Genetic screening of children with tumours can point to unexpected treatments, according to the findings of a UK project.
A small number of children were treated with drugs designed for adults as a result of the screening project, led by the Institute of Cancer Research London.
Researchers said that they faced regulatory problems which meant they were only able to provide a minority of children treatments that were identified through the testing.
More than 200 children took part in the trial and researchers said that half had gene mutations that could be treated with existing adult cancer drugs. Researchers studied 91 genes linked to the growth of cancer.
Just 7% of these children went on to receive treatments, the researchers report in the European Journal of Cancer.
These included three children with BRAF gene mutations, which were treated with the drugs dabrafenib and trametinib. The children all benefitted from their disease being held in check for periods of between nine and 15 months.
Researcher Dr Sally George said: “By testing tumours for specific gene mutations, we have shown it’s possible to identify new smarter, kinder treatment options for children, which may potentially give these patients much longer with their families after conventional therapies have failed.
“But our study also exposes the desperately frustrating barriers that children still face in receiving new treatments – barriers which lie in the regulations controlling how drugs for children are developed and approved.”
Fellow researcher Professor Louis Chesler, a paediatric oncologist, said: “In future, I want to be able to treat more children whose tumours have these targetable mutations with better drugs, as currently not all children have access. But gathering the molecular data is the first practical step to making this possible.
"This data, and more that we are continuing to collect, will be good evidence to more clearly guide use of the most appropriate drug for each child."
George SL et al. A tailored molecular profiling programme for children with cancer to identify clinically actionable genetic alterations. European Journal of Cancer 19 September 2019
https://www.ejcancer.com/article/S0959-8049(19)30446-0/fulltext
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