Government backing has been provided for a new project as part of the Department of Health’s $69 million funding of research to fight rare cancers and rare diseases.

Head of our Respiratory Research Unit Assoc Prof Rachel Thomson is part of a leading multidisciplinary team which plans to conduct further research into developing better treatment for an uncommon but devastating infection,  Mycobacterium abscessus.

These multi-drug resistant organisms are becoming increasingly common, particularly for sufferers of cystic fibrosis, and can result in severe lung infection in vulnerable individuals. Traditionally found in the environment and drinking water there is now evidence that they can spread between people and can survive for long periods on surfaces despite disinfection.

Current treatment options are complex, involving months of intravenous therapy, which is expensive and often poorly tolerated, meaning that patient outcomes are variable.

“As it stands, there is little current evidence base on which to determine management,” Dr Thomson says, “Our team are hoping to change this.”

On top of the impact to health, rare diseases frequently come with treatment complications for sufferers. There are significant challenges such as diagnostic delays, lack of treatment options and difficulty in finding the appropriate care.

Thanks to the Department of Health’s funding, this project will establish a platform adaptive trial that will test different drug combinations and provide evidence on which to base management by determining signatures in the blood for disease progression and treatment response.

“Once established, these clinical trials will recruit patients both nationally and internationally to enable a comprehensive evaluation of multiple treatments for people suffering from this disease,” Dr Thomson says.

CLICK HERE for more information about the work of the GMRF Respiratory Research Unit.