Children living with dementia have the same symptoms as adults with dementia, they can experience memory loss and face a gradual decline in health, with most not reaching adulthood.
There are around 700,000 children living with childhood dementia around the world, which is caused by more than 70 rare genetic conditions.
However, a new research effort involving the Centre for Molecular Medicine and Innovative Therapeutics (CMMIT) at Murdoch University offers hope to those families, with scientists exploring whether genetic ‘whiteout’ might be an effective therapy.
“There are drugs being used to reduce the fat accumulation in the brain that causes childhood dementia,” Dr May Aung-Htut, who is leading Murdoch University’s involvement, explained.
“However, while this is helping with the condition there are significant side effects.
“We believe the antisense or ‘gene patching’ drugs that we have developed and successfully applied to Duchenne muscular dystrophy can be designed to reduce this fat accumulation with no or few side effects,” Dr May Aung-Htut said.
These drugs exploit the cell machinery to trick cells that there is no gene message, acting as genetic ‘whiteout’. Professors Sue Fletcher and Steve Wilton (CMMIT, Murdoch University) originally developed the therapies at the Perron Institute.
Dr Aung-Htut, co-head of the Molecular Therapy lab at CMMIT and Perron Institute, is working on the research with Associate Professor Tony Cook at the University of Tasmania and colleagues from Menzies Institute for Medical Research, the Tasmanian School of Medicine and Tasmanian Health Service.
Associate Professor Cook was awarded $599,977 from the Medical Research Future Fund for the project to develop new approaches for the treatment and care of children with dementia.
“Many genetic diseases that cause childhood dementia involve accumulation of specific fat molecules within brain cells, causing them to become dysfunctional and die,” Associate Professor Cook said.
“Inhibiting production of these fat molecules using traditional drugs has shown promise for these conditions in the laboratory, but these drugs have limitations and side effects that mean they are unsuitable as a therapy.
“Working collaboratively across UTAS and Murdoch University, we will develop and conduct pre-clinical testing of a new type of drug that overcomes these limitations, which we anticipate will improve care of children with dementia.”
Dr Kris Elvidge, Head of Research at the Childhood Dementia Initiative, said the project offers hope to those living with the disease.
“Around 100 babies are born every year in Australia with a genetic condition that causes childhood dementia, and 75% of children with dementia will die before they turn 18,” Dr Elvidge said.
“Effective treatments and cures are desperately needed.”
CMMIT is a joint research centre with the Perron Institute and is part of Murdoch University’s Health Futures Institute, bringing together a multidisciplinary team of scientists and our local WA community to transform how long and how well people live, not just in Australia, but around the world.
Article and photo source: CMMIT, Murdoch University.