发新的精准药物和诊断测试的方式, 推动科学发现的新时代.
Understanding the genome: 患者数据库通过帮助将基因概况和基因突变与特定的健康结果相匹配，正在推进澳门葡京赌博游戏的知识. Ongoing advances in genome technology allow us to characterise patients into subgroups based upon their underlying disease mechanisms and identify the most appropriate genetic targets. 澳门葡京赌博游戏还对……进行了重大投资 multi-omic technologies (genomics, transcriptomics, proteomics, metabolomics, lipidomics) that are key to building a more complete picture of the complexities of disease, 哪些可以为新的测试和治疗提供信息.
Finding novel targets: We are combining our rich datasets with external sources of patient data and applying AI and machine learning to discover associations between data and disease, achieving healthcare breakthroughs more rapidly than ever before. For example, we are partnering with Benevolent AI 创建知识图谱，使澳门葡京赌博游戏能够分析大量的科学数据，看到基因目标之间潜在的相互作用, expression and disease.
Revolutionising imaging: 利用先进的成像技术，澳门葡京赌博游戏现在可以在分子水平上捕捉细胞之间的相互作用，这些相互作用可以定义一种疾病, 以及监测基于组织生物标志物对药物反应的调节的治疗效果.
We are taking these advances in imaging technology into our clinical trials, redefining endpoints to demonstrate disease modification with targeted treatment.
加速设计新的诊断测试; Finding the right patients depends on reliable diagnostic tests. 理想情况下，这些应该能够检测早期疾病和微创，以便在症状出现之前用于筛查患者.
We are collaborating with external partners to identify biomarkers and develop them into non-invasive tests, which could be validated for use in helping to diagnose patients.
为患者争取更美好、更健康的未来
Across the globe, 卫生保健系统正在寻求新的策略来管理越来越多的复杂疾病合并症患者的影响. 精准医疗为患者提供了明显的优势, healthcare systems and those involved in the provision of care – through earlier diagnosis, 避免不必要的治疗, 疾病过程的改善和更好的结果.
精准医疗的潜力
Patients
Reduced trial and error
Physicians
患者受益于改善的结果
Payer
只支付正确的治疗费用
Regulators
Efficacy and safety
Science
了解疾病，量身定制治疗
通过使治疗能够针对正确的患者, 精准医疗代表了一种新的医疗模式, 从目前往往依靠“试错”来找到最佳治疗方法的无针对性方法发展而来. 因为不太可能有效的治疗方法被避免了, there is less drug wastage, fewer side effects and total healthcare utilisation including length of hospital stays is reduced, providing savings to already stretched healthcare systems and building more sustainable care for everyone.
Precision Medicine FAQs
精准医疗是如何运作的?
精准医疗是一种在正确的时间为正确的病人配上正确的药物的方法. Typically, 这包括使用诊断测试，可以根据疾病的生物学特征来识别不同的患者群体. Right now, 病人通常根据他们的症状进行治疗, 这可能不能反映出他们患病的原因. In many cases this results in poor symptom control and does nothing to slow, 阻止或逆转疾病进展.
For example, 针对哮喘患者的标准药物旨在防止病情恶化和控制症状，但哮喘可能有许多不同的原因. 精准医学旨在提供诊断测试，帮助医生更多地了解每位患者哮喘的潜在原因. As a result of the tests, different groups of patients can then be given treatments that target the underlying causes of illness.
精准医疗的好处是什么?
Precision medicine can help more people to gain greater control over their illness and better quality of life faster by providing more accurate disease diagnosis and using treatments that target the causes of disease.
The development of specific diagnostic tests can help doctors to understand the causes of illness for each patient and this should allow more patients to be given treatments specifically designed to target the biological processes that are making them ill.
As a result, precision medicine reduces the guess work involved in diagnosing patients and means that more patients can be given highly effective treatments that won’t just control their symptoms but that could help to slow, 阻止甚至逆转他们的疾病.
为什么精准医疗很重要?
Precision medicine has the potential to transform healthcare. Since precision medicines target the underlying biology of disease there is potential to develop more effective treatments and to treat people who currently have no options. By incorporating diagnostics and specifically targeting the causes of disease, precision medicines also have the potential to detect more diseases earlier and to stop, or even reverse disease progression meaning that more people can get treatment sooner, 经历更少的症状，甚至可能在疾病严重影响他们的生活之前被治愈. 这对患者有好处，但也将改善临床医生和付款人的医疗保健，并具有全球可持续性效益.
什么时候使用精准医疗?
There are many diseases where patients have typically all been treated the same even though their illness can have many different causes and can affect their health in different ways. For any of these diseases a precision medicine approach could be beneficial. 哪里有现有的治疗方法, precision medicine may offer more effective alternatives by targeting the underlying biology of disease.
Precision medicine could also help people who currently have no treatment options. A precision approach would focus on a more specific subpopulation of patients so could result in effective treatments for specific groups where it was impossible to find a single treatment that was effective for everyone.
精准医疗从何而来?
Precision medicine is the result of our deeper scientific understanding of the causes of illness. Supported by new technologies, advances such as the sequencing of the human genome have shown us that the causes of chronic diseases are complex and that the same illness can have many different causes, 每种都需要不同的处理方法. 这些更深刻的见解也使澳门葡京赌博游戏有可能开始开发专门针对疾病原因的药物.
What is the difference between precision medicine and personalised medicine?
Although the terms are sometimes used interchangeably, many experts use them differently. We use the term precision medicine to describe an approach to treatment that involves matching specific groups of patients to the most appropriate treatments based on scientific insights into the underlying causes of their illness.
为什么澳门葡京赌博游戏需要精准药物?
采用精准医疗方法有很多好处, most importantly it offers the potential for a better quality of life for patients and it has the potential to reduce pressure of global healthcare systems.
Currently, people diagnosed with chronic diseases generally have to live with their effects for the rest of their lives. 通过瞄准疾病的起因, precision medicine has the potential to control the symptoms of disease and, in some cases, could even provide a cure.
Better disease control for patients also means less demand for healthcare services, which are being put under growing pressure due to factors such as increasing population, ageing, 肥胖和COVID-19大流行的后遗症. 连同其他创新, 精准医疗可以减少对广泛医疗的需求, long-term healthcare support, 这意味着更少的预约和更少的住院时间.
我现在能得到精准药物吗?
Precision medicines are already available for the treatment of some cancers and rare genetic diseases. Currently, precision medicines for chronic diseases are also being researched. Across all areas, precision medicine approaches are being explored for more than 90% of our R&D pipeline.
为什么澳门葡京赌博游戏还没有更精准的药物呢?
To develop precision medicines we need an in-depth understanding of the biological changes that cause diseases. This requires extensive scientific insights and advanced technologies, 直到最近才有. For example, genetic sequencing and genome editing have helped to identify which genes are involved in different diseases and this is making it possible to develop precision medicines targeting these genes.
精准医疗只针对癌症吗?
No. 许多最早的精准药物是针对癌症开发的，但同样的方法也可以应用于许多其他疾病. Generally, precision medicine is relevant for any disease that can have multiple different causes that all result in a similar set of symptoms. We are currently investigating precision medicine for a wide range of chronic diseases including asthma, 慢性阻塞性肺病，糖尿病以及心血管疾病, renal, 肝脏和免疫疾病.
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References
1. AstraZeneca Pharmaceuticals. Data on file. Budesonide/formoterol: Annual Rate of Exacerbations Globally (ID:SD-3010-ALL-0017).
2. World Health Organisation. 慢性阻塞性肺病(COPD). Available at: http://www.who.int/news-room/fact-sheets/detail/chronic-obstructive-pulmonary-disease-(copd)
3. 雷内拉ME，拉撒路JV，拉齐乌V et al. NAFLD命名共识组. A multi-society Delphi consensus statement on new fatty liver disease nomenclature. Hepatology. 2023 Jun 24. doi: 10.1097/HEP.0000000000000520.
Veeva ID: Z4-59521
筹备日期:2023年11月