Award Data

PROJECT SUMMARY . Collecting complete and accurate outcome data directly from research participants is becoming increasingly important. Clinical researchers needs a cost-effective approach to capture high-quality patient-reported outcomes. Typically, data captured directly from participants is through self-administered questionnaires or through a human interviewer, each with their own advantages ...

Project Abstract An estimated 5.7 million Americans have Alzheimer’s disease or a related dementia (ADRD) and one in ten people age 65 or older show symptoms of Alzheimer’s disease. As ADRD progresses so does the level of dependency on others. As the disease progresses, higher levels of dependence in people with Alzheimer’s disease are associated with significantly increased burden and costs ...

Abstract The 90 kD heat shock proteins (Hsp90) are molecular chaperones that are responsible for the folding of select proteins, many of which are directly associated with cancer progression. Consequently, inhibition of the Hsp90 protein folding machinery results in a combinatorial attack on numerous oncogenic pathways. Seventeen small molecule inhibitors of Hsp90 have entered clinical trials for ...

PROJECT SUMMARY The long-term goal of the current proposal is to generate a new class of small molecules to treat familial hypercholesterolemia. Familial hypercholesterolemia (FH) patients suffer from excessively high levels of Low Density Lipoprotein Cholesterol (LDL-C), which if left untreated results in death from severe cardiovascular disease. FH is caused primarily by mutations in the LDL rec ...

Neurodon LLC proposes to conduct lead optimization and candidate-seeking activities on a novel series of neuroprotective small molecules that shows efficacy in a transgenic model of Alzheimer’s disease (AD). AD is a leading cause of death in the United States, with some estimates ranking it as high as third behind cardiovascular disease and cancer. Despite the enormity of this national public he ...

PROJECT SUMMARY Chemical risk assessment is widely applied in industries and regulatory agencies as an important tool to evaluate chemical toxicity in support of chemical registration, safety evaluation, and exposure limitation development. One of the most notable improvements in dose-response assessment - a required quantitative step in risk assessment - is the development of benchmark dose (BMD) ...

Project Abstract/Summary Pyruvate dehydrogenase complex (PDC) deficiency (PDCD) is a rare disease of mitochondrial energy failure in which the life expectancy of affected children is severely truncated from unrelenting lactic acidosis and/or from progressive neurological and neuromuscular degeneration. Treatment of PDCD remains a serious, unmet, challenge. Dichloroacetate (DCA) represents the firs ...

Abstract Tuberculosis (TB), caused by Mycobacterium tuberculosis (Mtb), is the biggest killer among infectious diseases. TB is also responsible for a quarter of all deaths associated with Antimicrobial Resistance (AMR). It is projected that by 2050, 75 million people, or one person every 12 seconds, will die due to AMR associated with TB. Long treatment times and increasing resistance to TB drugs ...

ABSTRACTUp to 30% of low birth weight preterm infants manifest some form of periventricular white matter injury (PWMI) making it the most common form of brain injury affecting premature infants. PWMI is associated with significant morbidity, as affected individuals may have profound intellectual impairment and cerebral palsy. Highlighting the magnitude of PWMI, each year more than 400,000 infants ...

PROJECT SUMMARY Thiopurines such as mercaptopurine are S-substituted antimetabolites used as functional analogs to natural nucleobase precursors. They are highly effective against hematologic malignancies (leukemia and lymphoma) and autoimmune diseases (inflammatory bowel diseases [IBD]), with an estimated patient population andgt;350,000 in the US. However, the cytotoxicity of thiopurines depends ...