Skip to main content

NF Bites: Progress in NF1 Optic Pathway Gliomas

By April 20, 2010December 18th, 2023Awareness, Science & Research

Welcome to the eighth and final in a series of “NF Bites” – providing snapshots of individual areas of neurofibromatosis research and how the Children’s Tumor Foundation is advancing this. Today: where are we with optic pathway glioma research progress?


Optic pathway gliomas (OPGs) can occur in a small percentage of children with NF1, but these tumors can require surgical removal often leading to sight loss.   Additional impact of these tumors in the brain can extend to impacting hormone fluctuations and precocious puberty. Most commonly OPGs have been managed through surgery. Treatment with chemotherapy and radiation are also used though these have been somewhat controversial. Like the majority of NF1 tumors, OPGs are benign and the concern is that many chemotherapy approaches might be too harsh and that radiation might trigger advancement to malignancy.

The good news is that researchers now understand quite a bit about OPG biology and this is helping to inform the selection and testing of candidate drug regimes in mice that might be advanced to humans.

·         Through the CTF NF Preclinical Consortium, a $4M multi-year initiative to accelerate the most promising candidate drugs to the clinic, the laboratory of David Gutmann (Harvard/ Brigham & Women’s Hospital) is testing candidate drugs in genetically-engineered mouse models of OPG, including through a collaboration with Genentech.

·         Joshua Rubin (Washington University School of Medicine) has received two Drug Discovery Initiative Awards – one, our only Advanced DDI Award (a special $50,000 Award for most promising DDI projects) to date, highlighting the importance of this research.  Based on understanding the biology of cells during OPG growth, Dr Rubin has used a mouse model to evaluate the use of the anti—inflammatory drug Rolipram as a co-therapy for treatment of optic pathway tumors. The goal of this research is to be able to reduce the amount of temozolomide that needs to be used to treat these tumors, and to take this forward to a clinical trial.

·         Young Investigator Awardee Sutapa Banerjee (Washington University School of Medicine) is examining the cell signaling element mTOR in regulating growth of optic pathway glioma tumors and in 2009 published early findings in the International Journal of Cancer.  Dr. Banerjee also published a paper in early 2010 in Cancer Research highlighting a new candidate drug target, called STAT3, for treating OPG.

·         Outside of CTF funding, an optic pathway clinical trial of Everolimus will commence in 2010 through the CDMRP NFRP Phase II Clinical Trials Consortium.


Thank you for your enthusiastic responses to the NF Bites series. Look for an updated series in the future!