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Emeritus Professor Sue Fletcher and Professor Steve Wilton - changing outcomes for patients with Duchenne muscular dystrophy
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In 1996, Murdoch University’s Professor Steve Wilton AO and Emeritus Professor Sue Fletcher AO began working to develop a drug that would eventually save lives.
Amongst the first scientists to recognise natural ‘exon-shipping’ - the ability of cells to skip over genetic errors and still function – the pair created Eteplirsen, which first came to fruition in 2004.
Eteplirsen addresses a subset of specific genetic mutations that cause Duchenne muscular dystrophy in around 12 per cent of cases.
Duchenne muscular dystrophy (DMD) is a rapidly progressing disease that predominantly affects boys, with symptom onset around age four years.
The condition is relentlessly degenerative, with life expectancy of 25-30 years of age.
Although considered a rare disease, DMD is the most common of the childhood muscular dystrophies, and prior to Eteplirsen, was considered untreatable.
The journey to treating the untreatable
Professor Wilton and Emeritus Professor Fletcher’s work to design the lead morpholino compound – led to Eteplirsen being patented and licensed to rare disease pharmaceuticals company Sarepta Therapeutics as ExonDys51 ®.
In 2016, this became the first FDA-approved drug made specifically to treat DMD – and the only drug to have altered the natural history of the disease.
For the first time, boys and young men living with DMD were walking and leading productive lives, even into their early 20s.
Next, the team’s additional compounds, which addressed different subsets of DMD mutations, were approved by the US FDA.
Golodirsen (VyonDys53 ®), was approved in 2019, and Casimersen (VyonDys45 ®), was approved in 2021.
With all three compounds approved, the number of DMD patients that could be treated went from 12% up to 30%.
The implications of these discoveries were enormous and changed the lives of sufferers of DMD and their loved ones in ways that had never seemed possible.
Recognition for lifesaving discoveries
In 2021, Professor Wilton and Emeritus Professor Fletcher were inducted as Officers of the Order of Australia for their life-changing contributions.
The following year, the pair received a major life sciences industry award from Australia’s pre-eminent industry body, AusBiotech.
The AusBiotech and Johnson & Johnson’s Industry Leadership Award recognised the pair's achievements, which included the development of DMD treatments along with other drugs made to treat rare diseases.
In 2022, Emeritus Professor Fletcher was also inducted as a fellow of the Australian Academy of Health and Medical Sciences.
Today, Professor Wilton and Emeritus Professor Fletcher’s research continues to be held as some of the industry’s most groundbreaking DMD research, and as a foundation for future research.
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Emeritus Professor Sue Fletcher and Professor Steve Wilton - changing outcomes for patients with Duchenne muscular dystrophy
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