MDA AWARDS $368K TO AUSTRALIAN RESEARCHER ADVANCING EXON SKIPPING STUDIES

TUCSON, Ariz., Aug. 18, 2010 – Fourteen years ago, Professor Steve Wilton had a revolutionary idea:  Why not use “molecular scalpels” to excise the flawed parts of a gene message, the way a surgeon removes diseased tissue from the body?  At the time, the idea sounded far-fetched, even to many scientists.  “I heard the words ‘party trick’ used,” recalled Wilton, head of the Molecular Genetic Therapies Group at the University of Western Australia. 

However, Muscular Dystrophy Association scientific advisors embraced the novel idea and today, Wilton’s technology, termed exon-skipping, is one of the best hopes for a therapy for Duchenne muscular dystrophy and several other genetic-based diseases.

Coming on the heels of more than a decade of almost continual funding from MDA, Wilton has been awarded a new three-year $368,100 MDA grant to further improve exon-skipping technology. The new project also will test the suitability of a “next generation” form of Wilton’s genetic band-aids, known as methoxyethyl oligomers (MOE).

Professor Steve Wilton

The technology uses high-tech molecules called anti-sense oligonucleotides.  Wilton likes to call them “genetic band-aids” and he uses them to trick the body’s gene-transcript processing machinery to “skip over” the flawed parts of the dystrophin pre-mRNA that lead to Duchenne muscular dystrophy, enabling muscles to produce nearly fully-functional dystrophin protein in some cases.  “It’s been very gratifying to go from a concept that we’ve demonstrated in vitro to completion of a clinical trial. It seems like it has taken forever, but in drug-development terms it is very quick,” explained Wilton.

Rapid progress should not be a surprise for this affable scientist who stays grounded by traveling the globe to meet families affected by muscular dystrophy as he keeps them and the scientific community informed about his pioneering efforts.

“I’d estimate I meet with about 1,000 Duchenne families a year from around the world,’’ said UWA Professor Wilton from his laboratory in Perth. “It’s a way of keeping very, very motivated to keep driving this work forward.’’

“Steve Wilton is a true champion for exon skipping as a therapeutic approach,’’ said R. Rodney Howell, M.D., Chairman of the MDA Board of Directors. “We’re optimistic that exon skipping could ultimately yield valuable treatments for a number of diseases.”

Dr. Wilton is one of 38 research leaders now receiving multi-year awards from MDA. His project is part of the $14.1 million in new research funding approved during the Association’s July board meeting.  This is his fifth MDA grant, and he’s previously tested other antisense oligomers that induce exon skipping in Duchenne muscular dystrophy. 

“I’ve been very lucky.  Essentially, I’ve been funded by the MDA for the exon-skipping work since the late 1990s,’’ Dr. Wilton said. “Without MDA’s support we would have not gotten as far as we have.”           
To learn more about Dr. Wilton and his current project, listen to his podcast.  

Research grant applications are peer-reviewed twice yearly by MDA’s Medical and Scientific Advisory Committees, comprising world-renowned experts in neuromuscular disease research.  The most promising of some 500 applications received each year are recommended for funding to the MDA Board of Directors.