An international consortium of researchers has demonstrated that a novel DNA-based therapy is safe and acts against certain advanced head and neck cancers. City of Hope’s Center for Biomedicine & Genetics (CBG) produced the therapeutic DNA for the clinical trial.

The study appeared in the March 10 issue of the Journal of Clinical Oncology.

The Center for Biomedicine & Genetics produces therapeutic DNA for innovative clinical studies. (Photo by Walter Urie)

Antisense DNA therapy employs a piece of DNA that mirrors a section of the genetic code in a target gene. This antisense DNA couples to the target DNA much like a jigsaw puzzle piece, tying it up and preventing the cancer cell from reading the code.

The clinical researchers injected the antisense DNA directly into tumors in 17 patients. None of the patients experienced severe toxicity, and although the study’s main goal was not to measure the therapy’s effectiveness, seven patients did show some response to the therapy, including two who went into complete remission.

The researchers tapped the CBG to make the antisense DNA because at the time the facility was a National Gene Vector Laboratory, one of only three in the country and the only one sanctioned by the National Institutes of Health, or NIH, to produce plasmids for gene therapy studies in the United States.

Plasmids are small circles of DNA that researchers use to carry genes, including therapeutic genes, into cells.

To be given to humans, any therapeutic product must be produced using “current good manufacturing practices.” The Food and Drug Administration, or FDA, sets these standards.

“We’re fully equipped to meet the FDA’s rigorous requirements,” said Larry A. Couture, Ph.D., senior vice president of the Sylvia R. and Isador A. Deutch Center for Applied Technology Development at City of Hope and director of the CBG. Couture was a co-author on the report.

Since the study began, the NIH dismantled its gene vector lab program in favor of other funding priorities.

Despite the NIH’s decision to cut support, several former NIH-backed gene vector labs saw a need to continue offering services to academic institutions exploring gene therapies as well as other experimental biological treatment methods.

These labs pooled resources to form the Association of Academic Biologics Manufacturers in 2007.

“The dissolution of the National Gene Vector Laboratories provided an opportunity for us to form a network of facilities that can offer a wider range of services,” said Couture.

The association comprises 14 nonprofit, academic facilities nationwide that use current good manufacturing practices to prepare products ranging from plasmid DNA for gene therapy to islet cells for transplantation in diabetes patients.

According to Couture, one of the main advantages of the association is the opportunity it provides for collaboration.

“Clinical researchers can come to us and find a partner for their research,” he said. Each of the member facilities also can tap the others for best practices, adding value for the researcher, Couture added.

More information about the Association of Academic Biologics Manufacturers is available at www.aabmonline.org.