A Sanford Research scientist is using an innovative pig model to better understand a rare genetic disorder that causes tumors in the nervous system, thanks to a more than $1.7 million grant from the Children’s Tumor Foundation (CTF). Jill Weimer, Ph.D., and her team received the award as part of CTF’s Synodos for NF 1 program, a unique collaboration of world-class scientists and clinicians from diverse expertise areas who work together with patients and share information in real time resulting in a faster and more efficient drug discovery and development process.
The Children’s Tumor Foundation is a not-for-profit organization dedicated to finding effective treatments for the millions of people worldwide living with neurofibromatosis (NF).
Weimer is a scientist and director of the Children’s Health Research Center at Sanford Research who studies nervous system development, neural development disorders and neurodegenerative diseases. She will lead a collaborative team of researchers from the University of Iowa, the University of Arizona and Exemplar Genetics, an Iowa-based biotechnology company that specializes in porcine models. This collaborative research crew includes experts in genetics, neuroscience, pathology medicinal chemistry, molecular biology and biomedical engineering. The group has already demonstrated success creating pig models for diseases like cystic fibrosis and ataxia telangiectasia.
“Scientists have experienced limited success replicating NF1 in mouse models; there are some characteristics of the disorder that have been difficult to model,” said Weimer. “It is becoming vital as we move toward developing effective therapies for NF1 that we develop reliable large animal models.”
NF1, which affects one in 3,000 worldwide, is caused by a mutation in the neurofibromatosis type 1 gene. It can cause tumors to grow on nerves throughout the body and may lead to blindness, bone abnormalities, cancer, deafness, disfigurement, learning challenges and disabling pain.
Earlier this year, Weimer received a nearly $440,000 grant to support her research of Batten disease, a rare pediatric neurodegenerative disease. She was also the recipient of a $1.75 million grant from the National Institutes of Health in 2014 to study intracellular trafficking in neurological disorders.
Weimer received her doctorate from the University of Rochester and completed a postdoctoral fellowship at the University of North Carolina. She joined Sanford Research in 2009.
About Children’s Tumor Foundation
The Children’s Tumor Foundation is a 501(c)(3) not-for-profit organization dedicated to finding effective treatments for the millions of people worldwide living with neurofibromatosis (NF), a term for three distinct disorders: NF1, NF2, and schwannomatosis. NF can cause tumors to grow on nerves throughout the body and may lead to blindness, bone abnormalities, cancer, deafness, disfigurement, learning disabilities, and disabling pain. NF affects one in every 3,000 people, more than cystic fibrosis, Duchenne muscular dystrophy, and Huntington’s disease combined. The Children’s Tumor Foundation funds critical research into neurofibromatosis. In addition to benefiting those who live with NF, this research is shedding new light on several forms of cancer, brain tumors, bone abnormalities, and learning disabilities, ultimately benefiting the broader community. For more information, please visit the Children’s Tumor Foundation website.
About Sanford Research
Sanford Research is a non-profit research organization and is part of Sanford Health, an integrated health system headquartered in the Dakotas. Sanford Health is one of the largest health systems in the nation with a presence in nine states and three countries. More than $600 million in gifts from Denny Sanford has provided for an expansion of research initiatives in type 1 diabetes, breast cancer and genomics in internal medicine.
With a team of more than 200 researchers, Sanford Research comprises several research centers, including Children’s Health Research, Edith Sanford Breast Center, Cancer Biology, Center for Health Outcomes and Prevention and Sanford Sports Science Institute.