By Arlene Weintraub Kathy Anderson has always been afraid that the breast cancer she survived eight years ago would come back. The statistics certainly are grim: 15% of the 1.2 million women diagnosed with the disease each year relapse later in life. But on Oct. 7, Anderson got welcome news from her doctor: The experimental drug she had been taking for the past two years, Novartis' (NVS) Femara, had been shown to slash the risk of recurrence by 43%. "I was amazed and encouraged," says Anderson, 50, an elementary school principal in Toronto.
Femara is just one of a multitude of "maintenance" therapies that cancer patients may be able to take for years at a stretch and rely on for longer-term survival. These include new forms of chemotherapy that don't cause dire side effects and vaccines that can be administered repeatedly to boost the immune system while the patient is in remission.
Although maintenance therapies probably won't be definitive cures -- especially for killers such as colon and lung cancer -- they could become powerful new tools for oncologists seeking to treat cancer as a chronic disease. "The ideal for us is to make this a condition that patients can live with, like heart disease," says Dr. George Somlo, an oncologist with the City of Hope in Duarte, Calif. "It really is a new paradigm."
CLOSE-RANGE ATTACK. One of the key goals of clinicians and cancer researchers is to eliminate chemotherapy's debilitating side effects. Cell Therapeutics (CTIC), a Seattle biotech company, plans to seek Food & Drug Administration approval next year for a drug called Xyotax, which combines the chemo agent paclitaxel (generic Taxol) with a protein found in green leafy vegetables. The protein causes the chemo to zoom in on tumor cells, so less of the drug ends up seeping into healthy tissues. That may reduce severe nausea and hair loss, and possibly prevent patients from becoming resistant to chemo. The drug has shown promise in treating ovarian cancer and some forms of lung cancer.
Improving the comfort factor for cancer patients has become a major push in oncology labs across the world. Powerful new painkillers obviously have a role to play. But sick patients are only one part of the equation. "We also need to look for novel nontoxic treatments of patients who are in remission, in the hopes of delaying or preventing relapse," says Dr. Paul Sabbatini, a physician specializing in gynecological oncology at Memorial Sloan-Kettering Cancer Centers.
Research institutes and Big Pharma, meanwhile, continue to invest in completely new compounds designed to block individual cancer-causing agents. The goal -- assuming the disease can't be erradicated -- is to limit the growth and spread of tumors. Abbott Laboratories (ABT) has a dozen anticancer agents in its pipeline, including one that suppresses a protein called endothelin, which stimulates tumor growth. And in late September, Eli Lilly (LLY) filed for FDA approval of a drug that slows the replication of tumor cells in some forms of lung cancer by blocking three key enzymes.
EXTRA PROTECTION. Most maintenance therapies drawing the attention of oncologists are cancer drugs that are already on the market, or improved versions of them. Femara, for example, was first approved in 1996 for patients with advanced breast cancer, but the recent trial provided the first evidence that it might also prevent the disease from recurring in post-menopausal women.
The once-daily pill suppresses the hormone estrogen, a key stimulant of tumor growth. The drug could provide an extra layer of protection for breast-cancer survivors after they stop taking the standard treatment, tamoxifen, which appears to provide no further benefit after five years.
Another approved drug being tested for long-term therapy is Genentech's (DNA) Herceptin. It blocks a protein that causes one form of breast cancer. And it triggers few side effects because it attaches only to the receptor on a cell that secretes that protein. "Prior therapies were so toxic [that] oncologists couldn't imagine using them to keep patients in remission," says Dr. Susan Desmond-Hellmann, Genentech's chief medical officer. "Now everyone talks about maintenance therapy."
COMMUTED SENTENCES. The quest to move these new medicines out of the concept stage and into cancer clinics will be challenging. Proving that the drugs actually extend life -- a key metric for FDA approval -- can take more than five years of trials, involving thousands of patients. And in aggressive forms of the disease, new treatments may increase survival by only a few months. So patients may have to take one drug after another, or several in combination, to get any benefit.
The potential windfall for companies that clear the many hurdles is enormous. Novartis' Gleevec, for chronic myeloid leukemia, brought in $515 million in sales in the first half of this year alone. But cancer researchers say it's the patients that keep them motivated. "These people were given death sentences," says Dr. David Epstein, president of Novartis' global oncology division. "Now they go to work. They play with their grandkids."
Naturally, longer drug regimens also signify increased revenues for manufacturers -- but patients and their families aren't likely to turn cynical as long as the new treatments keep coming. Weintraub is a BusinessWeek correspondent in Los Angeles
With Michael Arndt in Chicago