Abstract
Spinal and bulbar muscular atrophy (SBMA) is an adult-onset neuromuscular disease caused by the expansion of a trinucleotide CAG repeat in the androgen receptor (AR) gene. The ligand-dependent nuclear accumulation of pathogenic AR protein is central to the pathogenesis. Leuprorelin, a luteinizing hormone-releasing hormone (LHRH) analogue that suppresses testosterone production from testis, inhibits toxic accumulation of pathogenic AR, thereby mitigating histopathological and behavioral impairments in a mouse model of SBMA. A randomized placebo-controlled multi-centric phase 3 clinical trial showed an effect of the drug on motor functions, particularly there was a significant improvement of swallowing function. In addition, a long-term follow-up study compared with natural history group of patients revealed that the progression of motor dysfunction and occurrence of pneumoniae due to swallowing dysfunction were very much diminished with leuprorelin treatment.
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