BREAST CANCER RESEARCH PAYING DIVIDENDS; TWO NEW DRUGS OFFER EXCITING POSSIBILITIES, COMPLEXITIES.

Byline: Lawrence D. Wagman and George Somlo

A revolution in the fight against breast cancer is well under way. The decision by an advisory committee of the U.S. Food and Drug Administration to recommend the approval of the drug tamoxifen to prevent cancer shows we are finally learning how to attack the disease before it occurs.

This marks a breakthrough in medicine that doctors and researchers have been working on for decades.

Women with a high risk, including those with a history of breast cancer in their families, often face a very tough choice. Should they undergo breast surgery - bilateral prophylactic mastectomy, before any signs of the disease are found?

Now these women may have another choice. Tamoxifen has already been approved as a treatment to prevent recurrences of breast cancer. Now it is on track to be actually prescribed to high-risk women as a preventive option.

The FDA typically follows the recommendation of advisory committees and that would mean that the company that makes tamoxifen could advertise the drug's preventive effects and, insurance companies would likely cover the costs for high-risk women who have never had cancer.

At City of Hope Cancer Center, a National Cancer Institute-designated comprehensive cancer center, we participated in the clinical trials for tamoxifen. We are also participating in an effort to provide patients with access to herceptin, another drug that the advisory panel recommended for approval for women with advanced breast cancer. Both of these drugs represent advances in treatment following years of research and testing.

The path to approval for these two life-saving and life-enhancing drugs is built on the progress researchers have made in understanding the underlying causes of cancer. This knowledge has allowed us to rationally develop treatments that are more effective and less toxic to the patient.

But caution is always called for when we look at new treatments. One thing we know about science - usually progress is made by a series of steps along what can seem like an erratic path. These two drugs offer exciting possibilities, as well as complexities and uncertainties that patients must understand.

Let's look at tamoxifen first. Yes, there is now significant evidence from a large study conducted in the United States that the drug, which helps prevent the hormone estrogen from stimulating breast tissue, reduces the chances of breast cancer.

But two smaller European studies failed to confirm the U.S. results, which caused some researchers to doubt the rush to widely prescribe tamoxifen as a preventive measure.

FDA approval will probably allow any physician to prescribe the drug and millions of women could be potential candidates. But this raises serious questions too.

For all the benefits of tamoxifen there are also hazardous side effects that include potentially fatal blood clots and cancer of the uterine lining. So tamoxifen cannot be automatically prescribed. Patients and their physicians must understand the comparative risks and make individual decisions.

Yet, make no mistake, this drug provides a noninvasive option that has the potential of reducing the chances of getting breast cancer.

Herceptin is not a cure for breast cancer, but used alone or with chemotherapy has been shown to delay the progression of the disease which can translate into prolonging life and improving the quality of life.

Herceptin would be the first genetically-engineered drug for the treatment of advanced breast cancer, but we need to see how the drug works with larger numbers of patients to pin down exactly how and when the drug should be used as part of a treatment plan.

There are also hopes that the drug can play a role in the treatment of less advanced breast cancer. But this concept needs to be tested in carefully designed clinical trials.

The move forward on tamoxifen and herceptin is an important milestone. These drugs are potentially powerful weapons in our arsenal against breast cancer. They represent promising new approaches in the prevention and treatment of the disease.

The nation's cancer research, led by the NCI, is indeed beginning to pay the dividends we've all hoped for.

Congress is currently considering increasing funding to the National Institutes of Health, the parent organization of the NCI. As an NCI-designated comprehensive cancer center, we are pleased that our leaders understand the importance of cancer research to the nation.