Tags: FDA | Approves | Miracle | Leukemia | Drug

FDA Approves Miracle Leukemia Drug

Thursday, 10 May 2001 12:00 AM

The drug, an oral medication called Gleevec, reduces the number of cancerous cells in the blood and bone marrow of patients with chronic myeloid leukemia. Manufacturer Novartis Pharma AG, in East Hanover, N.J., says Gleevec should be available by month's end.

Ron Paquette, a leukemia researcher at Jonsson Cancer Center at the University of California, Los Angeles, told United Press International: "This is the most promising drug for CML that's been developed. It's highly effective in terms of bringing their white blood counts into the normal range and in dramatically reducing the number of leukemia cells that are present in the blood and the bone marrow."

In a number of patients, study investigators lowered leukemia cells to below detectable levels, said Paquette, himself a researcher with about 100 patients in the Novartis-funded trial. Such results have been achieved within three months in some cases, he added.

A patient who was treated in the clinical trials at the Jonsson Cancer Center, Peter Barnes, told UPI: "Basically, it really has saved my life. I was going into the third and last stage of chronic myeloid leukemia, which is blast crisis, and I got onto the program just in time.

"They had given me about a year," he continued. "Now the Gleevec has reversed the process and put me back into the first stage of the disease, which is the chronic stage, the manageable stage. I feel fantastic. ... Literally, the next morning after I took the first dose I could feel some improvement."

According to the Food and Drug Administration, CML occurs when pieces from two different chromosomes break off and reattach on another chromosome, forming a structure of genetic material known as the Philadelphia chromosome. As a result, a blood cell enzyme is turned on all the time and potentially life-threatening levels of mature and immature white blood cells accumulate in bone marrow and blood.

The hallmark of leukemia is higher-than-normal white cell blood counts, but with standard treatments, CML patients can feel well for years. Eventually the cancer progresses to an aggressive phase that is no longer effectively treatable.

Gleevec trials treated the early chronic phase and also the later phase. About half of all patients have showed a response, the best coming with those still in the early stage.

Specifically, a once-a-day, 400-milligram pill of Gleevec reduced white blood cell counts in 98 percent of chronic-phase patients to normal range.

The drug was also tested in 58 patients in blast crisis, the most advanced phase of the disease, in doses ranging from 300 mg to 1,000 mg. Fifty-five percent of the patients showed a measurable response, and 11 patients underwent a complete remission.

It is not yet known if Gleevec treatments will generally extend life expectancy, Paquette said. The drug has been fairly well tolerated so far, with some patients experiencing fluid retention, swelling around the eyes and ankles, elevated liver enzymes, muscle cramps, stomach upset and nausea.

"We know that the abnormal chromosome in the disease produces an abnormal protein. The drug was designed to specifically inhibit the function of the abnormal protein. It binds to the abnormal protein and prevents its activity," Paquette said.

Because that protein function is required for the leukemia to advance and take over the bone marrow, by inhibiting the function of the protein it is possible to markedly suppress the leukemia.

John Stevens, vice president for research at the American Cancer Society, said in a statement, "We are very excited about the development of Gleevec, which represents the first example of a totally rational approach to cancer treatment based on molecular targeting."

After clinical trials, the drug was approved in less than three months by the FDA under regulations that allow for the rapid bringing to market of specific drugs. Gleevec is part of FDA's orphan-drug program that provides financial incentives for drugs developed to treat rare diseases, according to the FDA.

Marshall Lichtman, executive vice president of research and medical programs at the Leukemia and Lymphoma Society, said even at this early age of study, Gleevec appears to be a dramatic step forward for patients with CML and potentially for patients with other diseases as well.

"Because of its specificity, the drug provides several advantages: ease of administration, usually minimal side effects, little adverse effect on other tissues and a high response rate," he said. "It will take another five or so years to learn the impact on the restoration of good health. But the FDA's approval confirms the potential efficacy of treatment to the genetic lesion in cancer."

About one person in 100,000 each year in the United States develops CML, and about 10,000 have the disease. Its early symptoms include fatigue, joint pain and swelling of the spleen. Once the cancer reaches the end stages death usually occurs in three to four years.

Copyright 2001 by United Press International.

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The drug, an oral medication called Gleevec, reduces the number of cancerous cells in the blood and bone marrow of patients with chronic myeloid leukemia. Manufacturer Novartis Pharma AG, in East Hanover, N.J., says Gleevec should be available by month's end. Ron Paquette,...
Thursday, 10 May 2001 12:00 AM
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