New gene therapy may offer hope for people with deadly brain cancer

NUS team aiming to start clinical trial for glioblastoma patients from NUH by late 2025

- Shabana Begum

In the future, patients who suffer a relapse of the deadliest form of brain cancer may get a second shot at life with the help of a novel gene therapy developed by researchers from the National University of Singapore's Yong Loo Lin School of Medicine (NUS Medicine).

The scientists behind the stem cell-based gene therapy are aiming to start a clinical trial for glioblastoma patients of National University Hospital (NUH) by late 2025.

Glioblastoma is an incurable and aggressive brain tumour. Once diagnosed, patients are typically left with about two years to live.

This targeted therapy - designed to kill aggressive tumours and activate anti-cancer immunity builds on a similar drug that the NUS Medicine scientists first used to treat dogs and cats with terminal cancers.

A vial of the treatment comprises human stem cells carrying cancerkilling genes that naturally gravitate towards tumours.

The team from NUS Medicine's biochemistry department includes Associate Professor Too HengPhon, adjunct senior research fellow Sarah Ho and postdoctoral fellow Woo Jun Yung.

Prof Too's team initially worked on the therapy to combat aggressive human tumours until a vet who heard about it contacted them in 2018.

The first version of the treatment was used on 65 dogs and cats between 2018 and 2022. It had two parts first, the modified stem cells were injected into the animal, and then off-the-shelf anti-fungal drugs were taken orally.

The researchers developed a technology to insert large amounts of yeast-based genes into the stem cells that would react with the antifungal drugs.

This reaction would allow the cells to produce a chemotherapy drug called fluorouracil (5FU).

The modified stem cells act like suicide bombers, loading the toxic 5FU around the tumour to kill it, said Dr Ho.