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Newly Approved Drug Works Wonders for Many Leukemia Patients

A new pill used to treat leukemia is working wonders for most of the patients who take it. The US Food and Drug Administration (FDA) approved the cancer drug, Gleevec, in record time--2_ months. Dr. Emily Senay explains.

Previously known as STI 571, Gleevec has proven effective in halting the spread of leukemia in a large number of patients who don't respond to existing treatments. It is one of the first treatments of its kind to target specific cancer-causing enzymes within a cancer cell and avoid affecting the healthy cells.

The once-a-day pill regimen specifically targets chronic myeloid leukemia (CML). The disease is caused by a protein that produces an abnormal chromosome, which in turn triggers a huge increase in the number of white blood cells in the body. CML claims more than 2,000 US lives every year. Gleevec was designed based on the principle of molecular targeting--killing leukemia cells while leaving normal white cells alone.

Scientists say the idea of fighting cancer by designing a small molecule that will specifically attack a target protein should work against other forms of the disease as well. Gleevec has been hailed by some as "the wave of the future" in fighting cancer.

For leukemia patient Beverly Caplan, chemotherapy used to make the day-to-day routine of life with cancer unbearable. "I got neuropathy in my feet, which is extremely painful, and many a night I cried myself to sleep," says Caplan. "I have memory loss, balance problems, and no energy at all."

But her options were limited. Despite the side effects, Caplan continued to take interferon until it stopped working. Her doctors then suggested she try a new experimental drug called STI 571 (Gleevec).

"I knew I had to do it. I knew I had to get off the interferon because that wasn't really working as well as it did in the beginning," says Caplan. "I knew if I didn't do something, I wouldn't be here."

Gleevec stopped Caplan's leukemia in its tracks, and because it does not affect healthy cells, Caplan avoided the side effects so common with other forms of chemotherapy.

"Since I've been on the pills, I do whatever I want to do. It just made such a difference in my life," says Caplan.

Caplan's doctor, Carole Miller, an oncologist at the Johns Hopkins Oncology Center in Baltimore, Maryland, says Caplan has had a very good response. By the time of her 3-month visit, she was in complete remission, and she has stayed in that state of complete remission.

Miller says she believes that the science behind the new pill will produce many more therapies for many more types of cancer. "It kills mainly just the bad cells, whereas with conventional chemotherapy you have to kill many of the good cells along with the bad cells," says Miller. "We hope that this will serve as an opening of the door of a new way of treating cancer cells."

But for Beverly Caplan, the future is here every day, enjoying life with her grandkids, 1 day at a time. "I ike to be with them. They are fun to be with. They are funny and cute and keep me laughing all day long," says Caplan.

An obvious question is: Does the pill work on other cancers?

Yes. There is evidence that it works on a rare form of stomach cancer, and studies are gearing up to see if it will work with prostate and breast cancer, too.

Could this be a cure for cancer?

There's no such thing yet when it comes to cancer. But this is one of the most promising avenues of treatment to come along in a long time. It's exciting because the drug works to target specific cancer cells. The hope is that if you can identify which enzymes cause the many different types of cancer, you can tailor new drugs that target the enzymes particular to those cancers.

What is the cost of the drug?

It is expected to cost between $2,000 and $2,400 monthly, which according to officials of Novartis Pharmaceuticals, the maker of Gleevec, is comparable to other current cancer treatments.


But the company is setting up a program to make sure it is available to uninsured, low-income people. It will be priced at a sliding scale for people with annual incomes below $100,000 and would be free for those making under $40,000 a year.

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