Therapeutic/Technology: Small molecule therapy
Repurposing Aminoadamantane Nitrate Compounds to Inhibit SARS-CoV-2 Viral Activity and to Protect the Brain from Viral-Related Damage
Research Objective The objective is to screen a series of aminoadamantane nitrate compounds for their ability to protect hiPSC-derived neurons from SARS-CoV-2-related damage and to block SARS-CoV-2 activity. Impact If successful, our screen would identify a drug candidate for further development that would protect neurons from SARS-CoV-2-related damage and also inhibit SARS-CoV-2 activity. Major Proposed […]
Identifying a lead compound for COVID19 using high throughput screening with lung stem cell organoids
Research Objective We propose to use a lung stem cell based organoid to identify a new compound for COVID-19 by screening a library of FDA approved compounds that could be repurposed for COVID-19 infection. Impact If successful, we will find a therapy to treat COVID-19 infection and prevent the lung complications that are so deadly. […]
Stimulating endogenous muscle stem cells to counter muscle wasting
Drug discovery for gastrointestinal motility disorders using hPSC-derived enteric ganglioids
Research Objective Our goal is to use stem cell models to identify a small molecule drug candidate for GI motility disorders. Impact The candidate will be used for severe gastrointestinal motility disorders including chronic constipation, achalasia and gastroparesis. Major Proposed Activities Assessment of candidate compounds on stem cell derived models from diverse genetic backgrounds Evaluation […]
Developing a breast cancer stem cell drug
Research Objective To make a drug that kills breast cancer stem cells Impact Metastatic breast cancer is incurable. The goal is to make a drug leading to cures Major Proposed Activities Make a novel drug that potentially more effectively treats breast cancer with less toxicity Test the drug for efficacy and toxicity Breast cancer is […]
Vax-CT to promote formation of cancer-specific T memory stem cell for personalized cancer immunotherapy
Research Objective A vaccine booster to induce antigen-specific T memory stem cells that will help enhance the long-term immunity against cancer recurrence Impact Cancer recurrence presents an unmet medical need. Cancer vaccines are promising, but often lack a long-term protection. We will induce T memory stem cells (TMSCs) to boost the long-term immunity. Major Proposed […]
Reversal of dysregulated myelopoiesis in breast cancers and cancer stem cells to boost antitumor immunotherapy
Research Objective A new antiestrogen drug will be developed to stop breast cancer (BC) by direct effects on BC cells including stem cells and indirect action on specific procancer immune cells that surround the cancer. Impact Substantial numbers of patients with localized breast cancer (BC) and essentially all patients with advanced BC become resistant to […]
Pharmacological regenerative treatment of idiopathic pulmonary fibrosis targeting the senescent niche of lung progenitor cells.
Research Objective Novel selective pharmacological strategy targeting senescent lung stem cells Impact Idiopathic pulmonary fibrosis along with other interstitial and age-related lung diseases Major Proposed Activities Chracterize senescence cells, including senescent stem and progenitor cells in the lung of patients affected by idiopathic pulmonary fibrosis Screening of a Rubedo Life Science library of senolytic small […]
Development of novel small molecules against cancer stem cells in solid cancers
Research Objective To study and optimize lead compounds with multi-kinase activity against existing glioma stem cells and radiation-induced phenotype conversion of non-stem glioma cells into induced glioma stem cells. Impact Glioblastoma is a universally deadly disease. While radiotherapy prolongs survival in glioblastoma it has hit a critical barrier. The proposed study aims to improve the […]
Drug Development of Inhibitors of Inflammation Using Human iPSC-Derived Microglia (hiMG)
Research Objective We will screen for modifiers of the response to misfolded αSyn and Aβ, and their cognate antibodies. Development of drugs to combat this inflammation is important in neurodegenerative diseases. Impact Inhibiting the immune response to minimize NLRP3 inflammasome activation may prevent the neurotoxic effect of activated microglia, and attenuate disease progression in neurodegenerative […]