Women with highly aggressive, early-stage breast cancers will now be able to try experimental drugs before they have been tested for safety and efficacy in the sickest cancer patients. The new guideline is part of a new push by the Food and Drug Administration (FDA) to speed lifesaving drugs to patients faster.
Previous practice was to test cancer drugs on the sickest patients with end-stage disease. If the drug appeared to extend life and slow the progression of the disease, it was then approved for use in patients in earlier stages of breast cancer. But it could take nearly a decade for a drug to move through clinical trials before it is approved for other patients.
While the new process will deliver potentially lifesaving drugs to women earlier, they would also serve as guinea pigs of sorts, proving whether the drug is successful at fighting their cancer and revealing any safety issues or side effects that come from its use. To help mitigate these risks, the new guideline will apply to women with an especially deadly type of breast cancer known as triple-negative.
The most common type of breast cancer is estrogen-receptor positive, meaning it is fueled by the hormone estrogen. About 20 percent of breast cancers are known as HER2-positive, which involve a type of protein. A third type involves the hormone progesterone. There are effective treatments for all these caners, but not for triple-negative breast cancer. These cancers tend to grow and spread faster than others, and are more common in younger women and African American women.
The women will be put into two groups, with one group given chemotherapy and the other given chemotherapy plus an experimental drug. The medications will be administered a couple months before surgery to remove the tumors.
Doctors will be looking for a complete pathological response at the time of surgery, and these response rates will be compared in each group. If the experimental drug proves beneficial, it would receive a provisionary accelerated approval.
Women would also be tracked for several years to see how long they remain cancer-free. Good results could allow a drug to gain full approval by the FDA.