Tamoxifen Treatment in Patients With Motor Neuron Disease
Status:
Completed
Trial end date:
2019-09-18
Target enrollment:
Participant gender:
Summary
The aim of this study is to survey the effect of Tamoxifen in motor neuron disease (MND)
patients, amyotrophic lateral sclerosis (ALS) with regular riluzole usage. TDP-43 is related
to ALS. Increased the ubiquitinated or phosphorylated TDP-43 can cause animal model of ALS,
and TDP43 can be degraded either by proteasome or autophagy pathway system. Autophagy pathway
can be activated by mTOR inhibition, resulting in ameliorating TDP-43 accumulation and rescue
in motor function in animal model. Tamoxifen had shown ability of enhance both proteasome and
autophagy pathway, therefore the investigators assume that Tamoxifen probably can ameliorate
TDP-43 accumulation and inclusion body formation in ALS.