miCF AVATAR: Personalised Lung and Intestinal Mini-Organs to Predict the Effect of Cystic Fibrosis Therapies in Individual Patients

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1. Please give a summary of your research.

Cystic Fibrosis (CF) is a fatal, chronic disease linked to more than 2000 different mutations in the CFTR gene, a chloride channel. In the 1950s, children with CF were not expected to survive beyond 5 years of age. Advances in symptom management have delayed disease progression. The median age of death in Australian CF patients remains 31.6 years with poor quality of life beginning in early infancy.

Breakthrough drugs, CFTR modulators, have transformed therapeutic options for some CF patients. Improving a patient’s quality of life as well as their life expectancy to over 50 years. Modulators directly correct the dysfunctional chloride channel. However, due to the wide range of CFTR mutations it is impossible to develop a “one size” fits all treatment.

60% of the CF population - with common CFTR mutations - are currently eligible to receive approved modulators. Since we all respond differently to medications, not all of these patients will respond positively to the drug. Currently, no clinical test exists to predict who may or may not respond to the drug. Meanwhile, the remaining 40% of the CF population - with rare CFTR mutations - are left behind. The cost of modulators ($250,000/patient/year) makes their provision without evidence of efficacy economically unattainable. 

We are using a personalised mini-organ platform to predict individuals that are responsive to the CF modulator drugs. 

These mini-lung and guts act like an ‘AVATAR’ for a person. We test the CF patients AVATAR to find out the effectiveness of different therapies. When the treatment is successful in fixing the dysfunctional chloride channel in that CF patient, their AVATAR will swell. Distinguishing patients responsive to treatment from those who are non responders. Should one or more therapies prove effective in their AVATAR, they can be recommended for use as targeted therapies for the patient.

2. Please include any additional details you would like to share

Script from the video clip:

1: Only you are you, and your genes are unique to you.

2: When it comes to medication, everyone responds to drugs differently.

3: To put it simply, one size does not fit all.

4: The best practice is to match medications to one’s genetics. This is called personalised medicine. One way to find the perfect fit is by testing medications on cells collected from that person.

5: In cystic fibrosis (or CF), an inherited chronic and progressive disease, with symptoms from infancy, these cells are collected from the inside of a patient’s own nose, lungs or gut

6: The cells are then grown to form mini-organs.

7: These mini-organs act like an ‘avatar’ for a person. They can be expanded in the lab, so that millions of copies are available for different drugs to be tested on.

8: At the miCF Research center, we are using the miCF AVATAR platform to find the best fit medication for our patients with CF.

9: When the treatment is successful, the patints AVATAR will swell.

We can then determine which treatment works best on a patient’s avatar and from there suggest a personalized therapy.

 This approach will take the guesswork out of the game, resulting in improved patient care.

10: So join us, and invest in a future free from Cystic Fibrosis 



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Im a scientist at miCF Research Centre at UNSW. Our team's vision is to integrate science and medicine; to bring about next generation patient care; personalised medicine. Our team members include...

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