Study Highlights The Value of Precision Medicine for Childhood Cancer

According to a story from BioSpace, a recent study of cancer in children is emphasizing the fact that genetic testing is an essential component of cancer treatment, providing critical information that will allow caregivers to choose the most effective treatment options. While the study revealed that a significant portion of pediatric patients could see significant benefit from the use of targeted therapies, only a small number of patients had ready access to these drugs.

Genes and Precision Medicine for Pediatric Cancer

The study looked at genetic data from 200 children with some form of cancer. The results were remarkable as almost half of the patients carried mutations that could be targeted by cancer drugs available for adults. These drugs were either being tested in clinical trials or were already approved. Ultimately only a small number of the patients in the study went on to get the recommended treatments as revealed by genetic analysis. However, those that did saw significant benefit. 

As only seven percent of the child patients had access to targeted therapies, the study also highlighted the fact that funding and regulatory obstacles were preventing these patients from getting the most effective therapies (many targeted drugs are currently only approved for adults, as an example).

Study Process

The study was organized by the The Royal Marsden NHS Foundation Trust and The Institute of Cancer Research, London. The research process involved the use of a gene panel test. This test is designed to read sequences of DNA found in 91 genes that are known to facilitate the growth of cancer tumors. There were several genes that were mutated at higher rates than the others. The most common genes affected were CTNNB1, CDKN2A, and ATRX (12 patients), followed by MYCN (11 patients) and PI3K3CA (10 patients). 

The study also looked at gene tests for eight patients from when they were diagnosed and after they had begun treatment. Six of this child patients saw genetic changes to their cancers, developing new mutations as the cancer responded to treatment. The authors state that future research will look more extensively into how cancer tumors change genetically over time as they are exposed to different therapies. The scientists also hope to conduct a study of children with brain cancer using the cerebral-spinal fluid in order to identify potential drug targets.


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