Guanabenz May Slow Progression Of Rare Brain Disease In Children: Study

An existing blood pressure medication may slow the progression of a rare and potentially fatal brain disorder in children, according to a study led by Amsterdam University Medical Centres (Amsterdam UMC) in the Netherlands.

Published in The Lancet Neurology, the study examined guanabenz as a potential treatment for vanishing white matter (VWM), a rare hereditary neurodegenerative disorder that mainly affects children between the ages of one and six.

VWM progressively affects motor and intellectual abilities and can lead to early death.

There is currently no approved treatment that can stop or slow its progression.

Researchers followed children with VWM who received guanabenz and compared their progress over three years with 66 children of similar disease severity from an international registry who had not received the drug.

The researchers found that children treated with guanabenz became dependent on wheelchairs less frequently and at a slower rate than those in the comparison group.

None of the children receiving guanabenz died during the study period, while five of the 66 children in the comparison group died.

“This demonstrates for the first time that this fatal brain disease in children can be influenced,” said Marjo van der Knaap, the study’s first author and a retired professor of paediatric neurology at Amsterdam UMC.

Some children experienced side effects, including hallucinations, drowsiness, constipation and low blood pressure, mainly during the first few months of treatment.

Most tolerated the medication well after four to six months, and no child stopped treatment because of side effects.

Researchers said guanabenz is not a cure and its benefits may fade after treatment stops.

A follow-up study will assess longer-term effects and higher doses. VWM affects about one in 1,00,000 children worldwide.

Also Read: Holly Artificial Kidney: Could This Implant End The Routine Of Dialysis?