European Leukodystrophy Association (ELA) awarded Vivi Heine to study if modulation of Hyaluronan in the host brain microenvironment of a mouse model for Vanishing White Matter Disease (VWM) can improve stem cell therapy.
VWM is a relatively prevalent leukodystrophy. VWM can occur at all ages, but most often has an early childhood onset. Patients have progressive motor coordination deficits and spasticity, get wheelchair dependent and die within a few years of onset. VWM leads to failure of proper brain white matter development. The white matter of the brain is mainly composed of astrocytes and oligodendrocytes. The astrocytes have many supporting functions in the brain; the oligodendrocytes wrap a fatty substance called myelin around the extensions of neurons to support neuronal communication. There is no treatment for VWM. For most leukodystrophies cell therapy currently gives the best prospect of halting progression or obtaining repair. We study the prospects for glia replacement therapy for VWM.
This project aims to study whether modulation of hyaluronan levels in the host microenvironment will improve cell replacement strategies.
If stem cell therapy would prove to be beneficial in VWM mice, this would form a major breakthrough for the treatment of patients with VWM, probably numerous other leukodystrophies and probably also non-genetic white matter disorders, like MS, with wider implications for the field at large.