Dr. Matthias Karajannis, New York University’s, Lagone Medical Center just announced that enrollment for the Phase II Study of Sorafenib in Children (and young adults) with recurrent or progressive Low-Grade Astrocytoma (including optic pathway gliomas) is officially OPEN! Three other institutions, Children’s Hospital of Philadelphia, Children’s Hospital of Los Angeles and Johns Hopkins University are all set to open for enrollment shortly.

Sorafenib is a small-molecule multi-kinase inhibitor with activity against CRAF, BRAF (including the V600 activating mutation), VEGFR-2/3, etc. Therefore, sorafenib simultaneously targets several pathways identified in a high proportion of LGAs. In addition, to BRAF inhibition, sorafenib’s anti VEGFR-2/3, PDGFR and FGFR-1 activity may interfere with tumor growth via anti-angiogenic effects. Successful treatment of brain metastases has been reported, indicating that sorafenib crosses the blood-brain barrier and achieves active drug concentrations in the CNS.

Based on favorable phase III results, sorafenib was granted FDA approval for treatment of renal cell carcinoma in 2005 and for hepatocellular carcinoma in 2007. In addition, sorafenib is the only RAF-inhibior already undergoing early-phase clinical testing in pediatric patients.

This study will look at patients between the ages of 2 – 25 that have a recurrent/progressive optic pathway gliomas or either clinical or MRI criteria, after standard therapy or histologically confirmed recurrent or progressive low-grade glioma by either clinical or MRI criteria after standard therapy. Patients with neurofibromatosis-1 (NF1) are eligible.

Funding for this clinical trial has been provided in partnership between the PLGA Foundation and the Making Headway Foundation’s Talbot Family Research Fund.