Irish researchers make breakthrough in battle against incurable childhood cancer 

Geneticists from Trinity College Dublin discovered that the mutation – H3K27M – causes diffuse midline glioma (DMG), and – in lab studies working with model cell types – successfully reversed its effects to slow cancer cell growth with a drug. Picture: Pexels

Geneticists from Trinity College Dublin have pinpointed how a gene mutation causes an incurable childhood cancer and have successfully targeted tumour cells with a tailored drug.

The researchers discovered that the mutation – H3K27M – causes diffuse midline glioma (DMG), and – in lab studies working with model cell types – successfully reversed its effects to slow cancer cell growth with a drug.

Paediatric gliomas like DMG are among the most devastating cancers, with tumours typically arising in the brain and having extremely poor prognoses.

The landmark work, just published in the international journal, Nature Genetics, translates a new understanding of the genetics of DMG progression into a highly promising, targeted therapeutic approach and offers significant hope of improved treatments in the future.

Clinical trials

The scientists have now called for clinical trials to begin, in which an already approved class of drugs called “EZH2 inhibitors” can be assessed.

These drugs target the same key biological pathway involved in DMG as they do successfully in lymphomas and sarcomas, two cancers common in adults.

Professor Adrian Bracken, from Trinity’s School of Genetics and Microbiology, who led the project, said this research was a “huge step forward” for these tumour types.

“We propose these drugs could be impactful for children with DMG and, as a result, call for clinical trials to begin next,” he said.

Ultimately, we hope that our work – together with that of others focused in this area – will lead to curative clinical approaches for what is a truly terrible disease that can devastate families and for which there are currently no therapeutic options.” 

Dr Jane Pears, paediatric consultant oncologist at Our Lady's Children's Hospital, Crumlin, who treats children with this disease, said the best treatment offered to children currently "may extend survival for a few months but is not curative". 

"We are now entering an exciting era of expansion of our knowledge of this disease at a molecular level, which in turn will lead us towards more targeted treatments,” she said.

Dr Becky Birch, head of research at the Brain Tumour Charity, which helped fund the study, described the discovery as “really promising”.

“With average survival still heartbreakingly short at less than 12 months, we urgently need to find new options to help slow the growth of this rare and often-inoperable cancer and give children diagnosed more time to live,” she added.