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Gene therapy transforms cells into tumor killers

Experimental treatment saves two men dying of end-stage melanoma

updated 5:10 p.m. ET Aug. 31, 2006

WASHINGTON - Mark Origer entered the last-ditch experiment hoping to beat back his melanoma for a few months, long enough to walk his daughter down the aisle. He got far luckier: Almost two years later, his body shows no signs of the aggressive skin cancer.

Government scientists rescued Origer and one other man with advanced melanoma by genetically altering their own white blood cells to turn them into tumor fighters.

The treatment didn’t help 15 other melanoma victims. So scientists are trying to strengthen it to work better.

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Still, the National Cancer Institute called its experiment, unveiled Thursday, the first real success in the long quest for gene therapy for cancer — because it fought the disease’s worst stage, when it had spread through the body, not just single tumors.

And it did so in a way far different than today’s standard options, by harnessing patients’ immune systems to continually search out and kill tumors.

“It’s not like chemotherapy or radiation, where as soon as you’re done, you’re done,” said Dr. Steven Rosenberg, the NCI’s surgery chief who led the research published Thursday by the journal Science. “We’re giving living cells which continue to grow and function in the body.”

Doctors can’t predict how the therapy’s first two successful patients will fare long term. Melanoma, which kills almost 8,000 Americans annually, is notorious for returning years after patients think they’ve subdued it.

“I’m cured for now,” is how a grateful Origer, 53, of Watertown, Wis., puts it.

He recalls his doctors’ wide grins when, just a month after his December 2004 treatment, his tumors started to shrink. By his daughter’s wedding last fall, just one small cancerous spot remained, on his liver. Surgeons later cut it out. A checkup from NCI doctors this week confirmed that Origer is still cancer-free.

“I know how fortunate I am to have gone through this and responded to this. Not everybody’s that lucky,” he said.

Cancer specialists praised the work, but warned that years of additional research are needed.

“Clearly this is a first step,” cautioned Dr. Len Lichtenfeld of the American Cancer Society. “We have to be very cautious about not raising hopes too much.”

But, “it is exciting,” he added. “It certainly is a proof of concept that this approach will work.”

More importantly, the gene therapy can be customized to create cells that should attack more common cancers, said Dr. Patrick Hwu, melanoma chairman at the University of Texas M.D. Anderson Cancer Center, who once worked with the NCI team.

In a few months, NCI hopes to begin studying the approach in small numbers of patients with advanced breast, colon and other cancers.

White blood cells called T-lymphocytes hunt down germs and other foreign tissue. But cancerous cells look a lot like healthy cells, making it hard for those T-cells to spot a problem.


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