In a groundbreaking medical discovery led by Amsterdam University Medical Centers (Amsterdam UMC) in the Netherlands, researchers have found that Guanabenz, an older hypertension medication, can help slow the progression of Vanishing White Matter (VWM)—a rare and fatal genetic brain disorder affecting young children.
VWM causes progressive damage to the brain’s white matter, leading to severe deterioration of motor and cognitive skills. Over a three-year period, researchers monitored children receiving Guanabenz and compared their outcomes with an international registry cohort of 66 untreated VWM patients with similar disease severity.
The results showed that children treated with Guanabenz experienced significantly delayed wheelchair dependency. Furthermore, zero fatalities occurred in the Guanabenz-treated group during the study, compared to five deaths among the 66 patients in the control group. Published in The Lancet Neurology, the study represents the first evidence that the progression of this deadly neurological disease can be altered. While side effects such as drowsiness, low blood pressure, constipation, and occasional hallucinations were observed in the initial months, patients generally tolerated the medication after four to six months.
