AMSTERDAM, NETHERLANDS / RankWire.AI / – In a recent study, an existing medication for blood pressure demonstrated a 67% reduction in the risk of losing the ability to walk with support among children with vanishing white matter disease. Researchers at Amsterdam UMC evaluated guanabenz in a group of 33 pediatric patients suffering from this rare inherited neurological disorder. They compared these results to 66 closely matched children from an international historical registry. The study’s findings, published in The Lancet Neurology in August 2026, indicated a link between treatment and a reduced likelihood of losing walking ability.
Vanishing white matter disease, also known as VWM, primarily affects the white matter in the brain and typically manifests during childhood. Diagnostic confirmation for participants involved genetic testing and magnetic resonance imaging. All enrolled children showed symptoms by age six and had lived with the disease for no more than eight years. Before the trial, each child was able to walk at least 10 steps with limited assistance. Patients eligible for participation were enrolled from May 2021 through May 2024.
The primary focus of the analysis was on the duration children maintained the ability to walk with support. Each treated patient was matched with two untreated historical controls, based on disease onset and level of disability. The hazard ratio for reaching the primary walking endpoint was 0.33, indicating a 67% lower hazard among children treated with guanabenz. Brain imaging further revealed less deterioration of white matter in the treated group, with some children showing no progression during the follow-up period.
Study Monitors Mobility and Brain Changes
Children received guanabenz orally, beginning at 0.15 milligrams per kilogram of body weight daily. The dosage was gradually increased over approximately six weeks based on individual tolerance. The trial aimed for an optimal dose of 2 milligrams per kilogram daily. Out of 33 children enrolled, 31 completed the study, with a median treatment duration of 3.1 years. The most significant effects were observed in children whose symptoms started at age three or older.
Throughout the trial, safety assessments recorded 63 serious adverse events involving 25 participants. Investigators judged 30 of these events as likely or very likely related to guanabenz. Among these, 18 children experienced hallucinations mainly during the first four months of treatment. Severe constipation affected three children, and one experienced temporary low blood pressure with sedation. All these cases required brief hospital stays but later resolved. No participant discontinued treatment because of side effects, and there were no deaths reported during the study.
Extended Follow-up Research Is Ongoing Post Phase 1/2 Trial
The study design did not include random assignment of children to treated or untreated groups. Instead, the researchers compared guanabenz-treated patients with historical data from the Vanishing White Matter Registry. This meant there was no concurrent untreated control group enrolled during the same period. The research team emphasized that longer-term follow-up is needed to verify the potential disease-modifying properties of the medication. It’s important to note that guanabenz is not a cure for VWM, and it has not received approval from regulators as a treatment for this condition.
Amsterdam UMC is now conducting ongoing follow-up studies involving children from the original cohort. This extension aims to evaluate walking ability, neurological function, brain imaging, safety, and the effects of different guanabenz dosages over a longer period. Currently, guanabenz remains available solely within a research context for VWM. Originally developed to treat high blood pressure, the drug impacts cellular stress pathways associated with the disease. These initial results offer valuable clinical insights into the potential effects of treatment in children with early-onset vanishing white matter disorder.