Going Down Easy

HLA researchers make a pediatric cancer drug more effective—and tasty.

by Alicia Di Rado

While a spoon full of sugar is one way to help the medicine go down, researchers have found a way to make a common chemotherapy drug a little more palatable for kids—by turning it into a powder that tastes like raw cookie dough.

The formulation of fenretinide is the first one ever specifically designed to make it easier for pediatric cancer patients to take their medicine, a feat that garnered a prize at a national conference for the research team at the USC-CHLA Institute for Pediatric Clinical Research.

The research team at Childrens Hospital Los Angeles included Barry J. Maurer, M.D., Ph.D., assistant professor of pediatrics and cell and neurobiology at the Keck School of Medicine of USC, and C. Patrick Reynolds, M.D., Ph.D., professor of pediatrics and pathology at the Keck School, director of the Developmental Therapeutics Program at the Institute for Pediatric Clinical Research, and co-leader of the Developmental Therapeutics Program at USC/Norris.

David Yesair, Ph.D., of BioMolecular Products Inc., and Walter Shaw, Ph.D., of Avanti Polar Lipids Inc., were industry partners on the research team.

Maurer explains that improving drug formulations for children is important not only because adult forms may be unwieldy for kids, but also because there may be more effective ways to get the drugs into the system.

“A past problem with fenretinide has been that its large capsules are difficult for children to swallow and hard for the body to absorb,” Maurer says. In the new formulation, the fenretinide is encased in a sort of glove of fats—going by the trademark of Lym-X-Sorb—that helps carry the drug into the bloodstream.

The new fenretinide formulation can be “mixed with food or drinks to make it easy for children to take,” Maurer says. More importantly, the new fenretinide powder appears to be much better absorbed than the capsules, and the researchers hope that getting more drug into the body will increase its anti-tumor effect.

The new fenretinide formulation was awarded the 2004 Eurand Award Grand Prize for Novel Approaches in Oral Drug Delivery at the 31st Annual Meeting and Exposition of the Controlled Release Society in Honolulu in 2004. The Controlled Release Society is the world’s largest professional society dedicated to drug formulations.

The fenretinide formulation work is supported, in part, by the National Cancer Institute (NCI) Rapid Access to Intervention Development Program, which supports drug development by investigators in academic institutions. NCI investigators Rao Vishnuvajjala and Shanker Gupta also supported the project.

The USC-CHLA Institute for Pediatric Clinical Research was established in 2004 with an anonymous gift of $15 million to develop new strategies for the prevention and cure of serious childhood diseases.