Phase I/II study of MEK162 for children with progressive or recurrent low-grade gliomas and other central nervous system tumors

Award: $250,000 over 2 years (2020 – 2022)
Principal Investigators: Dr. Nathan Robison, Pediatric Neuro-Oncology Attending, Children’s Hospital Los Angeles, Dr. Mariella Gruber, Department of Pediatric Neuro-Oncology, Dana-Farber Cancer Institute, Dr. Karen Wright, Pediatric Neuro-Oncologist, Dana-Farber Cancer Institute
Funding Partners: Taylor Matthews Foundation, WhyNotMe? Foundation, Making Headway

Phase I of this trial was rolled out in 2016 with support from PLGA Fund and had a target validation component to determine the ability of MEK162 to penetrate into the tumor as well as assess the ability of MEK162 to affect its target. A short course pharmacokinetics and pharmacodynamic evaluation on these patients for correlation was performed to evaluate the following criteria: To quantify concentration of investigational compound in tumor tissue after treatment with MEK162 for 7-21 days and correlate with PK assessment in blood; to assess RAS-RAF-MEK-ERK pathway inhibition, as measured by ERK phosphorylation, in LGG after treatment with MEK162 for 7-21 days. Blood pharmacodynamic assessment will also be performed and correlated with tumor results. MEK162 demonstrated a positive response; with such promising initial results, researchers expanded the enrollment by 33% (from 75 patients to 100) and allowed current patients to remain on the trial for an additional year.