Matthew Herper, 05.16.05, 6:00 AM ET
ORLANDO, FLA. - At least for the moment, Genentech, the original biotechnology pioneer, is undeniably on top of the cancer world. But as gigantic rivals, including Pfizer and GlaxoSmithKline, try to steal its tricks, can the South San Francisco-based biotech maintain its dominance?
The 29-year-old firm has dominated this year's biggest cancer treatment conference, the annual meeting of the American Society of Clinical Oncology (ASCO), with its drugs Herceptin, Avastin and Tarceva. All of the medicines were invented through close attention to the genetic defects in cells that lead to cancer, and tested in large clinical studies that experts praise for their rigor. Now, in quick succession, Avastin has been shown to extend life by several months in lung, breast and colon cancer, when added on top of chemotherapy. The battery of results emerged in little more than a month (see "Genentech's Wall Of Data").
"It was the most fun five weeks I might ever have in my career," says Susan Desmond-Hellmann, who heads Genentech's (nyse: DNA - news - people ) clinical trials. "It's what you live for."
Doctors are impressed, especially with Avastin, which is designed to starve tumors of blood by blocking a protein called VEGF.
"It's powerful," says Nicholas Vogelzang, head of the Nevada Cancer Institute. Adds Len Lichtenfeld, deputy medical director of the American Cancer Society: "I happen to think these results are spectacular." Says Robert Mayer, director of the center for gastrointestinal oncology at the Dana-Farber Cancer Institute: "I think it's hard to be anything but enthusiastic about the results."
At the end of two years, 22% of non-small-cell lung cancer patients who received Avastin with their chemotherapy were alive, compared to 17% who received only chemotherapy. Before the meeting, some worried that the lung cancer data for the drug would be marred by a side effect: In earlier trials, 9% of patients had bled to death from their lungs while on Avastin treatment. But most of the bleeding occurred in patients who had a particular type of tumor, called a squamous cell tumor. By excluding patients at high risk of bleeding, the number of bleeding deaths was cut to a little more than 1% of those taking Avastin.
That won't prevent doctors from using the drug, says Roman Perez-Soler of Montefiore Medical Center in New York. "For this type of population," he says, "we are used to death from chemotherapy."
Data for use of Avastin in breast cancer and colon cancer were also presented. As in lung cancer, the drug extended the survival of at least half of the patients by several months or more. Dana-Farber's Mayer says he's encouraged that the results are similar in different kinds of tumors.
But that isn't all Genentech had to offer cancer doctors at the ASCO conference. Its Herceptin also extended the life of women with certain types of breast cancer.
"The differences that we're seeing there are among the greatest that I have seen for years and years and years," says Norman Wolmark of Pittsburgh's Allegheny General Hospital, in response to the Herceptin results. And Tarceva, a pill Genentech co-markets with OSI Pharmaceuticals (nasdaq: OSIP - news - people ), was the first drug to extend the lives of pancreatic cancer patients when added to Eli Lilly's (nyse: LLY - news - people ) Gemzar. To be sure, doctors do disagree over whether those results, though statistically significant, actually represent a meaningful increase in survival.
At some point, Herceptin, Avastin and Tarceva all looked like duds. But Desmond-Hellmann and her colleagues used clinical trials to look for hints of efficacy even when the treatments failed. Herceptin was found to work for tumors with a certain gene. When doubts emerged about Avastin and Tarceva, other clinical trials that ultimately proved the medicines' worth were already ongoing.
Now, far larger companies seem to be trying to steal from Genentech's playbook.
Pfizer (nyse: PFE - news - people ), the world's largest drug firm, may suddenly be becoming Genentech's biggest competitor in the cancer arena. Desmond-Hellmann claims she isn't worried about competition from companies whose research budgets rival Genentech's total annual spending. "I don't think it's about money," she says. "I think it's a way of doing things."
Pills from Pfizer and Bayer (nyse: BAY - news - people ) that hit not only VEGF, the Avastin target, but also other proteins in the cell, are showing incredible promise. Results from a VEGF pill being developed by Novartis (nyse: NVS - news - people ), however, were disappointing. Rivals such as Amgen (nasdaq: AMGN - news - people ) and the team of Sanofi-Aventis (nyse: SNY - news - people ) and Regeneron Pharmaceuticals (nasdaq: REGN - news - people ) are years behind.
"I haven't seen evidence that someone will have a pill that will replace Avastin," Desmond-Hellmann says.
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