TY - JOUR
T1 - A perspective on therapies for amyotrophic lateral sclerosis
T2 - can disease progression be curbed?
AU - Xu, Xiaojiao
AU - Shen, Dingding
AU - Gao, Yining
AU - Zhou, Qinming
AU - Ni, You
AU - Meng, Huanyu
AU - Shi, Hongqin
AU - Le, Weidong
AU - Chen, Shengdi
AU - Chen, Sheng
N1 - Funding Information:
This review work was supported by funding from the National Nature Research Foundation (81671241, 81771521); Shanghai Shuguang Plan Project (18SG15) and Shanghai outstanding young scholars Project.
Publisher Copyright:
© 2021, The Author(s).
PY - 2021/8/10
Y1 - 2021/8/10
N2 - Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease involving both upper and lower motor neurons, leading to paralysis and eventually death. Symptomatic treatments such as inhibition of salivation, alleviation of muscle cramps, and relief of spasticity and pain still play an important role in enhancing the quality of life. To date, riluzole and edaravone are the only two drugs approved by the Food and Drug Administration for the treatment of ALS in a few countries. While there is adequate consensus on the modest efficacy of riluzole, there are still open questions concerning the efficacy of edaravone in slowing the disease progression. Therefore, identification of novel therapeutic strategies is urgently needed. Impaired autophagic process plays a critical role in ALS pathogenesis. In this review, we focus on therapies modulating autophagy in the context of ALS. Furthermore, stem cell therapies, gene therapies, and newly-developed biomaterials have great potentials in alleviating neurodegeneration, which might halt the disease progression. In this review, we will summarize the current and prospective therapies for ALS.
AB - Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease involving both upper and lower motor neurons, leading to paralysis and eventually death. Symptomatic treatments such as inhibition of salivation, alleviation of muscle cramps, and relief of spasticity and pain still play an important role in enhancing the quality of life. To date, riluzole and edaravone are the only two drugs approved by the Food and Drug Administration for the treatment of ALS in a few countries. While there is adequate consensus on the modest efficacy of riluzole, there are still open questions concerning the efficacy of edaravone in slowing the disease progression. Therefore, identification of novel therapeutic strategies is urgently needed. Impaired autophagic process plays a critical role in ALS pathogenesis. In this review, we focus on therapies modulating autophagy in the context of ALS. Furthermore, stem cell therapies, gene therapies, and newly-developed biomaterials have great potentials in alleviating neurodegeneration, which might halt the disease progression. In this review, we will summarize the current and prospective therapies for ALS.
KW - Amyotrophic lateral sclerosis
KW - Autophagy
KW - Gene editing
KW - Motor neurons
KW - Stem cells
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U2 - 10.1186/s40035-021-00250-5
DO - 10.1186/s40035-021-00250-5
M3 - Review article
C2 - 34372914
AN - SCOPUS:85112336295
SN - 2047-9158
VL - 10
SP - 29
JO - Translational Neurodegeneration
JF - Translational Neurodegeneration
IS - 1
M1 - 29
ER -