New therapeutic approaches for the treatment of Huntington's disease.
The use of transplantation (TR) of fetal neural tissue as a therapeutic method started much later in patients suffering from Huntington's disease (HD) than in those with Parkinson's disease. The clinical trial, following a wide range of animal experiments (neurotoxic models and newly also transgenic mice), includes about 30 HD patients until now. Because of limited use of the human fetal tissue by ethical and technical concerns, there is necessity to search for the alternative sources for neural grafting. The first attempt with xenotransplantation (in 12 HD patients) and with TR of encapsulated genetically modified cells (in 6 HD patients) was performed, but no appreciable improvement of status in any of those patients was noted. Since no effective pharmacological treatment of HD is available, the TR of fetal neural tissue is now the only therapeutic approach which provides a reduction of symptoms in most of grafted patients. The possibilities are enormous offered by neural stem cells, optionally by embryonic stem cells, which could be expanded in cultures, cloned or genetically modified and then grafted into the patient's brain. On the other hand, the neural progenitor and stem cells, normally present within the subependymal layer of the lateral brain ventricles also in adulthood, might be induced to become an endogenous source of glia and neurons participating in the brain's repair.
Mazurová
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Department of Histology and Embryology, Charles University, Prague, Faculty of Medicine, Hradec Králové. mazurova@lfhk.cuni.cz
D000818:Animals; D016380:Brain Tissue Transplantation; D016332:Fetal Tissue Transplantation; D005818:Genetic Engineering; D015316:Genetic Therapy; D006801:Humans; D006816:Huntington Disease / Q000601:surgery / Q000628:therapy*; D009474:Neurons / Q000166:cytology / Q000637:transplantation; D033581:Stem Cell Transplantation; D014183:Transplantation, Heterologous