Wednesday, May 23, 2001

Gleevec attacks leukemia protein


Patient calls new drug 'phenomenal'

By Tim Bonfield
The Cincinnati Enquirer

        Two years ago, Elaine Pottenger wasn't counting on ever teaching again, or even seeing her 16-year-old son graduate from college. Sickened by leukemia, she had almost given up hope.

        Today, the Germantown, Ohio, woman is cancer-free, one of the first Southwest Ohio people to participate in tests of a breakthrough drug that's the talk of the medical world.

[photo] Elaine Pottenger has returned to teaching full-time after treatment with Gleevec wiped out her leukemia. Researchers are testing the drug on other cancers.
(Glenn Hartong photo)
| ZOOM |
        Scientists and researchers already are hoping that the drug's “smart bomb” attack on the protein that appears to cause leukemia like Mrs. Pottenger's will mean similar drugs for other cancers in the future.

        So successful were clinical trials that the drug, Gleevec, recently won unusually quick approval by the U.S. Food and Drug Administration.

        “When I was growing up, you heard the word leukemia and you started making funeral plans,” Mrs. Pottenger says. “Even with the treatments they have today, you have to put a lot of your life aside.”

        Mrs. Pottenger, 49, was diagnosed in 1993 with chronic myelogenous leukemia (CML), one of four main types. She underwent treatments, including chemotherapy, but they eventually lost effectiveness.

        About a year ago, her husband heard about limited clinical trials of Gleevec. Now Mrs. Pottenger is back to full-time work as a special education teacher at Valley View High School.

        She calls the drug — so new that it hasn't been added to many insurance plans' coverage lists — “phenomenal.”

        “In the first week, my blood counts were back to normal. In the first month, my bone marrow was 100 percent clear,” she says. “I was in such shock, I couldn't believe it.”

Targeting cancer cells
        Gleevec, manufactured by New Jersey company Novartis Oncology, is based on new understanding of how cancer works at the genetic level.

        The drug attacks CML more precisely than previous medications, targeting cancer cells directly with little or no damage to healthy cells. And it works as a pill, rather than being injected or administered intravenously.

        Gleevec appears to work extremely well. A recent study published in the New England Journal of Medicine reports that 51 of 53 patients who took the drug remained in remission at least a year. Test results were so impressive that the FDA approved Gleevec within 32 months, about half as long as most new drugs take to win approval.

        “This is a huge breakthrough for people with CML,” says Dr. Randy Broun, director of the stem-cell transplant program at Jewish Hospital, where many leukemia patients seek bone-marrow transplants to battle their disease.

        Gleevec was approved May 10, and shipments already have begun. More than 7,500 people have taken it worldwide, in 30 countries including the United States. The drug is expensive, costing $2,000 to $2,400 a month, and it must be taken indefinitely.

        “We are absolutely thrilled that the FDA has approved this drug. We have never seen a drug with these kinds of results in the past,” says Jay VanWinkle, executive director of the southern Ohio chapter of the Leukemia & Lymphoma Society.

        Nationwide, leukemia will strike an estimated 31,500 people this year; 4,700 of them will have CML, according to the American Cancer Society.

        CML is a chronic form of leukemia that strikes mostly adults. With standard chemotherapy using interferon-alpha, patients often live several years in stable health. But the treatment eventually loses effectiveness for many patients, and they slip into an acute phase of the disease that can kill within months.

        Mrs. Pottenger learned about Gleevec from a TV news report and her husband's research on the Internet. In January 2000, she enrolled in one of the few U.S. clinical trials of the drug, at the Karmanos Cancer Institute at the Detroit Medical Center.

        Seemingly overnight, Mrs. Pottenger's life was transformed.

        “My energy level is up. I'm working full-time,” she says. “We can make plans again, for college, for the future.”

        It will take at least five years before researchers can say Gleevec is a real cure for CML.

        The drug hasn't been studied long enough to know how long it will keep the disease at bay, or whether it has any surprise side effects.
       

"Smart-bomb' compounds
        Still, cancer experts hail Gleevec for several reasons.

        For one, Gleevec is a pill. People can take it at home with fewer side effects than chemotherapy treatments. For another, the science behind Gleevec offers hope for similar treatments for other types of cancer.

        Gleevec is designed to block a protein produced by the “Philadelphia chromosome,” a genetic abnormality found in people with CML. The protein is known to trigger leukemia's explosive growth of white blood cells.

        Gleevec's attack is so precise that it causes little damage to noncancer cells, producing fewer side effects than traditional treatments. What remains to be seen is how far the genetic concepts behind Gleevec can be extended, Dr. Broun says.

        Some studies already indicate that the same protein that plays a central role in CML is involved in the growth of other types of cancer cells.

        Perhaps more importantly, Gleevec's success has inspired a hunt for other “smart bomb” compounds that can block similar growth proteins in other cancers.

        “This is great for people with CML. But CML only affects about 5,000 people (in the United States) a year,” Dr. Broun says. “If this can work in something like lung cancer (which strikes about 170,000 people a year), then you'll really have a breakthrough.”
       



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