Abstract
During the past few years, bone marrow transplantation (BMT) has been performed for various lipidoses in both human patients and animal models (1–8). Most patients transplanted for lipidoses suffered from the non-neuropathic form of Gaucher’s disease. Since in this type of Gaucher’s disease the reticuloendothelial system is involved, possible beneficial effects of BMT may be explained by replacement of the Gaucher cells by enzymatically competent, donor-derived cells, since after BMT not only the blood cells, but also the tissue macrophages are being replaced by donor derived cells (9). Recently, improved psychomotor development has been reported in patients with metachromatic leukodystrophy (3, 4). The neurological improvement following BMT in these patients cannot be explained easily, since the blood-brain barrier prevents circulating enzyme to enter brain tissue (10), and the presence of donor-derived cells, which contribute to the increased enzyme activity in e.g. lung and liver tissue (11–12) , has not been reported in brain tissue so far. Preliminary studies in a patient with Niemann-Pick type B disease showed clinical improvement following BMT, but the long-term effects are not evaluable yet (13). In a mouse model for Niemann-Pick disease, BMT did not result in clinical improvement, despite decreased accumulation of sphingomyelin in liver and spleen (6).
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© 1988 Plenum Press, New York
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Hoogerbrugge, P.M., Poorthuis, B.J.H.M., Kobayashi, T., Wagemaker, G., Suzuki, K. (1988). Neurological Improvement Following Bone Marrow Transplantation in Twitcher Mice — Murine Globoid Cell Leukodystrophy. In: Salvayre, R., Douste-Blazy, L., Gatt, S. (eds) Lipid Storage Disorders. NATO ASI Series, vol 150. Springer, Boston, MA. https://doi.org/10.1007/978-1-4613-1029-7_99
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DOI: https://doi.org/10.1007/978-1-4613-1029-7_99
Publisher Name: Springer, Boston, MA
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