Funding to help sufferers of rare diseases

November 5, 2014

Murdoch University researchers from the Centre for Comparative Genomics (CCG) have received almost $800,000 in funding from the National Health and Medical Research Council (NHMRC) to develop genetic drugs to treat rare diseases.

Chief Investigator Professor Steve Wilton and Investigator Professor Sue Fletcher have previously had success in developing a therapy for people with Duchenne Muscular Dystrophy (DMD) and the new funding will allow them to extend their research to other rare conditions.

Over the last decade Professor Wilton and Professor Fletcher developed a new drug for DMD sufferers that works by “skipping over” the faulty part of the gene message, so that is can produce a functional version of the protein dystrophin that stabilises the muscle fibre.

“Clinical trials of the drug we developed for Duchenne Muscular Dystrophy have yielded some remarkable results,” Professor Wilton said.

“We believe we can use the same technology that we applied in development of the drug for DMD to treat other rare diseases such as cystic fibrosis, spinal muscular atrophy and Pompe’s disease.”

The (NHMRC) grant is worth $792,656 over four years starting in 2015.  Also on the research team are Director of the CCG, Professor Matthew Bellgard and Director Genetic Services & Familial Cancer Program of WA, Professor Jack Goldblatt.

Professor Fletcher said that it was crucial that universities researched drug development for rare diseases because the small number of patients requiring specific treatments is a disincentive to investment in therapy development.

“When you add up all the people in Australia suffering from a rare disease there are at least 400,000 which is actually more than the number of people living with cancer,” Professor Fletcher said.

“Yet despite the large number of people afflicted, there has been relatively little research on treatments for rare diseases.  Within Australia, WA Health has been leading the drive to help those living with a rare disease and there is also increasing community awareness and advocacy, driven by community groups.”

Professor Wilton and Professor Fletcher’s breakthrough treatment for DMD was recognized when they were awarded one of nation’s most prestigious awards last year, the Australian Museum Eureka Prizes, the NSW Health James Callachor Eureka Prize for Medical Research Translation.

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