Leukemia Pill Raved as `Holy Grail'

HOUSTON-A leukemia drug that patients say has fewer side effects than aspirin has shown surprising success in early trials, researchers at the University of Texas M.D. Anderson Cancer Center say.

The drug, STI571, a new treatment for chronic myelogenous leukemia, was a central topic at a weekend medical conference in San Francisco.

M.D. Anderson researchers reported that in all 31 CML patients given the drug at the center last year, the cancer went into complete remission. Lead investigator Dr. Moshe Talpaz called the pill ``the holy grail we are looking for in our business,'' the Houston Chronicle reported Sunday.

This year, 550 patients with chronic CML resistant to conventional treatment have been given the drug, and in more than 90 percent of the cases, their blood was normal after six months of treatment, according to research reported Saturday at the meeting of the American Society of Hematology.

``I will tell you, the hematology community is excited about the prospects for the approach this drug took,'' said Dr. Ed Benz, president of the hematology society and president of the Harvard-affiliated Dana-Farber Cancer Institute in Boston. ``We need many, many more studies and patients to know exactly how powerful this drug will be, but the early results are promising.''

CML causes a proliferation of white blood cells, which begin to outnumber other blood cells and can interfere with the functioning of organs. It affects about 4,500 Americans each year and is caused by a defect in a chromosome found in bone marrow cells. An abnormal enzyme is produced by the defect, which makes for the abundance of white blood cells.

The drug, developed by Oregon Health Sciences University researcher Dr. Brian Druker in collaboration with drug manufacturer Novartis Pharmaceuticals, focuses on shutting down the enzyme that causes the cancer without harming healthy cells.

Earlier this year, Druker was awarded a $7.5 million grant by the Leukemia and Lymphoma Society to oversee clinical trials of the pill at over 70 sites around the world.

``This has been a godsend,'' said Gerald Bourque, 62, a Houston sales engineer who began taking the pill for CML at M.D. Anderson in December. ``I have energy I didn't realize I had. It's unbelievable.''

The standard treatment for CML has been Interferon, a natural protein that revs up the body's immune defense but has side effects including pain and inflammation of the joints.

Novartis Pharmaceuticals, STI571's manufacturer, expects the new drug to become available commercially next year. A third-phase trial of the drug, involving more than 1,000 patients in the United States and Canada, is in progress.

The drug will also be used in clinical trials of glioblastoma, a brain tumor, and gastrointestinal stromal tumors, a rare cancer of the digestive system's connective tissue.