Amsterdam, August 16: A recent study from Amsterdam University Medical Centers has found that an existing blood pressure medication may slow the progression of a rare and deadly brain disease in children.
Published in *The Lancet Neurology*, the study examined the effects of the drug guanabenz, which is typically used to manage blood pressure. Researchers focused on Vanishing White Matter (VWM), a rare genetic neurodegenerative disorder primarily affecting children aged 1 to 6 years. This condition gradually impairs a child’s mobility and cognitive abilities, and it can be fatal as it progresses. Currently, there is no approved treatment to halt or slow down VWM.
During the study, researchers monitored children suffering from VWM who were administered guanabenz. The progression of their disease was observed over three years. Their results were then compared to a group of 66 similarly affected children who did not receive the medication.
The findings revealed that children taking guanabenz were less likely to become dependent on wheelchairs. Among those who did require wheelchairs, the onset of this condition developed more slowly compared to the control group. Notably, there were no deaths among the children receiving guanabenz during the study period, while five children in the non-medicated group passed away.
Some side effects of the drug were reported, including confusion, hallucinations, excessive sleepiness, constipation, and low blood pressure. These issues primarily occurred in the initial months of treatment, but most children began to tolerate the medication well after four to six months.
Retired Professor Marjo van der Kanap from Amsterdam UMC’s Pediatric Neurology department emphasized the importance of recognizing and managing side effects in very young patients. “It is crucial that side effects are identified and treated, and that they are temporary,” she stated.
However, researchers clarified that guanabenz is not a cure for VWM. A follow-up study is currently underway to monitor the children long-term and to investigate the effects of higher doses of guanabenz.
VWM is an extremely rare disease, with estimates suggesting that approximately one in every million children worldwide is born with it. In the Netherlands, about 1.3 individuals per million are affected.
–
Leave a Comment