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Cancer gene therapy news backgrounder: New ideas fuel next generation gene therapy research

are now."

Not only did the science need to advance, but there was "tremendous regulation" through reviews at the federal level and at M. D. Anderson.

Roth is already busy perfecting the liposome therapy by testing the delivery of other genes as well as modifying the liposome coating. "My goal is to move the use of this therapy away from patients who have no other options because these cancers are extremely difficult to treat and the response rates are always low," he says. "The best uses of gene therapy will probably be in earlier stages of cancer or as part of primary treatment when cancer is first diagnosed."

A virus for the brain

The old saw goes that if curing cancer in mice was the same as curing it in humans, we would have won the war on cancer a long time ago.

While the point is taken, researchers at M. D. Anderson who last year cured brain cancer in mice were amazed because no one had ever before tested a drug that had any effect on malignant glioma, the most deadly of brain cancers.

Testing a gene therapy in mice, likened to a "viral smart bomb," the M. D. Anderson scientists found only empty cavities and scar tissue where human glioma tumors had once been. The therapy, known as Delta-24-RGD, had moved like waves throughout the brains of the mice, killing the cancer while leaving normal tissue intact.

While the treatment employs an adenovirus, it does not seem to produce toxic effects in the brain, say researchers. In fact, the mice tested were considered clinically cured of their brain tumors with little known side effects.

These animal tests, reported last year, were considered so promising that the National Cancer Institute moved immediately to produce, in its own labs, a clinical-grade version of the therapy, and scientists with the FDA began collaborating.

"We've never seen this kind of response before with any other treatment tested in either animals or humans," says the
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Contact: Nancy Jensen
nwjensen@mdanderson.org
713-792-0655
University of Texas M. D. Anderson Cancer Center
5-May-2004


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