Dr. Widemann’s team conducted phase I/II clinical trials of selumetinib, a specific MEK inhibitor, in children with inoperable plexiform neurofibromas, which were causing clinical problems such as pain, disfigurement and
motor weakness. After a series of negative prior clinical trials with targeted therapies, selumetinib demonstrated unprecedented preclinical and clinical activity in NF1 plexiform neurofibromas. Selumetinib resulted in partial responses in 74 percent of patients and a progression free survival of 84 percent after three years on treatment.
The team observed tumor shrinkage in the majority of patients, which was unprecedented compared to previous clinical trials. In addition, and most importantly, the majority of patients experienced improvement in pain, quality of life, or function and thus direct clinical benefit.
Due to Dr. Widemann’s pivotal and pioneering work, selumetinib was approved by the Food and Drug Administration and is now available as the first treatment to help children with NF1 across the United States.
