University of California San Francisco
Helen Diller Family Comprehensive Cancer Center

Bringing Precision Cancer Medicine to Children

By Andrew Schwartz | | April 30, 2017

Bringing Precision Cancer Medicine to Children
A UCSF initiative aims to use high-throughput sequencing of pediatric tumors – including whole genome and RNA sequencing – to identify new therapeutic targets for pediatric cancers with high death rates.
UCSF Benioff Children’s Hospitals are poised to launch a Pediatric Cancer Genome Initiative to identify new therapeutic targets for pediatric cancers with high death rates. Despite remarkable successes for many childhood cancers, cancers remain among the leading causes of death for children under the age of 18.
Led by Mignon Loh, MD, and Alejandro Sweet-Cordero, MD – both Benioff chairs of Children’s Health – the initiative builds on the existing UCSF 500 Cancer Gene Panel, which already screens tumors for the most common cancer mutations.
When the program launches this June, any UCSF pediatric cancer patient with a high-risk tumor will have the opportunity to have his or her tumor sequenced at diagnosis or relapse using the UCSF 500 panel, as well as with whole genome and transcriptome (or RNA) sequencing. The latter two tests are not yet approved for direct clinical use, but the information they yield will grow an essential database, enhance understanding of these cancers and facilitate development of future therapies. A comprehensive pediatric-focused hereditary cancer clinic – which could identify mutations that provide information for entire families – will also be an important feature of this initiative.

Genomic Information Could Clarify Therapeutic Options

“Most childhood cancers are curable, but to overcome the next hurdles, we need smarter ways to kill cancer cells,” says Loh, the Deborah and Arthur Ablin Chair of Pediatric Molecular Oncology at UCSF.
Traditionally, finding better therapies for children has been a challenge. Industry concerns about the toxicity of experimental treatments and the complexities of creating liquid formulations make it difficult to open clinical trials for children.
Loh and Sweet-Cordero believe precision pediatric cancer medicine has the potential to change that dynamic. This initiative takes a critical first step by helping to establish large databases of pediatric tumors, as well as new processes that facilitate expert interrogation of those databases. The work will not only identify new therapeutic targets, but will offer immediate clinical applications as well. Recently, Loh’s team used the UCSF 500 panel to discover a novel mutation in a childhood leukemia patient. After review at the molecular tumor board, the patient received an Food and Drug Administration (FDA)-approved small molecule with excellent response.