Preclinical and Translational Neuroimaging Study Reveals Mitochondrial Dysfunction in ALS

A landmark PET/CT investigation linking mitochondrial and receptor alterations to ALS disease progression.

This peer-reviewed study, published in Neurobiology of Disease, demonstrates how advanced preclinical and translational PET/CT imaging can uncover the molecular mechanisms driving neurodegeneration in Amyotrophic Lateral Sclerosis (ALS). Using radiotracers [¹⁸F]BCPP-EF, [¹¹C]SA4503, and [¹¹C]UCB-J, the research evaluated mitochondrial complex 1 (MC1), sigma-1 receptor (S1R), and synaptic vesicle 2A (SV2A) expression in ALS patients versus healthy controls. Results revealed significant loss of MC1 and S1R in the amygdala, hippocampus, and insular cortex, correlating with disease severity and functional decline. Conducted through collaboration between academic and industry experts—including Perceptive Discovery, a leading preclinical imaging CRO — this work establishes a framework for biomarker development and early-phase therapeutic evaluation in neurodegenerative disease.

Why Read this Publication:

• Translational Impact: Demonstrates how preclinical imaging data can bridge into human PET studies to elucidate ALS mechanisms.
• Biomarker Advancement: Identifies mitochondrial and receptor signatures measurable with translational imaging platforms.
• Preclinical Imaging Expertise: Highlights the application of cutting-edge radiochemistry and imaging workflows used in preclinical and clinical settings.
• Therapeutic Development Relevance: Supports the design of biomarker-informed studies for mitochondrial and receptor-targeted therapies.
• Collaborative Science: Integrates expertise from the University of Exeter, Biogen, AbbVie, and Perceptive Discovery, reinforcing the value of CRO-academic partnerships.

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