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Packaging therapeutic RNAs for targeted treatment of breast cancer
Date:4/18/2012

BOSTON, Mass., April 18, 2012 /PRNewswire-USNewswire/ -- Researchers in the Program in Cellular and Molecular Medicine at Boston Children's Hospital and the Immune Disease Institute (PCMM/IDI) have developed a molecular delivery platform that overcomes one of the biggest obstacles to using RNA-based gene silencing technologies to treat cancer: Making sure the treatment gets to the right place and stays there.

Judy Lieberman, PhD, of the PCMM/IDI, and a former fellow from her laboratory, Erwei Song, MD, PhD, now at Sun Yat-Sen Memorial Hospital in Guangzhou, China, reported today in Science Translational Medicine that their platform—a combination of an antibody fragment and a packaging peptide—could successfully stabilize and deliver RNA-based molecules to tumors, silence them and prevent tumor growth and metastasis in a mouse model of breast cancer.

Breast cancer cells that display a protein called HER2 on their surface, which account for about 20 percent of all breast cancers, are highly aggressive. With the advent of targeted drugs like trastuzumab (Herceptin®) and lapatinib (Tykerb®), they have also become highly treatable. However, breast tumors that resist trastuzumab, relapse or do not have the HER2 protein can be quite difficult to treat.

To provide other targeted treatment options for breast cancers, Lieberman and Song have turned to a molecular phenomenon called RNA interference (RNAi), which relies on small pieces of RNA called small interfering RNAs (siRNAs) to silence the expression of individual genes. Originally observed in plants, RNAi was found to be active in mammals only a decade ago, but it is already the focus of many clinical investigations.

A stumbling block in the clinical use of RNAi, however,
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SOURCE Boston Children's Hospital
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