Therapeutic/Technology: Small molecule therapy
Cartilage Regeneration by the Chondrogenic Small Molecule PRO1 during Osteoarthritis
The ability to direct the differentiation of resident mesenchymal stem cells (MSCs) towards the cartilage lineage offers considerable promise for the regeneration of articular cartilage after traumatic joint injury or age-related osteoarthritis (OA). MSCs can be stimulated in vitro to form new functional cartilage. In the OA-affected joint, the repair is insufficient, leaving a damaged […]
Developing a drug-screening system for Autism Spectrum Disorders using human neurons
Autism and autism spectrum disorders (ASD) are complex neurodevelopmental diseases that affect 1 in 150 children in the United States. Such diseases are mainly characterized by deficits in verbal communication, impaired social interaction, and limited and repetitive interests and behavior. Because autism is a complex spectrum of disorders, a different combination of genetic mutations is […]
Preclinical development of a pan Bcl2 inhibitor for cancer stem cell directed therapy
Cancer is the leading cause of death for individuals under 85. Relapse and metastatic disease are the leading causes of cancer related mortality. Anti-apoptotic BCL2 family member overexpression has been shown to promote disease progression in both chronic myeloid leukemia (CML) and prostate cancer. Andr., the emergence of cancer stem cells (CSC) promotes apoptosis resistance […]
Stem Cell-mediated Therapy for High-grade Glioma: Toward Phase I-II Clinical Trials
Despite aggressive multimodal therapy and advances in imaging, surgical and radiation techniques, malignant brain tumors (high-grade gliomas) remain incurable, with survival often measured in months. Treatment failure is largely attributable to the diffuse and invasive nature of these brain tumor cells, ineffective delivery of chemotherapeutic agents to tumor sites, and toxic side-effects to the body, […]
Therapeutic Opportunities To Target Tumor Initiating Cells in Solid Tumors
Cancer is a major cause of human death worldwide. The vast majority of cancer patients suffer from solid tumors whose growth destroys vital organs. We propose to develop novel therapeutic drugs that target solid tumors affecting the brain, colon and ovaries. These cancers account for a significant proportion of currently intractable solid malignancies. Scientists have […]
Drug Discovery for Dilated Cardiomyopathy using Patient-Derived Human iPSCs
Research Objective Greenstone Biosciences will use patient-derived stem cells to discover safe and effective drugs for cardiovascular disease. Impact The use of patient-derived stem cells to discover novel drug targets for cardiovascular disease, identify drug candidates, and establish clinical trial in a dish to evaluate drug safety and efficacy. Major Proposed Activities Use patient-derived induced […]
COVID-19 Antiviral Therapy to Block Direct Cell Injury and Associated Tissue Damage
Translational Candidate Berzosertib (VE-822), a safe drug candidate for treatment against COVID-19, will be investigated. Area of Impact The outcome of the proposed studies will have a significant health benefit to COVID-19 affected patients. Mechanism of Action Our drug candidate, Berzosertib, works as a treatment against COVID-19 by blocking a critical step in virus replication. […]
Using hiPSC-derived lung organoids, a clinically-relevant system, to validate & winnow a list of approved drugs that inhibit SARS-CoV-2 cytopathy
Research Objective Using authentic in vitro models of the human lung, complete with inflammatory cells & vessels, we will validate drugs that might be rapidly repurposed for use in patients with COVID-19. Impact The impact will be the avoidance of an animal model once an approved medication hit has been verified by our model. The […]
Application of PGE2/BPV, a muscle stem cell targeting therapeutic, to the treatment of COVID-19 associated diaphragm atrophy
Research Objective Intramuscular delivery of 2 repurposed FDA approved drugs to activate diaphragm stem cells to augment regeneration and restore strength to COVID-19 patients with diaphragm atrophy from ventilation. Impact Currently effective treatments are lacking for diaphragm atrophy due to ventilation to treat COVID-19. Our treatment will promote full recovery of such patients. Major Proposed […]
Development of a novel PIKFYVE kinase inhibitor for the treatment of COVID-19
Research Objective Alveolar type II cells are the stem cells of the lung, and they are killed by SARS-CoV-2. We will determine if a novel PIKFYVE kinase inhibitor prevents SARS-CoV-2 infection of type II cells. Impact Although PIKFYVE inhibition blocks SARS-CoV-2 infection, no known PIKFYVE inhibitors have suitable drug-like properties. We would validate a novel […]