In Vivo Validation of High-Throughput Drug Screen Results for NF2
Principal Investigator:
FERNANDEZ-VALLE, CRISTINA
Institution Receiving Award:
CENTRAL FLORIDA, UNIVERSITY OF
Program:
NFRP
Proposal Number:
NF140044
Award Number:
W81XWH-15-1-0446
Funding Mechanism:
Investigator-Initiated Research Award - Optional Qualified Collaborator
Partnering Awards:
Award Amount:
$777,059.00
Period of Performance:
9/1/2015 - 8/31/2019
PUBLIC ABSTRACT
Neurofibromatosis type 2 (NF2) is a tumor disorder of the nervous system for which there is no drug therapy. Results of our drug screening campaigns revealed that two Food and Drug Administration-approved drugs reduced the viability of NF2 mutant mouse Schwann cells. Here we will profile the "best in class" SRC and MEK kinase inhibitors for efficacy in reducing viability of both mouse and our newly created human merlin-null Schwann cells. The best performing compounds will be evaluated in two animal models for NF2 schwannomas. Lastly, molecular studies will be conducted to compare treated and untreated mouse and human merlin-null Schwann cells to understand the pathways contributing to the drug responses. The outcome of this project will be to identify one or two compounds that could be advanced to clinical trials for NF2 and information that will guide future combination drug studies. |
