Intersection in marrow

How Gaucher's disease drugs could help multiple myeloma

An unlikely connection between Gaucher's disease and multiple myeloma has been uncovered by a U.K. group that found a common node in the pathways responsible for the bone disease symptoms that accompany both diseases. Suppression of myeloma-related bone erosion by the Gaucher's drug Zavesca miglustat suggests inhibiting sphingolipid synthesis could address the bone pathology in the cancer, and add a new angle to complement the growing list of myeloma therapies in development.

Zavesca is an inhibitor of GCS - an enzyme critical to glycosphingolipid synthesis - and is marketed by the UCB Group and Actelion Ltd. to treat Gaucher's disease and glycosphingolipid storage disorders.

While a host of late-stage clinical compounds for multiple myeloma (MM) are attracting attention - with efficacy data expected at the upcoming American Society of Clinical Oncology (ASCO) meeting - the field is still in search of new mechanisms to effectively address the bone lesions that occur in about 80% of patients.

The bone erosion is caused by activation of osteoclasts triggered by bone-resorbing factors in the tumor environment. In some cases the bone disorder is resolved during treatment by elimination

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