Gleevec: A Groundbreaking Example

Gleevec (imatinib mesylate) was initially approved by the FDA for the treatment of chronic myeloid leukemia (CML) and advanced or metastatic gastrointestinal stromal tumors (GIST) in 2001 and 2002, respectively. This marked the first approval of a drug that directly turns off the signal of a protein known to cause a cancer; Gleevec has an inhibitory effect against cell proliferation induced by BCRABL tyrosine kinase (TK) and against the KIT, ARG, and platelet-derived growth factor receptor (PDGFR) TKs.

Scientific evidence suggests that some TKs (such as PDGFR) are active in disease pathways for a number of hematological diseases. To explore Gleevec's full potential, Novartis AG conducted one...