Receptor for AGE (RAGE) Signal Transduction in Amyotrophic Lateral Sclerosis: In Vivo Imaging and Novel Therapeutic Approaches

Abstract

We hypothesized that the receptor for advanced glycation end products (RAGE) is implicated in the pathogenesis of ALS, at least in part through microglial perturbation. Our findings: (1) RAGE-positive Cd11b-positive cells are increased in the ventral horn of male and female SOD1G93A mouse lumbar spinal cord and male wild-type mice displayed higher proportions of RAGE-positive Cd11b cells than female wild-type mice. (2) In male SOD1G93A mice, microglia deletion of Ager in the ALS mouse background prolongs survival and slows loss of body weight and motor function. We identified an independent negative effect of the Cre recombinase mouse line in the ALS background (Cx3cr1ERT2 cre). At this time, we are finalizing all of the studies in the genetic model including new studies on limited numbers of male and female SOD1G93A mice and controls sacrificed pre-humane endpoint at day 120. (3) PET imaging using tracers to mark inflammation suggests higher spinal cord inflammation at day 100 and day 130 of life in SOD1G93A mice vs. controls. (4) Orally available (medicated chow) small molecule antagonists of RAGE/DIAPH1 are being tested in SOD1G93A mice. To date, the high dose treatment shows promise to reduce rate of weight loss and motor decline in combined male and female mice. Additional doses are underway at this time. Collectively, our data suggest deleterious roles for RAGE in ALS and indicate that further testing of this concept is warranted in this disease.

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Document Details

Document Type
Technical Report
Publication Date
Jul 01, 2020
Accession Number
AD1114506

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  • Ann M. Schmidt

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  • New York University

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