Irish research targeting childhood cancer’s ‘Achilles heel’ leads to clinical trial

28 Sep 2020

Image: © luchschenF/

A discovery made by TCD researchers regarding a potential treatment for a cancer that is common in children will be tested in clinical trials next year.

Researchers at Trinity College Dublin (TCD) focused on difficult-to-treat childhood cancers have said that one of their discoveries will be used in a drug trial next year. The team headed by Dr Gerard Brien confirmed that its discovery of an ‘Achilles heel’ in synovial tumours will now be used in clinical trials for a potential treatment by Boston-based pharma company C4 Therapeutics.

The original discovery was made two years ago in synovial sarcoma, a form of soft tissue cancer that is common in children. The TCD researchers used the gene-editing tool CRISPR to discover that the BRD9 gene is essential for synovial sarcoma tumour growth.

With this knowledge, the team developed drug candidates that could trick cancer cells into shutting off the gene and blocking the growth of synovial sarcoma tumours in pre-clinical testing. Approximately 60pc of patients diagnosed with synovial sarcoma die from the disease.

‘A super exciting time’

“It is incredibly motivating to see our work reaching patients so quickly as it often takes many years for laboratory-based research findings to have any tangible impact on patients,” Brien said.

“This is a super exciting time and we’re all crossing our fingers these trials are successful.”

Brien – who trained in childhood cancer research at the Memorial Sloan Kettering Cancer Center in New York and Harvard Medical School in Boston – said recent technological advances are transforming our ability to understand cancer. In research supported by Science Foundation Ireland and the Irish Cancer Society, Brien has discovered several new drug targets and has begun testing new approaches to treat different childhood cancers.

“We’re pretty excited by what we’ve found so far,” he said. “We’ve made some important discoveries and have several new approaches in testing. The new Trinity St James’s Cancer Institute is essential to integrate the laboratory and clinical sides of cancer research in Ireland.”

Colm Gorey was a senior journalist with Silicon Republic