Vanishing White Matter
"Vanishing White Matter falls within the so-called white matter diseases," says Prof. Marjo van der Knaap, principal investigator at Amsterdam Neuroscience." It is one of the more prevalent leukodystrophies in which the brain is always in the state of a stress response. In particular, the white matter can’t handle this continuous stress response very well. In almost all cases the disease leads to learning difficulties, spasticity and disability. Over time, the patients will experience motor problems, including difficulty with walking, swallowing and speaking. Many of the children pass away at a young age after a period of severe mental and physical disability.
Guanabenz trial
Previous research, co-funded by the Dutch Brain Foundation, has shown in which type of brain cells the stress response is observed and how this stress reaction can be inhibited. The researchers believe that the overly active stress response is the cause of Vanishing White Matter. "We have recently started a clinical trial with the drug Guanabenz, which is a drug for high blood pressure, which might inhibit the stress response in VWM patients. It could slow down or even stop the disease as a result," says van der Knaap.
A total of 30 children will be participating in the study. To measure the effect of Guanabenz properly, it is important that the brain has not yet suffered much damage, therefore, young children are included in the study. They are given Guanabenz daily and are checked once a year at Amsterdam UMC. The results of this new trial will be expected in mid-2026. If the drug seems to be effective, children around the world can be treated at relatively low cost.
Read more about the study in Dutch on the Dutch Brain Foundation website or the Vanishing White Matter Consortium website.