While we always provide the best possible care, it is important to understand that just because the treatment offered is currently the best one available, it does not mean that it is the best possible therapy. Unlike most common medical conditions that have a standard plan of care—or a known most effective therapy—there is no standard plan of care for the treatment. Because NF1 affects no two individuals exactly the same way, physicians and researchers must work together to find the most effective way to treat each individual living with NF1. The promise of personalized medicine – or treatments designed specifically for certain people and certain features seen in NF1 – may emerge from the pioneering studies underway at the Washington University NF Center. Combining investigations using mouse and human models of NF1 (Dr. Corina Anastasaki), clinical datasets (Dr. Stephanie Morris), and machine learning and bioinformatic approaches (Dr. Aditi Gupta and Dr. Philip Payne), we aim to identify the risk factors for specific problems arising in people with NF1, assess the response to treatment rapidly, and personalize our therapies to any given individual.
Morris SM, Gupta A, Kim S, Foraker RE, Gutmann DH, Payne PRO. Predictive Modeling for Clinical Features Associated With Neurofibromatosis Type 1. Neurol Clin Pract. 2021 Dec;11(6):497-505. doi: 10.1212/CPJ.0000000000001089. PMID: 34987881
Chatterjee J, Sanapala S, Cobb O, Bewley A, Goldstein AK, Cordell E, Ge X, Garbow JR, Holtzman MJ, Gutmann DH. Asthma reduces glioma formation by T cell decorin-mediated inhibition of microglia. Nat Commun. 2021 Dec 8;12(1):7122. doi: 10.1038/s41467-021-27455-6. PMID: 34880260
Anastasaki C, Wegscheid ML, Hartigan K, Papke JB, Kopp ND, Chen J, Cobb O, Dougherty JD, Gutmann DH. Human iPSC-Derived Neurons and Cerebral Organoids Establish Differential Effects of Germline NF1 Gene Mutations. Stem Cell Reports. 2020 Apr 14;14(4):541-550. doi: 10.1016/j.stemcr.2020.03.007. Epub 2020 Apr 2. PMID: 32243842