Fast-tracking new treatments into clinical trials for children with cancer

06 Sep 2021

Australian researchers have been awarded more than AUD$3 million by the USA’s National Cancer Institute (NCI) to test new treatment drugs for children with cancer over the next five years, so the most promising of these can be fast-tracked into clinical trials.

The Leukaemia Biology Group at Children’s Cancer Institute is the only non-US based research team to be included in this NCI-funded initiative, known as the Pediatric Preclinical In Vivo Testing Program (PIVOT). The goal of the PIVOT program is to accelerate the testing and approval of new childhood cancer drugs, since of the approximately 200 agents approved by the US Food & Drug Administration (FDA) for cancer treatment, fewer than 40 are approved for use in children.

“The vast majority of cancer treatments available for adults aren’t approved for use in children,” explains Professor Richard Lock, Head of the Leukaemia Biology Group at Children’s Cancer Institute. “Added to this, there are very few treatments that have been developed especially for children with cancer, and it can take several years before these become available in the clinic.”

The PIVOT program aims to improve this situation by connecting pharmaceutical companies that are developing new cancer therapies with drug testing centres that have the capacity to test these therapies in specialised models of childhood cancer.

Professor Lock and his group are known for developing the world’s most clinically relevant laboratory model of the most common cancer in children, acute lymphoblastic leukaemia (ALL), and are solely responsible for the ALL component of the PIVOT program. While the PIVOT program is new, Professor Lock’s group has more than 17 years’ experience working with several other testing centres involved in the PIVOT consortium on the testing of novel agents against paediatric cancer.

The main type of model used by the testing centres participating in the program is known as a patient derived xenograft, or PDX. This is a living model of disease, usually based on laboratory-bred mice growing human cancer cells − cells that have been taken directly from a patient with cancer.

“Our living model of ALL closely mimics the disease in children, making it possible to carry out meaningful experimental work without involving children,” Professor Lock explains. “By testing potential new treatments in our model, we can make sure that only safe and effective drugs proceed to testing in children, and we can prioritise those drugs that our tests show are the very best candidates.”

Media Contact

Tami Bradley
Children's Cancer Institute

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. More than 40 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 nearly 300 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 minimize side-effects and ultimately give children with cancer the best chance of a cure with the highest possible quality of life. More at