Drug first tested in Oregon may have effect on certain targeted tumors
Researchers at Oregon Health & Science University are beginning a clinical trial using Gleevec, a molecularly targeted anti-cancer drug, on a variety of cancers expressing structurally-similar proteins. Novartis Pharmaceuticals, maker of Gleevec, is sponsoring limited studies at 12 institutions around the world to see whether the drug will have an effect on certain cancers that are partially driven by proteins known to be inhibited by Gleevec.
"The premise is that we know Gleevec can stop the activity of these proteins in cells, and these proteins are present in certain cancers," said Michael Heinrich, M.D., associate professor of medicine at OHSU and principal investigator of the study. "What we don't know is whether stopping these two proteins, KIT and PDGF-R, will have a significant impact on the growth of these cancers, or if the proteins are only minor players in the growth of these mutant cells."
OHSU will accept up to 10 patients with several fairly uncommon diseases: chronic myelomonocytic leukemia with translocation involving the PDGF-R gene, dermatofibrosarcoma protuberans, mastocytosis, platinum refractory seminoma and synovial sarcoma. Patients accepted into the study will be at a life-threatening or advanced stage of their disease and not particularly responsive to chemotherapy treatment.
Gleevec, formerly known as STI571, was developed by Brian Druker, M.D., director of the OHSU Cancer Institute's Leukemia Center, in collaboration with Novartis, to specifically stop the abnormal protein that causes chronic myelogenous leukemia, or CML. That protein, BCR-ABL, is structurally related to KIT and PDGF-R, and Gleevec inhibits each of the proteins. The Food and Drug Administration approved Gleevec in May for use in the treatment of CML. In early studies the drug also has been shown to be effective in treating gastrointestinal stromal tumors, a deadly intestinal cancer that is driven primarily by KIT.
Novartis and the National Cancer Institute already have begun clinical trials with Gleevec on a rare brain cancer called glioblastoma, small-cell lung cancer and a form of prostate cancer. Because Gleevec has been determined to be safe in the trials for CML, all these studies will focus on determining its effectiveness in treating these other cancers.
Patients seeking information on Gleevec can call the OHSU Cancer Institute at 503 494-1080 or visit www.ohsu.edu/oci.