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Young Investigator Unravels New NF1 Drug Target

By February 4, 2010December 18th, 2023Awareness, NF1, Science & Research

Current Children’s Tumor Foundation Young Investigator Awardee Dr. Sutapa Banerjee (Washington University School of Medicine) is first author of a new publication* (links to abstract) in Cancer Research that identifies a new candidate drug target for treatment of NF1 tumors. Senior author Dr. David Gutmann is Dr. Banerjee’s mentor and also heads the optic pathway glioma site of the Children’s Tumor Foundation NF Preclinical Consortium.

The NF1 gene encodes neurofibromin, a tumor suppressor protein that suppresses the Ras cell signaling pathway. When the NF1 gene is mutated and functional neurofibromin protein is not made, these cell signals are hyperactivated. A number of these signals are being evaluated as candidate drug targets for NF1 treatments.  Dr. Banerjee’s study sought to identify additional new candidate drug targets. By screening a chemical drug library on NF1-deficient malignant peripheral nerve sheath tumor cells, the group found a natural product called cucurbitacin-I that inhibited growth. Research revealed this drug is targeting a cell signal called STAT3 which is hyperactive in NF1-deficient cells. The effects of the drug are independent of cell signals TORC1- and Rac1. This is important because sometimes blocking a drug target either blocks or stimulates another target which interferes with the effectiveness of drug.  This study however suggests STAT3 may be directly targeted with drugs to halt tumor growth without potential feedback to or interference from other cell signals.


* Banerjee et al. (2010) The Neurofibromatosis Type 1 Tumor Suppressor Controls Cell Growth by Regulating Signal Transducer and Activator of Transcription-3 Activity In vitro and In vivo.  Cancer Res; 70(4); 1356-66.