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

Read the full 1854 word article

How to gain access

Continue reading with a
two-week free trial.