A Pilot Study of Bevacizumab (Avastin) in Patients With Unresectable or Recurrent Hemangioblastoma From Von-Hippel-Lindau Disease

Von Hippel-Lindau (VHL) disease is an inherited syndrome manifested by a variety of benign
and malignant tumors. Hemangioblastomas are the most common lesion associated with VHL
disease affecting 60-84% of patients with a mean age at diagnosis of 29 years. Standard
treatment for this disease is by surgery or radiotherapy. No approved systemic therapy yet
exists. Patients with VHL have an increased growth factor production, specifically vascular
endothelial growth factor (VEGF), resulting in angiogenesis (growth of blood vessels).
Studies show that Bevacizumab inhibits the growth of VEGF protein and will block the
VEGF-driven angiogenesis and result in stabilization and regression of hemangioblastomas in
VHL disease patients. The dose of bevacizumab will be 10 mg/kg every two weeks for up to 6
months.