1Royal Victoria Infirmary, The Newcastle Hospitals NHS Foundation Trust, Newcastle Upon Tyne, United Kingdom.
2Mayo Clinic Cancer Center, Rochester, Minnesota; and.
Neurosurg Focus. 2024 Sep 1;57(3):E7. doi: 10.3171/2024.6.FOCUS24338.
Sonodynamic therapy (SDT) is gaining attention as a promising new noninvasive brain tumor treatment that targets and selectively kills tumor cells, with limited side effects. This review examines the mechanisms of SDT and ongoing clinical trials looking at optimization of sonication parameters for potential treatment of glioblastoma (GBM) and diffuse intrinsic pontine glioma (DIPG). The results in the first patient with recurrent GBM treated at the Mayo Clinic are briefly discussed.
The authors of this literature review used electronic databases including PubMed, EMBASE, and OVID. Articles reporting relevant preclinical and clinical trials were identified by searching for text words/phrases and MeSH terms, including the following: "sonodynamic therapy," "SDT," "focused ultrasound," "5-ALA," "ALA," "brain tumors," "diffuse pontine glioma," "glioblastoma," and "high grade glioma."
Preclinical and clinical trials investigating the specific use of SDT in brain tumors were reviewed. In preclinical models of high-grade glioma and GBM, SDT has shown evidence of targeted tumor cell death via the production of reactive oxygen species. Emerging clinical trial results within recurrent GBM and DIPG show evidence of successful treatment response, with minimal side effects experienced by recruited patients. So far, SDT has been shown to be a promising noninvasive cancer treatment that is well tolerated by patients. The authors present pilot data suggesting good radiological response of GBM to a single SDT treatment, with unpublished observation of a lack of off-target effects even after multiple (monthly) sonication outpatient treatments. The scope of the clinical trials of SDT is to investigate whether it can be the means by which the fatal diagnosis of GBM or DIPG is converted into that of a chronic, treatable disease.
SDT is safe, repeatable, and better tolerated than both chemotherapy and radiotherapy. It has been shown to have an effect in human cancer therapy, but more clinical trials are needed to establish standardized protocols for sonosensitizer delivery, treatment parameters, and combination therapies. The most appropriate timing of treatment also remains to be determined-whether to prevent recurrence in the postoperative period, or as a salvage option in patients with recurrent GBM for which redo surgery is inappropriate. It is hoped that SDT will also be developed for a wider spectrum of clinical indications, such as metastases, meningioma, and low-grade glioma. Further clinical trials are in preparation.
声动力学疗法（SDT）作为一种有前途的新型非侵入性脑肿瘤治疗方法，正在引起关注，它可以靶向并选择性地杀死肿瘤细胞，副作用有限。本综述探讨了 SDT 的作用机制，并对正在进行的临床试验进行了研究，这些临床试验旨在优化声振参数，以潜在治疗胶质母细胞瘤（GBM）和弥漫性内在脑桥胶质瘤（DIPG）。简要讨论了梅奥诊所首例复发性 GBM 患者的治疗结果。
本文作者使用了电子数据库，包括 PubMed、EMBASE 和 OVID。通过搜索文本词/短语和 MeSH 术语，包括以下内容，确定了报道相关的临床前和临床试验的文章：“sonodynamic therapy”、“SDT”、“focused ultrasound”、“5-ALA”、“ALA”、“brain tumors”、“diffuse pontine glioma”、“glioblastoma”和“high grade glioma”。
对专门用于脑肿瘤的 SDT 的临床前和临床试验进行了综述。在高级别神经胶质瘤和 GBM 的临床前模型中，SDT 通过产生活性氧显示出靶向肿瘤细胞死亡的证据。复发性 GBM 和 DIPG 的新兴临床试验结果表明，治疗反应成功，招募的患者经历的副作用最小。到目前为止，SDT 已被证明是一种有前途的非侵入性癌症治疗方法，患者耐受性良好。作者提出了初步数据，表明 SDT 单次治疗后 GBM 的影像学反应良好，并且即使在多次（每月）声振门诊治疗后，也未观察到非靶向效应。SDT 临床试验的范围是调查它是否可以成为将 GBM 或 DIPG 的致命诊断转变为慢性、可治疗疾病的手段。
SDT 安全、可重复且比化疗和放疗更能被患者耐受。它已被证明对人类癌症治疗有效，但需要更多的临床试验来建立声敏剂输送、治疗参数和联合治疗的标准化方案。治疗的最佳时机也有待确定——是在术后预防复发，还是在 redo 手术不合适的复发性 GBM 患者中作为挽救选择。人们希望 SDT 也能扩展到更广泛的临床适应证，如转移瘤、脑膜瘤和低级别胶质瘤。进一步的临床试验正在准备中。
