A Sex-dependent Response to dl-PHPB in an Amyotrophic Lateral Sclerosis Disease Mouse Model

Abstract

Amyotrophic Lateral Sclerosis is a fatal neurodegenerative disease affecting upper and lower motor neurons resulting in their death. There are currently only two FDA approved drugs for its treatment, and both offer mild effects on the disease. Hence the need for a continued search for a better and more effective treatment. dl-Potassium 2-(1-Hydroxypentyl)-benzoate (dl-PHPB) is a novel drug for the treatment of cerebral ischemia and has shown potential neuroprotective, anti-oxidative and anti-apoptotic benefits in several neurodegenerative diseases. In this study, the effects of dl-PHPB treatment are investigated in SOD1G93A mice, an animal model for ALS disease. Our results show that dl-PHPB has neuroprotective effects that are able to delay disease onset and progression, and also improve locomotor function particularly in female animals. Further investigation will be needed to confirm dl-PHPB as a drug candidate for the treatment of ALS and its effects on different gender groups.