CBSN

Gene Therapy Breakthrough

GENERIC DNA gene therapy syringe
CBS/iStockphoto
U.S. government scientists saved two men dying of melanoma by genetically altering the men's own white blood cells to attack their tumors — deemed the first major success in battling cancer with gene therapy.

While the men appear disease-free almost two years after the experimental therapy, it wasn't a panacea. Just two of the 17 treated victims are disease-free; 15 other melanoma victims were not cured. So the National Cancer Institute is trying to strengthen the shots.

Still, specialists proclaimed the work, published Thursday by the journal Science, as an important advance — gene therapy with the potential to fight cancer's worst stage, when it has spread through the body. And the cancer institute hopes to begin testing it soon against cancers more common than melanoma, such as advanced breast or colon cancer.

Doctors can't predict how the two men will fare long-term. Melanoma, the most aggressive skin cancer and killer of 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 Mark Origer, 53, of Wisconsin put it after a checkup from NCI doctors this week.

"I had what they call stage 4 of the disease," Origer told CBS News. "Prognosis was approximately about one year."

As a last resort, Origer enrolled in a clinical trial testing a new technique to help the body's own immune system kill the cancer," CBS News medical correspondent Dr. Jon LaPook reports.

Oringer, who thought his advanced melanoma would kill him, believed he'd never walk his daughter down the aisle at her wedding. But after the experimental treatment, hea was able to do so.

"I've been given my life back," Oringer said.

The approach remains highly experimental, requiring years of additional research.

"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."

"It's one of the first documented, effective cases of cancer gene therapy working," added Dr. Patrick Hwu, melanoma chairman at the University of Texas M.D. Anderson Cancer Center, who once worked with the NCI team.