NIH Press Release
National Cancer Institute

Tuesday, May 5, 1998

NCI Press Office
(301) 496-6641

Questions and Answers
About NCI's Role in the Development of Herceptin®

A recent large study has suggested that an experimental drug called Herceptin® can be beneficial for some patients with breast cancer that has spread to other parts of the body. Herceptin® is a monoclonal antibody, one of a group of drugs designed to attack specific cancer cells. Herceptin's targets are cancer cells that produce a protein called HER2 or HER2/neu, which occurs in high numbers in about 25 percent to 30 percent of breast cancers. The company that developed Herceptin®, Genentech in South San Francisco, Calif., is in the process of applying to the Food and Drug Administration (FDA) for permission to market this anti-HER2 monoclonal antibody.

The favorable study results have raised two issues: 1) the need to expand availability of Herceptin® while the FDA reviews Genentech's application, and 2) the need to expand clinical research with the drug. The National Cancer Institute (NCI) is working with Genentech to help make the drug available for testing in larger numbers of breast cancer patients and in more places around the country. NCI is also collaborating with Genentech to expand studies of Herceptin® for other indications and uses.

The purpose of this fact sheet is to describe NCI's role in this collaboration. More information about Genentech's work with Herceptin® and about the drug itself is available from the company at (650) 225-5759.


1. What is being done to increase supplies?

Current supplies of Herceptin® are quite limited. However, while awaiting FDA review, Genentech is increasing production of the drug and continuing to make it available through an "expanded access program." Under this program, which has been in effect for some time, patients who meet the Genentech eligibility criteria may enter a lottery to receive Herceptin®. Patients selected by lottery will become part of a non-randomized study. The lottery system was designed in consultation with breast cancer patient advocates, who consider a lottery the fairest way to make the currently limited supplies of the drug available.

2. What role will NCI play in expanding the availability of Herceptin®?

Initially, patients selected by the Genentech lottery were able to receive the drug as part of a study at about ten different hospitals. Now, the NCI is arranging to have the drug available in more locations through its Treatment Referral Center program. This program refers patients to receive experimental drugs at more than 50 NCI-designated cancer centers around the country. In the case of Herceptin®, some of these centers should be ready to accept eligible patients — those whose numbers are selected in the Genentech lottery — in mid-1998.

3. How can patients and physicians get more information?

Patients with advanced breast cancer who think they may be interested in entering HER2 trials, and their physicians, may call the NCI's Cancer Information Service (CIS) at 1-800-4-CANCER (1-800-422-6237). CIS information specialists will explain what steps a patient's physician should take to enter the patient in the Genentech lottery. Information specialists can also provide information on other trials.


4. What other studies of Herceptin® in breast cancer will NCI sponsor?

NCI will be working with Genentech to study the use of Herceptin® at different stages of breast cancer and in combination with other agents. These new breast cancer trials could start enrolling patients in mid-1998. These trials will be carried out by NCI's cooperative clinical trial groups and by investigators in other NCI-sponsored centers.

5. Is NCI supporting studies of Herceptin® to treat other cancers?

Yes. NCI and Genentech are already working together to study Herceptin® in tumors other than breast cancer. One current study is looking at the drug in a variety of solid tumors and another is assessing it in either ovarian cancer or cancer of the peritoneum (the lining cells of the abdomen). Both studies are being carried out through NCI-sponsored cooperative clinical trial groups. (These groups are networks of research centers around the country that conduct joint studies, using identical protocols and pooling their data.)

6. Will NCI launch more studies of Herceptin® in other kinds of cancer?

Yes. Once there is an adequate drug supply, NCI and Genentech hope to explore the use of Herceptin® in a variety of malignancies, including gastric, endometrial, salivary gland, non-small cell lung, pancreatic, prostate, and colorectal cancers. Not all patients with these types of cancer have high levels of HER2. But as many as 30 percent to 40 percent of patients with some of these tumor types may overexpress the protein and therefore be candidates for clinical trials with Herceptin®.

7. Is NCI supporting studies of other anti-HER2 antibodies?

Yes. NCI currently is involved in early trials of other monoclonal antibodies directed against the HER2 protein. For example, several phase I studies sponsored by NCI are testing a HER2 antibody, designated 520C9xH22, produced by the Medarex Corporation in Annandale, N.J. Other early, NCI-sponsored studies are evaluating a different HER2 antibody, 2B1, from Chiron Corporation in Emeryville, Calif.

Studies with other monoclonal antibodies targeted against different proteins on cancer cells are also under way. For more information, call the Cancer Information Service at 1-800-4-CANCER (1-800-422-6237). You can also search the NCI's clinical trial database, called PDQ, on the Internet at

For more information about cancer visit NCI's Website for patients, public and the mass media at or NCI's main website at