The recent death of Senator Ted Kennedy has focused new attention on brain cancer. Glioblastoma multiforme (GBM), the aggressive brain tumor that took Sen. Kennedy’s life, is one of the deadliest brain tumors that can occur in both adults and children.
It is also one of the most difficult tumors to treat. Oral drugs can’t cross the blood-brain barrier, and tumor removal surgery has limited effectiveness. Malignant gliomas are prone to infiltrating healthy tissue. Small pieces of tumor, invisible during surgery, may later take root and grow back into other tumors. Patients generally live less than a year after diagnosis.
Like many other companies in the field, NeoPharm of Lake Bluff, Ill. has encountered challenges in its efforts to develop a treatment for GBM. NeoPharm’s drug candidate, IL 13-PE38QQR (IL-13), is designed to kill malignant glioma cells while preserving healthy tissue. The company uses a method called convection-enhanced delivery to deliver IL-13 through catheters inserted in the brain tissue. Further trials for IL-13 were put on hold in late 2006 after the drug candidate failed to meet its primary endpoint in a Phase III clinical trial. The company went head-to-head with the current standard-of-care treatment, the Gliadel Wafer, developed by MGI Pharma. The NeoPharm study found that IL-13 was just as effective, but not more so, than the Gliadel Wafer.
In the meantime, NeoPharm is exploring other options for IL-13. The company recently signed a Cooperative Research and Development Agreement (CRADA) with the National Institute of Neurological Diseases and Stroke (NINDS), a division of the National Institutes of Health (NIH) to start a Phase I trial for various brain diseases. As part of the agreement, NeoPharm will be starting a Phase I trial for brain stem cancer. The trial will combine NeoPharm’s IL-13 with NINDS’ convection-enhanced delivery, which the agency had previously licensed to NeoPharm.
Other companies developing treatments for brain cancer include Boneca, ZGene, and ImmunoCellular Therapeutics.