Children's Medical Research Institute (CMRI) actively conducts fundamental (meaning fundamentally important) medical and biological research. Our scientists ask the difficult questions. What causes cancer? How can we stop it? Why does development go wrong and how can we prevent this? How does the brain work? How can what we’ve learned be used to treat cancer, epilepsy, and genetic diseases?

Our task is to make the future better

CMRI scientists ask the difficult questions in order to gain the important answers. This knowledge enables us to light the spark of discovery and to create a healthier future for all children.

What we’ve accomplished in the last 25 years:

• Embarked on a world-first project to transform cancer diagnosis and personalise treatment planning within 7 years, part of the 'Moonshot' program to end cancer
• Found a single genetic defect can cause cleft lip and palate
• Fate map of the early embryo to help us understand many developmental problems
• Identification of the components of telomerase, which will be important for treating 85% of all cancers
• Discovery of the Alternative Lengthening of Telomeres (ALT) mechanism, which can lead to treatments for the other 15% of cancers
• Developing a blood test for ALT cancers that will allow physicians to diagnose and plan treatments for cancers such as aggressive glioblastoma brain tumours
• Partnering with The Sydney Children’s Hospital Network on a cure for genetic liver disease, with clinical trials about to begin
• Discovering and developing a new class of drugs for treating epilepsy

What we plan to accomplish in the next 25 years:

• Develop epilepsy treatments that will help children (and adults) around the world
• Develop new treatments for kidney disease and diabetes
• Provide more gene therapy cures for inherited diseases in children
• New treatments for infectious diseases
• Develop treatments targeting Alternative Lengthening of Telomeres, which occurs in some aggressive cancers that are currently most difficult to treat
• Telomere research to help us understand predisposition to diseases
• Match cancer patients to the best treatment available
• Find new and better treatments for every type of cancer