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Pediatrics Cancer Prevention Drug

Texas Tech Cancer Center investigators develop new formulation of anti-cancer drug for children

Investigators at the TTUHSC School of Medicine Cancer Center have developed a new oral formulation of the anti-cancer drug, fenretinide, designed to make it easier for pediatric cancer patients to take their medicine.

Neuroblastoma is a form of cancer that starts in certain types of very primitive developing nerve cells found in an embryo or fetus, according to the American Cancer Society. This type of cancer occurs in infants and young children, and is rarely found in children older than 10 years.

Working with the New Approaches to Neuroblastoma Therapy Consortium (NANT), the investigators carried out a phase I clinical trial that documented the ability of the new drug formulation to achieve higher drug levels in children than the old formulation.

Children who participated in the clinical trial all had relapsed neuroblastoma, and in four of the children, no tumor was detectable after therapy with the new drug.

“A past problem with fenretinide has been that its large capsules are difficult for children to swallow and hard for the body to absorb,” said Barry J. Maurer, M.D., Ph.D., leader of the NANT clinical trial of the new fenretinide formulation in children with cancer. “The new fenretinide formulation is a powder that tastes like raw cookie dough and can be mixed with food or drinks to make it easy for children to take.”

The research team also includes Min Kang, Pharm.D., and C. Patrick Reynolds, M.D., Ph.D., of TTUHSC SOM as well as Judith Villablanca, M.D., and Araz Marachelian, M.D., of Children’s Hospital Los Angeles.

The fenretinide formulation research is supported in part by the National Cancer Institute (NCI) Rapid Access to Intervention Discovery program, as well as by NCI investigators Rao Vishnuvajjala, M.D., and Shanker Gupta, M.D.