Long-term Outcome of Bone Marrow Transplantation in a Mouse Model of Krabbe’s Disease 

Dr. Yoichi Kondo, University of Wisconsin, discussed the long-term outcome of bone marrow transplantation in a mouse model of Krabbe’s disease

Bone marrow transplantation (BMT) or umbilical cord blood transplantation are the only therapies available to date for globoid cell Leukodystrophy (GLD, Krabbe disease). However, they do not cure the disease. To discover why, Dr. Kondo investigated twitcher (twi) mice, a model of GLD. If BMT was performed on these mice 10 days after birth, the twi mice lived for an average of 168 days. Those animals that did not receive BMT lived for only about 51 days. When compared to control twi mice, animals that received BMT had better myelin formation at 45 days of age. However, at 200 days, the mice receiving BMT had extensive loss of myelin and displayed evidence of progressive neuronal damage. This study demonstrates that enzyme replacement by simple BMT is not sufficient for the long-term treatment of GLD. 

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Yiochi Kondo, PhD

Source: The Myelin Project Annual Meeting - 2008

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