Children's Medical Research Institute (CMRI) has around one hundred and sixty scientists and research students, working in teams that operate thirteen research units and world-leading expertise in four main areas of research: Cancer, Neurobiology, Embryology and Gene Therapy.

Our aim is to accelerate research into children’s genetic diseases, birth defects and childhood cancer to find better treatments and cures, so Australian kids can live normal, happy lives.

HOW WE'RE FIGHTING KIDS' CANCER

One of the most important differences between cancer cells and normal cells is that cancer cells can keep on multiplying an unlimited number of times, but normal cells eventually stop dividing. This difference is one area where we have chosen to focus our attention, as it has the potential to provide an answer to one of the most problematic issues in cancer treatment – how to kill cancer cells without harming healthy cells.

It is the progressive shortening of telomeres (the DNA at the ends of chromosomes) which sets a limit on the number of times normal cells can divide. This does contribute to aging, but also acts as a powerful protection against normal cells becoming cancerous.

Cancers evade this limitation, and effectively become ‘immortal’, by switching on one of two telomere lengthening mechanisms: an enzyme called telomerase or the Alternative Lengthening of Telomeres (ALT) mechanism, which was discovered by CMRI. If blocking telomere lengthening mechanisms is to become a viable option for cancer treatment, we need a thorough understanding of both mechanisms.

PARTNERING TO SAVE LIVES

The ACRF International Centre for the Proteome of Human Cancer (ProCan®) is our other area of Focus. This international research project is located at Children's Medical Research Institute (CMRI) in Sydney Australia. It was established with a $10 million grant from the Australian Cancer Research Foundation (ACRF) and has received subsequent funding from government, charitable organisations, and members of the community. This world-first initiative was developed by Professors Phil Robinson and Roger Reddel at CMRI.

ProCan has developed the world's largest database of cancer information to advance scientific discovery and enhance clinical treatment worldwide. This database will mean doctors can more precisely diagnose cancer and effectively narrow down the best type of currently available treatment to target a patient's individual cancer diagnosis. This is personalised, precision medicine.

ProCan will provide a major step forward for cancer diagnosis and treatment.

Children’s Medical Research Institute is working with leading cancer researchers, hospitals, universities, and other institutes throughout Australia and around the world to analyse tens of thousands of samples of all types of cancer from all over the world to grow this database. And they are working with government and pathology providers to ensure the ‘ProCan test’ for cancer eventually becomes routine.

Advanced data science and software engineering is used to compare the protein data ProCan generates with the de-identified information that is already available for each cancer sample, including clinical records such as pathology test results, genetic analyses, genome sequencing, and any previous responses to cancer treatment. This proteogenomic approach to understanding cancer is also crucial to speeding up the search for new and better treatments and cures.

The data gathered continuously improves our library of information on cancers, to advance scientific discovery and personalise cancer treatments by helping doctors rapidly choose the best type of currently available treatment for their patients’ individual diagnosis. All this without having to waste weeks and months trialling medication that may prove to be ineffective for treating that type of cancer.

The information generated by ProCan will be free for anyone to access. This will help scientists all over the world studying cancer. What’s more, the library of information created will be a point of reference that can be used now and, in the future, to aid rapid diagnosis and treatment planning.

ProCan was invited to join the ‘Cancer Moonshot’ initiative led by the United States’ Government, and this agreement was renewed in 2022. ProCan is also part of a project involving twenty countries called Individualised Paediatric Cure (iPC).

CMRI CANCER RESEARCH MAJOR ACHIEVEMENTS:

2023 NSW State Government Department of Health stated that ProCan’s database could save billions in healthcare costs.

2023 CMRI established an additional cancer lab, called ‘Nuclear Dynamics’ which is led by Dr Noa Lamm-Shalem whose aim is to better understand how cancers become resistant to chemotherapy and identify new vulnerabilities to target with new treatments.

2023 Dr Liz Conelly, a medical oncologist in CMRI’s ProCan team, has surveyed all available information about using proteomics to analyse sarcomas—difficult to treat cancers affecting children—and has outlined the way forward to make this useful in the clinic.

2022 Project launched by Dr Samantha Ginn of at CMRI to develop gene therapy treatments for Neurofibromatosis type 1 & 2, which causes nerve-associated tumours.

2022 ProCan completed the world’s largest database for predicting cancer treatment response based on proteins in cancer cell lines, with a panel of 949 cancer cell lines representing every type of cancer analysed.

2021 The ProCan team is a finalist in the 2021 Research Australia Health and Medical Research Awards for Data Innovation.

2020 ProCan achieved a world-first by developing a technology that could reliably analyse the proteins in cancer samples on a scale never seen before. The results were published in the prestigious scientific journal Nature Communications.

2020 A team of Australian scientists, including CMRI’s Prof Phil Robinson, became the first in the world to discover a way to target leukaemic cancer cells that are able to “hide’’ from chemotherapy and cause the cancer to relapse. Targeting these cells has the potential to cure leukaemias.

2019 ProCan was the first proteomics program in the world to analyse the proteomes of 10,000 human cancers.

2018 CMRI’s A/Prof Tony Cesare mde a ground-breaking discovery around regulation of telomere loop structure, which has major implications for conditions ranging from cancer to aging and heart disease.

2016 ProCan was established with a $10 million grant from the Australian Cancer Research Foundation (ACRF). ProCan is a world-first initiative developed by Professors Phil Robinson and Roger Reddel which launched in September 2016.

2015 CMRI discovered that cancer cells which switch on Alternative Lengthening of Telomeres (ALT) by losing ATRX gene function are much more fragile than normal cells when exposed to various forms of stress. We are using this knowledge to design treatments that will kill ALT-positive cancers with minimal side effects on normal cells.

2014 CMRI identified a gene that causes inherited bone marrow failure and uncovered a key anti-cancer target for ALT cancers - some of the most aggressive types.

2013 CMRI demonstrated that ALT has a normal counterpart in cells and developed a new model system for studying the ALT mechanism.

2012 Our scientists were involved in an international study identifying key genetic change (loss of ATP-dependent helicase) in ALT.

2011 We licensed C-circle assay for research use as a test for ALT cancers, helping identify the disease in kids easier and earlier.

2009 Our scientists developed C-circle assay for measuring ALT activity in cancer and discovered a telomere trimming mechanism that could one day be exploited to target and kill cancer cells.

2007 We were the first in the world to identify composition of active telomerase enzyme complex in human cells.

2000 CMRI demonstrated that ALT involves DNA recombination, becoming the first in the world to show this underlying mechanism.

1999 Our scientists found a diagnostic marker for ALT, called APBs, and also showed that unknown factors in normal cells can repress ALT cancers.

1995 We discovered ALT mechanism of telomere maintenance in cancer, which created an entirely new field of research.

For more information on CMRI, visit www.cmrijeansforgenes.org.au.