FDA Panel Backs Wider Use of Drug to Treat Early Stage Breast Cancer
THURSDAY Sept. 12, 2013 -- U.S. advisers endorsed on Thursday the broader use of a drug already used to treat advanced breast cancer that also appears to shrink early stage breast tumors.
This could potentially offer women with early stage breast cancer a first-of-its-kind treatment option and the hope of less-invasive surgical procedures.
If approved to treat early stage tumors, the drug, Perjeta, might result in less invasive surgical treatment for women with HER2-positive early stage breast cancer. In that role, Perjeta (pertuzumab) would be the first cancer-fighting drug approved as a first-step breast cancer therapy in the United States.
A U.S. Food and Drug Administration advisory panel voted 13 to 0 to recommend approval of the drug. The FDA is not bound to follow the recommendations of its advisory panels but it usually does.
An FDA report that was posted online Tuesday said the drug looked promising in trials. The report also said, however, that drug-related cardiac concerns will require additional research.
Advisory panel chairman Dr. Mikkael Sekeres, a professor of medicine at the Cleveland Clinic, called Thursday's vote "a historic moment," The Associated Press reported.
"We are supporting the movement of a highly active drug for metastatic breast cancer to the first-line setting, with the hope that women with earlier stages of breast cancer will live longer and better," Sekeres said.
The FDA is considering "fast-track" -- or accelerated -- approval of Perjeta, which is made by Roche's Genentech division, the AP reported. Fast-track approvals usually are given to drugs that haven't been thoroughly tested but show promising results in early trials.
Panel members insisted Thursday that Genentech carry out more trials to ensure that Perjeta will enable breast cancer patients to live longer, healthier lives, the news service said.
Genentech estimated that about 15,000 women with HER2-positive early stage breast cancer could receive early treatment with Perjeta each year, Bloomberg News reported.
Cancer specialists welcomed the news earlier this week of the preliminary results for Perjeta.
"This is tremendously exciting," said Dr. Amy Tiersten, associate professor in the division of hematology and medical oncology at Mount Sinai Medical Center in New York City.
"Pertuzumab was FDA approved in 2012 for the treatment of metastatic breast cancer when it was shown that it improved survival when added to standard regimens for HER2-positive metastatic breast cancer," Tiersten said. Metastatic breast cancer is cancer that has spread to other parts of the body.
"But the really exciting information is when this can be translated into earlier stages of disease, where we can completely eradicate the disease and cure more women," she said.
The preliminary trial under discussion showed that when Perjeta was added to standard pre-surgical regimens for HER2-positive breast cancer, the chance of finding no cancer after the treatment was nearly doubled, Tiersten said.
Women who have chemotherapy before surgery and who achieve complete remission -- no cancer at the time of breast surgery -- have a much greater chance of being completely cured of their disease, she said.
Dr. Aye Moe Thu Ma, attending physician in breast surgical oncology with St. Luke's and Roosevelt Hospitals in New York City, also voiced enthusiasm.
"We currently have limited options for [first-step] treatment of breast cancer," Ma said. "I'm excited that this may provide a more specific treatment for people with HER2-positive cancer."
This means women may be able to keep their breasts because the tumor size is reduced before surgery, and some women may prefer this, she said.
Genentech is hoping for fast-track approval of Perjeta. The FDA can expedite approval for groundbreaking drugs as long as the drug maker pursues more research to show that the medication prolongs disease-free survival.
Both short- and long-term side effects will need to be examined to fully evaluate risks and benefits of this medication if it is approved, Ma added.
Perjeta is one of many newer biologic agents used to treat breast cancer. "These biologic or 'targeted' treatments work on breast cancer cells in a more specific way than some older treatments so there are many fewer casualties to normal cells translating into many fewer side effects for our patients," Tiersten said.
About one in eight women in the United States will develop breast cancer in her lifetime.
According to the Mayo Clinic, HER2-positive breast cancer is a breast cancer driven by a protein called human epidermal growth factor receptor 2 (HER2), which promotes the growth of cancer cells.
HER2-positive breast cancers typically are more aggressive than other types of breast cancer. But there are effective drug treatments, including trastuzumab (brand name Herceptin) and lapatinib (Tykerb). Both drugs can produce side effects, including congestive heart failure, according to the Mayo Clinic.
The U.S. Department of Health and Human Services has more about early stage breast cancer.
Posted: September 2013