Leukemia Pill Continues to Show Remarkable Results

12/02/00    Portland, Ore.

Large Groups of Patients Show Rapid Improvement as FDA Submission Nears.

A pill developed by Novartis Oncology, in collaboration with Oregon Health Sciences University researcher Brian Druker, M.D., continues to produce astounding results in patients with chronic myelogenous leukemia (CML). Researchers will present data on the second phase of clinical trials, looking at more than 1,000 patients, at the American Society of Hematology annual meeting in San Francisco from Dec. 1 - 5.

"As hard as it is to imagine, the results from these larger studies are even more astounding than our initial studies," said Druker, associate professor of medicine in the OHSU School of Medicine and principal investigator of the initial studies. "These current studies show that a greater number of our patients have a complete disappearance of leukemia cells than in the first trial. We also have treated patients with more advanced disease with striking results.

"Just as exciting is the international cooperation and sheer size of this patient study," Druker added. After beginning as a small study at OHSU, UCLA's Jonsson Cancer Center and M.D. Anderson Cancer Center in Texas, the clinical trial of this drug has expanded to more than 30 sites in six countries. Further expansion to 27 countries is underway.

The drug, Gleevec, formerly known as STI571, targets an enzyme found only in the leukemia cells of CML patients. The result is a halt to the progression of the disease without the side effects of treatments like chemotherapy, which kills both normal and abnormal cells.

CML affects approximately 5,000 Americans each year and is characterized by an excessive growth of white blood cells. The often-fatal disease is caused by a chromosomal defect that produces an abnormal enzyme called bcr-abl, which causes the overproduction of white blood cells. Gleevec was engineered to inhibit the activity of bcr-abl.

Initial results presented last year showed that 31 of 31 patients in the study had a complete normalization of their white blood cell counts. Doctors also saw the molecular cause of the disease disappear from several patients.

"The potential significance of this type of research extends beyond leukemia," said Druker, whose research is supported in part by the Leukemia and Lymphoma Society and the National Cancer Institute. "One of the major goals of cancer research has been to identify differences between cancer cells and normal cells so that these differences can be targeted with more effective and less toxic treatments. That's exactly what we've seen happen in these patients."

Novartis Oncology plans to send data from the clinical trials to the Food and Drug Administration early next year for possible approval before the end of 2001. For more information about Gleevec clinical trials at OHSU, call 503 494-1117, or for studies in the U.S., call Novartis Oncology at 800 340-6843.