Amyotrophe Lateralsklerose (ALS) – Diagnose, Verlauf und neue Behandlungsoptionen

Meyer, Thomas

doi:10.1055/a-1562-7882

Cited by 17 publications

(8 citation statements)

References 16 publications

(11 reference statements)

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“…ALS is a progressive and fatal neurodegenerative disease characterized by upper motor neurons (UMN), including cranial motor nuclei in the pons and medulla and frontal cortex, and lower motor neurons (LMN), such as motor neurons in the anterior horn of the spinal cord damage, resulting in progressive muscle weakness and atrophy, with fasciculations, hyperreflexia, and spasticity [ 121 , 122 ]. The pathology is characterized by degeneration and death of motor neurons like pyramidal cells and Baez cells with intracellular inclusion bodies and proliferation of glial cells [ 122 , 123 ]. Previous studies also showed that mutations in C9orf72, SOD1, TARDBP/TARDBP/TAR DNA binding protein (TDP-43) and fused in sarcoma are the most frequent genetic forms of ALS [ 124 – 128 ].…”

Section: Pathological Roles Of Phase Separation In the Central Nervou...mentioning

confidence: 99%

Physiological and pathological effects of phase separation in the central nervous system

Wang,

Zhu,

Tian

et al. 2024

J Mol Med

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Phase separation, also known as biomolecule condensate, participates in physiological processes such as transcriptional regulation, signal transduction, gene expression, and DNA damage repair by creating a membrane-free compartment. Phase separation is primarily caused by the interaction of multivalent non-covalent bonds between proteins and/or nucleic acids. The strength of molecular multivalent interaction can be modified by component concentration, the potential of hydrogen, posttranslational modification, and other factors. Notably, phase separation occurs frequently in the cytoplasm of mitochondria, the nucleus, and synapses. Phase separation in vivo is dynamic or stable in the normal physiological state, while abnormal phase separation will lead to the formation of biomolecule condensates, speeding up the disease progression. To provide candidate suggestions for the clinical treatment of nervous system diseases, this review, based on existing studies, carefully and systematically represents the physiological roles of phase separation in the central nervous system and its pathological mechanism in neurodegenerative diseases.

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Section: Pathological Roles Of Phase Separation In the Central Nervou...mentioning

confidence: 99%

Physiological and pathological effects of phase separation in the central nervous system

Wang,

Zhu,

Tian

et al. 2024

J Mol Med

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“…Amyotrophic lateral sclerosis (ALS) is a fatal disease characterized by persistent degeneration of motor neurons, which is characterized by paralysis and death by affecting motor neurons in the motor cortex, brainstem and spinal cord ( Mazzini et al, 2016 ; Meyer, 2021 ). At present, some studies have proposed several pathological mechanisms of ALS-induced motoneuron death, including glutamate-induced excitotoxicity, abnormal cytoskeleton, protein aggregation, oxidative stress, mitochondrial dysfunction and extracellular SOD1 toxicity ( Peters et al, 2015 ; Shafiq et al, 2021 ).…”

Section: Nscs and Neurological Diseasesmentioning

confidence: 99%

Therapeutic role of neural stem cells in neurological diseases

Yang,

Liu,

Liu

et al. 2024

Front. Bioeng. Biotechnol.

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The failure of endogenous repair is the main feature of neurological diseases that cannot recover the damaged tissue and the resulting dysfunction. Currently, the range of treatment options for neurological diseases is limited, and the approved drugs are used to treat neurological diseases, but the therapeutic effect is still not ideal. In recent years, different studies have revealed that neural stem cells (NSCs) have made exciting achievements in the treatment of neurological diseases. NSCs have the potential of self-renewal and differentiation, which shows great foreground as the replacement therapy of endogenous cells in neurological diseases, which broadens a new way of cell therapy. The biological functions of NSCs in the repair of nerve injury include neuroprotection, promoting axonal regeneration and remyelination, secretion of neurotrophic factors, immune regulation, and improve the inflammatory microenvironment of nerve injury. All these reveal that NSCs play an important role in improving the progression of neurological diseases. Therefore, it is of great significance to better understand the functional role of NSCs in the treatment of neurological diseases. In view of this, we comprehensively discussed the application and value of NSCs in neurological diseases as well as the existing problems and challenges.

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“…Relyvrio, an FDA-approved treatment for amyotrophic lateral sclerosis (ALS), is a blend of sodium phenylbutyrate and taurursodiol [ 82 , 83 ]. ALS is so deadly that most patients have a life expectancy of only 3 to 5 years after onset of symptoms, and they typically die from respiratory failure, a progressive atrophy of the muscles used for respiration [ 84 – 87 ]. Relyvrio received early FDA approval due to the highly progressive nature and serious threat of ALS.…”

Section: Central Nervous System Drugsmentioning

confidence: 99%

Synthesis and clinical application of new drugs approved by FDA in 2022

Zhang,

Wang,

Sun

et al. 2023

Mol Biomed

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The pharmaceutical industry had a glorious year in 2022, with a total of 37 new drugs including 20 new chemical entities (NCEs) and 17 new biological entities (NBEs) approved by the Food and Drug Administration (FDA). These drugs are mainly concentrated in oncology, central nervous system, antiinfection, hematology, cardiomyopathy, dermatology, digestive system, ophthalmology, MRI enhancer and other therapeutic fields. Of the 37 drugs, 25 (68%) were approved through an expedited review pathway, and 19 (51%) were approved to treat rare diseases. These newly listed drugs have unique structures and new mechanisms of action, which can serve as lead compounds for designing new drugs with similar biological targets and enhancing therapeutic efficacy. This review aims to outline the clinical applications and synthetic methods of 19 NCEs newly approved by the FDA in 2022, but excludes contrast agent (Xenon Xe-129). We believe that an in-depth understanding of the synthetic methods of drug molecules will provide innovative and practical inspiration for the development of new, more effective, and practical synthetic techniques. According to the therapeutic areas of these 2022 FDA-approved drugs, we have classified these 19 NCEs into seven categories and will introduce them in the order of their approval for marketing.

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Amyotrophe Lateralsklerose (ALS) – Diagnose, Verlauf und neue Behandlungsoptionen

Cited by 17 publications

References 16 publications

Physiological and pathological effects of phase separation in the central nervous system

Physiological and pathological effects of phase separation in the central nervous system

Therapeutic role of neural stem cells in neurological diseases

Synthesis and clinical application of new drugs approved by FDA in 2022

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