Childhood dementia is a little-known but serious public health problem and now QIMR Berghofer researchers believe a new study has raised hopes of a breakthrough.
Associate Professor Anthony White’s team received a $49,600 grant from Australia’s Batten Disease Support and Research Association (BDRSA), to find drugs that can stop or slow the progression of the neurological disorder.
Batten disease is one of the most common forms of childhood dementia. It causes the progressive deterioration of brain cells and a series of serious symptoms such as loss of the ability to walk and speak.
“Childhood dementia proceeds in the same way as adult dementia. It’s still deadly and there are very few drug treatments available, so more research is desperately needed to improve outcomes,” said Professor A/White.
“At QIMR Berghofer, we strive to find drugs that treat Batten’s disease and other forms of childhood dementia, in order to slow or stop the progression of the disease and save these children valuable time and to their families. »
For Queensland mother Jessica Lyons, the research offers hope at a time when her family is facing a heartbreaking lack of options.
Her cheerful and cheeky daughter Emily was diagnosed with one of the rarest forms of Batten disease – CLN6. The eight-year-old is currently the only known case in Australia.
“Before Emily was diagnosed, we thought it would be enough to find the problem so she could get the right treatment and go on to live a reasonably normal life,” Ms Lyons said.
“But now we know that Emily won’t live to be an adult, she won’t go to college or anything like that, and accepting that is really hard. Every week we see it shrinking further, and there is absolutely nothing we can do.
“If researchers could find and stop the regression and save time for children with Batten disease, that would be amazing. Because at the moment we have no options.
The QIMR Berghofer research project focuses on drug repositioning. This involves identifying existing drugs approved for other conditions, which can be repurposed to treat Batten disease and other childhood dementia disorders.
“There are more than 70 childhood dementia diseases and they are all caused by different genetic mutations, but we think they may share some common genes and proteins to explain their similar symptoms,” Professor A/White said.
“If we can identify shared genes and proteins and find existing drugs that target them, we can treat multiple childhood dementia states. This could help slow or stop these diseases while more research is conducted to target their individual causes.
A/Prof White is Principal Investigator of the Batten Disease Research Team which includes fellow QIMR Berghofer researchers, Professor Eske Derks and Dr Zachary Gerring, who recently conducted a successful study identifying potential new treatments for Alzheimer’s disease.
The study will see Professor Derks and Dr Gerring use computer algorithms to analyze the genetics of childhood dementia and identify promising candidates for drug redirection.
Assistant Professor White will then test the drugs in tiny model brains, to validate the results and better establish the drugs’ impact on Batten disease and childhood dementia.
Dr Ineka Whiteman, Head of Research, Medical and Scientific Affairs at BDSRA Australia, said the association was delighted to support the important work of Prof A/White and his team.
“Their computational bioinformatics approach is sure to steer Batten disease and childhood dementia research in an exciting direction. This is a relatively new approach in the field of Batten disease and could help bring desperately needed effective treatments to patients much sooner,” said Dr Whiteman.
“We are intrigued to see what new potential therapeutic candidates they might uncover as the project progresses.”
The Childhood Dementia Initiative’s research director, Dr Kris Elvidge, expressed the hope that Professor A/Prof White’s approach would yield results for a variety of childhood dementia disorders.
“Every three days in Australia, a child is born with childhood dementia disorder. Unfortunately, the vast majority of these children will not live to adulthood,” Dr Elvidge said.
“This research project is exciting and we look forward to seeing this approach applied to the many types of childhood dementia to find treatments that give these children longer, more fulfilling lives.”