GBM, a.k.a. as grade IV astrocytoma, is the most common and most aggressive of the primary brain tumors. GBM usually spread quickly to other parts of the brain. For this reason, this disease is difficult to treat. Surgical intervention is essential in the initial treatment of GBM. Chemotherapy is the most common treatment option for recurrent malignant gliomas. GBMs account for 12-15% of intracranial tumors and 50-60% of primary brain tumors. Few patients with GBM survive longer than 3 years and only a handful survive 5 years. In 2005, 12,940 cases of GBM were expected to be diagnosed in the U.S.
Dr. Bruce Frankel, at the Medical University of South Carolina, has developed a novel therapy for GBM using intrathecal delivery of Ara-C (a.k.a. cytarabine or cytosine arabinoside). Pilot studies in patients have shown that treatment with this compound can result in significant responses, warranting further study of this treatment in a Phase I/II clinical trial.
Recently, Orphan Drug Status was obtained for using Ara-C to treat gliomas. Orphan drug status provides certain benefits, such as, among others, a 50 percent tax credit for expenditures on clinical trials and seven years of marketing exclusivity after the approval of the drug.