Phase I/II Study of MEK162 for Children with RAS/RAF Pathway Activated Tumors

Principal Investigator: Dr. Nathan Robison, Children’s Hospital of Los Angeles
Funding Partners: WhyNotMe? Foundation, Team Jack Foundation

With the discovery of new targets, the pipeline for PLGA clinical trials is ramping up to be more productive than ever before. The newest PLGA clinical trial, entitled Phase 1/II Study of MEK162 for Children with Ras/Raf Pathway Activated Tumors, opened for recruitment in June 2016. The main purpose of the phase I study is to determine the most effective dosage of the drug for children and adolescents with tumors that have recurred despite standard chemotherapy treatment, and to identify the most common side effects. Spearheaded by Dr. Nathan Robison at the Children’s Hospital of Los Angeles, the trial has already enrolled the first 2 cohorts of patients to evaluate optimal drug dosage and will continue to move forward. The phase II objectives will determine whether MEK162 causes NF1 patents and other tumors thought to be caused by abnormal activation of the Ras/Raf/MAP molecular pathway tumors in the pediatric population to shrink and/or stop growing. The study is projected to span 2 years for patient enrollment and will include 15 different institutions across the country.

Update: November 17, 2016

We’re excited to report that at the European Society for Medical Oncology (ESMO) 2016 Congress, held October 7–11 in Copenhagen, Denmark, Dr. Mark Kieran discussed the results of a phase I/II trial of dabrafenib therapy, which demonstrated higher response and lower toxicity in pediatric brain tumors. In this video clip, he discusses how the BRAF inhibitor has significant impact on gliomas in children, with none of the side effects seen in adult melanoma patients and introduces upcoming doublet trials alongside a MEK inhibitor. There are additional findings presented at ESMO 2016 about the effectiveness of targeted combination therapy for children with certain types of low-grade glioma brain tumors.

Funded in 2016.

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