Adelaide, “Childhood” and “dementia” are two words we wish we didn’t have. But sadly, approximately 1,400 Australian children and young people currently suffer from untreated childhood dementia.
Broadly speaking, childhood dementia is caused by any one of more than 100 rare genetic disorders. Although the causes differ from dementia acquired in late life, the progressive nature of the disease is similar.
Half of infants and children with childhood dementia will not reach their tenth birthday, and most will die before they turn 18.Yet there is a lack of awareness of this devastating condition, and importantly, there is a need to focus research towards treatments and a cure.
More information about causes
Most types of childhood dementia are caused by mutations (or mistakes) in your DNA. These mistakes lead to many rare genetic disorders, which in turn lead to childhood dementia.
Two-thirds of childhood dementia disorders are caused by "inborn errors of metabolism". This means that the metabolic pathways involved in the breakdown of carbohydrates, lipids, fatty acids and proteins in the body fail.As a result, neural pathways fail to function, neurons (nerve cells that send messages around the body) die, and progressive cognitive decline occurs.
What happens to children with childhood dementia?
Most children initially appear unaffected. But after a period of apparently normal development, children with childhood dementia gradually lose previously acquired skills and abilities, such as talking, walking, learning, remembering, and reasoning.
Childhood dementia also causes significant changes in behavior, such as aggression and hyperactivity. Severe sleep disturbances are common and vision and hearing may also be affected.Many children have seizures.
The age at which symptoms begin can vary, partly depending on the particular genetic disorder causing dementia, but the average is around two years. These symptoms are caused by significant, progressive brain damage.
Is there any treatment available?
Treatments for childhood dementia are currently under evaluation or approval, for a very limited number of disorders, and are only available in some parts of the world. These include gene replacement, gene-modified cell therapy, and protein O enzyme replacement therapy.Enzyme replacement therapy is available in Australia for a form of childhood dementia. These treatments attempt to "fix" the problems causing the disease, and show promising results.
Other experimental treatments include treatments that target faulty protein production or reduce inflammation in the brain.
lack of attention to research
The mortality rate for Australian children with cancer has almost halved between 1997 and 2017, thanks to research that has enabled the development of many treatments but in recent decades, nothing has changed for children with dementia.
In 2017–2023, childhood cancer research received four times more funding per patient than funding for childhood dementia.Although it is childhood dementia that causes the same number of deaths each year as childhood cancer.
The success of childhood cancer survivors in recent decades demonstrates that adequately funding medical research can improve patient outcomes.
Another barrier for childhood dementia patients in Australia is the lack of access to clinical trials. An analysis published in March this year showed that in December 2023, only two clinical trials in Australia were recruiting patients with childhood dementia.
However, 54 trials were recruiting around the world, meaning Australian patients and their families are watching patients in other parts of the world receive a potentially life-saving treatment who may have no recourse themselves.That said, we have seen a slow pace in recent years in the establishment of clinical trials for childhood dementia around the world.
Furthermore, we know from consulting families that current care and support systems are not meeting the needs of children with dementia and their families.
new research
Recently, we were awarded new funding for our research on childhood dementia, this will help us continue and expand studies that seek to develop life-saving treatments.
More broadly, we need to see increased funding in Australia and around the world for research to develop and translate treatments for a broader spectrum of childhood dementia conditions.(talk) NSA
Broadly speaking, childhood dementia is caused by any one of more than 100 rare genetic disorders. Although the causes differ from dementia acquired in late life, the progressive nature of the disease is similar.
Half of infants and children with childhood dementia will not reach their tenth birthday, and most will die before they turn 18.Yet there is a lack of awareness of this devastating condition, and importantly, there is a need to focus research towards treatments and a cure.
More information about causes
Most types of childhood dementia are caused by mutations (or mistakes) in your DNA. These mistakes lead to many rare genetic disorders, which in turn lead to childhood dementia.
Two-thirds of childhood dementia disorders are caused by "inborn errors of metabolism". This means that the metabolic pathways involved in the breakdown of carbohydrates, lipids, fatty acids and proteins in the body fail.As a result, neural pathways fail to function, neurons (nerve cells that send messages around the body) die, and progressive cognitive decline occurs.
What happens to children with childhood dementia?
Most children initially appear unaffected. But after a period of apparently normal development, children with childhood dementia gradually lose previously acquired skills and abilities, such as talking, walking, learning, remembering, and reasoning.
Childhood dementia also causes significant changes in behavior, such as aggression and hyperactivity. Severe sleep disturbances are common and vision and hearing may also be affected.Many children have seizures.
The age at which symptoms begin can vary, partly depending on the particular genetic disorder causing dementia, but the average is around two years. These symptoms are caused by significant, progressive brain damage.
Is there any treatment available?
Treatments for childhood dementia are currently under evaluation or approval, for a very limited number of disorders, and are only available in some parts of the world. These include gene replacement, gene-modified cell therapy, and protein O enzyme replacement therapy.Enzyme replacement therapy is available in Australia for a form of childhood dementia. These treatments attempt to "fix" the problems causing the disease, and show promising results.
Other experimental treatments include treatments that target faulty protein production or reduce inflammation in the brain.
lack of attention to research
The mortality rate for Australian children with cancer has almost halved between 1997 and 2017, thanks to research that has enabled the development of many treatments but in recent decades, nothing has changed for children with dementia.
In 2017–2023, childhood cancer research received four times more funding per patient than funding for childhood dementia.Although it is childhood dementia that causes the same number of deaths each year as childhood cancer.
The success of childhood cancer survivors in recent decades demonstrates that adequately funding medical research can improve patient outcomes.
Another barrier for childhood dementia patients in Australia is the lack of access to clinical trials. An analysis published in March this year showed that in December 2023, only two clinical trials in Australia were recruiting patients with childhood dementia.
However, 54 trials were recruiting around the world, meaning Australian patients and their families are watching patients in other parts of the world receive a potentially life-saving treatment who may have no recourse themselves.That said, we have seen a slow pace in recent years in the establishment of clinical trials for childhood dementia around the world.
Furthermore, we know from consulting families that current care and support systems are not meeting the needs of children with dementia and their families.
new research
Recently, we were awarded new funding for our research on childhood dementia, this will help us continue and expand studies that seek to develop life-saving treatments.
More broadly, we need to see increased funding in Australia and around the world for research to develop and translate treatments for a broader spectrum of childhood dementia conditions.(talk) NSA
