Status of ALS Treatment, Insights into Therapeutic Challenges and Dilemmas.
ALS trials platforms
gene targeted strategies
preclinical models
protein homeostasis inductors
riluzole
Journal
Journal of personalized medicine
ISSN: 2075-4426
Titre abrégé: J Pers Med
Pays: Switzerland
ID NLM: 101602269
Informations de publication
Date de publication:
28 Sep 2022
28 Sep 2022
Historique:
received:
17
08
2022
revised:
14
09
2022
accepted:
23
09
2022
entrez:
27
10
2022
pubmed:
28
10
2022
medline:
28
10
2022
Statut:
epublish
Résumé
Amyotrophic lateral sclerosis (ALS) is an extremely heterogeneous disease of motor neurons that eventually leads to death. Despite impressive advances in understanding the genetic, molecular, and pathological mechanisms of the disease, the only drug approved to date by both the FDA and EMA is riluzole, with a modest effect on survival. In this opinion view paper, we will discuss how to address some challenges for drug development in ALS at the conceptual, technological, and methodological levels. In addition, socioeconomic and ethical issues related to the legitimate need of patients to benefit quickly from new treatments will also be addressed. In conclusion, this brief review takes a more optimistic view, given the recent approval of two new drugs in some countries and the development of targeted gene therapies.
Identifiants
pubmed: 36294741
pii: jpm12101601
doi: 10.3390/jpm12101601
pmc: PMC9605458
pii:
doi:
Types de publication
Journal Article
Review
Langues
eng
Références
Neuromuscul Disord. 2012 Jan;22(1):73-5
pubmed: 21907581
Neurodegener Dis Manag. 2021 Dec;11(6):431-443
pubmed: 34816762
Science. 2006 Jun 2;312(5778):1389-92
pubmed: 16741123
Cells. 2022 Jan 25;11(3):
pubmed: 35159225
Nat Rev Mol Cell Biol. 2019 Aug;20(8):490-507
pubmed: 31147612
Neurology. 2016 Nov 22;87(21):2230-2234
pubmed: 27784774
Proc Natl Acad Sci U S A. 2009 Nov 3;106(44):18809-14
pubmed: 19833869
Neuron. 2011 Oct 20;72(2):257-68
pubmed: 21944779
Mult Scler. 1999 Aug;5(4):283-6
pubmed: 10467389
Proc Natl Acad Sci U S A. 2010 Sep 14;107(37):16320-4
pubmed: 20660762
Acta Neuropathol. 2016 Sep;132(3):391-411
pubmed: 26910103
Amyotroph Lateral Scler. 2008;9(1):4-15
pubmed: 18273714
Am J Med Genet. 1994 May 15;51(1):61-9
pubmed: 7913294
N Engl J Med. 2020 Sep 3;383(10):919-930
pubmed: 32877582
Muscle Nerve. 2021 Sep;64(3):309-320
pubmed: 34075589
Expert Rev Neurother. 2018 Nov;18(11):871-886
pubmed: 30273061
EBioMedicine. 2020 Sep;59:102844
pubmed: 32651161
J Neurol Neurosurg Psychiatry. 2003 Sep;74(9):1258-61
pubmed: 12933930
Exp Neurol. 2010 May;223(1):229-37
pubmed: 19682989
ACS Chem Neurosci. 2014 Sep 17;5(9):823-9
pubmed: 25001311
Biol Psychiatry. 2018 Feb 15;83(4):311-319
pubmed: 28967385
Mol Med. 2012 Jul 18;18:794-804
pubmed: 22481270
Neuron. 2013 Aug 7;79(3):416-38
pubmed: 23931993
Int J Mol Sci. 2021 Apr 27;22(9):
pubmed: 33925625
Brain. 2008 Jun;131(Pt 6):1540-50
pubmed: 18469020
J Neuromuscul Dis. 2021;8(1):25-38
pubmed: 33074186
Mol Neurobiol. 2022 Mar;59(3):1502-1527
pubmed: 34997540
Lancet Neurol. 2019 Feb;18(2):211-220
pubmed: 30663610
Ther Adv Neurol Disord. 2021 Jul 19;14:17562864211030365
pubmed: 34457038
Stem Cell Res Ther. 2014;5(2):32
pubmed: 25157751
Muscle Nerve. 2020 Aug;62(2):156-166
pubmed: 31899540
Cell. 2009 Mar 20;136(6):1001-4
pubmed: 19303844
J Med Chem. 2021 Mar 25;64(6):3026-3034
pubmed: 33703886
Ann Neurol. 2007 Jul;62(1):15-20
pubmed: 17455292
Science. 1994 Jun 17;264(5166):1772-5
pubmed: 8209258
Proc Natl Acad Sci U S A. 2016 Oct 18;113(42):E6496-E6505
pubmed: 27688759
J Neurochem. 2004 Feb;88(3):576-82
pubmed: 14720207
Curr Opin Neurol. 2012 Dec;25(6):689-700
pubmed: 23160421
Lancet. 2017 Nov 4;390(10107):2084-2098
pubmed: 28552366
JMIR Mhealth Uhealth. 2019 Dec 20;7(12):e13433
pubmed: 31859676
Ann Neurol. 1995 Jul;38(1):73-84
pubmed: 7611729
Front Mol Neurosci. 2017 Sep 08;10:251
pubmed: 28943839
FASEB J. 2003 Apr;17(6):725-7
pubmed: 12586733
Nature. 2021 May;593(7858):180
pubmed: 33963316
Sci Transl Med. 2019 Sep 25;11(511):
pubmed: 31554737
Ann Neurol. 2018 Jul;84(1):130-139
pubmed: 30014505
J Pers Med. 2020 Aug 25;10(3):
pubmed: 32854276
iScience. 2020 May 22;23(5):101087
pubmed: 32371370
Neuron. 2011 Oct 20;72(2):245-56
pubmed: 21944778
Neurobiol Dis. 2019 Feb;122:35-40
pubmed: 29981842
Proc Natl Acad Sci U S A. 1995 Jan 31;92(3):689-93
pubmed: 7846037
Muscle Nerve. 2012 Feb;45(2):200-8
pubmed: 22246875
Eur J Pharmacol. 2006 May 24;538(1-3):39-42
pubmed: 16674936
Biochemistry. 2003 Mar 18;42(10):3070-80
pubmed: 12627974
Stem Cells Int. 2019 Mar 10;2019:3675627
pubmed: 30956667
Brain. 2020 Dec 1;143(12):3526-3539
pubmed: 33427296
Stem Cells. 2012 Jun;30(6):1144-51
pubmed: 22415942
Curr Opin Neurol. 2014 Oct;27(5):515-23
pubmed: 25188012
J Neuroimmunol. 2009 May 29;210(1-2):73-9
pubmed: 19307024
Neurobiol Dis. 2008 Sep;31(3):395-405
pubmed: 18586098
Nature. 2015 Sep 3;525(7567):56-61
pubmed: 26308891
Amyotroph Lateral Scler Frontotemporal Degener. 2017 Feb;18(1-2):112-119
pubmed: 27819158
Ann Neurol. 2018 Sep;84(3):361-373
pubmed: 30048006
Curr Opin Neurol. 2021 Oct 1;34(5):765-772
pubmed: 34402459
Front Neurol. 2019 Mar 27;10:293
pubmed: 30972018
Muscle Nerve. 2022 Mar;65(3):291-302
pubmed: 34890069
Acta Neuropathol. 2013 Feb;125(2):273-88
pubmed: 22961620
Acta Neuropathol. 2019 Jun;137(6):859-877
pubmed: 30721407
Int J Mol Sci. 2021 Jul 30;22(15):
pubmed: 34360966
J Clin Invest. 2021 Feb 15;131(4):
pubmed: 33586684
Mol Omics. 2018 Feb 1;14(1):53-63
pubmed: 29570205
Trends Pharmacol Sci. 2020 Feb;41(2):99-109
pubmed: 31926602
Stem Cells Transl Med. 2019 Sep;8(9):887-897
pubmed: 31104357
Neurodegener Dis. 2012;9(1):38-52
pubmed: 21934272
Stem Cells. 2014 Dec;32(12):3163-72
pubmed: 25113670
Cell J. 2019 Jan;20(4):592-598
pubmed: 30124008
Neural Regen Res. 2022 Apr;17(4):875-880
pubmed: 34472488
Ann Neurol. 1997 Nov;42(5):783-93
pubmed: 9392578
N Engl J Med. 1994 Mar 3;330(9):585-91
pubmed: 8302340
EMBO J. 2016 May 17;35(10):1077-97
pubmed: 26951610
Exp Neurobiol. 2016 Oct;25(5):233-240
pubmed: 27790057
Neuroscience. 2013 Sep 17;248:333-43
pubmed: 23727509
Ann Neurol. 2015 Sep;78(3):426-38
pubmed: 26044557
Neuron. 2006 Oct 5;52(1):39-59
pubmed: 17015226
JAMA Neurol. 2016 Mar;73(3):337-44
pubmed: 26751635
Neurology. 2019 Jul 9;93(2):66-71
pubmed: 31171646
Ann Clin Transl Neurol. 2016 Sep 07;3(11):866-875
pubmed: 27844032
Amyotroph Lateral Scler Frontotemporal Degener. 2019;0(0):1-11
pubmed: 31081694
J Biol Chem. 2014 Apr 11;289(15):10769-10784
pubmed: 24515116
Science. 2014 Sep 5;345(6201):1192-1194
pubmed: 25103406
Nature. 2010 Aug 26;466(7310):1069-75
pubmed: 20740007
Neurobiol Dis. 2002 Aug;10(3):268-78
pubmed: 12270689
Am J Manag Care. 2018 Aug;24(15 Suppl):S320-S326
pubmed: 30207670
Cochrane Database Syst Rev. 2017 Oct 06;10:CD004427
pubmed: 28982219
JAMA Neurol. 2018 Jun 1;75(6):681-689
pubmed: 29507931
Neurotherapeutics. 2015 Apr;12(2):364-75
pubmed: 25567201
J Biol Chem. 2011 Dec 30;286(52):45083-92
pubmed: 22069317
Ann Neurol. 1993 Nov;34(5):661-9
pubmed: 8239560
Amyotroph Lateral Scler. 2011 Jul;12(4):264-71
pubmed: 21473708
J Neuromuscul Dis. 2021;8(6):869-884
pubmed: 34092651
Neuroimage Clin. 2021;30:102672
pubmed: 34016561
Cytotherapy. 2014 Aug;16(8):1059-72
pubmed: 24794182
Nature. 2016 Nov 10;539(7628):197-206
pubmed: 27830784
Lancet Neurol. 2018 May;17(5):423-433
pubmed: 29598923
N Engl J Med. 2020 Jul 9;383(2):109-119
pubmed: 32640130
Nat Rev Neurol. 2013 Sep;9(9):513-24
pubmed: 23917850
J Clin Invest. 2012 Sep;122(9):3063-87
pubmed: 22863620
Medicine (Baltimore). 2018 Jun;97(24):e11119
pubmed: 29901635
Acta Neuropathol. 2013 Dec;126(6):895-905
pubmed: 24166615
BMJ Open. 2019 May 30;9(5):e028486
pubmed: 31152038
Cytotherapy. 2007;9(5):414-26
pubmed: 17786603
Clin Neurophysiol. 2013 Mar;124(3):433-4
pubmed: 23098643
Lancet Neurol. 2017 Jul;16(7):505-512
pubmed: 28522181
Nat Neurosci. 2018 Feb;21(2):228-239
pubmed: 29311743
Mol Cells. 2018 Sep 30;41(9):818-829
pubmed: 30157547
EMBO J. 2000 Feb 1;19(3):453-62
pubmed: 10654943
Nat Genet. 2000 Feb;24(2):175-9
pubmed: 10655065
J Neural Transm (Vienna). 2020 Feb;127(2):189-203
pubmed: 31912279
J Cachexia Sarcopenia Muscle. 2022 Apr;13(2):1385-1402
pubmed: 35194965
Neural Regen Res. 2019 Feb;14(2):313-318
pubmed: 30531015
Nat Neurosci. 2008 Mar;11(3):251-3
pubmed: 18246065
Science. 2006 Oct 6;314(5796):130-3
pubmed: 17023659
Front Immunol. 2017 Aug 21;8:1005
pubmed: 28871262
Neuron. 2017 Oct 11;96(2):285-297
pubmed: 29024655
Sci Transl Med. 2012 Aug 1;4(145):145ra104
pubmed: 22855461