Gleevec's innovative targeted approach improves lives for people with these cancers
Today the U.S. Food and Drug Administration approved the drug, Gleevec developed by Oregon Health & Science University Cancer Institute researcher Brian Druker, M.D., to be used as an effective treatment for five more cancers.
The tumor cancer is dermatofibrosarcoma protuberans, or DFSP, a type of tumor that begins as a hard lump found in the skin of the chest, abdomen or leg.
The blood cancers covered by the new clearance are: Philadelphia chromosome-positive acute lymphoblastic leukemia, or Ph+ ALL, a rapidly progressive blood cancer; certain forms of myeloproliferative disorders, or MPD, diseases in which too many types of certain blood cells are made in the bone marrow; hypereosinophilic syndrome, HES, in which the body persistently makes too many of the white blood cells eosinophils; and aggressive systemic mastocytosis, or SM, in which the body has too many of a type of white blood cells called mast cells.
"Gleevec works on these cancers because they are driven by a target of Gleevec and these targets are the Achilles' heel in these cancers," said Druker, JELD-WEN Chair of Leukemia Research at OHSU Cancer Institute and Howard Hughes Medical Institute Investigator. Druker was an investigator in the Ph+All study.
Michael Heinrich, M.D., also a member of the OHSU Cancer Institute and Portland Veterans Affairs Medical Center, and Christopher Corless, M.D., Ph.D., vice chairman for research in the Department of Pathology and member of the OHSU Cancer Institute, were investigators on the DSFP study leading to the FDA approval.
"We were trying to find more applications for Gleevec. There was no known treatment for this type of sarcoma except for surgery. It typically takes several surgeries to completely remove the tumor. DFSP tumors are like weeds that have roots that go everywhere and keep coming back," Heinrich said.
Each of the eight patients in his clinical trial showed a positive response to Gleevec.
"It shows the targeted therapy works best. We have found the target and have a drug that deactivates it. It is important because it shows how research can lead to treatment, even for rare diseases. Next we hope to study the effectiveness of Gleevec and surgery," he said.
Gleevec, a yellow-colored pill marketed by Novartis since 2001, is the first approved drug to directly turn off the signal of a protein known to cause cancer. It was the first drug to demonstrate that molecular targeted therapy works. Initially used to treat patients with chronic myelogenous leukemia, also called CML, has also proved successful in treating GIST, gastrointestinal stromal tumors. Before Gleevec, there were few effective treatments for GIST patients.
"What this tells us is that even for the more common cancers, such as breast or prostate, we are on the right track. It means that if we can identify the Achilles' heels in those cancers, we can develop specific, targeted therapies for all cancers," Druker said.
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The OHSU Cancer Institute remains the only cancer center designated by the National Cancer Institute center between Sacramento and Seattle. It comprises some 120 clinical researchers, basic scientists and population scientists who work together to translate scientific discoveries into longer and better lives for Oregon's cancer patients. In the lab, basic scientists examine cancer cells and normal cells to uncover molecular abnormalities that cause the disease. This basic science informs more than 200 clinical trials conducted at the OHSU Cancer Institute.