Neurofibromatosis Clinical Trials Consortium

As part of an ongoing effort to facilitate neurofibromatosis research, UAB serves as the national coordinating center for the NF Clinical Trials Consortium. This collaborative group of 17 medical centers across the country is dedicated to conducting clinical trials of the most promising drug therapies for all forms of neurofibromatosis. Formed in 2006 with funding from the U.S. Army Medical Research and Material Command, the consortium helps to accelerate the pace of research by providing the opportunity for a high volume of patients to participate in clinical trials throughout the nation.

Identification of the genes associated with NF1, NF2, and some forms of schwannomatosis has paved the way for the testing of medications that may be effective in preventing or treating complications of neurofibromatosis. Many of these medications work by blocking specific cell signaling pathways, an intricate form of cell communication that is also involved in other disease processes, including cancer. During the cell signaling process, each cell receives a complex combination of signals which simultaneously trigger many different signaling pathways in a domino-like fashion. When specific signaling pathways become altered, cells respond with uncontrolled growth and an increased capability to invade surrounding tissues. New medications currently being tested in clinical trials block these altered cell signaling pathways, providing a potential means of shrinking existing neurofibromas or preventing others from growing.