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Getting the latest healthcare news for you
Getting the latest healthcare news for you
An old hypertension drug may offer new hope for kids with vanishing white matter disease. A phase 1/2 trial found that guanabenz cut the risk of losing the ability to walk by 67% compared to untreated historical controls — making it the first potential disease-modifying therapy for this devastating and currently untreatable neurological condition.
An old blood pressure medication may be the first disease-modifying treatment for vanishing white matter (VWM) disease, a rare, inherited, and progressive brain disorder that strikes young children and leads to severe neurological disability and early death. A phase 1/2 trial published in The Lancet Neurology found that guanabenz — an oral alpha-2 adrenergic antihypertensive — was associated with a 67% lower risk of children losing their ability to walk with support, compared to matched untreated historical controls.
The trial followed 33 children with confirmed VWM disease for up to 4 years. The benefit was most pronounced in children whose disease began at age 3 or later, with only 1 of 18 treated patients in that subgroup losing walking ability, versus 11 of 36 controls. Researchers believe early treatment — or even presymptomatic intervention — could yield even greater benefits.
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Why it matters: VWM disease has no approved therapies, making this the first trial to demonstrate potential disease modification. A long-term extension study is underway, and experts say gene therapy and combination approaches may further improve outcomes.
