New Leukemia Drug Works for Incurable Stomach Tumors, Too

From the WebMD Archives

May 13, 2001 (San Francisco) -- Todd Hendrickson says that last August he had "about 60 days to live. I had tumors the size of two footballs in my gut and I couldn't get out of bed unless my wife pulled me up." That changed on Aug. 3 when he became "patient No. 1" in a study of a new cancer drug code named STI571.

Today, Hendrickson spent his Sunday afternoon mowing his neighbor's lawn. And that, says Hendrickson, is a miracle

Just days after Gleevec -- the pill formerly known as STI571 -- received FDA approval to treat a type of leukemia called chronic myeloid leukemia, cancer researchers from the United States and Europe now report that the drug also works in patients with a previously untreatable type of stomach cancer called gastrointestinal stromal tumor or GIST.

John Mendelsohn, MD, president of the University of Texas M.D. Anderson Cancer Center in Houston, says this new drug is one of about a dozen new compounds that are taking cancer treatment in a new direction -- targeted therapy. These new "smart bombs" seek out the unique property that turns a cell malignant, he says.

But he adds that unlike chronic myeloid leukemia, or CML, "with solid tumors, it is unlikely that there will be a single target."

Hendrickson, a 43-year-old investment banker from Minnetonka, Minn., tells WebMD that taking 400 mg of the drug each day since Aug. 3, 2000, has "shrunk my tumors by 82%. Right now they are basically liquefied, and I think they will just be absorbed by my body."

He adds, however, that his doctors haven't told him what will happen because "we are flying in new territory now."

His cancer was diagnosed 10 days before the birth of his youngest son. His oncologist told him then to "get my affairs in order because I wouldn't live to see that child's second birthday." That was October 1998. A year and half earlier, Hendrickson's surgeons had removed a 10 cm tumor from his stomach, but by that day in October the cancer was back and it had spread.

Around that time, Hendrickson met University of Minnesota transplant surgeon Tim Sielaff, MD. "He told me that he could keep cutting me to keep me alive until I could find the science to cure me," says Hendrickson. As surgery followed surgery, Hendrickson tried six different chemotherapy regimens, but each one failed to stop the cancer. Then last year, an oncologist at UCLA "referred me to Chuck Blanke."

That's Charles Blanke, MD, an associate professor of medicine at the University of Oregon Health Sciences Center in Portland. Blanke's colleague Bruce Druker, MD, developed STI571. "Chuck sent me to Druker, who tested my tumors and they were full of c-kit."

Blanke tells WebMD that Gleevec hones in on c-kit, a gene that sends a signal that tells cells to grow. This gene is present in many cancer cells, he says. If c-kit is abnormal it causes uncontrolled cell growth and multiplication, which forms a tumor. The drug blocks the signal. Oncologist Michael Gordon, MD, of the Arizona Cancer Center in Tucson, says that Gleevec works like a circuit breaker to shut down the power to the cell.

In his study of 139 patients with GIST, more than half of the patients responded to treatment, says Blanke. Hendrickson was patient No. 1.

"They rushed the study because I was so near death," Hendrickson says. "I knew the drug was working right away because in seven days I went from severe pain to moderate pain and after 40 days my first CT scan showed that the tumor was reduced by 47%."

Allan T. van Oosterom, MD, professor of medicine at the University of Leuven in Belgium, says that a smaller study of 36 patients with GIST suggests that the most effective dose is actually 800 mg or twice what Hendrickson takes. "I have two patients who didn't respond at 400 mg; we increased to 800 mg and now they are responding," van Oosterom tells WebMD.

At a press conference Sunday where the Gleevec results were presented at the American Society of Clinical Oncologists meeting held here, he said "I have been waiting 30 years to report results like these."

Although the buzz among the 26,000 cancer specialists attending this meeting is definitely Gleevec, Blanke and van Oosterom both say they are worried that oncologists will try the drug on the wrong patients. The drug is approved for leukemia and "demonstrated efficacy in GIST", but this targeted therapy only works if the target is present. In solid tumors, that means that the tumor must test positive for c-kit and have the mutation that causes overproduction of cells.

Cancer experts worry, too, that patients will demand treatment with the new drug, which has received almost as much publicity about its ease of use as it has about its effectiveness. Unlike other cancer treatments, Gleevec is a pill. Patients should take it after meals, says van Oosterom.

Moreover, the side effects of the drug are minimal. "Sleep disturbances, puffiness around the eyes, a slight rash," says Hendrickson. And the side effects "usually disappear after eight weeks," says van Oosterom.

Blanke says that several other types of cancers express c-kit, and his center is beginning a trial in terminal lung cancer patients while other researchers are testing the drug in pancreatic cancers and brain tumors.

Meanwhile, in Minnesota, Hendrickson celebrated yet another holiday, Mothers' Day, with his wife and three sons. He observed, "I was knocking on death's door until this drug. This stuff is absolutely a miracle."