Drug shows promise in leukemia and intestinal cancer

In this story: Results in treating advanced leukemia Glivec and other cancers Pill might soon get marketing approval RELATED STORIES, SITES

By Rhonda Rowland
CNN Medical Unit

PORTLAND, Oregon (CNN) -- Initial tests of an experimental cancer pill show that the treatment is durable, or lasting, researchers said.

Glivec was studied in patients with chronic myelogenous leukemia (CML) who have failed standard therapy.

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"Results were for one full year of treatment," said Dr. Brian Druker, lead investigator, with Oregon Health Sciences University, in Portland. "Fifty-one of the 53 patients remain in remission."

The findings are published in the New England Journal of Medicine.

It's too early to talk about a cure, but there are hints it may be possible, according to Druker. In at least one patient, leukemia cells could not be detected -- if those results continue for a year or two, patients could be taken off treatment.

"I'm incredibly encouraged by our results. I can't think of another agent in all of oncology where we've seen these kinds of dramatic results," said Druker. "We've given patients hope and have restored them to good health."

Glivec, a pill that is taken once a day, targets only cancer cells while leaving healthy, normal cells virtually untouched. Patients so far have reported only minimal side effects, including nausea, muscle cramps and fluid retention.

"This is a drug that controls cancer with almost no side effects," said Dr. Harmon Eyre with the American Cancer Society. "So, for us in the world of oncology to have that is a huge breakthrough. It's a great drug, a great new discovery."

Results in treating advanced leukemia

A second study in the New England Journal of Medicine showed encouraging results with Glivec in patients with advanced leukemia, a condition known as "blast crisis."

"These are patients in which almost nothing works," Druker explained.

"They often receive chemotherapy and remain in the hospital for 4-6 weeks and are deathly ill. With Glivec, they were able to receive treatment on an out-patient basis."

Most patients responded to Glivec, but relapse was common after 3 to 4 months. Seven of 38 patients with myeloid blast crisis have remained in remission for up to a year.

"It's amazing that it's as active in the blast crisis as it is," said Eyre. "Essentially for all practical purposes that disease is basically untreatable. It was completely unexpected in my mind. It's possible that, if combined with chemotherapy, we may see even better responses."

Glivec and other cancers

Gastointestinal stromal tumor (GIST) affects about 2,000 people in the United States every year and almost nothing works in treating it. A case report of Finnish women shows that Glivec works against GIST as well. There is still no sign of cancer following one year of treatment,.

"The growth of GIST cells is driven by a similar abnormality that drives the growth of CML cells," said Druker.

Glivec appears to shut down proteins regulating cell growth.

Researchers say this case report provided the impetus for other GIST patients to be treated in clinical trials. Further results will be reported in May.

Additional studies will evaluate the effectiveness of the pill in glioblastoma, a lethal brain tumor, small-cell lung cancer and prostate cancer.

"If this targeted cancer therapy approach works in GIST and CML, it will work in each and every tumor," predicted Druker. "In these two cancers we understand what drives the growth and the drug shuts down the abnormality. We've been able to disable the cancer, without disabling the patient. There's no reason why it shouldn't work in every other cancer."

Druker said researchers need to understand critical early differences between each cancer type and normal cells. The mapping of the entire human genome will facilitate this process.

Pill might soon get marketing approval

Novartis, the makers of Glivec, filed in March for marketing approval with the U.S. Food and Drug Administration. Submissions have also occurred in the European Union, Canada, Switzerland and Australia. An application may be filed soon in Japan. Results in expanded trials with 1,230 CML patients continue to show promise. So far, Glivec has been studied in more than 5,000 patients in 30 countries.

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Chronic Myelogenous Leukemia (CML)
Leukemia Research Foundation
The Leukemia and Lymphoma Society
American Cancer Society
Novartis

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