Children with incurable brain tumors could benefit from potentially life-extending treatment if genetic testing was used to personalize therapy as it is in many adults, major new research reports.

Scientists analyzed the DNA of children taking an adult cancer drug on a clinical trial deemed to have ‘failed,’ and found that many with particular genetic traits had actually responded well to treatment.

Some of these children survived more than a year longer than others on the trial.

The international study—led by a team at The Institute of Cancer Research (ICR), London, and involving 51 centers in 14 countries—found that children whose tumors had mutations in the MAPK network of genes benefited from Avastin (bevacizumab) alongside standard treatment.

In these children, Avastin also appeared to cause immune cells to flood in to help destroy their tumors—raising the possibility that they could be good candidates for future immunotherapy.

Benefits of testing children for genetic mutations

Children’s aggressive, or ‘high grade,’ brain tumors are currently treated as one disease, but a recent genetic analysis by the same team at ICR showed there were actually at least 10 different diseases.

The new research shows the benefits of testing children for genetic mutations in their tumors to make sure they receive the treatment most likely to work.

Increasingly, treatment of adult cancers is shaped by genetic testing, but children’s cancer continues to lag behind.

The new independent academic study—published today in the prestigious journal Cancer Cell and funded by Roche—analyzed genetic, molecular, and immunology data from the HERBY phase 2 clinical trial after it had been completed.

The researchers will now look to confirm the findings in a clinical trial set up specifically to test the effectiveness of Avastin in children with these mutations. If successful, it would open up a whole new treatment option for a disease with very few effective therapies.