TITLE:
Comparative Effect of Different Treatments with a Bacterial Polyketide in a Mouse Model of ALS (Amyotrophic Lateral Sclerosis)
AUTHORS:
Enrique Wulff
KEYWORDS:
Amyotrophic Lateral Sclerosis (ALS), Mithramycin, SOD1G93A Transgenic Mouse Model
JOURNAL NAME:
Open Journal of Genetics,
Vol.16 No.3,
September
16,
2026
ABSTRACT: Amyotrophic lateral sclerosis (ALS) is characterized by motor deficits that affect voluntary movements. This research aims to assess the impact of mithramycin (MTM) therapy on these deficits. Mice were subjected to behavior tests to determine whether chronic MTM therapy, initiated by the third month of life, leads to measurable improvements in motor skills. Muscle viability was evaluated using rotarod and footprint tests. The overall quality of life for the animals was assessed by monitoring their total body weight. To examine potential increases in life expectancy associated with MTM therapy in this ALS mouse model, a Kaplan-Meier survival analysis was conducted. The results indicate that treatment beginning in the first month of life with a dosage of 30 µg/kg/day yields maximum values for parameters related to muscle atrophy reduction, weight gain, and increased life expectancy. Additionally, the findings suggest an enhancement in survival when a higher dosage of 150 µg/kg/day is administered starting at three months of age. These functional improvements are consistent with a proposed neuroprotective mechanism involving the NO/ROCK/Sp1/S100A10/TASK-1 pathway, though direct molecular evidence for this pathway was not obtained in this study.