Curing Childhood Cancer – a Global Approach to Improve Cancer Treatment
July 12, 2018
This week in the United States, a database of genetic information will be made publicly available of more than 270 patient-derived cancer models, encompassing 25 different childhood cancers. Assisted by funding from the Australian Federal Government Department of Health, Children's Cancer Institute contributed 90 leukaemia models to this global effort.
Although childhood cancer is relatively rare, it is the leading cause of death from disease in young children. The low numbers of childhood cancer patients who are available for clinical trials of new drugs means that it is essential to carry out extensive laboratory testing of new drugs (called preclinical drug testing) so that clinical researchers can better prioritise which drugs to pursue in paediatric clinical trials.
Children’s Cancer Institute in Sydney is a world leader in the development of patient-derived xenograft (PDX) models and their use for preclinical drug testing. These PDX models allow researchers to test known and novel drugs against childhood cancer samples in the laboratory to measure their effectiveness as treatments for individual children, opening up new options for access to novel drug clinical trials. It is essential that these PDX models undergo extensive molecular characterisation so that researchers can try to exploit their genetic vulnerabilities with new treatments.
The Children’s Cancer Institute team, headed by Professor Richard Lock, is the only non-US member of the Pediatric Preclinical Testing Consortium (PPTC). Funded by the National Cancer Institute, part of the National Institutes of Health in the US, the PPTC’s goal is to produce reliable, preclinical drug testing data using paediatric cancer PDX models. The Pediatric Preclinical Genomic Characterization Project is an effort to characterise those childhood cancer PDX models that lack full genomic data so that they can be used to test for better treatments and cures.
This week in the United States through the Baylor College of Medicine (Texas) and Nationwide Children’s Hospital (Ohio), a database of genetic information of more than 270 PDX models across 25 different childhood cancers, will be made publicly available by the PPTC so that researchers globally can hunt for genetic vulnerabilities in these cancers that can be targeted with new drugs and treatments. The data is available on the PedcBioPortal for Childhood Cancer Genomics (pedcBio portal) and raw characterization data is available on the database of Genotypes and Phenotypes (dbGaP).
Professor Lock and his team contributed 90 acute lymphoblastic leukaemia (ALL) child patient derived PDX models to this combined effort of more than 270 PDXs. ALL is the most common childhood cancer and 48 of the PDX models (including leukaemia, brain cancer and neuroblastoma) were sequenced using Australian Federal Government Department of Health funding awarded to Zero Childhood Cancer in November 2017. This funding was to support the development of a preclinical testing platform for the Zero Childhood Cancer personalised medicine program for children with high-risk cancer. Sequencing of the remaining xenograft models was possible due to additional gift funds to the PPTC from the Alex’s Lemonade Stand Foundation in the US.
According to Professor Lock, “the Australian Federal Government Department of Health funding will vastly improve our capacity to identify new targets in childhood cancer to develop and test novel individualised treatments and help to cure the disease”.
About Alex’s Lemonade Stand Foundation
Alex’s Lemonade Stand Foundation (ALSF) emerged from the front yard lemonade stand of cancer patient Alexandra “Alex” Scott (1996-2004). In 2000, 4-year-old Alex announced that she wanted to hold a lemonade stand to raise money to help find a cure for all children with cancer. Since Alex held that first stand, the Foundation bearing her name has evolved into a national fundraising movement, complete with thousands of supporters across the country carrying on her legacy of hope. To date, Alex’s Lemonade Stand Foundation, a registered 501(c)3 charity, has raised more than $150 million toward fulfilling Alex’s dream of finding a cure, funding over 800 pediatric cancer research projects nationally. For more information on Alex’s Lemonade Stand Foundation, visit AlexsLemonade.org.
About Children’s Cancer Institute
Originally founded by two fathers of children with cancer in 1976, Children’s Cancer Institute is the only independent medical research institute in Australia wholly dedicated to research into the causes, prevention and cure of childhood cancer. Forty years on, our vision remains unchanged – to save the lives of all children with cancer and to eliminate their suffering. The Institute has grown to now employ more than 220 researchers, operational staff and students, and has established a national and international reputation for scientific excellence.
Our focus is on translational research, and we have an integrated team of laboratory researchers and clinician scientists who work together in partnership to discover new treatments which can be progressed from the lab bench to the beds of children on wards in our hospitals as quickly as possible. These new treatments are specifically targeting childhood cancers, so we can develop safer and more effective drugs and drug combinations that will minimise side-effects and ultimately give children with cancer the best chance of a cure with the highest possible quality of life.
We are currently leading the establishment of the Zero Childhood Cancer national child cancer personalised medicine program for children with the most aggressive cancers, in partnership with the Sydney Children’s Hospitals Network. This program will revolutionise the way treatment decisions are made, with the aim of improving survivorship for those children at highest risk of treatment failure from their disease.