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 30 years:

• Embarked on a world-first project to transform cancer diagnosis and personalise treatment planning, 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 and other telomere research, which will be important for treating 85% of all cancers.
• Discovery of the Alternative Lengthening of Telomeres (ALT) mechanism and developing an in depth understanding which can lead to treatments for the other 15% of cancers.
• Partnering with The Sydney Children’s Hospital Network on a cure for genetic liver disease, with clinical trials happening in the UK.
  
• Discovering and developing a new class of drugs for treating epilepsy.
• Introducing dozens of tests for genetic eye diseases and enabling Australian-first gene therapy treatments.

What we plan to accomplish in the next 30 years:

• Provide more gene therapy cures for genetic disorders in children. Over 6,000 genetic diseases and other conditions could one day be cured with gene therapy.
• New epilepsy treatments that will help children (and adults) around the world.
• Precision medicine to better understand and treat birth defects and genetic disorders.
• Develop regenerative medicine that could one day enable lab-generated healthy cells or organs to be used for transplantation.
  
• Help clinicians match cancer patients to the best treatment available through our ProCan technology.
• Find new and better treatments for every type of cancer with our telomere and genome biology research, including 'broad spectrum' cancer treatments that could work on 95% of all cancers with fewer side effects.