Aug 16, 2017

‘Childhood Alzheimer’s’: Groundbreaking Drug Gives Hope to Young Families

Alzheimer’s disease is often thought about as a condition that older people get. However there is a rare neurodegenerative condition which is often referred to as “childhood Alzheimer’s”.

This condition, also known as Niemann-Pick type C (NPC), causes cholesterol build up in the neurons which leads to enlarged organs, lung damage, muscle stiffness, seizures, dementia and difficulty speaking.

People with “childhood Alzheimer’s” also have a short lifespan with most dying before they turn 20.  

The reason it is compared to Alzheimer’s disease is because the symptoms that the children have are similar to that of dementia symtoms: loss of brain function, including loss of motor control, hearing, speech and cognition.

NPC affects about one in 10000 births, it is under diagnosed and genetic testing suggests it is close to one in 40,000.

New research has given hope to families affected by childhood Alzheimer’s, who believe they have found a drug that could slow the disease in its tracks.

What the drug trial, which was led by researchers at Washington University School of Medicine and the Eunice Kennedy Shriver National Institute of Child Health and Human Development of the National Institutes of Health, focussed on was a sugar molecule called cyclodextrin.

Cyclodextrin is a sugar molecule that has been used as a minor ingredient for other pharmaceuticals because it helps compounds dissolve in water.

It is also an active ingredient in odour elimators like Febreze.

The sugar molecule, when administered therapeutically, showed to return some brain function in NPC patients.

In a study published on August 10 scientists outline the positive effects the drug has shown.

“We were surprised to see evidence that this therapy could slow progression of the disease and, in some cases, get back some function – speech in particular,” said head author Dr Daniel S Ory.

In a most neurodegenerative disease, therapies can’t recover the neurons that have died. But if some brain cells are dysfunctional, it seems this treatment can potentially recover some of that function.

The trial had the cyclodextrin drug administered 14 NPC patients between the ages of four and 23.

11 of the 14 had cyclodextrin administered into their spinal column once a month for 12 to 18 months, while the other three patients had it administered every two weeks for 18 months.

After the 18 month treatment, seven of the 14 patients saw improvements in their gait, cognition and speech.

‘Some of the patients began this trial without the ability to speak, and now they speak,’ Dr Ory said.

However, there were some negative side effects to the treatment as it was found that hearing loss could be caused.

‘Cyclodextrin therapy accelerates the hearing loss that is already a part of the natural progression of this disease,’ said Dr Ory, adding that the researchers expected to see hearing loss as a side effect, based on testing of the drug in animals.

‘A therapy that causes hearing loss is not ideal. But since the disease itself causes hearing loss, we felt that this side effect may be a reasonable trade-off, given the alternative decline and death that the disease also causes.’

Dr Ory added that the patients were able to use hearing aids to maintain quality of life. 

If such treatment work on returning cognition to those who have “childhood Alzheimer’s”, there is potential to use similar treatment modes on other types of dementia in treating the symptoms.

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