Therapeutic/Technology: Small molecule therapy
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 […]
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 […]
Development of COVID-19 Antiviral Therapy Using Human iPSC-Derived Lung Organoids
Research Objective To develop a new therapy for COVID-19 using human iPSC-derived lung organoids that targets SARS-CoV-2 protease known as the virus’ “Achilles Heel” Impact Our work, if successful, will bring a class of new drugs directly targeting viral enzyme and open the door for future COVID therapies. Major Proposed Activities Complete synthesis and testing […]
Drug Development for Autism Spectrum Disorder Using Human Patient iPSCs
Research Objective We will use human patient induced pluripotent stem cell (hiPSC)-based models to screen for a drug that activates a transcription factor critical to the treatment of Autism Spectrum Disorder (ASD). Impact Our goal is to develop a small molecule to treat Autism Spectrum Disorder (ASD), which currently affects 1/68 children born in the […]
Small Molecule Proteostasis Regulators to Treat Photoreceptor Diseases
Research Objective We will discover small molecule compounds that correct disease in eyecups (retinal organoids) differentiated from patient iPSCs with photoreceptor diseases. Impact Our small molecule agents will provide new treatments for achromatopsia and cone-rod dystrophy. These are rare hereditary blinding diseases with no cures Major Proposed Activities Transcriptomic and proteomic profiling of control and […]
Stimulating endogenous muscle stem cells to counter muscle atrophy
Research Objective Intramuscular delivery of two repurposed FDA approved drugs will activate resident muscle stem cells. This therapeutic strategy will augment regeneration and restore strength to atrophied muscles. Impact Currently effective treatments are lacking for localized muscle atrophy due to nerve injury (eg., Carpal Tunnel Syndrome) or immobilization after trauma or surgery (eg., hip or […]