For decades, UCSF has led the way in understanding the fundamental mechanisms of neurofibromatosis biology to build the next generation of diagnostic tests and therapeutic approaches. We are active members of the UCSF Brain Tumor Center SPORE, the UCSF Cancer Health Outcomes SPORE, and the multi-institutional DHART SPORE. The main goal of our work is to translate discoveries safely, effectively, and expeditiously to the clinic in order to help people with neurofibromatosis.
Translational Research
Led by Translational Research Director Harish Vasudevan, MD, PhD, our lab studies neurofibromatosis biology from the bench to the bedside and is supported by funding from the National Cancer Institute/National Institutes of Health, Department of Defense, Gilbert Family Foundation, Neurofibromatosis Therapeutic Acceleration Program, and Children's Tumor Foundation. Recent highlights of our work include:
- Defining molecular groups of neurofibromatosis-associated nerve sheath tumors (Vasudevan et al., Nature Communications 2024)
- Investigating KRAS inhibition as a potential target for NF1 (Vasudevan et al., PNAS 2025)
- Identifying molecular signatures underlying MEK inhibitor response in gliomas (Pan et al., JCI Insight 2025)
- Understanding how the immune microenvironment mediates radiation response in malignant peripheral nerve sheath tumors (MPNSTs) (Zhu et al., JCI 2026)
- Using spatial transcriptomics to understand mechanisms of malignant transformation (Mirchia et al., Neuro-Oncology 2026)
Clinical research
Our physicians lead and enroll patients in NFCTC, PNOC, and industry-sponsored trials, including a study of binimetinib in NF1 led by Alyssa Reddy, MD, and a multi-institutional study (NF-118) combining radiation and MEK inhibition for high-grade gliomas for which Harish Vasudevan, MD, PhD, serves as radiation oncology chair.
Neurocognitive and quality-of-life research
Our neuropsychology team contributes to national standards for measuring cognition and quality of life in NF through the REiNS (Response Evaluation in Neurofibromatosis and Schwannomatosis) International Collaboration and the NFCTC neurocognitive committee.