Mesothelioma (MM) is an aggressive and lethal disease with few therapeutic opportunities. Platinum-pemetrexed chemotherapy is the backbone of first-line treatment for MM. The introduction of immunotherapy (IO) has been the only novelty of the last decades, allowing an increase in survival compared to standard chemotherapy (CT). However, IO is not approved for epithelioid histology in many countries. Therefore, therapy for relapsed MM remains an unmet clinical need, and the prognosis of MM remains poor, with an average survival of only 18 months. Increasing evidence reveals MM complexity and heterogeneity, of which histological classification fails to explain. Thus, scientific focus on possibly new molecular markers or cellular targets is increasing, together with the search for target therapies directed towards them. The molecular landscape of MM is characterized by inactivating tumor suppressor alterations, the most common of which is found in CDKN2A, BAP1, MTAP, and NF2. In addition, cellular targets such as mesothelin or metabolic enzymes such as ASS1 could be potentially amenable to specific therapies. This review examines the major targets and relative attempts of therapeutic approaches to provide an overview of the potential prospects for treating this rare neoplasm.
间皮瘤是一种侵袭性强且致命的疾病，治疗手段极为有限。铂类联合培美曲塞化疗是间皮瘤一线治疗的基础方案。过去数十年来，免疫疗法的引入成为该领域唯一突破性进展，相较于标准化疗可延长患者生存期。然而，许多国家尚未批准免疫疗法用于上皮样组织学分型。因此，复发间皮瘤的治疗仍是亟待满足的临床需求，其预后依然较差，中位生存期仅为18个月。越来越多的证据揭示了间皮瘤的复杂性和异质性，而组织学分类已无法充分解释这些特征。因此，学界日益聚焦于潜在的新型分子标志物或细胞靶点，并致力于开发针对这些靶点的精准疗法。间皮瘤的分子特征主要表现为抑癌基因失活突变，其中最常见于CDKN2A、BAP1、MTAP和NF2基因。此外，间皮素等细胞靶点及ASS1等代谢酶也可能成为特异性治疗的潜在目标。本综述通过探讨主要治疗靶点及相关治疗尝试，旨在系统阐述这一罕见肿瘤治疗的潜在前景。
肿瘤(癌症)患者之家