— Disease indication – neuroprotection in stroke/ischemia with potential for treating other neurodegenerative diseases
Drug format – neurosteroid
Drug class – drug repurposing (25-hydroxycholesterol)
Research stage and Preliminary data:
In vitro – The inventors demonstrated…
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About 70 million computed tomography (CT) scans are performed in the United State alone each year. Traditional helical scanners use a single point x-ray source that rotates around the patient, acquiring images in sequential slices, which results in high quality images. But these helical scanners a…
— Background
Niemann-Pick type C is a rare, progressive neurodegenerative disease characterized by accumulation of cholesterol and other lipids in the viscera and central nervous system. For cases caused by mutations in the NPC1 or NPC2 gene, egress of lipids from endosomes or lysosomes are impaired, resulting in clinical symptoms such as abnormal eye movements, hearing loss, cerebellar ataxia, difficulty swallowing, and cognitive impairment. However, patients with Neimann-Pick disease have highly variable clinical presentation and symptom progression, making diagnosis challenging.
Technology Summary
A panel of oxygenated forms of cholesterol (oxysterols) in plasma that can be used as a diagnostic test for Neimann-Pick type C1 disease. The mass spectrometry-based panel is able to discriminate between subjects with NPC1 and normal control subjects. The oxysterol biomarkers have clinical utility in newborn screening for early diagnosis of NP1C disease, as markers for disease severity, and as biomarkers to aid in evaluation of new therapies for NPC1.
Key Advantages
High sensitivity
Rapid diagnostic for screening infants
Patents
US 8,497,122 Issued
US 9,012,216 Issued
Publications
Porter et al., 2010 – Cholesterol oxidation products are sensitive and specific blood-based biomarkers for NPC1 disease.
— Technology Description
A team of researchers at Washington University in St. Louis have developed patented antibodies designed to identify MIOX (myo-inositol oxygenase), a renal-specific biomarker that can be detected immediately following acute kidney injury (AKI). AKI can be caused by surgery, s…
— Compounds with high affinity for aggregated alpha-synuclein, after radiolabeling with C-11 or F-18, 1- 125, 1-123 and Br-76, could be used as PET tracers to measure the level and distribution of aggregated alpha-synuclein in patients with Parkinson’s disease and related diseases such as DLB…
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