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The gene-based therapy hits only malignant cells, researchers say
by: Robert Preidt

An experimental gene-based drug is able to "trick" its way into prostate cancer cells and kill them, U.S. researchers report.

The drug, which is the first of its kind, uses a type of genetic material called "targeting RNA" to enter cancer cells and another type, "silencing RNA," to halt the expression of a protein called PLK1, which keeps prostate cancer cells alive.

Researchers at Duke University Medical Center in Durham, N.C., tested the drug in mice with prostate cancer and found that it shrank the size of their tumors by half and caused no side effects. Tumors in control mice that did not receive the drug continued to grow.

The findings were published in the August issue of the journal Nature Biotechnology.

"This study represents the first step in creating an RNA-based drug for cancer. It provides a 'proof of principle' that an entirely RNA-based drug can work with minimal side effects, and it shows it is possible to overcome many of the obstacles that have hampered the development of RNA-based drugs," study lead author James McNamara, a postdoctoral fellow in experimental surgery, said in a prepared statement.

Previous experimental RNA-based drugs were nonspecific. They targeted all cells in the body, not just cancer cells, resulting in unwanted side effects.

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