Between 2021 to 2024, patients were screened, now study finds blood pressure-hypertension medicine may treat rare and potentially fatal brain disease in children

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

The study, published in the latest issue of The Lancet Neurology, examined the effects of guanabenz on vanishing white matter (VWM), a rare hereditary neurodegenerative disorder that primarily affects children between the ages of 1 and 6. Patients were screened before being enrolled for the trial between May 31, 2021 and May 31, 2024.

VWM causes progressive loss of motor and intellectual abilities and can lead to early death. There is currently no approved treatment that can stop or slow the progression of the disease, according to Amsterdam UMC.


Researchers followed children with VWM who received guanabenz and compared their disease progression over three years with that of 66 children with similar disease severity from an international registry who had not received the drug, reports Xinhua news agency.

They found that children treated with guanabenz became dependent on wheelchairs less frequently and less rapidly than those in the comparison group.

"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 pediatric neurology at Amsterdam UMC.

None of the children treated with guanabenz died during the study period, compared with five of the 66 children in the comparison group, according to the study.

Side effects, including hallucinations, drowsiness, constipation and low blood pressure, occurred mainly during the first few months of treatment. After four to six months, the children generally tolerated the medication well, and none discontinued treatment because of side effects.

"Precisely because we are dealing with young children, it is important that side effects are recognisable, treatable and temporary," van der Knaap said.

The researchers stressed that guanabenz is not a cure for VWM and that its beneficial effects appear to disappear after treatment is discontinued. They also cautioned that the study did not include a simultaneous untreated control group.

A follow-up study is underway to monitor the children over a longer period and investigate the effects of higher doses of guanabenz, according to Amsterdam UMC.

VWM is extremely rare, with an estimated one in 100,000 children worldwide born with the disease. In the Netherlands, about 1.3 people per million are living with the condition.