About Us

Ks for Kids is all about getting active and helping find answers for the 6000 childhood genetic diseases and cancers that impact 1 in 20 Aussie kids.

Children's Medical Research Institute (CMRI) is an independent research organisation with over one hundred and seventy scientists and research students working in thirteen research teams, with world-leading expertise in four main areas: 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.

Did you know?

  • 1 in 20 children face a birth defect or genetic disease
  • Cancer is triggered by genetic changes and is a leading cause of death in children aged 4–14
  • 30% of all admissions to children’s hospitals are due to genetic conditions and are the leading cause of death in children under 4
  • There are over 6000 different genetic diseases

A history of saving lives

CMRI was founded in 1958 thanks to Australia’s first telethon. This and other community support made CMRI possible, establishing Australia’s first research organisation focused on improving children’s health. Since then, CMRI has saved countless lives by:

  • dramatically improving the survival rate for premature babies
  • developing the paediatric heart and lung life support machine
  • pioneering microsurgery to repair tiny blood vessels in the heart and nerves, also making infant organ transplants possible
  • introducing rubella vaccinations
  • advocating against alcohol use during pregnancy
  • conducting early research on cystic fibrosis

The future now

CMRI launched Jeans for Genes in 1994 to fund genetic research which was at that time considered science fiction: gene therapy. Gene therapy can treat or cure genetic diseases by addressing the problem at the source—the DNA.

Thanks to community support through Jeans for Genes, CMRI is a leader in gene therapy in Australia and globally. Some achievements in collaboration with The Children’s Hospital at Westmead include:

  • 2001: Conducted the first ever gene therapy clinical trial for a genetic disease in Australia, in collaboration with the Children’s Hospital Paris, curing SCIDX1-deficiency, better known as “boy in the bubble” disease
  • 2009-11: In collaboration with the Australian pharmaceutical industry, prepared a gene transfer vector and anti-cancer drug formulation for paediatric clinical trial use
  • 2018: Successfully worked with NSW Health to have Spinal Muscular Atrophy (SMA) added to newborn screening.
  • 2019: Began Australian trial for SMA gene replacement therapy.
  • 2021 Made the first gene therapy for inherited blindness possible in collaboration with Novartis
  • 201: Established the Australian Genome Therapeutics Centre (AGTC), a collaborative effort which will transform the treatment of children with serious inherited diseases and contribute to the development of exciting new treatment options for a wide range of other diseases, including cancer, across all age groups.

CMRI’s cancer research is equally world leading. Since our discovery of the Alternative Lengthening of Telomeres (ALT) mechanism used by 15% of all cancers, we expanded our world-leading work in this field, being the first to identify the components of telomerase (a key factor in the other 85% of cancers). To date, CMRI hosts the greatest concentration of telomere researchers in the world, with their work contributing to the discovery of new and better treatments for every type of cancer, improved understanding of aging, plus better diagnosis and treatments for aplastic aenemia and other short telomere syndromes.

The ACRF International Centre for the Proteome of Human Cancer (ProCan) was established at CMRI in 2016, quickly attracting the interest of the US Cancer Moonshot program, becoming its first international member. ProCan's 7-year plan is to analyse tens of thousands of examples of all types of cancer from all over the world to develop a library of information to advance scientific discovery and enhance clinical treatment worldwide. Already, the program has made international records, analysing more than 10,000 cancer samples and establishing new methods for analysing big data, resulting in ProCan winning Highly Commended in the Data Innovation category at the 2021 Research Australia awards.