2014
DOI: 10.1517/13543784.2014.933807
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Experimental trials in amyotrophic lateral sclerosis: a review of recently completed, ongoing and planned trials using existing and novel drugs

Abstract: The challenge remains for developing effective targeted therapeutic interventions for ALS. However, with improved recognition of the complex interplay of several factors that may contribute to ALS pathogenesis, in addition to improved patient selection criteria, outcome measures and biomarkers for drug development, advancements may be made in the future.

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Cited by 36 publications
(30 citation statements)
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“…Drugs targeting neuroinflammation such as celecoxib, ceftriaxone, thalidomide, and minocycline were reported to enhance survival in transgenic mice, yet none were effective in human ALS trials [142][143][144]. Some critics have suggested suboptimal animal study design such as inadequate powering or administration of drug to mice prior to onset of signs of disease, while in patients with ALS administration can only begin well after the appearance of disease symptoms and signs [145,146].…”
Section: Als Immunomodulation Treatment Strategiesmentioning
confidence: 99%
“…Drugs targeting neuroinflammation such as celecoxib, ceftriaxone, thalidomide, and minocycline were reported to enhance survival in transgenic mice, yet none were effective in human ALS trials [142][143][144]. Some critics have suggested suboptimal animal study design such as inadequate powering or administration of drug to mice prior to onset of signs of disease, while in patients with ALS administration can only begin well after the appearance of disease symptoms and signs [145,146].…”
Section: Als Immunomodulation Treatment Strategiesmentioning
confidence: 99%
“…Injections of AAV9 -GFP-SOD1-shRNA. Presymptomatic rats at P70 were injected with adeno-associated virus serotype 9 containing the green fluorescent reporter protein (AAV9 -GFP) detailed by Foust et al (2009) or with AAV9 -GFP-SOD1-shRNA (AAV9 -SOD1-shRNA; SOD1-shRNA: -CATGGATTCCATGTTCATGA-) detailed by . Virus particles at 5 ϫ 10 12 were injected in 2 l of viral solution per site using a glass micropipette attached to a 10 l Hamilton syringe, controlled by a Hamilton microinjection pump.…”
Section: Ctip2mentioning
confidence: 99%
“…For neonatal mouse injections, P1 SOD1 G93A pups (n ϭ 5) were used. A total volume of 50 l containing 5 ϫ 10 11 (3.6 ϫ 10 14 vg/kg) DNase-resistant viral particles of AAV9 -scrambled-SOD1-shRNA (Virapur) was injected through the temporal vein as described previously (Foust et al, 2009). After a correct injection was verified by blanching of the vein, pups were returned to their cage.…”
Section: Ctip2mentioning
confidence: 99%
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“…No effective treatments exist for ALS; over 50 well-designed clinical trials have been conducted in ALS over the last 25 years and with the exception of the riluzole trial, all have failed [150]. However riluzole only modestly extends survival time.…”
Section: General Summarymentioning
confidence: 99%