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FDA Grants Orphan Status to Brain Cancer Surgery Aid

Emperor scorpion

Blaze Bioscience’s BLZ-100 is based on a peptide derived from scorpion venom. (Rosa Pineda, Wikimedia Commons)

7 July 2015. The U.S. Food and Drug Administration is granting orphan drug status to an engineered peptide that illuminates brain cancer cells making them easier to surgically remove. The treatment, code-named BLZ-100, is made by Blaze Bioscience, a biotechnology company in Seattle.

Blaze Bioscience is a spin-off enterprise from Fred Hutchinson Cancer Research Center, also in Seattle, to develop and commercialize drug candidates based on engineered peptides that better target tumor cells than conventional cancer drugs. The company is commercializing research begun at the Hutchinson center on optides — short for optimized peptides — engineered protein molecules that more precisely target cancer cells than most of today’s chemotherapy drugs. The better that cancer treatments can zero-in on and bind to tumor cells, the fewer the side-effects, often severe, that affect cancer patients undergoing chemotherapy.

BLZ-100 refines the optide technology into what the company calls tumor paint that combines optides with a fluorescent dye, emitting light in the near-infrared range. BLZ-100 is based on a peptide variant called chlorotoxin, originally derived from scorpion venom, which in its natural state has been shown to bind to some tumors. Blaze says treatments with BLZ-100 provide high-resolution visualization of cancer cells during surgery, making possible more precise and complete removal.

According to the company, FDA granted orphan drug status to BLZ-100 for its use in surgery on malignant brain tumors. Blaze is recruiting participants to test BLZ-100 in an early stage clinical trial of adults with glioma, a type of cancer affecting glial cells that surround and support the brain’s nerve cells. Cancers of this type can be aggressive and highly malignant, and account for about one-third of all brain cancers.

A second early-stage trial also to test BLZ-100 is recruiting children and young adults (up to age 30) with tumors in their central nervous systems. In both trials, investigators are looking primarily at safety of the treatments, but also concentrations of BLZ-100 in the blood up to 4 hours following injection, as well as the ability of BLZ-100 to illuminate cancerous brain tissue.

Orphan drug designation is granted by FDA to treatments being developed for diseases affecting fewer than 200,000 people in the U.S. Therapies, both drugs and biologics, designated as orphan drugs qualify for incentives such as tax credits for clinical trials and exemptions from marketing application fees.

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