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Background: Intrathecally (IT) delivered antisense oligonucleotides (ASOs) are promising therapies that can reduce tau pathology in Alzheimer's Disease (AD). However, current plasma and CSF sampling methods to estimate brain tissue exposure of ASOs are inherently limited, hampering ASO clinical developmental plans. We developed the PET tracer [F]BIO-687, which binds ASO conjugates (ASO-Tz) in vivo, allowing us to image ASO distribution in a living brain using "pretargeted" imaging.
View Article and Find Full Text PDFBackground: Intrathecally (IT) delivered antisense oligonucleotides (ASOs) are promising therapies that can reduce tau pathology in Alzheimer's Disease (AD). However, current plasma and CSF sampling methods to estimate brain tissue exposure of ASOs are inherently limited, hampering ASO clinical developmental plans. We developed the PET tracer [18F]BIO-687, which binds ASO conjugates (ASO-Tz) in vivo, allowing us to image ASO distribution in a living brain using "pretargeted" imaging.
View Article and Find Full Text PDFCerebrospinal Fluid and Serum Neuron-Specific Enolase in Niemann-Pick Disease Type C1.
Am J Med Genet A
December 2024
Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), National Institutes of Health, Bethesda, Maryland, USA.
Niemann-Pick disease, type C1 (NPC1) is an ultra rare, autosomal recessive disorder characterized by impaired intracellular cholesterol trafficking. This study assessed neuron-specific enolase (NSE) as a biomarker for disease status and treatment response in individuals with NPC1. We also evaluated the concordance between serum and cerebrospinal fluid (CSF) NSE measurements.
View Article and Find Full Text PDFTreatment of intractable paradoxical herniation by invasive mechanical ventilation with increased positive end-expiratory pressure: a case report.
Brain Inj
November 2024
Department of Neurosurgery, Beijing Shunyi District Hospital, Capital Medical University, Beijing, China.
Article Synopsis
- Paradoxical herniation (PH) is a dangerous complication that can occur after a decompressive craniectomy, and treating it involves strategies to increase intracranial pressure.
- A 59-year-old patient with a severe brain injury experienced intractable PH due to cerebrospinal fluid leaks from numerous lumbar punctures, leading to ineffective conventional treatments.
- The patient's condition improved dramatically when invasive mechanical ventilation with increased positive end-expiratory pressure (PEEP) was implemented, successfully elevating intracranial pressure and leading to recovery.
Intrathecal anti-PD-1 treatment in metastatic melanoma patients with leptomeningeal disease (LMD): real-world data and evidence.
J Neurooncol
December 2024
State Key Laboratory of Oncology in South China, Collaborative Innovation Center for Cancer Medicine, Sun Yat-sen University Cancer Center, Guangzhou, PR China.
Purpose: Leptomeningeal disease (LMD) is a severe complication of melanoma with a very poor prognosis. Despite improved treatment strategies and prolonged survival, the incidence of LMD has increased over the past decade. This real-world study aims to evaluate the efficacy and safety of intrathecal anti-PD-1 treatment in melanoma patients with LMD.
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