It has been a year like no other. As we all have learned to adjust to life during the COVID-19 pandemic, we have also had to grapple with inherent racial inequities in our country. It is not an understatement to say that these times have served as a flashpoint, prompting deep re-examination, extraordinary accommodations, and renewed focus. Over the past year, we have also had to make changes and adaptations. With great sadness, we made the decision to postpone the 2020 Washington University NF Center Research Symposium until it is safe for us all to gather again in person. In the clinic, we are now using telemedicine to care for our families with NF, while in the research laboratories, we have established ways to remain productive through virtual laboratory meetings and streamlined operations.
ADVANCING CLINICAL CARE
On a very positive note, this year witnessed the first Federal Drug Administration (FDA)-approved drug for Neurofibromatosis Type 1 (NF1). Based on promising studies spearheaded by Dr. Brigitte Widemann at the National Cancer Institute, the MEK inhibitor, Selumetinib, is now available to treat children and adults with growing plexiform neurofibromas. In addition, we have seen remarkable progress in our understanding and management of brain tumors in people with NF1. Dr. Jasia Mahdi recently published a study on brain tumors in children with NF1 arising in locations other than the optic pathway, forming the foundations for future studies to develop practice care guidelines for these tumors. As a part of a larger international team, we also established new recommendations for the management of brain tumors in children and adults with NF1, as well as worked with the World Health Organization and National Institutes of Neurological Disorders and Stroke to update their resources on Neurofibromatosis. These advances are important for our families whose children with brain tumors are cared for by Dr. Nicole Brossier, our pediatric neuro-oncologist specializing in the treatment of children with NF1-associated brain tumors.
ADVANCING NF RESEARCH
In the laboratory, we celebrated the graduation of two star PhD students, Michelle Wegscheid and Xiaofan (Gary) Guo. Dr. Wegscheid is currently completing her medical training at the Washington University School of Medicine, and hopes to become a neurosurgeon-neuroscientist. In her most recent publication, Michelle used human induced pluripotent stem cells to demonstrate that different NF1 mutations create different defects in human mini-brain development. Dr. Guo is now a Neurology resident at Loma Linda University in California. Gary’s major project was also published this spring, in which he found that Nf1 optic glioma growth in mice is controlled by interactions between nerves and immune system cells, suggesting potential future avenues for brain tumor treatments. In addition, researchers in the NF Center identified new mutations in high-grade gliomas (Dr. Wing Wong) and malignant peripheral nerve sheath tumors (Dr. Angela Hirbe).We have also been fortunate to attract several new members to our research team, including Kelly Hartigan (future MD-PhD trainee), Olivia Cobb (staff bioinformatician), Dr. Alex Chen (new postdoctoral fellow), Alice Bewley (bioinformatics student), and Ji-Kang Chen (visiting scientist).
RAISING NF AWARENESS
Using a combination of in-person and virtual tours, visitors to the NF Center learned how laboratory studies have advanced our understanding of the health problems affecting children and adults with NF. Furthermore, the NF Center has been able to pivot and transition all of its Complementary Care programs to a virtual format. Whether providing our toddler’s with a virtual Beat NF program, participating in a virtual Club NF session, or continuing our Teen NF program via Zoom meetings; we continue to provide free, high-quality therapy programs to our NF families during these uncertain times.
David H. Gutmann, MD, PhD, FAAN, FANA
Donald O. Schnuck Family Professor
Director, Washington University NF Center
Vice Chair for Research Affairs, Neurology