ALS Drug Discovery Studies

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that progressively and irreversibly affects motor movement due to the death of motor neurons in the brain and spinal cord. ALS is characterized by stiff muscles, muscle twitching, and gradual muscle atrophy resulting in difficulty speaking, swallowing, and eventually breathing. About 90% of ALS cases have no known etiology, while the remaining 10% have a genetic cause. Mutations in over 20 genes have been associated with familial ALS, with mutations in 4 genes accounting for the majority of familial cases – SOD1, FUS, TDP-43, and C9orf72. Charles River has validated the transgenic SOD1-G93A model and is in the process of validating the C9orf72 model.

Transgenic SOD1 (superoxide dismutase 1) mice express a G93A mutant form of human SOD1. SOD1 mice (TgN-SOD1-G93A-1Gur) exhibit a phenotype similar to amyotrophic lateral sclerosis (ALS) in humans.

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Validated Endpoints

Basic Monitoring

  • Body weight
  • Kaplan-Meier survival plot
  • Disease onset and clinical scoring

Behavioral Testing

  • Gait, balance, and posture changes using fine motor kinematic analysis
  • Rotarod tests
  • Grip strength
  • Open field tests
  • Rearing frequency

Imaging

  • MRI to detect neurodegeneration in the brain stem and spinal cord
  • MRS to assess metabolic changes in the brain stem
  • FDG-PET imaging to assess metabolic changes

Testing/Validation Data

Click below to see a sample data set.