2013 NSW Health Jamie Callachor Eureka Prize for Medical Research Translation

Helping children walk.

Children with a deadly muscle-wasting disease are regaining the ability to walk and potentially avoiding life-threatening complications, thanks to a new treatment developed by researchers at Perth’s Murdoch University.

The treatment, developed by Murdoch Professors Steve Wilton and Sue Fletcher, targets the genetic defect responsible for Duchenne muscular dystrophy, a condition which affects around one in 3500 boys.

For bringing their drug from laboratory bench to bedside, Professors Wilton and Fletcher have won the 2013 Australian Museum NSW Health Jamie Callachor Eureka Prize for Medical Research Translation.

Their work gives hope for sufferers of the previously incurable disease, and leads the way for personalised genetic therapies for conditions such as spinal muscular atrophy, cystic fibrosis, progeria and thalassaemia.

“This is a radically new treatment that is already improving young lives,” the Director of the Australian Museum, Frank Howarth said. “The best measure of success for a treatment is how it benefits patients, and it’s exciting to hear that they’re seeing real benefits to children in their phase 2 trial,” he says.

Professors Wilton and Fletcher found a way to skip mutations in the genes associated with Duchenne muscular dystrophy. The disease only attacks boys, leaving them in a wheelchair by the age of 12 and usually dead by 19. The new novel drug, eteplirsen, is in the second year of a Phase 2 clinical trial, and is greatly improving patients’ muscular function, as measured by their ability to walk.

The two other finalists for the prize were chosen for a system for detecting hearing loss in young children, and a diagnostic tool for leukaemia relapse.

Associate Professor Robert Cowan of the HEARing CRC and HEARworks Pty Ltd, and Adjunct Professor Harvey Dillon of the National Acoustic Laboratories, have developed HEARlab®, a computerised tool that non-specialists can use to detect hearing loss in infants and young children.

Professors Michelle Haber and Murray Norris of the Children's Cancer Institute Australia and Professor Glenn Marshall of the Sydney Children's Hospital have developed a sensitive test for leukaemia that has halved the relapse rate in children at highest risk.

The Australian Museum Eureka Prizes reward excellence in the fields of research and innovation, leadership and commercialisation, science journalism and communication, and school science. This year the 17 sponsored prizes include awards for agriculture, defence, infectious diseases and innovative use of technology.

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Kea Lambert , Project Officer, Eureka Prizes
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