New screening approach for GBM and the mechanics of ONC201 revealed

6 min read


Study finds improved survival for incurable brain tumour, providing 'a crack in the armour'. H3K27M-mutant diffuse midline glioma (DMG) is a type of childhood brain tumour for which there are no effective treatments. This study reports on the results of two early-stage clinical trials, revealing that the drug, ONC201, nearly doubled survival for patients with DMG or DIPG, compared to previous patients. Furthermore, it revealed the underlying mechanisms behind the compound’s success, which were previously unknown as ONC201 took an unconventional route to clinical trial.  

Published in Cancer Discovery, the paper reported that ONC201 got into tumour cells and affected mitochondria, producing more L-2HG than untreated tumour cells. The team found that the increased L-2HG levels reversed tumour-defining signals which caused the tumour cells to differentiate more and divide less. The longer the patients were on ONC201, the more tumours exhibited these reversals. More clinical trials are underway. 

FDA Grants Orphan Drug Designation to Treatment of Malignant Glioma.

Lisata Therapeutics has announced that LSTA1, for the treatment of malignant glioma, has been granted Orphan Drug Designation by the FDA. Lisata Therapeutics created LSTA1 to activate a new uptake pathway, allowing co-administered or tethered anti-cancer drugs to combat solid tumours more successfully. The drug can also modify the tumour microenvironment to make tumours more vulnerable to immunotherapies. 


New approach to screening has potential to extend survival in glioblastoma brain cancer. A new form of screening may improve survival rates among people with glioblastoma (GBM) by helping identify those most likely to benefit from certain treatments.  


Published in the journal Cancers, the research in mice models demonstrated a molecular imaging technique that can reveal the presence of a protein called PD-L1 (programmed death ligand-1). High levels of PD-L1 have been associated with GBM invasiveness and immuno-resistance. The study, undertaken by researchers at The Institute of Cancer Research, could help direct treatment decisions, particularly predicting responses to immune checkpoint inhibitors, potentially leading to better patient outcomes. 

Optical blood-brain-tumour barrier modulation expands therapeutic options for glioblastoma treatment

The treatment of glioblastoma has limited clinical progress over the past decade, partly due to the lack of effective drug delivery strategies across the blood-brain-tumour barrier. 

Researchers in this study showed that pulsed laser excitation of vascular-targeted gold nanoparticles. (optoBBTB) non-invasively and reversibly influenced blood-brain-tumour barrier permeability and enhanced the delivery of the anticancer drug, paclitaxel, in two models. The treatment reduced tumour volume by 6 and 2.4-fold and prolonged the survival by 50% and 33%, respectively. Since paclitaxel does not penetrate the blood-brain-tumour barrier and is abandoned for glioblastoma treatment following its failure in early-phase clinical trials, these results, published in Nature Communications, raise the possibility of reevaluating a number of potent anticancer drugs by combining them with strategies to increase blood-brain-tumour barrier permeability. 

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