UF researcher aims to improve pediatric liver cancer treatment

A University of Florida researcher has received three grants to study gene therapy techniques that target hepatoblastoma, or HB, the most common pediatric liver cancer in the U.S.

The disease is typically diagnosed in children under age 2, and while treatments have improved in recent years, nearly one-third of patients still die from the illness within 10 years of diagnosis. The most common treatment for the disease is a liver transplant, but donor shortages and post-transplant rejection can limit the widespread use of this procedure. As a result, new and more effective therapies are needed.

Chen Ling, Ph.D., a research assistant professor of pediatrics in the University of Florida College of Medicine, aims to develop novel gene-delivery vehicles called recombinant adeno-associated virus vectors, or rAAV, for use in gene therapy to selectively and efficiently target and destroy HB cells while minimizing the risk of additional damage.

Using specific DNA elements, Ling’s research will explore various gene therapy techniques to maximize the targeting of malignant cells without affecting normal ones. Where as this type of therapy has proved successful in inherited genetic diseases such as hemophilia, using it to treat cancer cells is a unique idea.

“I believe that he has developed a unique AAV vector system, with which the potential of gene therapy to treat human liver cancer is likely to become feasible,” said Arun Srivastava, Ph.D., chief of the division of cellular and molecular therapy in the department of pediatrics.

For these efforts, the Bankhead-Coley Cancer Research Program’s New Investigator Research Grant awarded Ling a three-year, $374,000 grant. Ling was one of just 15 recipients selected from among 78 entrants.

In addition, he received $100,000 from the Alex’s Lemonade Stand Foundation’s Young Investigator Award. Founded in 2005, the award is designed to fill the critical need for start-up funds for new researchers and physicians to pursue promising research ideas, primarily into new treatments and cures.

Lastly, Ling received $7,500 from the University of Florida’s Clinical and Translational Science Institute Pilot Trainee Project, a one-year grant. The program provides intramural grants to support the growth of interdisciplinary and investigator-initiated research across the university’s broad range of scientific disciplines.

All of the grants began in July.

“We are honored to have Chen Ling as a member of our department,” said Scott Rivkees, M.D., chair of the UF College of Medicine department of pediatrics. “Early in a very promising career, Ling is already making great strides in the development of innovative therapies for cancer.”