Professor Borden Awarded St. Baldrick’s Grant

Darrell Yamashiro, MD/PhD (PI), and Mark Borden, PhD, were recently awarded a research grant from St. Baldrick's Foundation to investigate a novel gene therapy strategy to treat children's cancers.  The funding will allow the multi-disciplinary team to continue developing microbubbles as gene carriers for targeted delivery to solid pediatric tumors.

Gene therapy is an approach to treat cancer by re-introducing a missing gene or introducing a new gene that restores natural function or induces cell death.  While gene therapy has been gaining momentum over the past decade, a significant challenge has been to efficiently and selectively deliver DNA molecules to cancer cells without inducing an allergic response.  Dr. Borden's lab has developed a multi-tiered approach to address the challenges of immunogenicity, tissue selectivity, cellular delivery and intracellular trafficking. "We are designing and engineering advanced microbubble constructs to carry and deliver genes to the tumors," he said.  "A microbubble is a tiny gas sphere, about the same size as a red blood cell, which contains a surfactant shell that can be loaded with DNA.  DNA-loaded microbubbles circulate in the blood stream and enter the tumor vessels, where focused ultrasound is then applied to cavitate the microbubbles and deliver their genetic cargo."

Dr. Yamashiro is the co-Director (with Dr. Jessica Kandel) of the Pediatric Tumor Biology Laboratory at the Columbia University Medical Center; he has extensive research experience in the modeling and treatment of pediatric tumors, such as Wilm's tumor and neuroblastoma.  These solid tumors are often difficult to remove surgically, but they may be accessible to ultrasound waves for treatment.  Dr. Yamashiro will lead the effort to evaluate gene delivery at the cellular level in the tumors, and to develop clinically translatable therapeutic strategies.

The proposed research will not only result in advanced gene delivery technology, but also will better characterize the underlying mechanisms of ultrasound-microbubble gene delivery, and thus would move this concept and technology rapidly toward clinical practice in the treatment of children with cancer.

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