The Neurofibromatosis Network and its member organizations, NF Northeast, NF Michigan, Washington State NF, NF Central Plains, NF North Central and our colleagues from The NF Team are proud to be supporters of the University of Wisconsin-Madison NF1 Swine project. We are optimsitic that this research will speed up the development of NF1 therapies to the benefit of the NF1 community. The following article, written by Charles Konsitzke, Assistant Director University of Wisconsin-Madison Biotechnology Center, describes the progress of this exciting research.
NF1 Translational Research Initiative at University of Wisconsin-Madison:
Despite the availability of NF1 mouse research models, hurdles exist in translating the data from mouse studies to the clinical setting. The overwhelming genetic, anatomic, and physiological similarities of the swine to human, make the swine the ideal research model for accelerating discovery and development of new therapies for NF1.
The research team at UW-Madison understand that NF1 is a genetically complex disease and that each individual with NF1 is unique in the way that the disease affects them. Therefore, a single swine model of NF1 simply cannot represent all those with NF1. To address this hurdle, the team has created a number of NF1 swine models and are in midst of studies to understand various aspects of the disease. Concurrently, they have launched an effort to create child-specific NF1 swine models that will allow for drug development, screening and selection to occur, prior to the use of the therapy in a child.
To complement the research in these swine models, the research team is also developing a pig stem cell-based platform for rapidly discovering and testing new therapies. This platform will allow the team to create NF1 tumors carrying a large diversity of mutations of the gene, and then by performing comprehensive analysis, find common targets for drugs that may be effective across multiple number of mutations. This platform will allow for rapid and high-throughput discovery, development and testing of drugs prior to focused testing in the live pig models and subsequent use in the clinical setting.
It is the central mission of the NF1 Research Team at UW-Madison to make the swine models and research tools that they develop available to other NF researchers in the United States and the world. They are also aware that many of these research institutions do not have the necessary expertise, facilities and resources for conducting research in swine models. Thus, they are committed to make these aspects of UW-Madison available for collaborative research. The team fundamentally believes that sharing of research ideas, expertise, tools and resources is the most effective way to make rapid and significant advancements that benefit NF1 patients and their families.