Synodos for NF1 Research Published in Leading Journal
Children’s Tumor Foundation research led by its Synodos for NF1 team has demonstrated the ability to replicate NF1 manifestations in pigs, as reported in JCI (Journal of Clinical Investigation), a leading peer-reviewed publication of the American Society for Clinical Investigation. These manifestations include café-au-lait spots, neurofibromas, axillary freckling, and learning and memory neurological deficiencies. The significance of this advancement is that it allows researchers and clinicians, for the first time, to test and study potential NF treatments in large animal models that mimic human manifestations of neurofibromatosis.
Traditional NF1 research models utilize mice, which can replicate certain aspects of NF1, but not in a comprehensive manner, and hence are not as easily replicable for human application. This new, innovative approach conducts NF studies in pigs, which allows for more relevant and accurate analysis, and aids in better understanding of the disease, thereby increasing the potential for effective treatments.
This research was initiated out of the Children’s Tumor Foundation’s Synodos initiative, a collaborative team science and open data model that brings together a multidisciplinary ‘dream team’ of leading experts to solve the complex problems of NF. This team included researchers from Sanford Research, the University of Iowa, and the University of Arizona.
Why Pigs? And Next Steps
There are many similarities between pigs and humans in terms of metabolism, biochemistry, physiology, weight, lifespan and genetics. As demonstrated in other disease areas, including cystic fibrosis, pig models offer a unique ability to monitor disease progression and have shown better predictability in human drug response and behavior. The pigs in this study are born with NF1, and exhibit human-like NF characteristics, such as café-au-lait spots. By 10 months of age, the pigs start showing neurological impairments similar to that in humans with NF. These new models are now an invaluable tool in furthering and accelerating NF1 research and therapies, and will open the door for the very first time to the testing of drugs that improve the neurological impairments of NF1.
Lead funding for the Synodos for NF1 project was provided by James and Laurée Moffett.
To read the paper in JCI Insight, click here.
To read an interview with Jill Weimer, PhD, of Sanford Research and one of the lead researchers on this project (“Pig Models and NF1: The New Research Frontier”), click here.