Page last updated: 2024-12-08

arimoclomol

Description Research Excerpts Clinical Trials Roles Classes Pathways Study Profile Bioassays Related Drugs Related Conditions Protein Interactions Research Growth Market Indicators

Description

arimoclomol: a coinducer of heat shock proteins, delays disease progression in ALS mice [Medical Subject Headings (MeSH), National Library of Medicine, extracted Dec-2023]

Cross-References

ID SourceID
PubMed CID208924
CHEMBL ID4760607
SCHEMBL ID2324741
MeSH IDM0466770

Synonyms (26)

Synonym
arimoclomol
289893-25-0
n-[(2r)-2-hydroxy-3-piperidin-1-ylpropoxy]-1-oxidopyridin-1-ium-3-carboximidoyl chloride
brx 345
arimoclomolum [inn-latin]
eut3557rt5 ,
brx-345
brx220
n-((2r)-2-hydroxy-3-(1-piperidyl)propoxy)pyridine-3-carboximidoyl chloride, 1-oxide
who 8300
arimoclomolum
arimoclomol [usan:inn]
unii-eut3557rt5
289893-23-8
arimoclomol [inn]
arimoclomol [mi]
arimoclomol [who-dd]
3-pyridinecarboximidoyl chloride, n-((2r)-2-hydroxy-3-(1-piperidinyl)propoxy), 1-oxide
arimoclomol [usan]
n-[(2r)-2-hydroxy-3-(1-piperidyl)propoxy]pyridine-3-carboximidoyl chloride, 1-oxide
DB05025
SCHEMBL2324741
Q4790694
CHEMBL4760607
AC-36932
(3z)-n-(2-hydroxy-3-piperidin-1-ylpropoxy)-1-oxidopyridin-1-ium-3-carboximidoyl chloride

Research Excerpts

Overview

Arimoclomol is a co-activator that prolongs the binding of activated HSF1 to heat shock elements (HSEs) in the promoter regions of inducible Hsp genes. It was safe and well-tolerated in a pilot study of inclusion body myositis.

ExcerptReferenceRelevance
"Arimoclomol is an oral co-inducer of the cellular heat shock response that was safe and well-tolerated in a pilot study of inclusion body myositis, reduced key pathological markers of inclusion body myositis in two in-vitro models representing degenerative and inflammatory components of this disease, and improved disease pathology and muscle function in mutant valosin-containing protein mice."( Safety and efficacy of arimoclomol for inclusion body myositis: a multicentre, randomised, double-blind, placebo-controlled trial.
Amato, AA; Barohn, RJ; Blaettler, T; Bonefeld, K; Carstensen, TD; Ciafaloni, E; Dimachkie, MM; Freimer, M; Gibson, SB; Hanna, MG; Heim, AJ; Herbelin, L; Jones, SM; Jørgensen, AN; Levine, TD; Lloyd, TE; Machado, PM; McDermott, MP; Mozaffar, T; Phonekeo, K; Rosholm, A; Shaibani, AI; Sundgreen, C; Wicklund, M, 2023
)
1.94
"Arimoclomol is a hydroxylamine derivative, a group of compounds which have unique properties as co-inducers of heat shock protein expression, but only under conditions of cellular stress. "( The role of heat shock proteins in Amyotrophic Lateral Sclerosis: The therapeutic potential of Arimoclomol.
Greensmith, L; Kalmar, B; Lu, CH, 2014
)
2.06
"Arimoclomol is a co-activator that prolongs the binding of activated HSF1 to heat shock elements (HSEs) in the promoter regions of inducible Hsp genes."( Induction of heat shock proteins in differentiated human neuronal cells following co-application of celastrol and arimoclomol.
Brown, IR; Deane, CA, 2016
)
1.37
"Arimoclomol is an investigational drug for amyotrophic lateral sclerosis (ALS) that amplifies heat shock protein gene expression during cell stress. "( Arimoclomol at dosages up to 300 mg/day is well tolerated and safe in amyotrophic lateral sclerosis.
Barber, JR; Brown, RH; Cudkowicz, ME; Grasso, D; Schoenfeld, D; Shefner, JM; Shui, A; Simpson, E; Wieland, S; Yu, H; Zhang, H, 2008
)
3.23

Effects

ExcerptReferenceRelevance
"Arimoclomol has been found to be neuroprotective in a number of neurodegenerative disease models, including Amyotrophic Lateral Sclerosis (ALS), and in mutant Superoxide Dismutase 1 (SOD1) mice that model ALS, Arimoclomol rescues motor neurons, improves neuromuscular function and extends lifespan."( The role of heat shock proteins in Amyotrophic Lateral Sclerosis: The therapeutic potential of Arimoclomol.
Greensmith, L; Kalmar, B; Lu, CH, 2014
)
1.34

Treatment

Treatment with arimoclomol from early (75 days) or late (90 days) symptomatic stages significantly improved muscle function. Treatment delayed the appearance of these changes, increased innervation, cholinergic enzyme activities and endplate size.

ExcerptReferenceRelevance
"Arimoclomol-treated SOD1(G93A) mice show marked improvement in hind limb muscle function and motoneuron survival in the later stages of the disease, resulting in a 22% increase in lifespan."( Treatment with arimoclomol, a coinducer of heat shock proteins, delays disease progression in ALS mice.
Burnstock, G; Dick, JR; Greensmith, L; Kalmar, B; Kieran, D; Riddoch-Contreras, J, 2004
)
1.4
"Treatment with arimoclomol from early (75 days) or late (90 days) symptomatic stages significantly improved muscle function."( Late stage treatment with arimoclomol delays disease progression and prevents protein aggregation in the SOD1 mouse model of ALS.
Cheetham, ME; Gray, A; Greensmith, L; Kalmar, B; Margulis, B; Novoselov, S, 2008
)
0.99
"Treatment with arimoclomol was reported to improve survival and muscle function in a mouse model of motor neuron disease."( Arimoclomol: a potential therapy under development for ALS.
Barber, J; Cudkowicz, M; Lanka, V; Wieland, S, 2009
)
2.14
"Treatment with arimoclomol delayed the appearance of these changes, increased innervation, cholinergic enzyme activities and endplate size and reversed muscle fibre transformation."( Treatment with a coinducer of the heat shock response delays muscle denervation in the SOD1-G93A mouse model of amyotrophic lateral sclerosis.
Edet-Amana, E; Greensmith, L; Kalmar, B, 2012
)
0.72

Toxicity

ExcerptReferenceRelevance
" Adverse events occurred in 30/34 patients (88."( Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatment.
Andreasen, AK; Blaettler, T; Da Riol, RM; Day, S; Del Toro, M; Deodato, F; Gautschi, M; Geist, MA; Grunewald, S; Grønborg, S; Hansen, T; Harmatz, P; Havnsøe Torp Petersen, N; Héron, B; Í Dali, C; Ingemann, L; Kirkegaard, T; Maier, EM; Mengel, E; Patterson, MC; Roubertie, A; Santra, S; Tylki-Szymanska, A, 2021
)
0.93
" Arimoclomol is an oral co-inducer of the cellular heat shock response that was safe and well-tolerated in a pilot study of inclusion body myositis, reduced key pathological markers of inclusion body myositis in two in-vitro models representing degenerative and inflammatory components of this disease, and improved disease pathology and muscle function in mutant valosin-containing protein mice."( Safety and efficacy of arimoclomol for inclusion body myositis: a multicentre, randomised, double-blind, placebo-controlled trial.
Amato, AA; Barohn, RJ; Blaettler, T; Bonefeld, K; Carstensen, TD; Ciafaloni, E; Dimachkie, MM; Freimer, M; Gibson, SB; Hanna, MG; Heim, AJ; Herbelin, L; Jones, SM; Jørgensen, AN; Levine, TD; Lloyd, TE; Machado, PM; McDermott, MP; Mozaffar, T; Phonekeo, K; Rosholm, A; Shaibani, AI; Sundgreen, C; Wicklund, M, 2023
)
2.13
" 126 completed the trial on treatment (56 [77%] and 70 [90%], respectively) and the most common reason for treatment discontinuation was adverse events."( Safety and efficacy of arimoclomol for inclusion body myositis: a multicentre, randomised, double-blind, placebo-controlled trial.
Amato, AA; Barohn, RJ; Blaettler, T; Bonefeld, K; Carstensen, TD; Ciafaloni, E; Dimachkie, MM; Freimer, M; Gibson, SB; Hanna, MG; Heim, AJ; Herbelin, L; Jones, SM; Jørgensen, AN; Levine, TD; Lloyd, TE; Machado, PM; McDermott, MP; Mozaffar, T; Phonekeo, K; Rosholm, A; Shaibani, AI; Sundgreen, C; Wicklund, M, 2023
)
1.22

Dosage Studied

This study provides Class II evidence that arimoclomol is safe and well-tolerated at a dosage of 200 mg tid for up to 12 months.

ExcerptRelevanceReference
" Serum pharmacokinetic profiles support dosing of three times per day."( Arimoclomol at dosages up to 300 mg/day is well tolerated and safe in amyotrophic lateral sclerosis.
Barber, JR; Brown, RH; Cudkowicz, ME; Grasso, D; Schoenfeld, D; Shefner, JM; Shui, A; Simpson, E; Wieland, S; Yu, H; Zhang, H, 2008
)
1.79
"This study provides Class II evidence that arimoclomol is safe and well-tolerated at a dosage of 200 mg tid for up to 12 months."( Randomized, double-blind, placebo-controlled trial of arimoclomol in rapidly progressive
Andersen, PM; Atassi, N; Benatar, M; Cudkowicz, M; David, W; Schoenfeld, D; Wuu, J, 2018
)
0.99
"This study provides Class II evidence that arimoclomol is safe and well-tolerated at a dosage of 200 mg tid for up to 12 months."( Randomized, double-blind, placebo-controlled trial of arimoclomol in rapidly progressive
Andersen, PM; Atassi, N; Benatar, M; Cudkowicz, M; David, W; Schoenfeld, D; Wuu, J, 2018
)
0.99
[information is derived through text-mining from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Research

Studies (34)

TimeframeStudies, This Drug (%)All Drugs %
pre-19900 (0.00)18.7374
1990's0 (0.00)18.2507
2000's11 (32.35)29.6817
2010's18 (52.94)24.3611
2020's5 (14.71)2.80
[information is prepared from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Market Indicators

Research Demand Index: 46.49

According to the monthly volume, diversity, and competition of internet searches for this compound, as well the volume and growth of publications, there is estimated to be strong demand-to-supply ratio for research on this compound.

MetricThis Compound (vs All)
Research Demand Index46.49 (24.57)
Research Supply Index3.66 (2.92)
Research Growth Index4.69 (4.65)
Search Engine Demand Index68.78 (26.88)
Search Engine Supply Index2.00 (0.95)

This Compound (46.49)

All Compounds (24.57)

Study Types

Publication TypeThis drug (%)All Drugs (%)
Trials4 (11.76%)5.53%
Reviews6 (17.65%)6.00%
Case Studies0 (0.00%)4.05%
Observational0 (0.00%)0.25%
Other24 (70.59%)84.16%
[information is prepared from research data collected from National Library of Medicine (NLM), extracted Dec-2023]