The Hague: An existing blood pressure medication may help slow the progression of a rare and often fatal childhood brain disorder, according to a study led by researchers at Amsterdam University Medical Centers (Amsterdam UMC) in the Netherlands.The findings, published in the latest issue of The Lancet Neurology, examined the effects of guanabenz on vanishing white matter (VWM) disease, a rare inherited neurodegenerative condition that primarily affects children between the ages of one and six.VWM causes progressive deterioration of motor and cognitive functions and can result in early death. According to Amsterdam UMC, there is currently no approved treatment capable of halting or slowing the disease.Researchers monitored children with VWM who received guanabenz and compared their outcomes over a three-year period with those of 66 children with similar disease severity from an international registry who did not receive the medication.The study found that children treated with guanabenz became dependent on wheelchairs less frequently and at a slower rate than those in the comparison group.“This demonstrates for the first time that this fatal brain disease in children can be influenced,” said Marjo van der Knaap, the study's lead author and a retired professor of paediatric neurology at Amsterdam UMC.None of the children receiving guanabenz died during the study period, compared with five deaths among the 66 children in the comparison group.Researchers reported that side effects, including hallucinations, drowsiness, constipation and low blood pressure, were mainly observed during the first few months of treatment. After four to six months, most children tolerated the drug well, and none stopped treatment because of side effects.“Precisely because we are dealing with young children, it is important that side effects are recognizable, treatable and temporary,” van der Knaap said.The researchers stressed that guanabenz is not a cure for VWM and noted that the beneficial effects appear to diminish once treatment is discontinued.They also cautioned that the study did not include a simultaneous untreated control group, highlighting the need for further research.A follow-up study is already underway to assess longer-term outcomes and evaluate the impact of higher doses of guanabenz.Vanishing white matter disease is extremely rare, affecting an estimated one in 100,000 children worldwide. In the Netherlands, approximately 1.3 people per million are living with the condition.
