Disease Focus: Cancer
Targeting pancreatic cancer stem cells with DDR1 antibodies.
Research Objective A therapeutic antibody to DDR1 for targeting pancreatic cancer stem cells to overcome resistance to chemotherapy and potentiate the treatment of advanced cancer. Impact PDAC is lethal cancer that poorly responds chemotherapy to which it becomes resistant. DDR1 antagonistic Abs should improve chemotherapy responsiveness and may cause tumor regression as a monotherapy. Major […]
Modulation of oral epithelium stem cells by RSpo1 for the prevention and treatment of oral mucositis
Research Objective Locally delivered formulation of RSpo1 protein as an activator of Lgr5+ epithelial stem cells in chemotherapy- or radiation therapy-induced oral mucositis Impact Oral mucositis Major Proposed Activities RSpo1 formulation design and selection for optimal oral delivery Activation of Wnt pathway by formulated RSpo1 in-vitro Production of RSpo1 protein Oral stem cell expansion by […]
Development of a novel stem-cell based carrier for intravenous delivery of oncolytic viruses
Research Objective Develop a stem cell-based platform that safely and efficiently delivers viruses that specifically kill tumor cells and restore immune activity in patients with advanced cancer. Impact Overcome the inherent limitations that prevent efficient intravenous delivery of tumor killing viruses to metastatic tumors. Major Proposed Activities Compare the relative efficacy of native or cargocyte-delivered […]
Preclinical development of an exhaustion-resistant CAR-T stem cell for cancer immunotherapy
Research Objective The expected outcome is an exhaustion-resistant CAR-T cell, which persists long-term in a functional progenitor T cell state in the tumor microenvironment and can be used for cancer immunotherapy. Impact CAR-T cells are effective in B cell cancer, but less than 50% of patients experience long-term disease control. Exhaustion-resistant CARs may provide long-term […]
Generating deeper and more durable BCMA CAR T cell responses in Multiple Myeloma through non-viral knockin/knockout multiplexed genome engineering
Research Objective We will use integrated gene editing techniques to develop a new CAR-T cell therapy for multiple myeloma treatment Impact Develop an improved CAR-T cell therapy for patients with refractory multiple myeloma and a new manufacturing strategy that circumvents the costs and inefficiencies of viral production. Major Proposed Activities Establish and optimize a CRISPR […]
Human iPSC-derived chimeric antigen receptor expressing macrophages for improved cancer treatment.
Research Objective These studies will produce a new CAR-targeted iPSC-derived macrophage-based cell therapy product for treatment of refractory malignancies such as ovarian cancer. Impact These studies eliminate a bottleneck in macrophage production and enable these cells to be engineered and manufactured in a standardized, off-the-shelf manner, rather than on a patient-specific basis. Major Proposed Activities […]
Stem cell-derived extracellular vesicles to reverse radiation-induced brain injury
Research Objective These preclinical studies will discover the efficacy of stem cell-derived, nanoscale, extracellular vesicles (candidate) to treat adverse effects of cancer therapy on brain function and cognition. Impact Stem cell-derived extracellular vesicles will address the confounders of stem cells (tumors, immunorejection, immunosuppression) & mitigate debilitating side-effects of cancer therapy on the brain. Major Proposed […]
Targeting Critical Regulators of Cancer Stem Cells
Research Objective We will develop a small molecule inhibitor that blocks the growth of human pancreatic cancer and AML cancer stem cells in vitro and in vivo. Impact This work will lead to a new treatment for cancer stem cell driven diseases such as AML and pancreatic cancer. In addition, it will improve the prognosis […]
Human iPSC-derived chimeric antigen receptor-expressing macrophages for cancer treatment
Research Objective These studies will produce a new CAR-targeted iPSC-derived macrophage-based cell therapy product for treatment of refractory malignancies such as ovarian cancer. Impact These studies eliminate a bottleneck in macrophage production and enable these cells to be engineered and manufactured in a standardized, off-the-shelf manner, rather than on a patient-specific basis. Major Proposed Activities […]
Preclinical Development of An HSC-Engineered Off-The-Shelf iNKT Cell Therapy for Cancer
Research Objective The expected outcome is a therapeutic candidate, allogeneic HSC-engineered HLA-I/II-negative human iNKT cells, that can potentially be used as an off-the-shelf cellular therapy for treating cancer. Impact The proposed off-the-shelf HSC-engineered iNKT therapy has the potential to become a general cancer immunotherapy for treating multiple cancers and a large population of cancer patients. […]