Page 6 - FSANZ AUTUMN -2023

Clinical trial funding offers a beacon of
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Australia’s Medical Research Future Fund has announced $15 million in funding for a clinical trial of mitochondrial donation.
The trial will be only the second in the world to advance the technique that offers new hope for families affected by mitochondrial disease.
Around 50 Australian children are born every year with the devastating disease that is painful and debilitating severely limiting an expected lifespan, sometimes to only about five years.
Mitochondrial DNA is inherited via a mother’s egg and is responsible for helping to generate energy in all the body’s cells. If mitochondrial DNA carries variants that lead to faulty energy production, this can cause mitochondrial disease.
Mitochondrial donation aims to correct this genetic timebomb through an IVF-based technique that allows women with mitochondrial DNA disease to have children without passing on their faulty mitochondrial gene.
It involves removing the nuclear DNA from a patient’s egg containing faulty mitochondria and inserting it into a healthy donor egg, which has had its nuclear DNA removed.
The mitoHOPE (Healthy Outcomes Pilot and Evaluation) Program involves mitochondrial and clinical geneticists at Murdoch Children’s Research Institute, fertility specialists and clinical embryologists from Monash IVF working together with reproductive scientists from Monash University and the University of Adelaide.
The team also includes Monash clinical trial experts and a team of social science researchers to engage with the community. The consortium is led by Professor John Carroll, Director of the Monash Biomedicine Discovery Institute (BDI) and an expert in mitochondrial activity in egg and embryo development.
The mitoHOPE trial will be based in Melbourne, but available to eligible women all over the country to help improve their chances
of having healthy children
“Our entire mitoHOPE team is committed to making this a success for all families who have mitochondrial genetic disease,” Professor Carroll said.
The trial will be based in Melbourne, but available to eligible women from all over the country.
Professor John Cristodoulou, with Professor David Thorburn of the Murdoch Children Research Institute, will lead recruitment into the clinical trial. A national network of clinical geneticists will facilitate entry to the trial.
Monash IVF Group Medical Director and FSANZ President, Professor Luk Rombauts, said the five-year project would allow eligible women across Australia to use assisted reproductive technology to improve their chances of having healthy children.
“By introducing mitochondrial donation in the clinical IVF laboratory, we can prevent some children from suffering from this life-threatening disease and reduce the burden of mitochondrial disease on families and our health system,” he said.
Mitochondria generate energy in the body's cells
Pushing the boundaries
of current knowledge
Announcing the $15 million funding for the mitochondrial donation pilot program, the Federal Minister for Health, Mark Butler, said: “This is a positive step for all those patients and families who campaigned for Maeve’s Law.
“This funding is about increasing our understanding of the disease so we can better support women and create a brighter future for themselves and their families. The Albanese Government is funding Australia’s researchers to push the boundaries of current knowledge to deepen our understanding of health conditions so we can tackle the issues that challenge us the most.”