The current strategy for clinical management of plexiform neurofibromas (pNFs) is observation. While many pNFs remain stable over an individuals’ lifetime, a significant portion will progress. These symptomatic tumors can result in structural disfigurement, pain, airway compression, spinal cord compression, and loss of neurologic function. For patients with symptomatic pNFs that require treatment, surgery is the current standard of care.

However, because pNF tumors grow along normal nerves, they are almost never amenable to full surgical resection as complete removal of the tumor carries a risk of nerve damage. In addition, in many cases, surgery is not feasible due to the location of the tumor relative to other vital organs.

Partial removal or debulking that maintains some of the normal nerve fibers is generally possible, but is often of limited utility as these tumors tend to regrow. Furthermore, with each surgery, there is increased risk of permanent nerve damage.

Radiation therapy is reserved for patients with proven malignancy and plays a very limited role in the management of pNF tumors.

There are currently no effective drug therapies to prevent, halt, or reverse the progression of pNFs. Neurofibromatosis Therapeutic Acceleration Program (NTAP) is dedicated solely to addressing this urgent medical need.