Nfm-14. Identification of a Therapeutic Time Window That Improves Vision in an Nf1-deficient Optic Pathway Glioma Mouse Model
Year: 2018
Type: conference-abstract
Abstract: Optic pathway gliomas (OPGs) are glial tumors that arise early during development in 15-20% of patients with Neurofibromatosis Type I (NF1). While chemotherapy is often used for patients who have growing tumors and/or vision loss associated with the tumor, a minority of patients have improvement in their vision following treatment. Thus, a new approach to treatment is needed to improve the visual outcomes in this patient population. In our laboratory, we have a Nf1-deficient conditional knockout mouse model that develops optic pathway gliomas around 60 days of age (P60). These mice have associated axon degeneration, loss of retinal ganglion cells, and evidence of vision loss on behavioral testing, thus making this a good model to use in order to identify the optimal therapeutic time window that improves or prevents vision loss. In this study, we treated NF1-deficient mice with a Mek-inhibitor during different developmental time windows and measured response to treatment in terms of nerve cellularity, axon integrity, and retinal ganglion cell loss. We found that response to treatment is highly dependent on when the therapy is initiated during OPG development, and the best response occurred when therapy was initiated early during this process. This study suggests that the timing of therapy initiation is critical for optimal outcomes in NF1-associated OPGs.
Source: Neuro-Oncology
Institutions: Children's National, University of Maryland, Baltimore
Language: English
Subfield: Neurology
Field: Medicine
Domain: Health Sciences
Open Access status: green
OpenAlex ID: W2809039398