A decades-old blood pressure medicine may help slow the progression of Vanishing White Matter (VWM), a rare inherited neurological disorder that can cause progressive loss of motor and cognitive abilities in children.
Researchers at Amsterdam University Medical Centers have been studying guanabenz as a potential treatment for VWM. The disorder affects the brain’s white matter and can lead to worsening movement problems and other neurological disabilities. Amsterdam UMC says there is currently no curative treatment for VWM, while research into disease-modifying therapies is ongoing.
According to the study findings provided, researchers followed children receiving guanabenz for approximately three years and compared their disease progression with 66 children in an international registry who had a comparable disease severity but had not received the drug.
The researchers reported that children treated with guanabenz appeared to develop wheelchair dependence less frequently and later than those in the comparison group. No deaths were recorded among the children receiving the medicine during the study period, while five deaths occurred in the comparison group.
The findings are significant because treatment options for VWM remain extremely limited. Amsterdam UMC has described its guanabenz study as the first clinical treatment trial for VWM and says the research is intended to determine whether the medicine can slow or stabilise disease progression.
However, the results should not be interpreted as proof that guanabenz is an established treatment for VWM. Amsterdam UMC’s trial documentation states that guanabenz cannot be prescribed for VWM outside the research setting and that its effectiveness in patients still needs to be established.
Researchers also reported side effects associated with the medicine, including drowsiness, constipation and low blood pressure, with some patients experiencing hallucinations. The side effects were reportedly more common during the early months of treatment and generally became easier to tolerate over time.
Guanabenz has traditionally been used as a blood-pressure medicine. Researchers became interested in its potential for VWM after laboratory studies suggested that it could improve disease-related changes and motor function in mouse models.
Amsterdam UMC continues to work with international researchers on clinical trials and potential treatments for VWM, a rare genetic disorder for which there remains no established cure.
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