Molecular Insight Pharmaceuticals has announced the initiation of a clinical trial of Azedra in pediatric neuroblastoma patients. Neuroblastoma is a type of neuroendocrine cancer that primarily affects children and is the most common solid tumor in children outside of the brain.

The drug Azedra is a small, targeted radiotherapeutic molecule that binds to the norepinephrine transporter, a protein highly expressed on neuroendocrine tumors such as neuroblastoma. Because of this preferential binding, Azedra can deliver a greater amount of radiation to the tumor cell, thus increase tumor killing while reducing side effects.

“Neuroblastoma is an aggressive and difficult to treat form of neuroendocrine cancer that usually affects children under five years of age,” said Katherine Matthay, M.D., Chief of Pediatric Hematology and Oncology at the University of California San Francisco Children’s Medical Center. “Unfortunately, the prognosis for many of these children is quite poor. There currently are no FDA-approved treatments available for patients who have progressive, recurrent or refractory disease, and our current treatment options are extremely limited.”