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Gold and BBB Opening in ALS (The GOALS Trial): A Phase 2a, Open-label, Single-arm Clinical Trial of Oral CNM-Au8 Coupled with Primary Motor Cortex Blood Brain Barrier Opening Using Transcranial MR-guided Focused Ultrasound in Amyotrophic Lateral Sclerosis

  • Grant Type

    Team grants

  • Area of research

    Neurodegeneration

  • Disease Area

    ALS

  • Competition

    ALS Canada - Brain Canada Discovery Grants

  • Province

    Ontario

  • Start Date

    2022

  • Total Grant Amount

    $125,000

  • Health Canada Contribution

    $62,500

Project Overview

Rationale: Mitochondrial dysfunction has been identified as a contributing factor for bioenergetic dysregulation and neurodegeneration in the motor cortex and spinal cord of patients with ALS. CNM-Au8 is an investigational compound designed to promote biocatalytic activity and is effective in improving mitochondrial function and survival in preclinical ALS models.  Oral…

Determination of hypothalamic neuropathology and metabolic defects in ALS

  • Grant Type

    Team grants

  • Area of research

    Neurodegeneration

  • Disease Area

    ALS

  • Competition

    ALS Canada - Brain Canada Discovery Grants

  • Province

    Ontario

  • Start Date

    2022

  • Total Grant Amount

    $125,000

  • Health Canada Contribution

    $62,500

Project Overview

Amyotrophic lateral sclerosis (ALS) is a motor neuron disease leading to paralysis and death within 2-5 years from disease onset. Other hallmark features include weight loss and metabolic dysfunction. Several lines of clinical evidence indicated that higher energy expenditure (hypermetabolism) is associated with weight loss in ALS patients. Recent clinical…

Novel biomarkers of SOD1 pathology in familial and sporadic ALS

  • Grant Type

    Team grants

  • Area of research

    Neurodegeneration

  • Disease Area

    ALS

  • Competition

    ALS Canada - Brain Canada Discovery Grants

  • Province

    Québec

  • Start Date

    2022

  • Total Grant Amount

    $125,000

  • Health Canada Contribution

    $62,500

Project Overview

Mutations in the SOD1 gene, leading to the misfolding and toxic aggregation of SOD1 protein, are the second leading cause of Familial Amyotrophic Lateral Sclerosis (FALS-SOD1) and emerging literature has detected misfolded SOD1 (mSOD1) in the cerebrospinal fluid (CSF) and post-mortem tissue of Sporadic ALS (SALS) patients. Based upon the…

Identification of Protein Interactions That Regulate FUS Mislocalization and Aggregation in ALS

  • Grant Type

    Team grants

  • Area of research

    Neurodegeneration

  • Disease Area

    ALS

  • Competition

    ALS Canada - Brain Canada Discovery Grants

  • Province

    Ontario

  • Start Date

    2022

  • Total Grant Amount

    $125,000

  • Health Canada Contribution

    $62,500

Project Overview

Amyotrophic lateral sclerosis (ALS) is a debilitating and terminal disease. A common hallmark of its familial and sporadic forms is the accumulation of insoluble protein aggregates in the cytoplasm of motor neurons. FUS RNA binding protein (FUS) is found in cytoplasmic protein aggregates of post-mortem spinal cords and brains in…

Imaging a New ALS Biomarker in the Eye

  • Grant Type

    Team grants

  • Area of research

    Neurodegeneration

  • Disease Area

    ALS

  • Competition

    ALS Canada - Brain Canada Discovery Grants

  • Province

    Ontario

  • Start Date

    2022

  • Total Grant Amount

    $125,000

  • Health Canada Contribution

    $62,500

Project Overview

Current biomarkers of ALS used by clinicians to help care for ALS patients have many drawbacks that may include cost, accessibility, and invasiveness of procedures. An imaging biomarker that is non-invasive, non-contact, and widely accessible would be a welcome addition to the current armamentarium of clinical tools used to diagnose,…