Multicenter efficacy study of BLZ-100 tumor paint in pediatric tumors of the central nervous system

Brain Cancer
Sarah Leary, MD, MS
Seattle Children's

Brain tumors are the leading cause of death in children. Those who survive the surgeries that excise tumor tissue often suffer a decreased quality of life. It is important to remove as little healthy brain tissue as possible. Surgeons usually rely on their visual and tactile clues to distinguish brain cancer from normal brain.

BLZ-100 is a tested tumor-binding molecule that is attached to a fluorescent molecule. Surgeons are able to discern the fluorescent tumor tissue and excise more precisely, leaving more normal brain tissue intact. This drug has been tested successfully for safety in a pediatric population in a Phase 1 study.

This is a single-arm multicenter Phase 2 efficacy study. The Pacific Neuro-Oncology Consortium (PNOC) is comprised of fifteen of the leading pediatric brain tumor centers in the United States. Per FDA guidance, the study’s endpoints will determine if BLZ-100 successfully reduces the removal of healthy brain tissue in children with brain tumors. They will enroll 96 subjects and gather data related to histology, molecular diagnostics, tumor cell proliferation, extent of tumor resection and survival.

BLZ-100 could become the standard of care for pediatric brain tumors and greatly increase the post-surgical quality of life for these children.

Clinical Summary:

Precise neurosurgery can result in an increase chance of cure and decrease chance of brain damage for children diagnosed with a brain tumor. Currently, surgeons rely on their visual and tactile clues to distinguish brain cancer from normal brain, but unfortunately this is often very difficult. Brain tumors are now the leading cause of cancer death and the majority of survivors have decreased quality of life related to surgery and toxic treatment.

BLZ-100 Tumor Paint is a fluorescent molecule that is given as an injection prior to neurosurgery, attaches to tumor tissue and allows the surgeon to distinguish the fluorescent tumor tissue from healthy brain. BLZ-100 Tumor Paint has been demonstrated to be safe for pediatric use and this study proposes to conduct a phase 2 clinical trial to evaluate BLZ-100 Tumor Paint at 15 of the leading pediatric brain tumor centers in the United States that make up the Pacific Neuro- Oncology Consortium (PNOC).

No drug has ever been initially developed for pediatric brain tumors. Dr. Leary’s study team, in collaboration with Blaze Bioscience, is working with the FDA to determine the most efficient way to evaluate BLZ-100 Tumor Paint so that, if effective, it may immediately become a standard of care for all children with brain tumors. Dr. Leary’s team can maximize the efficiency of this study by working within the established PNOC infrastructure.