Cell Culture Model Initiative
A rapid, cost-effective way to predict the efficacy of potential drug candidates alone and in combination.
When NTAP convened scientists from around the world at its 2012 symposium, the lack of cellular model systems was identified as a major limiting factor in NF1 research. While animal models were remarkably predictive for NF1 research, they were also expensive and slow. The combination of high cost and long lead times meant that too few reagents were being studied.
To solve this problem, NTAP established the Cell Culture Model Initiative, which funded a series of projects to develop cell models and offer a rapid, cost-effective way to predict the efficacy of potential drug candidates alone and in combination.
As a result of this successful initiative, today NTAP is able to offer NF1 researchers all over the world access to cell model systems, often with full reimbursement. This early breakthrough is just one more example of how NTAP is leading the way in accelerating the developing of therapeutic answers for patients with NF1.
Cell model system studies conducted
Development of a Novel Plexiform Neurofibroma Farming System for 96-well Plate Drug Screening.
- Development of Plexiform Neurofibroma Cellular Assay for High-Throughput Screening.
- Perpetuating NF1+/- and NF1-/- plexiform neurofibroma-derived tumor cells through the generation of induced pluripotent stem (iPS) cells.
- A primary plexiform neurofibroma cell culture model for use in cell-based high-throughput screens.
- Plexiform Neurofibroma Model Systems for Preclinical Drug Screening.
- Transition to confirmatory and secondary screening of plexiform neurofibroma models in 96-well format.
- CIDr/NTAP Collaboration: (1) Genetic characterization studies (Cell Line Authentication, SNP Array, Exome Sequencing, and RNA Sequencing) of NF1-/-, NF1-/+, and NF1+/+ cell-lines to be available as tool-set for community, and (2) Analyses conducted by Sage Bionetworks.
- Screening (Single Agent MIPEs, combo): As a result of a collaboration between UFL, NTAP, and NCATS, a panel of cell culture systems that represented cNF complexity was used to screen new compounds (as single and combination agents) and identify novel therapeutic targets.
- Validation Studies to validate the results of preliminary HTS at NIH/NCATS by Dr. Ferrer.