Trial model could fast track cancer drugs


Taking a stroll with Laurel May, you'd probably never guess that she's been locked in a fight for her life. A little more than a year ago, she was diagnosed with advanced breast cancer, which had also spread to her lungs. As part of her treatment, she began receiving a drug called Perjeta.

"I feel amazing. No one knows I'm sick or that any thing's wrong with me unless I tell them," says May.

Perjeta was developed by the Bay Area's Genentech and first approved last year for a specific form of advanced breast cancer. Chief Medical Officer Hal Barron, M.D., says it works by helping to block a protein believed to drive the cancer.

"Perjeta is a very, very exciting drug," says Barron. "It can actually stop the signaling of this very, very bad protein from causing problems with women who overexpress this protein."

In fact, it was successful enough that Genentech hoped to fast track its approval for early stage breast cancer as well as advanced -- a process that normally can take years. But what happened next could signal a major shift in the way cancer drugs are tested and just as importantly, how quickly they reach patients.

The company employed an emerging clinical trial method known as neoadjuvant. It calls for starting breast cancer patients on a drug therapy before surgery rather than after. That allows researchers to gauge the drug's effectiveness at shrinking the tumor. The model has been championed by UCSF breast cancer researcher Laura Esserman, M.D.

"Those of us who are trying to push the envelope and shorten the time it takes to get effective drugs to patients are cheering this," says Esserman. "The whole idea is as soon as you know something is really effective, we want to get it to patients as quickly as possible."

The neoadjuvant model produced compelling results in the Genentech trial.

"By adding Perjeta on top of those regimens, we were able to significantly increase the number of women who at the time of surgery had no evidence of tumor in their breast," says Barron.

This fall, the FDA granted accelerated approval for Perjeta for early HER-2 positive breast cancer patients. Supporters of neoadjuvant trials hope that new cancer drugs and new combinations of existing drugs could quickly follow.

"This is a new paradigm," says Barron. "This is a new way of developing drugs that could allow not only Genentech, but any company to be able to take a promising new therapy and test it in early settings, way in advance of what normally occurred."

"This is a huge first step," adds Esserman. "Shortening the timeline maybe three to five years, just by showing OK, we can shrink the tumor."

Cancer patients like May are grateful for this new approach.

"I think I can speak for anyone with cancer. Any breakthrough for anyone is a breakthrough for all us and I just can't wait to see where the technology is going to lead us from here," she says.

Perjeta is used in combination with another Genentech drug called Herceptin. The company is now conducting a follow-up study to examine the long term benefits for patients.

Written and produced by Tim Didion

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