Therapeutics accelerator, world-first trial, and INDIGO results

4 min read

Brain Tumour Research is excited to announce that applications for the first cycle of the  Brain Tumour Research Novel Therapeutics Accelerator are now open! 

The accelerator programme provides independent and holistic guidance to help researchers position their therapeutic or technology along a realistic pathway to clinical trials. For the applicant guide click here or here for the website with more information and the pre-application form. Deadline for the pre-application form is Friday 21st July 2023.


INDIGO: Vorasidenib Offers Patients With IDH-Mutant Low-Grade Glioma a Means to Delay Chemotherapy and Radiotherapy The trial, named INDIGO, is the first randomised phase 3 study of a targeted therapy in grade 2 IDH1/2-mutated glioma, and represents the first targeted agent to improve patient outcomes in low-grade glioma in more than 20 years. The results published in the New England Journal of Medicine, showed that vorasidenib led to a 61% reduction in the risk of tumour progression or death, and significantly delayed the need for more toxic therapies such as chemotherapy or radiotherapy when compared with a placebo. 83% of patients receiving vorasidenib did not require additional treatment at 24 months, compared to 27% who had the placebo. Vorasidenib also demonstrated a manageable safety profile.

World-first trial for paediatric brain cancer A world-first clinical trial has been launched at Sydney Children’s Hospital, Randwick for children with ependymoma. The Deflexifol® At Relapse Trial (DART), will test Deflexifol® - a new Australian-developed co-formulation of a chemotherapy called 5-FU and leucovorin - in treating children with high-risk or relapsed ependymoma. The treatment is expected to be a less toxic and more effective chemotherapeutic compared with standard of care versions of these drugs commonly used to treat adult cancers.


New study gives early indication certain drugs might be repurposed to treat brain tumours A new study has shown early indications that several existing FDA-approved drugs could be repurposed as possible treatments for brain tumours. The study, published in Biomedicines, looked into the changes in gene expression in glioblastoma tumours at first surgery compared to at the time of relapse. The research team further tested these genes in a connectivity mapping analysis using software developed at Queen’s University (QUADrATiC) and were able to predict the possibility that some FDA-approved drugs might be effective against glioblastoma.

A metabolic process in cancer cells could unlock a possible treatment for glioblastoma A study led by researchers at the UCLA Jonsson Comprehensive Cancer Center has found that targeting a metabolic process in people with a specific genetic mutation could help treat glioblastoma. The genetic alteration — a deletion in a gene called CDKN2A — is present in about 60% of people who have glioblastoma. They found that the gene rewires lipid (fat) metabolism to work in a different way than cells without that genetic alteration, and when the CDKN2A gene is missing, the way cancer cells process fats makes them more vulnerable to cell death, or ferroptosis. The discovery, published in Cancer Cell, suggests that this metabolic vulnerability could be a future target of therapy for glioblastoma patients.


2023 Queen Square Brain Tumour Hands-on Simulation Course. This advanced neurosurgical oncology simulation course is aimed at trainees and consultants and will take place on 16th June 2023 at the National Hospital for Neurology and Neurosurgery. For more information, and to register click here.

NCRI’s CTRad: Radiotherapy Proposal Guidance – Proposal Submission.  CTRad is inviting radiotherapy research ideas for open discussion and peer input prior to application to funding bodies at the next virtual CTRad Proposal Guidance meeting on 3 July 2023 (9am-12pm). There will be opportunities for investigators to present and discuss their concepts with CTRad experts at this meeting. The deadline for submissions is Sunday 18 June at 11.59pm. Click here for more information, or contact CTRad at

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