蛛网膜转移癌代表了一种具有极少持久治疗选择的侵袭性癌症阶段。在过去的二十年里，对癌症生物学、肿瘤对致癌驱动突变的依赖和复杂的免疫系统相互作用的理解的不断深入，促成了癌症定向治疗的爆炸式增长，包括小分子抑制剂和免疫检查点抑制剂。然而，这些治疗大多数在患有蛛网膜转移癌的患者中未被充分探索，这限制了从脑外甚至颅内功效转化到独特的蛛网膜空间的推断。进一步混淆对蛛网膜中药物活性的解释的是我们对脉络丛血脑脊液屏障中药物穿透的了解还不完整。尽管如此，多项回顾性研究和有前途的前瞻性试验提供了一些小分子和免疫检查点抑制剂的蛛网膜活性的证据，并强调了进一步治疗发展的潜在领域，以治疗患有蛛网膜疾病的患者。© 2022. The Author(s).

Leptomeningeal metastases represent an aggressive stage of cancer with few durable treatment options. Improved understanding of cancer biology, neoplastic reliance on oncogenic driver mutations, and complex immune system interactions have resulted in an explosion in cancer-directed therapy in the last two decades to include small molecule inhibitors and immune checkpoint inhibitors. Most of these therapeutics are underexplored in patients with leptomeningeal metastases, limiting extrapolation of extracranial and even intracranial efficacy outcomes to the unique leptomeningeal space. Further confounding our interpretation of drug activity in the leptomeninges is an incomplete understanding of drug penetration through the blood-cerebrospinal fluid barrier of the choroid plexus. Nevertheless, a number of retrospective studies and promising prospective trials provide evidence of leptomeningeal activity of several small molecule and immune checkpoint inhibitors and underscore potential areas of further therapeutic development for patients harboring leptomeningeal disease.© 2022. The Author(s).