Understanding steroid hormone signaling in tumor cells will help predict responses to such therapies in order to establish guidelines. The in vivo work will produce an animal model for testing such therapies. Many selective estrogen receptor modulators are being developed for this purpose in other tumor types that could be useful in NF1 if there is a research-based rationale supporting clinical trials. Our work will address this need. NF1 is characterized by abnormal cell growth, the most common form being the neurofibroma, a generally slow-growing benign nerve tumor composed mostly of Schwann cells.
Dermal neurofibromas can cause disfigurement and affect function, depending on location and size.
Plexiform neurofibromas can grow very large and may be disabling or fatal. This is an area of great interest in research of cancer. However there has been virtually no research in this field in NF1 or Schwann cells. This project will determine scientifically whether the hormones estrogen or progesterone have a growth-promoting effect in neurofibromas. These cultures can grow after transplantation into the mouse nerve, developing a small version of the original human tumor.