Early phase trial results for GBM and chordoma tumours

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Preclinical and early clinical studies of a novel compound SYHA1813 that efficiently crosses the blood–brain barrier and exhibits potent activity against glioblastoma. Glioblastoma (GBM) is the most common high-grade brain tumour in adults. Previous studies have shown that both immune cells (macrophages) and blood vessel formation (angiogenesis) play significant roles in GBM progression, and co-targeting of CSF1R and VEGFR is likely to be an effective strategy for GBM treatment. Therefore, this study, published in Acta Pharmaceutica Sinica B, developed a novel and selective inhibitor of CSF1R and VEGFR, SYHA1813, possessing potent antitumour activity against GBM.  As a clinical proof of concept, SYHA1813 achieved confirmed responses in patients with recurrent GBM in an ongoing first-in-human phase I trial. The data of this study supports the rationale for an ongoing phase I clinical study. 

Pilot Study of High-Dose Pemetrexed in Patients with Progressive Chordoma. Chordomas are ultra rare tumours of the axial spine and skull-base without approved systemic therapy. Adult patients with previously treated, progressive chordoma participated in an open-label, single-institution, single-arm, pilot clinical trial of intravenous pemetrexed 900 mg/m2 every 3 weeks and supportive medications of folic acid, vitamin B12, and dexamethasone. High-dose pemetrexed appears tolerable and shows objective antitumour activity in patients with chordoma. Phase II studies of high-dose pemetrexed are warranted. 


Association between viral infections and glioma risk: a two-sample bidirectional Mendelian randomisation analysis. Glioma is one of the leading types of brain tumour, but few causes of primary glioma have been identified. Previous observational research has shown an association between viral infection and glioma risk. In this study, published in BMC Medicine, researchers used Mendelian randomisation (MR) analysis to explore the causal relationship between viral infection and glioma. They provide evidence that genetically predicted herpes zoster (shingles) caused by VZV (varicella zoster virus/chicken pox) infection can reduce the risk of low-grade glioma. They conclude that this area needs further research. 

The long non-coding RNA OTX2-AS1 promotes tumour growth and predicts response to BCL-2 inhibition in medulloblastoma. The current molecular characterisation of medulloblastoma, the most common high-grade paediatric brain tumour, is mainly based on protein-coding genes, while little is known about the involvement of long non-coding RNAs (lncRNAs). This study aimed to uncover the role of the lncRNA called OTX2-AS1 in medulloblastoma. The researchers revealed a pro-tumorigenic role of OTX2-AS1 in medulloblastoma and identified BCL-2 inhibition as a potential therapeutic approach to target OTX2-AS1 overexpressing medulloblastoma cells. 

Incidence, management, and outcome of incidental meningioma: what has happened in 10 years? The aim of this study, published in the Journal of Neuro-Oncology, was to study the incidence of meningioma in our population, clarify changes in clinical handling over time, and potential differences in management and outcome. They concluded that despite the same number of unique patients undergoing brain scans in the time periods, there was a trend towards more patients diagnosed with an incidental asymptomatic meningioma in the more recent years. This difference may be attributed to more contrast enhanced scans and more scans among the elderly but needs to be further studied. 

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