Gene linked to brain defect could point to a new brain tumour drug target

Research into a rare genetic disease could help scientists understand a type of brain tumour, according to a US study.

Seckel syndrome can cause microcephaly, a condition in which infants are born with an abnormally small head.

And researchers at the University of North Carolina Lineberger Comprehensive Cancer Centre in the US have linked the way this genetic condition limits brain growth to how a particular type of tumour, called medulloblastoma, develops.

One form of Seckel syndrome is caused by faults in the ATR gene, which cause inadequate brain growth.

As medulloblastoma causes part of the brain to continue growing, and Seckel syndrome limits brain growth, researchers wanted to see whether one could cancel the other out.

Professor Tim Gershon, an author on the study, said that by understanding how Seckel syndrome limits brain growth, researchers can adapt the process to tackle brain tumours.

They found that targeting the ATR gene in mice could help slow the growth of medulloblastoma, according to the study published in the journal Development.

This suggests that ATR is important for the medulloblastoma cells to survive.

The researchers said more work is needed to understand how safe the approach might be in people.

Dr Simona Parrinello, a Cancer Research UK expert on brain tumours, said: “This interesting study in mice with medulloblastoma is especially striking and suggests that blocking ATR might be an effective strategy for treating this type of tumour.

“What is particularly intriguing is that blocking ATR also kills cells with mistakes in a gene called p53, which is frequently faulty in cancer.

“So this approach may be more effective in targeting a broader range of cancer cells than other chemotherapy drugs.”