Bemdaneprocel

Bayer is successfully executing on an ambitious growth strategy Five pivotal approvals worldwide in 2025 underscore a landmark year of strategic execution Bayer will drive significant global regulatory expansions and market penetration across its pharmaceutical portfolio, with multiple high-impact launches across oncology, cardiology, and women’s health Focus continues to accelerate a modality-rich pipeline across oncology, cardiology, neurology and immunology, complemented by existing high-value commercial products SAN FRANCISCO--(BUSINESS WIRE)--On the occasion of the 44th J.P. Morgan Healthcare Conference in San Francisco, Bayer AG today unveiled the strategic focus for its Pharmaceuticals Division in 2026. Capitalizing on new launch momentum, the company will drive significant global regulatory expansions and market penetration across its pharmaceutical portfolio. High-value commercial products and an accelerating modality-rich pipeline across oncology, cardiology, neurology, and immunology have firmly cemented the Pharmaceuticals Division’s growth for the coming years. Five pivotal worldwide approvals in 2025 underscore a landmark year of strategic execution, validating the successful advancement of Bayer’s ambitious pharmaceutical growth strategy. “We are now clearly seeing the success of our strategy. We have fundamentally accelerated Bayer Pharmaceutical’s growth runway with multiple high-impact launches across oncology, cardiology, and women’s health,” said Stefan Oelrich, Member of the Board of Management, Bayer AG, and President of Bayer’s Pharmaceuticals Division. “Our strategic focus remains on delivering transformative medicines to patients faster, powered by our cutting-edge R&D expertise, dynamic operating model, partnership-driven innovation, AI-enabled development, and commitment to commercial excellence. Our Pharmaceuticals Division is poised for sustainable growth over the coming years.” Innovating to capture additional market opportunity in cardiovascular and cerebrovascular medicine Bayer is advancing its global leadership in cardiovascular disease management through world-class science, strategic partnerships, and a high-value pipeline designed to deliver both patient and shareholder impact. Asundexian, an investigational, once daily, oral Factor XIa inhibitor, has been investigated as a potential treatment for secondary stroke prevention. Each year, approximately 12 million people worldwide will experience a stroke. Of these, 20-30% will be a recurrent stroke. 1,2 Despite available secondary stroke prevention options, the risk of secondary stroke remains high; one in five stroke survivors will have another stroke within five years. 3 Stroke is the second leading cause of death globally, and recurrent ischemic strokes tend to be more disabling and carry a higher mortality risk than the first stroke. 2,4,5 Topline data from the Phase III OCEANIC-STROKE study showed it met its primary safety and efficacy endpoints. OCEANIC-STROKE is the first successfully completed Phase III study of a Factor XIa inhibitor and these results underscore the blockbuster potential of asundexian in secondary stroke prevention. Asundexian has been granted Fast Track Designation by the U.S. Food and Drug Administration (FDA) as a potential treatment for stroke prevention in patients after a non-cardioembolic ischemic stroke. Additionally, Bayer is driving forward its global leadership in cardiovascular and renal disease, leveraging integrated capabilities and robust partnerships to unlock new value streams. The FDA and Japan’s Ministry of Health, Labour and Welfare (MHLW), among other regulatory authorities, have approved finerenone (marketed as Kerendia ® ) for patients with heart failure with left ventricular ejection fraction (LVEF) of ≥40%, based on the positive results from the pivotal Phase III study FINEARTS-HF, further building on its established efficacy in chronic kidney disease (CKD) associated with type 2 diabetes. Applications in HF with LVEF ≥40% are under review in additional markets, including the EU and China. Ongoing, dedicated Phase III heart failure and chronic kidney disease programs (MOONRAKER and THUNDERBALL) continue to evaluate Kerendia’s potential across a broad spectrum of patients and clinical settings of Cardiovascular-Kidney-Metabolic (CKM) conditions. In November 2025, late-breaking, topline data from the Phase III study FINE-ONE presented at the American Society of Nephrology’s Kidney Week showed it met its primary safety and efficacy endpoints. For important risk and use information about KERENDIA, please see the full Prescribing Information . The European Commission granted marketing authorization for Beyonttra™ (acoramidis), marketed by Bridge Bio in the U.S., to treat wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). Beyonttra slows the amyloidogenic process that results in ATTR-CM. Beyond current successes, Bayer is actively shaping the future of cardiology with a robust and diversified pipeline, strategically positioned to address critical unmet needs and drive significant long-term value. This includes AB-1002, an investigational single-dose gene therapy in development for congestive heart failure (CHF) developed together with AskBio Inc., a wholly owned subsidiary of Bayer. AB-1002 is progressing well in Phase II. Its Phase I results were published in Nature Medicine and it has been granted Pioneering Regenerative Medical Product designation (SAKIGAKE) by Japan’s MHLW. Additionally, Bayer has initiated the Phase II SIRIUS study of BAY-3018250, a first-in-class anti–alpha-2 antiplasmin (α2AP) antibody designed to enable targeted thrombolysis in deep vein thrombosis (DVT), and a Phase I clinical study of BAY-3670549, an investigational, highly selective G-protein-coupled inwardly rectifying potassium channel 4 (GIRK4) inhibitor, with the potential to help control the electrical activity of heart cells in patients with atrial fibrillation. Bayer’s commitment to cutting-edge science is further underscored by the company’s licensed dilated cardiomyopathy program with Dewpoint Therapeutics, which leverages condensate biology to develop genotype-guided therapies for dilated cardiomyopathy patients with specific genetic mutations. Concurrently, securing exclusive rights in Japan to aficamten from Cytokinetics—a cardiac myosin inhibitor in development for hypertrophic cardiomyopathy—strengthens Bayer’s precision cardiology footprint and expands its strategic market access. Oncology growth engine: Leveraging blockbuster assets and precision pipeline for future leading position Bayer’s oncology portfolio is delivering significant patient benefit and commercial success, with blockbuster Nubeqa ® (darolutamide) continuing its exceptional global uptake, progressing towards a market-leading position. With over 200,000 patients treated worldwide and a third approval in prostate cancer in both the U.S. and Europe, Nubeqa continues its strong growth trajectory. Total global sales are on the rise in all markets. Third approval in China is anticipated in 2026. Its comprehensive clinical development program is exploring Nubeqa’s potential in earlier prostate cancer settings, with two additional Phase III readouts expected in 2027 and 2028. Darolutamide, under the brand name Nubeqa is approved for the treatment of patients with metastatic castration-sensitive prostate cancer (mCSPC) in combination with ADT, with or without chemotherapy, and with ADT alone in non-metastatic castration-resistant prostate cancer (nmCRPC) in patients who are at high risk of developing metastatic disease. For important risk and use information about Nubeqa, please see the full Prescribing Information . Adding to this momentum in oncology, the FDA granted accelerated approval for Bayer’s Hyrnuo ® (sevabertinib) for adult patients with locally advanced or metastatic non-squamous HER2 -mutant non-small cell lung cancer (NSCLC), who have received prior systemic therapy. Approval followed Breakthrough Therapy Designations from both the FDA and China’s CDE and was based on results from the SOHO-01 study. Bayer received Breakthrough Therapy Designation in the US and China for sevabertinib as a first-line treatment for patients with HER2 -mutant non-small cell lung cancer. The Breakthrough Therapy Designations are supported by preliminary clinical evidence from cohort F (patients who had not previously received treatment) of the ongoing Phase I/II SOHO-01 study. Sevabertinib addresses unmet needs for a patient population with limited treatment options. In addition to SOHO-01 the clinical program for sevabertinib includes ongoing trials evaluating its effectiveness as a first-line treatment for HER 2-mutant NSCLC and in patients with metastatic solid tumors harboring HER 2 activating mutations, excluding advanced NSCLC. For important risk and use information about Hyrnuo, please see the full Prescribing Information. As pioneers in Targeted Radionuclide Therapy (TRT), Bayer was instrumental in helping establish Targeted Alpha Therapies (TATs) as a standard of care for patients with metastatic castration resistant prostate cancer (mCRPC) with bone metastases. The EORTC PEACE III study evaluating radium-223 dichloride (marketed as Xofigo ® ) in combination with enzalutamide met its primary endpoint. An additional Phase III clinical study with radium-223 dichloride will be completed in 2026 and will guide decisions regarding future regulatory steps. Radium-223 dichloride is approved under the brand name Xofigo in over 50 countries for mCRPC patients with symptomatic bone metastases and no known visceral metastatic disease. For important risk and use information about Xofigo, please see the full Prescribing Information . Bayer’s oncology pipeline is advancing precision-driven therapies, making pivotal progress by targeting challenging tumor types and previously undruggable mechanisms, proteins or pathways resistant to current therapies. This includes multiple investigational agents for KRAS-mutant cancers (an internal SOS1 inhibitor, and a partnership with Kumquat for a KRAS G12D inhibitor), next-generation TATs (225Ac-GPC3 for advanced hepatocellular carcinoma), and a PRMT5 inhibitor (from Suzhou Puhe BioPharma) for MTAP-deleted tumors, all of which have entered Phase I. Further strengthening its discovery engine, Bayer secured worldwide rights to the clinical-stage Werner helicase inhibitor (VVD 214) via Vividion, whose R&D capabilities were expanded through new facilities and the acquisition of Tavros Therapeutics. These strategic investments underscore Bayer’s commitment to driving groundbreaking innovation and securing long-term growth in oncology. Menopause management: advancing care for women with diverse needs Bayer achieved a big step forward in women's health with the recent regulatory approvals of Lynkuet ® (elinzanetant) for the treatment of moderate to severe vasomotor symptoms (VMS) due to menopause. It is the only hormone-free therapy approved in the EU for both moderate to severe VMS associated with menopause or caused by adjuvant endocrine therapy for breast cancer. These milestones follow consistent positive results across all four Phase III clinical studies (OASIS 1- 4). For important risk and use information about Lynkuet, please see the full Prescribing Information . Developing a comprehensive cell and gene therapy (CGT) portfolio Multiple regulatory priority designations granted by authorities in the U.S., Europe and Japan and across Bayer’s CGT portfolio reflect the potential of these programs to deliver transformative patient impact. Bemdaneprocel, an investigational cell therapy designed to replace the dopamine producing neurons that are lost in Parkinson’s disease, developed with Bayer’s wholly owned subsidiary BlueRock Therapeutics, entered pivotal evaluation in a Phase III study. AskBio’s ametefgene parvec (AB-1005), an investigational gene therapy, is being assessed in a Phase II study in moderate stage Parkinson’s disease. The first participant has been randomized in Germany while recruitments in the U.S., UK and Poland are ongoing. In ophthalmology, BlueRock’s OpCT-001, an iPSC-derived photoreceptor cell therapy for primary photoreceptor diseases, received FDA Fast Track designation and is progressing in its Phase I/IIa study. Radiology: Advancing innovation and expanding into Molecular Imaging Bayer continues to advance innovation in medical imaging with gadoquatrane, its investigational low-dose magnetic resonance imagining (MRI) contrast designed to reduce the gadolinium dose by up to 60% compared to other macrocyclic MRI contrast agents. Building on positive Phase III results, Bayer has submitted marketing authorization applications in key markets worldwide, including the U.S., EU, Japan, and China. Complementing progress in its existing portfolio, Bayer announced the strategic acquisition of two investigational radiotracers – AT-01 (a Positron Emission Tomography or PET tracer in Phase III of clinical development) and AT-05 (a tracer for Single Photon Emission Computed Tomography, or SPECT, currently in Phase I) – for the diagnosis of cardiac amyloidosis. This step marks Bayer’s entry into diagnostic tracers and underscores its ambition to expand in molecular imaging, aiming to broaden diagnostic options and enhance patient outcomes. About Bayer Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, “Health for all, Hunger for none,” the company’s products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2024, the Group employed around 93,000 people and had sales of 46.6 billion euros. R&D expenses before special items amounted to 6.2 billion euros. For more information, go to . Find more information at Follow us on Facebook: Forward-Looking Statements This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer’s public reports which are available on the Bayer website at . The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments. 1 Feigin VL, et al. World Stroke Organization (WSO): global stroke fact sheet 2022. International Journal of Stroke. 2022 Jan;17(1):18-29. 2 Feigin VL, et al. Pragmatic Solutions to Reduce Global Burden of Stroke. Lancet Neurol. 2023;22:1160–1206. 3 Kolmos M, et al. Recurrent ischemic stroke–a systematic review and meta-analysis. Journal of Stroke and Cerebrovascular Diseases. 2021 Aug 1;30(8):105935. 4 Rochmah TN, et al. Economic burden of stroke disease: a systematic review. International journal of environmental research and public health. 2021 Jul 15;18(14):7552. 5 Skajaa N, et al. Risks of stroke recurrence and mortality after first and recurrent strokes in Denmark: a nationwide registry study. Neurology. 2022 Jan 25;98(4):e329-42. Contacts Contact for media inquiries : Elaine Colón Phone +1-732-236-1587 Email: elaine.colon@bayer.com Contact for investor inquiries: Bayer Investor Relations Team, phone +49 214 30-72704 Email: ir@bayer.com

1. 引言：帕金森病概述与研究背景 帕金森病（Parkinson's Disease, PD）是全球第二大神经退行性疾病，我国患病人数预计2050年将达1050万，居全球首位。其核心临床表现为静止性震颤、运动迟缓等运动症状，及嗅觉减退、认知障碍等非运动症状，随病情进展导致患者丧失自理能力，给家庭与社会带来沉重负担。 传统治疗依赖左旋多巴等药物控制症状，但长期使用易引发运动并发症且无法阻断疾病进展。近十年，在遗传因素、神经炎症、蛋白质错误折叠等领域的研究突破，为开发新型疗法提供了契机。2025年我国科学家在《Science》发现PD全新治疗靶点FAM171A2，成为该领域里程碑事件。本报告系统梳理2015—2025年PD研究核心进展，为多领域相关人员提供参考。2.帕金森病病理机制研究进展2.1 遗传因素研究突破 迄今已发现17个家族性PD相关单基因（Park1~18），其中12个致病基因被克隆，LRRK2、GBA、SNCA为核心基因。LRRK2是最常见常染色体显性遗传致病基因，2025年研究确认其p.L1795F变异通过增强激酶活性致病，G2019S突变则增加患者认知障碍风险2-3倍。 GBA基因突变是重要风险因素，其p.N409S变异在PD患者中携带率达10%—15%，通过损害溶酶体功能导致α-突触核蛋白清除障碍。SNCA基因编码的α-突触核蛋白是路易小体核心成分，其突变或多拷贝引发早发性PD，2025年FAM171A2受体研究揭示其介导病理性α-突触核蛋白细胞内摄取的新机制。 全基因组关联研究（GWAS）成果显著，2024年多祖先荟萃分析确定78个全基因组显著性位点，我国研究发现rs61204179等新风险位点，多基因风险评分（PRS）最高五分位数个体发病风险是最低者的2.56倍，为精准风险评估奠定基础。2.2 神经炎症机制核心发现 神经炎症是PD发病关键环节，活化的小胶质细胞与星形胶质细胞释放TNF-α、IL-1β等促炎因子，形成神经元损伤的恶性循环。小胶质细胞兼具促炎与神经保护双重作用，其cGAS-STING通路过度激活会引发I型干扰素介导的炎症级联反应。 α-突触核蛋白与小胶质细胞的相互作用是研究热点：其聚集形式可激活NLRP3炎症小体，同时损害小胶质细胞吞噬功能与自噬通路，形成病理蛋白积累的正反馈。2024年研究证实CXCR4过表达通过刺激小胶质细胞促炎因子释放，触发多巴胺能神经元凋亡，为抗感染治疗提供新靶点。 神经炎症与α-突触核蛋白传播密切相关，病理蛋白可通过外泌体、隧道纳米管等途径扩散，小胶质细胞在此过程中既是受害者也是传播载体，这一发现为阻断病理进展提供了新思路。2.3 蛋白质错误折叠机制新认知 α-突触核蛋白错误折叠与聚集是PD核心病理特征。生理状态下其为无序蛋白，参与突触囊泡循环；病理状态下转变为富含β－折叠的异常结构，经寡聚体、原纤维阶段形成路易小体，其中寡聚体具有强神经毒性。 2025年复旦大学郁金泰团队的突破性研究发现，神经元膜受体FAM171A2可选择性结合病理性α-突触核蛋白并介导其内化，诱导细胞内单体蛋白错误折叠，这一机制被《Science》审稿人称为领域“圣杯”。该蛋白的发现为开发阻断病理传播药物提供了精准靶点。 α-突触核蛋白与线粒体功能障碍密切相关，其可通过VDAC转运至线粒体内膜，抑制离子交换与能量代谢，诱发活性氧（ROS）过量产生。液－液相分离（LLPS）是近年发现的新聚集机制，不同剪接异构体通过该途径组装成淀粉样聚集体，成为聚集早期干预的关键靶点。 蛋白质质量控制系统障碍加剧病理进程：GBA基因突变导致溶酶体功能受损，泛素-蛋白酶体与自噬－溶酶体系统异常共同促进α-突触核蛋白积累。此外，α-突触核蛋白构象多样性决定其毒性差异，Mini-P纤维因结构特性具有更强的神经元播种活性。2.4 其他关键病理机制 线粒体功能障碍是PD重要病理基础，PINK1和Parkin基因突变直接证实线粒体质量控制的关键作用。α-突触核蛋白病理可导致多巴胺能神经元线粒体基因表达下调、ATP生成障碍，激活凋亡通路。 氧化应激与铁代谢异常形成协同损伤：多巴胺氧化产生大量自由基，而PD患者抗氧化系统功能下降；黑质铁沉积增加可通过催化自由基生成加剧氧化损伤，7T MRI已能清晰显示黑质铁沉积变化。 兴奋性毒性与血脑屏障障碍也参与疾病进展：谷氨酸释放增加过度激活NMDA受体引发钙超载；长期左旋多巴代谢产生的醌类毒素可诱导小胶质细胞释放炎性因子，破坏血脑屏障导致外周免疫细胞浸润。自噬－溶酶体系统功能异常则影响病理蛋白清除与线粒体质量控制，成为疾病进展的核心环节。3. 帕金森病治疗方法发展3.1 传统药物治疗优化进展 传统药物仍是PD治疗基石，近年在剂型创新、给药优化与联合策略方面取得显著进展。《中国帕金森病治疗指南（第四版）》明确“不刻意推迟左旋多巴使用”理念，强调早期干预的重要性。 左旋多巴制剂呈现多剂型发展：标准片起效快适用于症状波动者，缓释片延长作用时间改善剂末现象，口腔速溶片便于吞咽困难患者，肠道凝胶通过持续泵入适用于晚期患者。2024年美国FDA对24小时皮下输注的ND0612进行重新审查，预计2025年底完成审批。 多巴胺受体激动剂以非麦角类为主，2024年7月注射用罗替高汀微球在我国获批，每周注射一次显著减少用药频率与浓度波动；透皮贴片则提升了吞咽困难或认知障碍患者的依从性。MAO-B抑制剂方面，沙芬酰胺作为双重作用药物，兼具多巴胺能与抗谷氨酸作用，2025年5月已在我国正式临床应用。 COMT抑制剂领域，奥匹卡朋于2024年11月在我国提交上市申请，与左旋多巴联用可显著延长“开”期时间。针对左旋多巴诱导的异动症（LID），金刚烷胺缓释制剂与伊曲茶碱（腺苷A2A受体拮抗剂）成为有效治疗选择。联合用药策略日益精细化，指南推荐根据患者年龄、症状特点制定个性化方案。3.2 基因治疗临床试验进展 基因治疗通过AAV载体等工具递送治疗性基因，成为PD治疗前沿方向。AskBio公司的AB-1005（AAV2-GDNF）获FDA再生医学先进疗法认定，2025年2月启动欧洲Ⅱ期试验，其递送的GDNF可促进多巴胺能神经元存活与功能恢复，I期试验显示良好安全性与初步疗效。 AAV-GAD基因治疗通过向苍白球内侧核递送谷氨酸脱羧酶基因，调节基底节环路功能，是唯一完成两项阳性结果假手术对照试验的中枢神经基因治疗。多巴胺合成相关基因治疗中，VY-AADC01通过AAV递送AADC基因，提升左旋多巴转化效率，显著减少“关”期时间。 RNA干扰技术靶向SNCA基因降低α-突触核蛋白表达，CRISPR/Cas9技术则可直接编辑LRRK2、PARK2等致病基因突变，虽目前多处于临床前阶段，但展现出根治遗传性PD的潜力。我国在该领域成果显著：瑞宏迪医药RGL-193注射液完成8例患者入组，天泽云泰VGN-R09b通过双基因递送实现神经保护与功能改善，均显示良好前景。3.3 细胞治疗临床突破 细胞治疗通过移植功能性细胞替代受损神经元或提供神经保护，近年取得多项突破性进展。2025年《Cell》发表的研究显示，胚胎干细胞来源的A9-DPC细胞在I/IIa期试验中，实现运动症状改善且疗效呈剂量依赖性，安全性良好。 诱导多能干细胞（iPSCs）技术避免免疫排斥问题，BlueRock公司BRT-DA01作为全球首个PD干细胞疗法进入Ⅲ期试验，2025年启动exPDite-2试验并完成首例给药。我国在该领域处于国际领先地位：2025年11月中科大附一院施炯团队利用异体干细胞移植使6例患者恢复正常生活，其中37岁患者成为全球首例“治愈”的早发性PD患者；浙江完成首例自体iPSC衍生多巴胺前体细胞移植，症状改善显著。 神经干细胞与间充质干细胞治疗各具优势：北京协和医院通过鼻黏膜途径移植人源神经干细胞，改善中晚期患者症状；基因工程化间充质干细胞可稳定分泌多巴胺，实现神经保护与免疫调节双重作用。睿健医药NouvNeu001基于化学诱导技术，2025年4月启动Ⅱ期试验，其亚型NouvNeu003专门针对早发型患者。细胞治疗长期随访显示，经严格质量控制可有效规避肿瘤形成等风险。3.4 其他新兴治疗技术 神经调控技术实现智能化升级：自适应深部脑刺激（aDBS）通过AI算法实时监测脑活动，自动调整刺激参数，疗效优于传统DBS。磁共振引导聚焦超声（MRgFUS）作为无创技术，已在欧洲获批用于药物难治性震颤治疗。 免疫治疗聚焦α-突触核蛋白靶向：罗氏普拉西珠单抗虽IIb期试验未达主要终点，但在早期患者中显示潜在获益；主动免疫治疗UB-312与AFFITOPE® PD01A在I期试验中展现良好安全性与免疫原性，抗体应答者可获得显著获益。 线粒体靶向治疗与肠道菌群调节成为新方向：天玑济世TJ0113作为线粒体自噬诱导剂，Ⅱ期试验正在推进；研究证实肠道炎症加速α-突触核蛋白聚集，菌群调节策略进入临床试验。A2A受体拮抗剂VG081821AC完成Ⅱ期试验，通过非多巴胺能机制实现症状改善与潜在疾病修饰作用。4. 临床实践应用现状4.1 诊断技术革命性进步 生物标志物检测实现早期预警：2018—2023年种子扩增试验（SAA）以97%准确率检测脑脊液α-突触核蛋白，2025年徐评议教授团队研发的QSAA血液检测技术，仅需2-3毫升血液即可在一天内完成检测，准确率超90%，实现7—10年疾病预警。 神经影像技术精度显著提升：7T MRI空间分辨率达0.4毫米，通过SWI技术清晰显示黑质铁沉积；神经黑色素MRI联合QSM成像提高PD分期与认知评估效能；18F-FPCIT PET扫描早期即可发现纹状体多巴胺转运体不对称性摄取降低，敏感性达92.3%。 人工智能推动诊断革新：凝动医疗MODAS系统通过自然图像AI技术，实现毫米级震颤检测，评估效率提升4倍，准确率超95%；MultiParkNet深度学习框架整合五种异构数据源，测试准确率达96.74%。经颅超声作为经济无创手段，以黑质高回声为标志，敏感性达80%—90%，适合基层筛查。 多模态诊断策略成为主流，整合影像、电生理、运动功能等指标建立综合模型。嗅觉测试作为前驱期筛查手段被纳入指南，UPSIT评分≤15分成为重要诊断依据，显著提高早期识别率。4.2 治疗策略系统化升级 个体化治疗成为核心原则，指南推荐根据年龄分层用药：早发型患者（<65岁）优先选择非麦角类激动剂或MAO-B抑制剂，晚发型患者（≥65岁）首选左旋多巴。持续多巴胺能刺激（CDS）理念广泛应用，左旋多巴肠凝胶与皮下输注系统通过稳定血药浓度，显著改善晚期运动波动。 多学科团队（MDT）模式全面推广，2024年国家老年疾病临床医学研究中心分中心落地北大国际医院，整合神经内外科、康复科等资源提供一体化服务。中西医结合治疗规范化发展，《帕金森病中西医结合诊疗指南》发布，中药、针灸等手段有效改善非运动症状。 非药物治疗地位提升，太极拳、节律步态训练等运动疗法改善平衡与运动功能；康复治疗针对吞咽、语言障碍提供专业训练；心理干预缓解抑郁焦虑情绪。全程管理体系逐步建立，实现早期诊断、中期优化、晚期关怀的连续性服务。4.3 中国诊疗特色与发展 我国诊疗指南持续完善，第四版指南新增前驱期诊断标准，纳入嗅觉减退、REM睡眠行为障碍等核心指标。中西医结合成为独特优势，中国中医科学院西苑医院等机构的临床实践证实其在症状改善与病情延缓方面的效果。 专科诊疗体系日益成熟，北京宣武医院、上海瑞金医院等形成多学科协作优势，清华大学玉泉医院提出“病－脑－身”三维诊疗体系。药物可及性显著提升，罗替高汀微球、沙芬酰胺等新药陆续在华获批，奥匹卡朋上市申请已获受理。 基层诊疗能力逐步增强，《帕金森病基层诊疗指南2025》强调非运动症状的早期识别价值。科研创新成果丰硕，除FAM171A2发现外，基因与细胞治疗临床试验数量位居全球前列，展现我国在该领域的国际竞争力。医保政策持续优化，PD纳入慢性病管理，减轻患者经济负担。5. 社会影响与展望5.1 患者与家庭的多维负担 PD对患者身体功能造成渐进性损害，美国研究显示患者日常生活活动能力评分显著降低，我国约20%患者需长期护理，远超其他疾病的7%。非运动症状危害突出，半数患者伴有抑郁焦虑，15%—30%晚期出现认知障碍，显著增加护理难度。 经济负担沉重：美国PD患者3年增量成本达20,832美元，我国患者年均自付医疗费用6000-20,000元，晚期联合用药费用可增至5000-12,000元/年。家属照护压力巨大，每周平均提供31小时无偿照护，需承担三项以上生活护理，半数护理者周工作时长超100小时。 患者社会参与受限，逐渐退出社交活动导致孤独感加剧；心理问题双向传导，患者担心成为家庭负担，家属则面临照护与工作的双重压力，形成恶性循环，导致患者与家庭生活质量全面下降。5.2 未来趋势与挑战 精准医学成为发展核心，多组学技术推动PD分型精细化，PRS实现风险精准预测，基因指导的个体化治疗逐步落地。疾病修饰治疗有望突破，FAM171A2等新靶点为开发阻断病理传播药物提供可能，预计5-10年内将有1-2种疾病修饰药物获批。 人工智能深度赋能诊疗：可穿戴设备结合AI实时监测症状变化，手术导航系统提高DBS精准度，药物发现平台加速新药研发。联合治疗策略成为主流，药物与神经调控、基因与细胞治疗、中西医结合等模式实现疗效协同。 新型给药系统不断涌现，纳米递送、脑内植入装置等技术实现药物精准靶向。早期干预体系逐步建立，高危人群筛查与生活方式干预成为预防重点。 同时面临多重挑战：疾病异质性增加诊疗难度；药物研发转化率低，动物模型与临床存在差距；新技术成本高昂限制可及性；基因编辑等技术涉及伦理争议；基层专业人才短缺。国际合作与政策支持至关重要，需通过资源共享、技术转移、医保优化等措施推动全球PD防治进展。6. 结论与建议 2015—2025年PD研究取得里程碑式进展：病理机制方面，遗传因素、神经炎症、蛋白质错误折叠等领域的突破深化了疾病认知；治疗领域，传统药物优化与基因、细胞治疗的临床转化为患者带来新希望，我国在细胞治疗领域实现全球领先；临床实践中，早期诊断技术与个体化诊疗体系显著提升患者获益。 针对不同群体提出建议：科研工作者需加强基础与转化研究，推动多学科融合与国际合作；临床医生应更新知识体系，践行精准诊疗与MDT模式；政策制定者需加大科研投入，完善医保与长期照护制度，提升基层服务能力；患者与家属应积极配合治疗，参与康复训练与患者组织；社会各界需消除歧视，完善无障碍设施，支持公益事业。 展望未来，随着技术进步与社会协同，预计2035年实现疾病修饰治疗突破，2040年早期干预显著降低疾病影响，2050年PD成为可防可控的慢性疾病。通过全社会共同努力，将为PD患者点亮希望之光，最终实现疾病的有效控制与攻克。