治疗脑肿瘤一直是个挑战，因为中枢神经系统(CNS)功能障碍可能会导致治疗选择有限及各种不良后果。本综述聚焦于该领域的最新发展，着重于临床治疗。不断增长的临床试验景观反映了癌症免疫学和基因组学的先进见解，以及需要处理分子和临床异质性的必要性。最近的第三期试验研究抗PD-1免疫疗法，尤其是尼伐珠单抗，在胶质母细胞瘤中未能证明其改善生存期的效果，强调了需要更好地理解CNS免疫监视复杂性的必要性。相反，靶向治疗在批准美国食品和药物管理局批准的脑肿瘤方面占有一定份额，尤其是针对BRAF V600E突变和TRAK融合的疗法，因此需要定期筛查这些罕见的分子异常的患者。对于原发性CNS淋巴瘤，人们开始关注巩固疗法的长期结果，最近的研究突显了高剂量化疗和干细胞移植所提供的出色疾病控制。脑膜瘤仍然是研究重点，通过八肽素联合mTOR抑制剂和单药Pembrolizumab免疫治疗观察到了初步有益结果。最后，质子放射线治疗已成为来自实体肿瘤的蛛网膜下腔转移的一种新颖替代手段，可通过颅脊神经放射线治疗来更安全地治疗，并通过脑脊液中循环肿瘤细胞的计数来监测生物标志物。总的来说，这些渐进的进展已经改善了选择性脑肿瘤患者的预后，而持续的临床试验将为更多脑肿瘤患者带来有意义的进展和突破。 ©2023美国癌症学会。

Management of brain tumors has been challenging given the limited therapeutic options and disabling morbidities associated with central nervous system (CNS) dysfunction. This review focuses on recent developments in the field, with an emphasis on clinical management. The growing clinical trials landscape reflects advanced insights into cancer immunology and genomics and the need to address molecular and clinical heterogeneity. Recent phase 3 trials investigating anti-PD-1 immunotherapies, particularly nivolumab, have failed to demonstrate improved survival in glioblastoma, underscoring the need to better understand the complexity of CNS immunologic surveillance. Conversely, targeted therapies have accounted for several US Food and Drug Administration approvals extended to brain tumors, particularly therapies directed to BRAF V600E mutations and TRAK fusions, underscoring a need to routinely screen patients for these rare molecular abnormalities. In primary CNS lymphoma, attention has turned to long-term outcomes of consolidation therapies, and recent studies have highlighted the excellent disease control afforded by high-dose chemotherapy and stem cell transplantation. Meningiomas remain a focus of investigations, with preliminary promising results observed with octreotide combined with mTOR inhibition, and immunotherapy with single-agent pembrolizumab. Finally, proton radiotherapy has emerged as a novel alternative for leptomeningeal metastases from solid tumors, which can now be treated more safely with craniospinal irradiation and monitored by the enumeration of circulating tumor cells in the cerebrospinal fluid as a biomarker. Taken together, these incremental advances have improved outcomes in select brain tumor patient populations, whereas ongoing clinical trials hold the promise of meaningful advances and breakthroughs for larger proportions of patients with brain tumors.© 2023 American Cancer Society.