by Murdoch University

Children living with dementia have the same symptoms as adults with dementia, in that they are losing memories and face a gradual decline in health, with most not reaching adulthood. Credit: Murdoch University

There are about 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 Center for Molecular Medicine and Innovative Therapeutics (CMMIT) 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's involvement, explained. "However, while this is helping with the condition there are significant side effects. We believe the antisense, or gene patch, drugs that we have developed and successfully applied to Duchenne's Muscular Dystrophy can be designed to reduce this fat accumulation with no or few side effects."

These drugs exploit the cell machinery to trick cells that there is no gene message, acting as genetic "whiteout." They were originally developed by Professors Sue Fletcher and Steve Wilton of CMMIT and the Perron Institute.

Dr. Aung-Htut 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.

"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."

Provided by Murdoch University