Blood-pressure drug shows promise in slowing rare childhood brain disease

A common blood-pressure medication may slow the progression of vanishing white matter, a rare and fatal brain disorder in children, according to a study by Amsterdam University Medical Centers. The findings suggest a potential new avenue for treatment of this hereditary neurodegenerative condition.

A blood-pressure medicine already in use may help slow a severe, rare brain disorder in children, according to researchers at Amsterdam University Medical Centers. The work, published in The Lancet Neurology, focused on guanabenz and vanishing white matter, a hereditary neurodegenerative disease that most often appears in early childhood and can steadily erode movement and thinking.

In the study, children with vanishing white matter who were given guanabenz were tracked over three years and compared with 66 similar patients from an international registry who had not taken the drug. According to Amsterdam UMC, the treated group became dependent on wheelchairs less often and at a slower pace. Marjo van der Knaap, the study’s first author and a retired professor of paediatric neurology at Amsterdam UMC, said: “This demonstrates for the first time that this fatal brain disease in children can be influenced.”

The researchers also reported that none of the children receiving guanabenz died during the study period, compared with five deaths in the comparison group. Side effects such as hallucinations, drowsiness, constipation and low blood pressure were mainly seen in the first few months of treatment, but most children later tolerated the medicine well and none stopped taking it because of adverse reactions. Van der Knaap said: “Precisely because we are dealing with young children, it is important that side effects are recognizable, treatable and temporary.”

The team stressed that guanabenz is not a cure and that its benefit appears to fade if treatment stops. They also noted that the research did not include a simultaneous untreated control group. Amsterdam UMC has said it has begun a further trial involving 30 children to test whether the drug can dampen the brain’s stress response and possibly slow or halt disease progression. The condition remains extremely rare, affecting an estimated one in 100,000 children worldwide, with about 1.3 people per million living with it in the Netherlands.