Chemotherapy drugs proven to work against the brain tumor ependymoma are lacking. Current treatment is built on surgery and radiation. So, one report from St. Jude Children’s Research Hospital was welcome news. Scientists found that a drug widely used against colon cancer may also be effective against ependymoma.
The drug is 5-fluorouracil. Working in laboratory models, researchers had shown that giving the drug rapidly in a single dose called a bolus, rather than infusing it slowly, magnified the drug’s benefits. But more information was needed about how children would react and to determine the effective dose to kill brain tumors.
Researchers used applied math, supercomputing and other tools to analyze lab data to find the best starting bolus dose for children. That dose was well tolerated and had an anti-cancer effect when used in a phase I study of children and young adults whose brain tumors had returned.
“The modeling and simulation used in this study streamlined identification of the optimal bolus dose of 5-fluorouracil for young ependymoma patients,” said Clinton Stewart, PharmD, of St. Jude Pharmaceutical Sciences. “The approach is a template for drug development, especially for rare diseases like ependymoma.”
The research was published in the journal CPT: Pharmacokinetics and Systems Pharmacology.