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NF Bites: Progress in Understanding NF1 Malignancies

By April 13, 2010December 4th, 2023NF1

Welcome to the fifth in a series of “NF Bites” – providing snapshots of individual areas of neurofibromatosis research and how the Children’s Tumor Foundation is advancing this. Over the coming days and weeks we will focus on different aspects of neurofibromatosis research. Today: where are we with research progress in NF1 malignancies?

Malignant tumors are estimated to occur in up to 15% of persons with NF1 and may occur in plexiform neurofibromas or other cell type. However it is not readily predictable when and where malignancy will occur; therefore it is critical to understand how and why malignant transformation occurs and how malignant tumors might be treated or even prevented.  The Children’s Tumor Foundation funds a number of studies to unravel what causes a tumor to transform from benign plexiform neurofibroma to malignancy and to assess candidate drug treatments for MPNSTs.

Through the CTF NF Preclinical Consortium, a $4M multi-year initiative to accelerate the most promising candidate drugs to the clinic, the laboratory of Karen Cichowski (Harvard/ Brigham & Women’s Hospital) is testing a pipeline of candidate MPNST drugs in collaboration with Novartis and Genentech.
David Wiemer(University of Iowa) received two Drug Discovery Initiative Awards to develop candidate MPNST treatments called schweinfurthins; these studies led to two publications in the Journal of Organic Chemistry and the schweinfurthins are now being commercialized through startup biotech Terpinoid Therapeutics.
Mark Philips (New York University Langone Medical Center) has a Drug Discovery Initiative Award to test new drug entities called Icmt inhibitors on MPNST cells to see if they halt growth. This work is in progress.
Among our Young Investigator Awardees, Jody Fromm (Harvard/Brigham and Women’s Hospital) is currently testing combined drugs Sunitinib and Rapamycin in mice with MPNSTs. Young Investigator Awardee Johanna Buchstaller (University of Michigan) is looking for those cells within a plexiform neurofibroma that drive tumors from benign to malignant and has identified a gene, Hmga2 that may be involved.
Look for more NF Bites in the coming days and weeks!