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急性髓系白血病药物递送与治疗策略的最新进展

英文原题:Recent advances in drug delivery and treatment strategies for acute myeloid leukemia.

PubMed 2025/08/18(内容时间) Int J Pharm Q1 · IF 6(JCR 2025)

研究概要

急性髓系白血病(AML)仍是一种高度异质性、侵袭性强且预后不良的血液系统恶性肿瘤。

中文摘要

急性髓系白血病(AML)仍是一种高度异质且侵袭性强的血液系统恶性肿瘤,预后较差。尽管化疗方案、靶向疗法(如 FLT3 和 BCL2 抑制剂)及抗体药物偶联物和 CAR-T 等免疫治疗取得显著进展,治疗结局仍不理想,原因包括化疗耐药、靶外毒性、疾病复发和生物利用度有限。为解决这些局限,纳米医学和药物递送系统成为提高疗效、减少不良反应的有前景方法。本综述全面概述现有 AML 治疗,介绍其进展和持续局限,并重点关注正在研究的基因疗法。随后探讨多种纳米载体平台,主要是脂质和聚合物纳米颗粒,分析其改善药物稳定性、生物利用度和白血病细胞靶向能力、从而克服 AML 临床治疗挑战的潜力。近期靶向制剂创新包括抗体、肽和纳米抗体偶联药物递送系统,旨在提高 AML 特异性。最后,综述讨论 AML 治疗的关键挑战和未来方向,强调需持续推进生物材料创新、制剂优化和精准靶向方法,以改善患者结局。

展开英文摘要原文

Acute myeloid leukemia (AML) remains a highly heterogeneous and aggressive hematologic malignancy with a poor prognosis. Although significant advancements have been made in chemotherapeutic regimens, targeted therapies (e.g., FLT3 and BCL2 inhibitors), and immunotherapies such as antibody-drug conjugates and CAR-T cells, treatment outcomes remain unsatisfactory, due to chemoresistance, off-target toxicities, disease relapse, and limited bioavailability. To address these limitations, nanomedicine and drug delivery systems have emerged as a promising approach to enhance therapeutic efficacy and minimizing adverse effects. This review provides a comprehensive overview of current AML treatments, highlighting both achievements and persistent limitations, with a particular focus on gene therapies under investigation. We then delve into various nanocarrier platforms, mainly lipid-based and polymer-based nanoparticles (NPs), examining their potential to overcome existing clinical challenges in AML therapy by improving drug stability, bioavailability, and leukemic cell targeting. Recent innovations in targeted formulations, such as antibody-, peptide-, and nanobody-conjugated drug delivery systems, have been designed to improve AML specificity. Finally, we discuss the key challenges and future directions in AML treatment, emphasizing the need for continued research in biomaterial innovation, formulation optimization, and precision-targeted approaches to enhance patient outcomes.

论文信息

作者
Pan Q、Mahato RI
第一作者单位
Department of Pharmaceutical Sciences, The University of Nebraska Medical Center, Omaha NE68198, USA.United States
通讯作者单位
Department of Pharmaceutical Sciences, The University of Nebraska Medical Center, Omaha NE68198, USA. Electronic address: ram.mahato@unmc.edu.United States
文献类型
综述
期刊
International journal of pharmaceutics2025 Oct 15
原文标识
PubMed 40834936 · DOI 10.1016/j.ijpharm.2025.126078