Personalised Mini-Organs: Revolutionising Treatment in Children with Cystic Fibrosis

Personalised mini-organs are changing the way health experts understand Cystic Fibrosis (CF), the most common inherited disorder affecting Australian children.

Professor Adam Jaffe, John Beveridge Professor of Paediatrics and Head of School of Women and Children’s Health at the University of New South Wales (UNSW) and Clinical Academic, Respiratory Medicine at Sydney Children’s Hospitals Network along with Dr Shafagh Waters, Molecular and Integrative Cystic Fibrosis (miCF) Research Centre ® laboratory group leader and senior research associate at UNSW and Sydney Children’s Hospital discussed the importance of these mini-organs in a recent interview on ABC Radio National.

“1 in 25 people in Australia unknowingly carry a mutation in a single copy of the gene that causes CF. This gene is important as it encodes for a protein that controls a chloride channel in the cell. With two copies of the mutations, the gene does not work properly, causing the airway to become really thick as it gets built up with mucous. This will then lead to an infection, respiratory failure and can ultimately be fatal.” – Professor Jaffe

Approximately 400 out of 2000 mutations in the gene causes CF – through the miCF Research Centre ®, a new Australian CF Avatar Platform uses gut and respiratory tissue from patients with CF to create mini organ models (organoids) which acts like an avatar for each child and can be tested to see how it will respond to various medicines.

“Growing organoids can be somewhat similar to growing plants – we are able to grow them in petri dishes provided with specialised media as food source, propagate them to make a lot of copies so we have enough cells to perform the tests we need to fully understand the disease.” – Dr Waters

12 years old, Ruby was one of the first patients to take part in this research, having her own organoid grown and tested to see how it responded to various medicines. This has changed Ruby and her family’s life as it allowed Ruby to take the right drug specific to her genetic built up.

“It has revolutionised her life, it really has – we have no sick days, she is like every other kid and it’s really hard as she has just started high school. Honestly this is just excellent as this is the closest thing to a cure for her.” – Shelby, Ruby’s mother

Professor Jaffe adds that though some drugs are highly effective for specific mutations in some patients, it is not always the case for many others. The high cost of personalised medicine is also a concern.

“In saying this, we hope that through the CF Avatar Platform, we are able to have appropriate patient selection to ensure best valued healthcare is being delivered especially for patients with CF.”

Click here to listen to the full interview on ABC Radio National.