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Vinceti, M.* ; Urbano, T.* ; Filippini, T.* ; Bedin, R.* ; Simonini, C.* ; Sorarù, G.* ; Trojsi, F.* ; Michalke, B. ; Mandrioli, J.*

Changes in cerebrospinal fluid concentrations of selenium species induced by tofersen administration in subjects with amyotrophic lateral sclerosis Carrying SOD1 gene mutations.

Biol. Trace Elem. Res., DOI: 10.1007/s12011-024-04311-4 (2024)
Verlagsversion DOI PMC
Open Access Hybrid
Creative Commons Lizenzvertrag
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease affecting the brain and spinal cord motor neurons. On 25 April 2023, the drug tofersen, an antisense oligonucleotide, received the US Food and Drug Administration approval for treating ALS in adults carrying mutations of the SOD1 gene. We aimed at assessing whether cerebrospinal fluid concentrations of selenium, an element of both toxicological and nutritional interest possibly involved in disease etiology and progression, are modified by tofersen administration. We determined concentrations of selenium species by anion exchange chromatography hyphenated to inductively coupled plasma-dynamic reaction cell-mass spectrometry and overall selenium by using inductively coupled plasma sector-field mass spectrometry, at baseline and 6 months after active tofersen treatment in ten Italian ALS patients carrying the SOD1 gene mutation. Concentrations of total selenium and many selenium species substantially increased after the intervention, particularly of inorganic (tetravalent and hexavalent) selenium and of the organic species selenomethionine and a compound co-eluting with the selenocystine standard. Overall, these findings suggest that tofersen treatment markedly alters selenium status and probably the redox status within the central nervous system, possibly due to a direct effect on neurons and/or the blood-brain barrier. Further studies are required to investigate the biological and clinical relevance of these findings and how they might relate to the pharmacological effects of the drug and to disease progression.
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Publikationstyp Artikel: Journalartikel
Dokumenttyp Wissenschaftlicher Artikel
Schlagwörter Amyotrophic Lateral Sclerosis ; Sod1 ; Selenium ; Selenium Compounds ; Tofersen; Antisense Oligonucleotide Tofersen; Human Serum; Selenoproteins; Neurotoxicity; Speciation; Toxicity; Therapy; Humans; Health; Trial
Sprache englisch
Veröffentlichungsjahr 2024
HGF-Berichtsjahr 2024
ISSN (print) / ISBN 0163-4984
e-ISSN 1559-0720
Zeitschrift Biological Trace Element Research
Verlag Springer
Verlagsort Campus, 4 Crinan St, London, N1 9xw, England
Begutachtungsstatus Peer reviewed
Institut(e) Research Unit BioGeoChemistry and Analytics (BGC)
POF Topic(s) 30202 - Environmental Health
Forschungsfeld(er) Environmental Sciences
PSP-Element(e) G-504800-002
Förderungen Ministero dell'Istruzione, dell'Universit e della Ricerca
DOI 10.1007/s12011-024-04311-4
WOS ID 001270435700001
Scopus ID 85198848014
PubMed ID 39017978
Erfassungsdatum 2024-07-18
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