Professors Steve Wilton AO and Sue Fletcher AO have received a major life sciences industry award from Australia’s pre-eminent industry body, AusBiotech.
The AusBiotech and Johnson & Johnson’s Industry Leadership Award is in recognition of Professors Wilton and Fletcher’s lifetime achievements in the development of novel drugs for the treatment of Duchenne muscular dystrophy and other diseases.
The scientists were amongst the first to recognise the origin of natural ‘exon-skipping’ – the ability of cells to skip over genetic errors and still function – and the potential of antisense drugs to treat genetic disorders.
Their initial focus was Duchenne muscular dystrophy, where their pioneering approach resulted in the development of three ‘exon-skipping’ medicines now approved by the US Food and Drug Administration (FDA) that overcome specific genetic mutations that are the cause of Duchenne in about 30% of cases.
Mainly affecting boys, Duchenne is an inherited disorder that causes progressive muscle weakness and loss of muscle mass. People with this disease are typically wheelchair dependent by age 12 and many do not survive beyond their mid-20s.
Although a rare disease, Duchenne is the most common of the childhood muscular dystrophies and was long considered untreatable. The drugs developed by Professors Wilton and Fletcher challenged this – and changed lives.
Rather than being wheelchair-bound, some boys on the drugs are still walking and leading normal lives, even in their early 20s. This is Australian medical science at its best – genuinely innovative research that is life-changing for patients.
The three drugs for Duchenne, developed at the Perron Institute and UWA, are being manufactured under license by the US company Sarepta Therapeutics.
Duchenne was the first step on an even longer journey as antisense drugs are being developed to treat a range of disorders. Drugs for literally dozens of rare inherited and acquired diseases are under development in laboratories in Perth, including where the duo is based, at Murdoch University’s Centre for Molecular Medicine and Innovative Therapeutics (CMMIT).
Professor Wilton leads this research centre and is also Director of the Perron Institute for Neurological and Translational Science, Western Australia’s oldest medical research institute. Professor Fletcher is also based at CMMIT and also Chief Scientific Officer of the Perth-based biotechnology company, PYC Therapeutics.
Perron Institute CEO Steve Arnott said the award, announced at AusBiotech’s annual conference in Perth, is recognition of the tremendous contribution to life sciences that Professors Wilton and Fletcher have made.
“To be truly meaningful, medical research must aim ultimately to improve the lives of patients. Sue and Steve’s research has achieved this in spades,” said Mr Arnott.
“The Perron Institute and our partner, Murdoch University, are justly proud of Steve and Sue’s achievements and look forward to the day when antisense drugs are available to treat a whole range of different diseases.”
Article source and photo credit: CMMIT, Murdoch University.