Unmet Need: Glioblastoma (GBM) is the most common malignant brain tumor in adults, affecting 17K patients in the US annually. GBM stem cells underlie GBM recurrence, leading to treatment resistance and poor patient outcomes. Standard treatments remove the primary tumor cells but leave space for GBM stem cells to cause tumorigenesis. There is a need for therapies that can specifically target this stem cell population to prevent GBM recurrence and treatment resistance.
Technology Overview: Johns Hopkins inventors have developed a novel therapeutic that specifically targets GBM stem cells and prevents their pathogenic proliferation. The invention is a targeted nanoparticle for the delivery of miR-603, a tumor suppressor in which its expression is dysregulated in GBM stem cells. The inventors have shown that their technology can reduce the neurosphere clonogenic potential of the cancer stem-cells in vitro and sensitize patent-derived GBM stem-like cells to ionizing radiation (IR), thus demonstrating the potential of this technology as a therapeutic against GBM.
Stage of Development: The inventors are planning to test their technology in animal models and in combination with known chemotherapy drugs and IR.
Publications:
1) Shabana, et al. Targeted liposomes encapsulating miR-603 complexes enhance radiation sensitivity of patient-derived glioblastoma stem-like cells. Pharmaceutics, July 21, 2021.
2) Ramakrishnan et al. Radiation-induced EV release of miR-603 promotes IGF1-mediated stem cell state in glioblastoma. EBioMedicine. June 7, 2020.