Current State and Future Directions in the Therapy of ALS

卷:12

Review

关键词: AMYOTROPHIC-LATERAL-SCLEROSIS;    QUALITY-OF-LIFE;    ANTISENSE OLIGONUCLEOTIDE TOFERSEN;    BRAIN-COMPUTER INTERFACE;    NONINVASIVE VENTILATION;    NEUROPROTECTIVE AGENT;    NUCLEOCYTOPLASMIC TRANSPORT;    ADENOASSOCIATED VIRUS;    PROGNOSTIC BIOMARKER;    DISEASE PROGRESSION;   

DOI  :  10.3390/cells12111523

来源: SCIE

【 摘 要 】

Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disorder affecting upper and lower motor neurons, with death resulting mainly from respiratory failure three to five years after symptom onset. As the exact underlying causative pathological pathway is unclear and potentially diverse, finding a suitable therapy to slow down or possibly stop disease progression remains challenging. Varying by country Riluzole, Edaravone, and Sodium phenylbutyrate/Taurursodiol are the only drugs currently approved in ALS treatment for their moderate effect on disease progression. Even though curative treatment options, able to prevent or stop disease progression, are still unknown, recent breakthroughs, especially in the field of targeting genetic disease forms, raise hope for improved care and therapy for ALS patients. In this review, we aim to summarize the current state of ALS therapy, including medication as well as supportive therapy, and discuss the ongoing developments and prospects in the field. Furthermore, we highlight the rationale behind the intense research on biomarkers and genetic testing as a feasible way to improve the classification of ALS patients towards personalized medicine.