The Amsterdam Leukodystrophy Center at the Amsterdam University Medical Centers (Amsterdam UMC) is conducting a clinical trial in children with Vanishing White Matter (VWM). In this trial, they want to evaluate whether a drug called Guanabenz is effective in slowing progression, stabilizing, or even improving the brain white matter abnormalities in VWM.
Guanabenz is an old and well-known medicine that has been used for decades for the treatment of high blood pressure. It has been approved by the FDA, the American medicines agency. Given the mechanism of action of Guanabenz, we estimated it likely to be beneficial in VWM. We have laboratory mice with VWM and have treated them with Guanabenz. The results indicate that long-term high-dose Guanabenz treatment ameliorates VWM in these mice and leads to both important improvements of motor function and amelioration of brain pathology (more information in Dooves et al, 2018).
Because we have shown previously that the disease mechanism of VWM and the mode of action of Guanabenz are the same in mice and patients, our expectation is that long-term treatment with high doses of Guanabenz could also be beneficial in patients with VWM.
However, mice are different from humans and the effect of Guanabenz in humans with VWM still has to be investigated. As Guanabenz has been used for years by adults for the treatment of high blood pressure and has proven to be safe in this patient population, and research also supports the safe use of Guanabenz in teenagers, we want to treat children with VWM with Guanabenz.
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