A groundbreaking clinical trial conducted by Canberra Health Services researchers, Professor Walter Abhayaratna and Dr Edwin Lee, offers hope to those fighting a rare heart condition.

ATTR amyloidosis is caused by abnormal deposits of a protein called TTR in heart muscles.  The diagnosis of the life-threatening condition, leading to heart failure, has been on the increase.

No specific treatment has been available to date. Recent advances in medical research are about to change that.

MAGNITUDE, a phase 3 clinical trial led by Professor Walter Abhayaratna at Canberra Health Services aims to halt the disease progression in one single dose of treatment.

Professor Abhayaratna describes the treatment as a 'potential game-changer'.

'MAGNITUDE is leading the way in gene-based technology for this heart condition known as ATTR-CM,' he explains.

'This innovative treatment targets a specific protein called transthyretin (TTR). It uses gene-based technology to turn off the abnormal gene responsible for producing too much TTR protein.'

'We're testing this therapy in a clinical trial where participants are randomly assigned to receive the treatment or not. The goal is to recruit more participants and speed up research.'

'The overall aim is to reduce new TTR protein production by over 90%. If successful, this could significantly improve treatment for people with this heart condition,' Professor Abhayaratna said.

The study participants will be followed for a minimum of 18 months to allow for a comprehensive evaluation of long-term effects.

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