Finding cures for children's genetic diseases


Research Explained

CMRI conducts both basic science research and medical research —
What does that mean?

Basic Science

This is the foundation for understanding everything. Thanks to basic science, we know how blood cells carry oxygen so we can breathe; we know that DNA has all the information needed to make an entire person; and we know how to transform an unwanted infectious virus into a cure for an inherited disease.

'Basic' clearly doesn't mean unimportant.

In fact, basic science research is the crucial first step towards understanding how the body works, what goes wrong in diseases like cancer, and how to fix it. Today's medicines and tomorrow's revolutionary new treatments depend upon it.

Medical Research

While all research relies on basic science, CMRI's research has the goal of helping to improve the health and wellbeing of people, especially children. That means it is often classified as 'medical research'.

Much of CMRI's research over the last 55 years has become a part of standard medical practice (see Our History and Achievements).

What's more, we have many research programs ongoing right now that have advanced beyond discovering the crucial basics and are producing tangible benefits: 
  • Our drug development programs are perfecting new and better treatments for cancer, epilepsy and other diseases.
  • The Gene Therapy Research Unit (a joint initiative with The Children's Hospital at Westmead), conducted the first ever gene therapy clinical trial to cure a genetic disease, and they have several clinical trials underway to help children with cancer and rare diseases.
  • The newly-established Telomere Analysis Centre can help clinicians better diagnosis telomere-related diseases, like DKC, which can cut short the lives of children as young as 6-years-old.

Why Does it Take So Long?

Research is a very long and expensive process, and many people don't understand why it takes so long. Why can't we find a treatment for our loved one right now?

We wish it were instantaneous, or even easy. But 'research' by definition means we don't know the answer. Sometimes we don't even know the right question to ask. And, often, technological advances in microscopes and other equipment are needed in order to make the advances needed.

Finding a cure for cancer is like going to the moon. A massive, international effort, involving scientists, engineers, new technology and specialised know-how, is required to reach the goal we have so clearly in our sights. But that goal is achievable.

Learn More

Use the links on this page to learn more about the science behind our main research areas: cancer, embryology, neurobiology, and gene therapy. Also visit our Science Education section for fun educational activities for kids and links to other learning resources.