Therapeutic/Technology: Small molecule therapy
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 […]
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 […]
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 […]
Novel methods to eliminate cancer stem cells
Research Objective Our goal is to develop and optimize novel drugs that can attack blood cancer stem cells. These drugs interfere with a target protein, and will prevent relapse of disease. Impact By targeting blood cancer stem cells, these compounds can be used to treat and prevent recurrence of cancer in patients. In the future, […]
Therapeutics to overcome the differentiation roadblock in Myelodysplastic Syndrome (MDS)
Research Objective This proposal will deliver a small molecule therapeutic candidate for the treatment of Myelodysplastic Syndromes and will act by inducing differentiation on mutated hematopoietic stem cells. Impact This application will enable development of a therapeutic candidate for the treatment of Myelodysplastic Syndromes, a preneoplastic hematological condition of HSCs. Major Proposed Activities Determine the […]
Improving the efficacy and tolerability of clinically validated remyelination-inducing molecules using developable combinations of approved drugs
Research Objective The candidate is a fixed dose binary small molecule drug combination, consisting of two agents that act synergistically on a multipotent stem cell population in the CNS to stimulate remyelination. Impact The proposed studies will address bottleneck issues related to the effect size and tolerability of clinically validated remyelination drug classes. Major Proposed […]
Development of a new therapeutic for directing target specific stem cell migration and treatment
Research Objective A drug-stem cell combination therapy wherein the drug will direct and promote the delivery and distribution of stem cells to the disease site for the optimal therapeutic effect of the stem cells Impact Amyotrophic lateral sclerosis (ALS) and the way to deliver and enhance stem cell-based treatment of ALS Major Proposed Activities Complete […]
Small Molecules to inhibit Nemo-like Kinase for Treatment of Diamond Blackfan Anemia
Research Objective We propose to study small molecules that inhibit Nemo-like Kinase, to improve the production of red blood cells in bone marrow stem cells of children with Diamond Blackfan Anemia (DBA). Impact If small molecule NLK inhibitors are identified that are effective in improving the anemia of DBA and nontoxic, then treatment and transfusions […]