Breaking barriers with tofersen: Enhancing therapeutic opportunities in amyotrophic lateral sclerosis

被引:13
作者
Saini, Aniket [1 ]
Chawla, Pooja A. [1 ,2 ]
机构
[1] ISF Coll Pharm, Dept Pharmaceut Anal, Moga, Punjab, India
[2] ISF Coll Pharm, Dept Pharmaceut Chem & Anal, Moga 142001, Punjab, India
关键词
ALS; adverse drug reactions; amyotrophic lateral sclerosis; clinical trial; FDA-approved drugs; tofersen; ANTISENSE OLIGONUCLEOTIDE TOFERSEN; ALS-LINKED SOD1; SUPEROXIDE-DISMUTASE; MUTANT SOD1; ER STRESS; MUTATIONS; SURVIVAL; DISEASE; TRIAL; AGGREGATION;
D O I
10.1111/ene.16140
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
Background and purposeAmyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that primarily affects adults, characterized by muscle weakness resulting from the specific death of motor neurons in the spinal cord and brain. The pathogenesis of ALS is associated with the accumulation of mutant superoxide dismutase 1 (SOD1) proteins and neurofilaments in motor neurons, highlighting the critical need for disease-modifying treatments. Current therapies, such as riluzole and edaravone, provide only symptomatic relief. Recently, tofersen gained approval from the US FDA under the brand name Qalsody as the first and only gene therapy for ALS, addressing a significant pathological aspect of the disease.MethodsWe carried out a literature survey using PubMed, Scopus, National Institutes of Health, and Biogen for articles published in the English language concerned with "amyotrophic lateral sclerosis", pathophysiology, current treatment, treatment under clinical trial, and the newly approved drug "tofersen" and its detailed summary.ResultsA comprehensive review of the literature on the pathophysiology, available treatment, and newly approved drug for this condition revealed convincing evidence that we are now able to better monitor and treat ALS.ConclusionsAlthough treatment of ALS is difficult, the newly approved drug tofersen has emerged as a potential therapy to slow down the progression of ALS by targeting SOD1 mRNA, representing a significant advancement in the treatment of ALS.
引用
收藏
页数:11
相关论文
共 50 条
[1]   Profiling tofersen as a treatment of superoxide dismutase 1 amyotrophic lateral sclerosis [J].
Santos, Miguel Oliveira ;
de Carvalho, Mamede .
EXPERT REVIEW OF NEUROTHERAPEUTICS, 2024, 24 (06) :549-553
[2]   Tofersen for SOD1 amyotrophic lateral sclerosis: a systematic review and meta-analysis [J].
Hamad, Abdullah Ashraf ;
Alkhawaldeh, Ibraheem M. ;
Nashwan, Abdulqadir J. ;
Meshref, Mostafa ;
Imam, Yahia .
NEUROLOGICAL SCIENCES, 2025, :1977-1985
[3]   Therapeutic Development in Amyotrophic Lateral Sclerosis [J].
Bucchia, Monica ;
Ramirez, Agnese ;
Parente, Valeria ;
Simone, Chiara ;
Nizzardo, Monica ;
Magri, Francesca ;
Dametti, Sara ;
Corti, Stefania .
CLINICAL THERAPEUTICS, 2015, 37 (03) :668-680
[4]   Serious Neurologic Adverse Events in Tofersen Clinical Trials for Amyotrophic Lateral Sclerosis [J].
Lovett, Alexandra ;
Chary, Sowmya ;
Babu, Suma ;
Bruneteau, Gaelle ;
Glass, Jonathan D. ;
Karlsborg, Merete ;
Ladha, Shafeeq ;
Mayl, Keith ;
Mcdermott, Christopher ;
Bucelli, Robert C. ;
Chio, Adriano ;
Ferguson, Toby A. ;
Cochrane, Thos ;
Fradette, Stephanie ;
Smirnakis, Karen ;
Inra, Jennifer ;
Malek, Sohail ;
Fanning, Laura .
MUSCLE & NERVE, 2025,
[5]   ASO drug Qalsody (tofersen) targets amyotrophic lateral sclerosis [J].
Jin, Jing ;
Zhong, Xiao-bo .
TRENDS IN PHARMACOLOGICAL SCIENCES, 2023, 44 (12) :1043-1044
[6]   Recent Progress of Antisense Oligonucleotide Therapy for Superoxide-Dismutase-1-Mutated Amyotrophic Lateral Sclerosis: Focus on Tofersen [J].
Moriyama, Hidenori ;
Yokota, Toshifumi .
GENES, 2024, 15 (10)
[7]   Multidisciplinary care and therapeutic advances in amyotrophic lateral sclerosis [J].
Matamala, Jose Manuel ;
Moreno-Roco, Javier ;
Acosta, Ignacio ;
Hughes, Ricardo ;
Lillo, Patricia ;
Casar, Juan Carlos ;
Earle, Nicholas .
REVISTA MEDICA DE CHILE, 2022, 150 (12) :1633-1646
[8]   Drug discovery and amyotrophic lateral sclerosis: Emerging challenges and therapeutic opportunities [J].
Soares, Pedro ;
Silva, Catia ;
Chavarria, Daniel ;
Silva, Filomena S. G. ;
Oliveira, Paulo J. ;
Borges, Fernanda .
AGEING RESEARCH REVIEWS, 2023, 83
[9]   Oxidative Stress as a Therapeutic Target in Amyotrophic Lateral Sclerosis: Opportunities and Limitations [J].
Park, Hee Ra ;
Yang, Eun Jin .
DIAGNOSTICS, 2021, 11 (09)
[10]   Is Dutasteride a Therapeutic Alternative for Amyotrophic Lateral Sclerosis? [J].
Proano, Belen ;
Casani-Cubel, Julia ;
Benlloch, Maria ;
Rodriguez-Mateos, Ana ;
Navarro-Illana, Esther ;
Maria Lajara-Romance, Jose ;
de la Rubia Orti, Jose Enrique .
BIOMEDICINES, 2022, 10 (09)