Existing blood pressure drug may slow rare childhood brain disease: Study

A recently published study conducted by Amsterdam University Medical Centers in the Netherlands suggests that an existing blood pressure medication, guanabenz, may help slow the progression of a rare and potentially fatal childhood brain disease known as vanishing white matter (VWM). The study, published in The Lancet Neurology, examined the effects of guanabenz on children between the ages of 1 and 6 who suffer from this hereditary neurodegenerative disorder.

VWM causes a progressive loss of motor and intellectual abilities, often leading to early death and has no approved treatment to halt or slow its progression. Researchers followed children with VWM who received guanabenz over a three-year period, comparing their disease progression with that of 66 children from an international registry who had similar disease severity but did not receive the drug.

The findings revealed that children treated with guanabenz were less likely to become dependent on wheelchairs and did so at a slower rate than those in the comparison group. Notably, none of the children treated with guanabenz died during the study period, while five of the 66 children in the comparison group did.

Side effects were generally mild and occurred mainly during the first few months of treatment, including hallucinations, drowsiness, constipation, and low blood pressure. However, after four to six months, the children typically tolerated the medication well, and none discontinued treatment due to side effects. The researchers emphasized that guanabenz is not a cure for VWM and that its beneficial effects diminish after treatment is discontinued.

They also cautioned that the study did not include a control group receiving no treatment, and a follow-up study is underway to monitor children over a longer period and investigate the effects of higher doses of guanabenz. VWM is an extremely rare condition, affecting approximately one in every 100,000 children globally, with about 1.3 people per million living with the disease in the Netherlands.