Amsterdam researchers find remedy for fatal child brain disease

Researchers from the Emma Children’s Hospital at Amsterdam UMC have for the first time found a way to treat a rare genetic brain disease found mostly in children – vanishing white matter, or VWM.

According to The Lancet Neurology, which published the findings, VWM is a neurodegenerative disease “with onset mostly in children aged 1–6 years that causes early death and has no effective therapy.” It’s caused by a genetic defect and occurs in 1 in 100,000 people in the Netherlands.

But now the blood pressure drug guanabenz has been found to inhibit the disease.

In a trial at Amsterdam UMC, 33 children from various countries received guanabenz for four years, while the comparison group included 66 children who did not receive it. Children who received the drug stabilised and ended up needing a wheelchair less often and less quickly.

They also lived – as compared to five patients in the comparison group who died.

“We saw that the disappearance of the white matter in the brain slowed down or even stopped,” chief researcher Marjo van der Knaap told broadcaster NOS.

Moving forward, “the disease-modifying effect of guanabenz should be confirmed in a long-term extension study,” wrote the researchers. The drug also needs the approval of the European Medicines Agency to be used as a treatment for VWM.