Facing up to childhood dementia

By Kayla Olaya 3 years ago | In Industry
  • 3 years ago
Childhood Dementia Initiative staff 'Face it' to raise awareness

22 September 2021

Childhood dementia is more common than most people think, with around one in every 2,800 children born with a disorder that leads to childhood dementia, yet treatment options are few.

Marking its first Childhood Dementia Day today (Wednesday), the Childhood Dementia Initiative (CDI) is raising awareness of what is largely an unknown disease that impacts thousands of children.

“I ran a patient organisation called the Sanfilippo Children’s Foundation for seven years that funds research into a rare condition called Sanfilippo Syndrome,” Childhood Dementia Director and CEO Megan Donnell tells MedNews.

“Sanfilippo Syndrome is a form of dementia and my children have the condition. The Childhood Dementia Initiative came about because I saw the opportunity to drive a lot more focus and attention for all conditions that present with dementia in children.

“We wanted to move the focus from the individual underlying genetic cause of the 70 different conditions that present with dementia to focus on the commonality, which is dementia.

“Whether it’s in a research setting, clinical care or a support setting, what we’re advocating for is collective consideration of childhood dementia disorders.”

The size of the problem

Just as in adults, childhood dementia is neurocognitive decline resulting in a loss of developmental and cognitive skills and neuromotor decline over a period of time. Of the 70 genetic disorders that can lead to childhood dementia, only three or four have an effective treatment meaning of the 2,273 Australian children with dementia, most have no viable treatment. More than 90 die prematurely every year.

“At this stage, we understand that there are about 700,000 children around the world living with dementia and around 48,000 will die this year alone,” Donnell said.

“There’s a very big gap in terms of any sort of access to intervention for these families and it’s critical that we accelerate the development of therapies as soon as possible.”

Wilson disease, biotinidase deficiency and holocarboxylase synthetase deficiency are among the few diseases that do have treatment options that can be effective if diagnosed and treated early, while a handful of other disorders have treatments such as enzyme replacement therapies that may slow progression. However, they often don’t treat the neurological aspects of the disease.

BioMarin’s BRINEURA (cerliponase alfa) is one exception and is funded through the LSDP for a subtype of Batten Disease that, if left untreated, can result in childhood dementia. It has been shown to slow the progression of motor and language deficits but its long-term effectiveness against disease progression is unknown.

“There are gene therapy trials for Sanfilippo syndrome, Battens disease, some of the leukodystrophies, and they are showing some promise,” CDI’s Head of Research, Kris Elvidge says. “Early treatment is also very important and gene therapy is a really exciting prospect.”

Donnell agrees that gene therapy is providing “a lot of hope” for parents.

“If you look at gene therapy, it’s moving ahead in leaps and bounds for single genetic fault disorders of which most childhood dementias are,” she says. “There is a big opportunity with emerging technologies and advanced therapeutics to make a real difference for these kids.”

Overlapping disease mechanisms found across childhood dementia disorders include inflammation and lysosomal dysfunction, providing opportunities for common drug treatments that target and slow disease progression and improve quality of life.

Journalist Kayla Olaya joins in the awareness campaign.

‘Face it’ for childhood dementia

‘Childhood Dementia: It’s time to face it’ is the organisation’s first disease awareness campaign. It runs in September, coinciding with Dementia Awareness Week and asks people to reconnect with their childhood by painting their faces, representing facing up to childhood dementia.

“We’ve chosen face painting because we think it’s a really lovely way of adults getting to be children again by painting their faces and doing something quite fun,” Donnell says. “In reverse, a treatment or cure could allow kids with dementia to grow up and become adults someday.

“Awareness is key. We need researchers, doctors, policymakers – everybody that’s involved in the diagnosis and care of these kids throughout their short life to understand that dementia is something that affects children.”

To participate in CDI’s disease awareness campaign, just paint your face and upload a photo to social media using the hashtags #faceit and #childhooddementia.

“I’m just one mother of 700,000 around the world but our kids deserve better than what they have currently,” says Donnell.

“There is an opportunity for future generations to live a different life to the one that myself and hundreds of thousands of other families currently live. We need to do things differently.”

©MedNews 2021

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