In a world first, researchers from the Victor Chang Institute have used gene-based therapy to successfully reverse an inherited heart rhythm disorder in members of an entire family, some of whom had previously been told they had only months to live.
This gene-therapy, practised as part of the new era of personalised medicine, achieved remarkable results. The research is published in the Journal of the American College of Cardiology.
Rather than using conventional heart failure medicine, the researchers identified and treated the gene mutation that caused their heart rhythm disorder, dilated cardiomyopathy (DCM).
Dilated cardiomyopathy is a condition where the heart cannot pump enough blood to the body, making it a major risk factor for stroke and heart failure. Around 1 in 2000 Australians are affected by DCM*.
Associate Professor Diane Fatkin (pictured above), lead author on the study, says the results have been outstanding, with many patients with severe disease returning to full health within 6 months.
“This is game-changing research. Whilst we can only claim to have treated one particular mutation that causes cardiac dysfunction, and there is still much more to do to find the genetic causes of heart disease in every family, this is a huge step in the right direction.”
The research was launched to coincide with the arrival of the Paceline riders, who completed an epic 1100km journey from Coolangatta to Sydney to raise awareness and funds for research into cardiac arrhythmias.
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