Professor Sanford Simon

Sanford “Sandy” Simon, The Gunter Blobel Professor at The Rockefeller University, had focused his work on fundamental cell biology with an emphasis on studying single events from studies of single channels for translocating proteins across membranes (Cell 1991, 1992), for exporting filamentous phage (Science 1999), single proteins moving in the cell (Biophys J 1999), single vesicles fusing or endocytosing (JCB 1999, Cell 2008), the assembly of single viruses (Nature 2008, Nature Cell Bio 2011, eLife 2018).  

Now he studies a single individual well-defined cancer fibrolamellar carcinoma (FLC). When his daughter was 12, she was diagnosed with FLC, a usually lethal liver cancer (Science 2014). He has identified the driver, the transcriptome, proteome to understand pathogenesis in a paediatric cancer (Science 2014, PNAS 2015, PNAS 2017, Cancer Discovery 2021) and is actively preparing therapeutics using drug-repurposing (Cancer Discovery 2021), antisense technology (Clinical Cancer Research 2023, Molecular Therapeutics 2023) and PROTACS. The lab is a cross-disciplinary mix of chemists, bioinformaticians, paediatric oncologists, paediatric surgeons, cell biologists, hepatologists and patients with FLC, who are working at the bench. Sandy is also a founder and chair of The Fibrolamellar Registry, an IRB-approved non-profit patient advocacy group that is an interface between the science and the patients.