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Support for fast-tracking new drugs into clinical trials for childhood acute leukemia

Children's Cancer Institute Australia

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IMAGE: Professor Richard Lock is Head of the Leukaemia Biology Program at Children's Cancer Institute. view more

Credit: Children's Cancer Institute

Children's Cancer Institute welcomes its inclusion, by the US National Cancer Institute (NCI), in a systematic program of drug evaluation in childhood cancer.

Children's Cancer Institute, under the direction of Principal Investigator, Professor Richard Lock, Head of the Leukaemia Biology Program, has been awarded close to US$2 million for 5 years under the Pediatric Preclinical Testing Consortium (PPTC), created by NCI to prioritise and fast-track new drugs into clinical trials in children with aggressive cancers.

Professor Lock will be testing drugs in preclinical models of acute lymphoblastic leukaemia (ALL), the most common childhood cancer. He previously received funding from NCI for 10 years as part of the Pediatric Preclinical Testing Program (PPTP), and was the only PPTP Principal Investigator located outside the USA. His invitation to become a member of the newly-formed PPTC attests to the value NCI places on his contributions to-date. PPTC will consist of 5 testing sites; 4 located within the United States as well as Children's Cancer Institute in Sydney.

Using their respective models of childhood cancer, the 5 PPTC sites will each systematically test drugs that are considered to have the potential to improve the treatment of childhood cancer, facilitating acceleration of the most effective drugs into clinical trials.

Professor Lock is extremely gratified that Children's Cancer Institute is seen as a world-leader in preclinical drug testing for ALL. "Our research proposal underwent a rigorous competitive review process, and our inclusion in PPTC is substantial recognition for our work," he said.

"I feel honoured to have been involved with NCI for 10 years, and am delighted to be able to continue this work with their support.

"NCI is not only supremely rational and systematic in its approach, it has brokered a ground-breaking agreement between academia and members of the pharmaceutical industry that ensures all results are published, including negative results.

"It's very important to identify inactive drugs as well as active drugs - because every time you evaluate an inactive drug in a child with cancer, it could prevent the evaluation of an active drug.

"Through the PPTP, NCI has also raised the bar in terms of stringency in preclinical drug testing, ensuring that testing criteria are as close to the clinical setting as possible.

"An unbiased approach is taken, and drugs that don't meet very stringent measures of response are discarded.

"It's already possible to match some drugs with specific molecular signatures in tumours. In future, we hope to be able to target every tumour with exactly the right therapy."

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