AMSTERDAM, NETHERLANDS / RankWire.AI / – An existing blood pressure medicine has shown evidence of slowing vanishing white matter disease in children. Researchers at Amsterdam UMC tested guanabenz in 33 children with the rare inherited brain disorder. They compared the participants with 66 closely matched patients from an international historical registry. The study linked treatment with a lower risk of losing the ability to walk with support. Researchers published the phase 1/2 findings in The Lancet Neurology in August 2026.
Vanishing white matter disease, also called VWM, damages the brain’s white matter and often begins during childhood. Genetic testing and magnetic resonance imaging confirmed the diagnosis for children enrolled in the study. Participants had developed symptoms by age six and had lived with the disease for no more than eight years. Each child could also walk at least 10 steps with limited assistance before entering the trial. Researchers enrolled eligible patients from May 2021 through May 2024.
The main analysis measured how long children retained the ability to walk with support. Researchers matched every treated patient with two untreated historical controls based on disease onset and disability. The resulting hazard ratio was 0.33 for reaching the study’s main walking endpoint. That represented a 67% lower estimated hazard for children receiving guanabenz. Brain scans also showed less white matter deterioration in treated participants, including some children with no detectable progression during follow-up.
Study tracks walking ability and brain changes
Children took guanabenz by mouth, starting at 0.15 milligrams per kilogram of body weight each day. Researchers gradually raised doses over about six weeks according to each child’s tolerance. The trial set 2 milligrams per kilogram per day as the optimal target dose. Thirty-one of the 33 enrolled children completed the study, with a median treatment period of 3.1 years. Researchers reported the strongest treatment effect among children whose symptoms began when they were three years old or older.
Safety monitoring identified 63 serious adverse events among 25 participants during the study. Investigators considered 30 of those events likely or very likely related to guanabenz. Hallucinations affected 18 children and occurred mainly during the first four months of treatment. Severe constipation affected three children, while one experienced temporary low blood pressure with sedation. Those four cases required brief hospital stays and later resolved. No participant stopped treatment because of side effects, and the trial recorded no deaths.
Longer-term research continues after phase 1/2 trial
The study did not randomly assign children to treated and untreated groups. Researchers instead compared guanabenz recipients with historical patients from the Vanishing White Matter Registry. That approach meant the trial had no untreated control group enrolled at the same time. The researchers said longer follow-up should confirm the medicine’s disease-modifying effect. Guanabenz does not cure VWM, and regulators have not approved it as a treatment for the disorder.
Amsterdam UMC is continuing follow-up research involving children from the original study. The extension will examine walking ability, neurological function, brain imaging, safety and different guanabenz doses over a longer period. Guanabenz remains available for VWM only within a research setting. The medicine was originally developed for high blood pressure and acts on cellular stress pathways linked to the disease. The current findings provide measured clinical data on treatment effects in children with early-onset vanishing white matter disease.