Shah Muhammad Hamza, Ranganathan Sruthi, Thomson Nirupama, Dorcas Adeleke Oluwaseun
School of Medicine, Dentistry & Biomedical Sciences, Queen's University Belfast, Belfast, UK.
School of Clinical Sciences, University of Cambridge, Cambridge, UK.
Ann Med Surg (Lond). 2025 Jul 16;87(8):5356-5359. doi: 10.1097/MS9.0000000000003546. eCollection 2025 Aug.
Gliomas remain one of the most formidable challenges in neuro-oncology due to their diffuse infiltration, treatment resistance, and high recurrence rates. A major subset of these tumours harbour mutations in isocitrate dehydrogenase (IDH), which lead to epigenetic reprogramming and impaired homologous recombination (HR) DNA repair, presenting a unique vulnerability to synthetic lethality-based interventions. Poly (ADP-ribose) polymerase (PARP) inhibitors, which target HR-deficient cells, have demonstrated preclinical efficacy against IDH-mutant gliomas. Despite the promise of both IDH and PARP inhibitors, monotherapy has yielded limited durable clinical responses. This paper explores the rationale for a dual-target approach combining IDH and PARP inhibitors, supported by mechanistic insights, preclinical studies, and emerging clinical trial data. It also addresses the challenges posed by tumour heterogeneity, clonal evolution, and toxicity management. Furthermore, it evaluates the potential of integrating immune checkpoint inhibitors and radiotherapy as part of combinatorial strategies. We conclude that while a dual-target approach holds great promise, its clinical success will depend on biomarker-driven stratification, adaptive trial designs, and a deeper understanding of glioma biology.
由于胶质瘤具有弥漫性浸润、治疗抵抗和高复发率,它们仍然是神经肿瘤学中最严峻的挑战之一。这些肿瘤的一个主要亚群携带异柠檬酸脱氢酶(IDH)突变,这会导致表观遗传重编程和同源重组(HR)DNA修复受损,从而对基于合成致死性的干预措施表现出独特的易感性。靶向HR缺陷细胞的聚(ADP - 核糖)聚合酶(PARP)抑制剂已在临床前研究中显示出对IDH突变型胶质瘤的疗效。尽管IDH抑制剂和PARP抑制剂都有前景,但单药治疗产生的持久临床反应有限。本文探讨了联合使用IDH抑制剂和PARP抑制剂的双靶点方法的原理,这一方法得到了机制见解、临床前研究和新出现的临床试验数据的支持。它还讨论了肿瘤异质性、克隆进化和毒性管理带来的挑战。此外,它评估了将免疫检查点抑制剂和放疗作为联合策略一部分的潜力。我们得出结论,虽然双靶点方法前景广阔,但其临床成功将取决于生物标志物驱动的分层、适应性试验设计以及对胶质瘤生物学的更深入理解。