Assessing Therapeutic Potential of a New Drug ICG-001 in Glioblastoma
Glioblastoma multiforme (GBM) is the most prevalent and malignant form of brain cancer. Currently, standard care for GBM patients consists of surgical resection, radiation treatment, and chemotherapy. Despite these aggressive treatments, median survival time is only 15 months1, primarily due ineffective elimination of cancer cells and rampant tumor recurrence. While multiple promising drug candidates are in clinical trials, therapeutic potentials of some of these drugs have already been found to fail expectation2. Clearly, new therapeutic avenues for GBMs have to be explored.
This pilot project aims to determine whether a novel compound (ICG-001) can target an unique population of tumor cells called cancer stem cells (CSCs), cancer cells with defining stem cell characteristics. CSCs have been shown to be resistant to current therapies and they have the unique ability to initiate a tumor upon transplantation, suggesting that they are responsible for therapy resistance and tumor recurrence. Our preliminary data suggest that ICG-001 is a potent inhibitor of GBM stem cells in culture. We propose to determine its efficacy in a larger number of independent patient derived GBM stem cell lines to test its general applicability, and to assess the efficacy of the drug in live mice carrying human GBM tumors in their brains.
The Maine Cancer Foundation Pilot Grant program is ideal to fund this proof-of-principle research on testing the efficacy of a new drug (ICG-001) that has the potential to eliminate GBM stem cells. A second-generation compound of ICG- 001 is currently in clinical trial for other cancers (colon and leukemias); therefore, the potential for translating our findings to the clinic is very high and imminent. The preliminary data gathered in this study will prepare us for future grant applications (NIH R21 or R01) to translate our findings to the clinic.