Defects in the enzyme
alpha-galactosidase lead to the buildup of globotriaosylceramide, causing
Fabry's disease. The pharmaceutical drug
migalastat enhances the function of alpha-galactosidase and is used to treat Fabry's. Globotriaosylceramide is also one of the targets of
Shiga toxin, which is responsible for pathogenicity of enterohemorrhagic
Escherichia coli (EHEC). The bacterial Shiga toxin can be used for targeted therapy of certain gastrointestinal cancers that express the receptor of the Shiga toxin. For this purpose a non-specific chemotherapeutic agent is conjugated to the B-subunit to make it specific. In this way only the tumor cells, but not healthy cells, should be destroyed during therapy. ==References==