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    Cannabidiol and Brain Cancer Treatment

    Research Led by CPMCRI Scientists Leads to FDA Orphan Drug Designation for Cannabidiol, to Treat Brain Cancer

    August 26, 2014

    A growing body of research suggests that non-psychotropic cannabinoid compounds, found in the Cannabis plant, are effective antitumor agents in multiple pre-clinical models of aggressive cancers.
    California Pacific Medical Center Research Institute (CPMCRI) scientists Sean McAllister (Ph.D.) and Pierre Desprez (Ph.D.) previously discovered that the cannabinoid, cannabidiol (CBD), inhibits breast cancer metastasis—and more recent research suggests a role in treating the most common and aggressive form of brain cancer, glioblastoma multiforme.

    “The preclinical trial data is very strong, and CBD has favorable safety profile in humans. There's a lot of research to move ahead with to get people excited,” said Dr. McAllister, who along with Dr. Desprez has been studying cannabinoids as potent inhibitors of metastatic cancer for the past decade.

    Glioblastoma multiforme is the most common and severe form of brain cancer. Each year in the U.S., 15,000 people are diagnosed with the illness. Even with standard-of-care radiation therapy, most patients have an expected survival of approximately 15 months after diagnosis.

    Because of the promising preclinical trial data, the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to CBD, for the treatment of glioblastoma. The approval significantly advances development of the compound and moves the research one step further toward phase 2 and 3 clinical trials to test the drug in human patients with the disease. Orphan drug approval of the compound represents the first time in the U.S. that a cannabinoid compound has been recognized by the FDA as a potential potent anti-cancer agent in treating this aggressive form of brain cancer.

    “Based on previous research conducted with CBD to treat brain tumors, we believe there is supportive evidence for the use of CBD as an adjunct treatment in glioblastoma and eagerly anticipate the results from our in-vivo models to further support clinical studies in humans,” said Dr. McAllister. He and other investigators at CPMCRI including Dr. Desprez and Liliana Soroceanu (M.D., Ph.D.) are conducting pre-clinical research into the ability of CBD to sensitize glioblastoma multiforme to current standard of care chemotherapy treatment.

    CPMCRI partnered with Insys Therapeutics, Inc. on a licensing agreement furthering the research which led to FDA orphan drug approval of CBD.

    Orphan drug designation is granted by the FDA to novel drugs or biologics that treat rare diseases or conditions affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven-year period of U.S. marketing exclusivity.

    Dr. McAllister says the next steps will be to work toward obtaining investigational new drug status for CBD, in treating glioblastoma in human patients.