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.
By Salvatore La Rosa, VP Research and Development
Early investments from the Children’s Tumor Foundation are paying off and delivering innovative and impactful research. It’s great to see research moving forward thanks to the support of both private and public funding. Our philosophy of betting on high-risk high-reward projects is proving successful and will eventually help us realize our vision of ending NF.
We are very proud to announce that the Children’s Tumor Foundation has been ranked as one of the best nonprofit organizations to work for by the leading publication for nonprofit management, The Nonprofit Times. This annual ranking is dedicated to finding and recognizing the best employers in the nonprofit industry.
Lai Man (Natalie) Wu, PhD, of Cincinnati Children’s Hospital Medical Center, is first author on a recently published article in Cancer Cell. This study, funded in part by a 2017 Young Investigator Award from the Children’s Tumor Foundation, suggests ways to attack MPNSTs.
Research funded by the Children’s Tumor Foundation (CTF) and led by Ludwine Messiaen, PhD, professor of genetics at the University of Alabama at Birmingham (UAB), has shown that missense mutations in a cluster of just five codons in the NF1 gene are an important risk factor for severe symptoms of the genetic disorder neurofibromatosis (NF) type 1, thereby improving the predictability of this condition when these specific mutations are present in an individual. This work was published today in the American Journal of Human Genetics.