ROCKVILLE, Md--Because advanced pancreatic cancer is a devastating
disease with no effective treatment, members of the FDA Oncologic
Drugs Advisory Committee (ODAC) voted to recommend approval of
the nucleoside analog Gemzar (gemcitabine hydrochloride, Eli Lilly)
as a first-line treatment for patients with nonresectable stage
II, stage III, or metastatic (stage IV) adenocarcinoma of the
The agent is currently available under a treatment investigational
new drug program for selected patients with advanced or metastatic
pancreatic cancer, which will continue while the company awaits
the FDA's final decision on approval.
In the United States, 27,000 people die each year of pancreatic
cancer, usually less than a year after diagnosis. "Previous
clinical trials have shown that fluorouracil, the most commonly
used treatment for pancreatic cancer, in combination with other
chemotherapeutic agents is no better than fluorouracil alone,"
said Daniel Von Hoff, MD, Cancer Therapy and Research Center,
San Antonio. Therefore, fluorouracil was the drug against which
Gemzar was tested.
Lilly presented two clinical trials: a phase III randomized, blinded
(but not double blind) study comparing Gemzar with fluorouracil,
and a phase II single-arm study of patients whose disease had
progressed while they were receiving fluorouracil.
New Surrogate Endpoint
Dr. Von Hoff noted that it is difficult to evaluate advanced pancreatic
cancer patients following surgery, radiation, and chemotherapy
using traditional endpoints such as the rate of tumor growth or
shrinkage, since these often do not provide a meaningful analysis
of a patient's overall health.
"Because clinical endpoints are so hard to measure in this
disease, we wanted to set up a trial to determine the effect of
Gemzar on what bothers patients most," he said.