Skip to main content

From Mouse to Man: Monday’s Highlights from the Neurofibromatosis Conference

By June 13, 2011December 5th, 2023Awareness, NF Conference

Today at the NF Conference a number of presentations focused on how we can harness the biology of NF1 tumors to select and test candidate drug treatments.  A key focus was on the extent to which we can draw information from mouse models and apply it to the human situation to make rational decisions about which drugs emerging from mouse studies should be advanced to clinical trials.

This year’s NF Conference co-chair Dr. Nancy Ratner(University of Cincinnati) a member of the CTF NF Preclinical Consortiumhas developed a unique and valuable mouse model that develops NF1 plexiform tumors over many months, thereby reflecting the slow growth of these tumors seen in the human. This makes the mouse a good model for testing candidate drug therapies that may eventually advance to humans. Dr. Ratner showed promising though very preliminary findings from testing of a MEK targeted drug in these mice once tumor has established, and found the drug slowed further tumor growth in a proportion of the mice.   Dr. Kevin Shannon (UCSF), also a member of the CTF NFPC, described his work on mice that develop NF1-related leukemias.  Dr. Shannon has also had preliminary success with MEK inhibitors; in the case of the mouse leukemias the drug helps to restore normal blood cell proliferation and circumvents leukemia progression.  Dr. Wade Clapp (Indiana University) described his work focused on understanding the biology and role of mast cells in plexiform tumors, as mast cells are inflammatory cells thought to play a key role in promoting tumor growth. (Gleevec/Imatinib, currently in clinical trials for NF1 plexiform neurofibromas , targets mast cell signaling).

Dr. Brigitte Widemann (NCI) described two new clinical trials for NF1 tumors. The MEK inhibitor AZD6244 will be assessed as a treatment for plexiform neurofibromas, and a combination of mTOR inhibitor RAD001 + VEGF inhibitor bevacizumab (Avastin) will be assessed as a treatment for MPNSTs. Dr. Widemann emphasized the importance of  preclinical researchers collaborating closely with clinical trialists to ensure the most promising drugs advance to the clinic.  Dr. Scott Plotkin(Harvard/MGH) gave thought-provoking presentations offering some cautionary notes on the extent to which we can draw information from mice and apply it to humans, emphasizing the importance of well validated models that truly represent the human condition.