AMSTERDAM, NETHERLANDS / RankWire.AI / – In Amsterdam in August 2026, a common medication for high blood pressure demonstrated promising signs of slowing the progression of vanishing white matter disease in children. Researchers affiliated with Amsterdam UMC conducted a trial involving 33 young patients diagnosed with this rare inherited neurological disorder. These participants were compared to 66 matched controls from an international historical registry. The study found that treatment correlated with a decreased risk of losing the ability to walk with support. The phase 1/2 findings were published in The Lancet Neurology during August 2026.
Vanishing white matter disease, also known as VWM, leads to damage in the brain’s white matter and typically manifests during childhood. Diagnosis was confirmed through genetic testing and magnetic resonance imaging for the enrolled children. All participants exhibited symptoms by age six and had lived with the condition for no longer than eight years. Prior to the trial, each child was able to walk at least 10 steps with some assistance. Eligible patients were recruited between May 2021 and May 2024.
The primary focus of the analysis was on the duration children retained the ability to walk with support. Each treated child was matched with two historical controls based on disease onset and degree of disability. The hazard ratio for reaching the primary walking endpoint was 0.33, indicating a 67% lower risk for children treated with guanabenz. Brain imaging further revealed less white matter deterioration among the treated group, including some children who showed no progression during follow-up.
Study Monitors Walking Capacity and Brain Changes
Participants received guanabenz orally, starting at 0.15 milligrams per kilogram of body weight daily. Doses were gradually increased over approximately six weeks, tailored to individual tolerance. The trial set a target dose of 2 milligrams per kilogram per day as optimal. Out of the 33 children enrolled, 31 completed the study, with a median treatment duration of 3.1 years. Researchers observed the most significant treatment effects in children who developed symptoms at age three or older.
During safety assessments, 63 serious adverse events were recorded among 25 participants. Investigators judged that 30 of these events were likely or very likely related to guanabenz. Hallucinations were reported in 18 children, mostly during the first four months of therapy. Severe constipation impacted three children, while one experienced temporary hypotension accompanied by sedation. All four cases resulted in brief hospital stays and later resolved. No participant discontinued treatment due to side effects, and there were no deaths reported during the trial.
Extended Follow-Up Continues Post-Phase 1/2 Trial
Since the study lacked randomization, researchers compared children treated with guanabenz against historical data from the Vanishing White Matter Registry. Consequently, there was no concurrently enrolled untreated control group. The investigators noted that longer-term follow-up is necessary to verify whether the medication truly alters disease progression. Although promising, guanabenz does not cure VWM, and it has not yet received regulatory approval for this indication.
Follow-up research at Amsterdam UMC involving original trial participants is ongoing. This extension aims to evaluate walking ability, neurological health, brain imaging results, safety, and different dosing regimens over an extended period. Currently, guanabenz remains experimental for VWM and is only accessible within research settings. Originally developed to treat hypertension, the drug influences cellular stress pathways associated with the disease. The recent findings offer valuable clinical insights into how treatment may impact children with early-onset vanishing white matter disorder.