Paediatric Precision Medicine

Project Overview

Paediatric Precision Medicine (PPM) will transform children’s health care in New South Wales (NSW) by bringing precision medicine to all children with cancer and rare genetic diseases. By leveraging game-changing technologies and integrating research with clinical care, it will enable children across the state access to cutting-edge treatments and truly personalised care, no matter how rare or complex their condition.

The Luminesce Alliance Paediatric Precision Medicine program is made up of 6 streams across pre-clinical, clinical and health systems research, which together will build capacity and enable new technologies to be integrated into personalised clinical care.

What is precision medicine?

Everyone of the nearly 8 billion people living on planet Earth is unique, a one-off combination of our genes and our personal history. What if our healthcare could be tailored to our individual needs, preventing disease, or responding with the best possible treatment, at the optimal dose, first time?

Precision medicine allows healthcare to be finely tuned to each individual. It takes into account a person’s genes, behaviours, and environment and the role these factors have in a person’s health, disease, or condition. This may also be referred to as personalised medicine.

By combining this knowledge and information, doctors and researchers will be able to accurately predict which treatments will be most effective and safe, or possibly how to prevent the illness from starting in the first place. Properly implemented, precision medicine has the potential to shift the focus of the health system from the treatment of illnesses to the protection of health.

How can precision medicine improve the health of children?

Admissions to tertiary paediatric hospitals in NSW are dominated by genetic conditions (about 40-50% are estimated to have a major genetic contributory factor) and around 10-15% of childhood cancers appear to be associated with an underlying genetic predisposition. New forms of screening can identify, prevent or manage otherwise undetected diseases and enable a better quality of life for children and families, and lower costs to the healthcare system.

The majority of drugs used today in the treatment of childhood cancers, for example, are generally cytotoxic drugs that also affect normal tissues. The lack of specificity of these drugs in targeting cancer cells results in adverse side effects and significant healthcare costs. Importantly, for those children who survive their disease, 30% will suffer one or more life-altering on-going health conditions as a result of the toxicity of current treatment protocols and often with associated socioeconomic and psychosocial consequences.

Precision medicine can help us better understand the biological and genetic basis of diseases and develop new and improved treatments delivering the right drugs in the right dose to the right patient at the right time. This will reduce the burden of disease and improve long term outcomes overall.

Summary

Paediatric precision medicine supports the development of new strategies for prevention, diagnosis and treatment, thus improving outcomes for treatment and their families.