Herceptin Offers Hope for Some Women With Advanced Breast Cancer

From the WebMD Archives

March 14, 2001 -- Wall Street's passion for biotech may have cooled, but patients who are being treated with the first of the wave of new "designer" drugs feel differently.

Ginger Empey was the first patient to volunteer for an experimental study of the drug Herceptin, the results of which are published in the March 15 issue of The New England Journal of Medicine. "Before I got into the clinical trial, I was getting sicker and sicker because my breast cancer had spread. My life was going down the tube. My doctor told me there was no treatment that would help me, to go home, get my affairs in order, and not bother making another appointment. Yet here I am 5 years and 8 months later, with all of my liver tumors shrunk so much they have been too small to measure since 1997. I'm happy to see Spring again, and I expect to see many more," says Empey, of Bakersfield, Calif.

Partly as a result of this trial, Herceptin was approved by the FDA in 1998 for treatment of a very dangerous type of breast cancer. The cells of these cancers have high levels of the "HER2" growth factor. As a result, they grow more quickly than other types of breast cancer and often do not respond to standard breast cancer treatments. About 25-30% of breast cancers have these high levels of HER2. Women who have these aggressive cancers are likely to have early breast cancer relapses and shorter survival.

Herceptin, a bioengineered antibody developed by Genentech Inc., clicks onto the HER2 molecules that studded the surface of Empey's stubborn breast cancer cells. This blocks HER2 from sending the signals that help cancer cells grow and spread. The result is often that the tumors shrink and wither away.

"I had three big tumors that had spread to my liver. The result was that my liver was greatly enlarged, and I had a huge amount of fluid accumulated in my abdomen. When I started taking Herceptin, I looked like I was 6 months pregnant. By the end of the third month of weekly doses, the fluid was gone and my liver metastases had shrunk by 25%. My pain improved after the very first dose. Within 2 years, the tumors had been reduced to tiny spots too small to measure, and they have stayed that way since 1997," Empey tells WebMD.


Larry Norton, MD, one of the study investigators, says that Herceptin had two important effects in the clinical trial: It increases the number of patients who respond to treatment for their breast cancer, and it makes those responses last longer.

"Survival advantages of this kind are hard to find in stage IV [advanced] breast cancer such as the patients in this trial," says Norton, who heads the solid tumor oncology division at Memorial Sloan-Kettering Cancer Center in New York City.

Empey had to fight to be one of those study participants. "My previous doctor -- the one who had told me to essentially just go home and die -- was not encouraging about my chances of getting in trial," she says. "I managed to get the name of the study out of him, then I went to the Rhonda Fleming Mann Resource Center for Women with Cancer at UCLA to investigate. They had information about the study and a tape of a speech about it by Dennis Slamon, MD, the UCLA oncologist who headed the study. When I listened to the tape, I realized that I very likely had HER2-overexpressing breast cancer, since my cancer had spread very quickly and had not responded to two different chemotherapy programs. I besieged Dr. Slamon's office with calls over the summer before he started the study, and I was the first patient to take Herceptin. If you are in my situation, pursue information about clinical trials yourself. Nobody else is going to do it for you. And don't sit around and wait for people to call you back. Clinical trials offer hope, but we have to remember that we are in charge of our own health."

Norton stresses that Herceptin is recommended only for women who have advanced breast cancer and whose cancers have high levels of HER2. Without HER2, the drug does not work.

The clinical trial showed that Herceptin works even better when used in combination with standard cancer chemotherapy. The combination increased survival in women with high levels of HER2 and metastatic breast cancer by 24% compared to chemotherapy alone.