News on ATRT, DIPG, GBM – just three of over 120 types of brain tumour

19 September 2020

Atypical teratoid rhabdoid tumours (ATRT) are a rare, fast-growing brain tumour that usually strikes children aged three and below, although they can occur in older children and adults. There are treatments options, but no definitive standard of care and long-term survival is poor. In this paper, researchers describe how the functional loss of a single gene negatively impacts neural development and promotes the growth of this particularly deadly form of paediatric brain cancer.

Last week we looked at orphan drug designation ( you can click here to see last week’s update) which can also be called rare disease designation and that is what has been granted to Ziopharm Oncology for Ad-RTS-hIL-12 with veledimex to be used in the investigational treatment of diffuse intrinsic pontine glioma (DIPG).

A new clinical trial is testing a promising treatment to repair brain injury and improve quality of life in survivors of childhood brain cancer. The trial will test whether the diabetes drug metformin can boost cognitive function and enhance recovery in childhood brain cancer survivors. If successful, this new treatment could lead to a change in care from helping children and families manage potential long-term cognitive challenges to actively preventing and treating them at their root.

Here’s a TV news item from the US with a scientist talking about his life’s work and how he has designed a medicine that fights DIPG, the drug is called OKN-007 and its primary use was to treat glioblastoma in adults however results have shown promise in younger patients as well. If approved, OKN-007 would become the only US Food and Drug Administration (FDA) approved treatment for DIPG.

To conclude this week I really recommend having a look at this piece. Cancer Research Heats up with Biotechs Attacking Tumour ‘Comfort Zone’ – the comfort zone in question is the tumour microenvironment and this links to GBM work at our QMUL centre, which we have reported on previously, but is worth looking at again in the light of the article above. New research shows GBMs turn good guys bad.