Miplyffa (arimoclomol)

To treat Niemann-Pick disease type C

FDA Approval:

Research Synopsis

  • Miplyffa (arimoclomol) is a co-inducer of heat shock proteins (HSPs) being studied primarily for its potential therapeutic effects in amyotrophic lateral sclerosis (ALS).
  • Recent research indicates that arimoclomol enhances HSP gene expression, which helps cells cope with stress and may protect motor neurons in neurodegenerative diseases.
  • Studies in mouse models of ALS have shown that arimoclomol significantly slows disease progression, improves muscle function, and enhances motoneuron survival.
  • Clinical trials have reported that arimoclomol is well-tolerated at doses up to 300 mg/day, supporting its safety for potential human treatment.
  • Research also suggests that arimoclomol may delay muscle denervation and reduce harmful protein aggregation in ALS models, indicating its multi-faceted protective roles.
  • Laboratory studies demonstrate that treatment with arimoclomol improves cognitive and motor functions in stress conditions like hypoxia, suggesting broader neuroprotective effects.
  • A systematic review identified arimoclomol as a promising candidate amidst 113 compounds evaluated for ALS treatment, with ongoing Phase II trials focused on its efficacy for both familial and sporadic forms of the disease.
  • The emerging consensus in research highlights arimoclomol’s dual role in neuroprotection and enhancement of cellular resilience in neurodegenerative conditions, pointing to its potential beyond ALS to other related disorders.

Related articles

Research articles about Miplyffa (arimoclomol)

Miplyffa (arimoclomol)

Nontoxic heat shock protein coinducer BRX-220 protects against acute pancreatitis in rats.

London, UK

2 hours ago

1 Received

  • Nontoxic heat shock protein (HSP) inducers like BRX-220 show potential for therapeutic applications by enhancing the body's protective mechanisms against stress.
  • The study investigated the effects of BRX-220 on acute pancreatitis induced by cholecystokinin (CCK) in male Wistar rats, comparing treated and control groups.
  • Results indicated that BRX-220 significantly increased pancreatic HSP levels, improved protein content, and enhanced enzyme activities, suggesting it may have protective effects against pancreatitis-related damage.

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Effect of BRX-220 against peripheral neuropathy and insulin resistance in diabetic rat models.

London, UK

2 hours ago

1 Received

  • Bimoclomol (BML) is a drug developed to manage symptoms of diabetic neuropathy and retinopathy, while BRX-220 is a new compound targeting diabetic complications and insulin resistance.
  • In a study on rats with induced type 1 diabetes, BRX-220 significantly improved nerve function, with motor and sensory nerve conduction velocities increasing by up to 91.3% and 93.2% respectively after a month of treatment.
  • Additionally, BRX-220 effectively reduced severe insulin resistance in both the STZ-induced diabetic rats and Zucker diabetic fatty rats, indicating its potential benefits for managing diabetes-related issues.

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Upregulation of heat shock proteins rescues motoneurones from axotomy-induced cell death in neonatal rats.

London, UK

2 hours ago

1 Received

  • Heat shock proteins (hsps) help cells survive stress, and this study investigated the effects of BRX-220, which increases hsp expression, on injured motoneurones in rat pups.
  • After crushing the sciatic nerve at birth, rats treated daily with BRX-220 showed a significant increase in motoneurone survival (39% vs. 21%) compared to those treated with saline.
  • Additionally, BRX-220 treatment enhanced the expression of hsps (hsp70 and hsp90) in glia and neurones, leading to improved muscle function and fewer motoneurone losses over 10 weeks.

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The effect of treatment with BRX-220, a co-inducer of heat shock proteins, on sensory fibers of the rat following peripheral nerve injury.

London, UK

2 hours ago

1 Received

  • The study investigates the impact of BRX-220, a co-inducer of heat shock proteins, on sensory changes after peripheral nerve injury in rats.
  • After treating injured rats with BRX-220, researchers observed improved preservation of crucial sensory markers (CGRP and IB4) compared to those that only received a vehicle treatment.
  • Although BRX-220 did not fully prevent pain responses, long-term treatment was linked to reduced pain-related behaviors, indicating it might aid recovery and enhance sensory function post-injury.

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Treatment with arimoclomol, a coinducer of heat shock proteins, delays disease progression in ALS mice.

London, UK

2 hours ago

1 Received

  • Amyotrophic lateral sclerosis (ALS) is a terminal neurodegenerative disease that causes the death of motoneurons, leading to progressive paralysis and usually death within 1-5 years of diagnosis.
  • Approximately 20% of familial ALS cases are linked to mutations in the SOD1 gene, and studies in transgenic mice with a specific SOD1 mutation show symptoms resembling those of human ALS.
  • Treatment with arimoclomol, which boosts heat shock proteins, has been found to significantly slow disease progression in SOD1(G93A) mice, improve muscle function, enhance motoneuron survival, and increase lifespan by 22%, suggesting a potential new therapy for ALS and similar diseases.

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Putting the heat on ALS.

London, UK

2 hours ago

1 Received

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Neuroprotective agents for clinical trials in ALS: a systematic assessment.

London, UK

2 hours ago

1 Received

  • Riluzole is the only FDA-approved treatment for ALS, but its survival impact is limited, prompting a search for additional neuroprotective agents.
  • Researchers identified 113 compounds with potential for ALS treatment, narrowing the list to 24 based on efficacy and safety, with 20 selected for further development.
  • Some compounds, like talampanel and tamoxifen, are already in early trials, while others need more testing before entering large-scale human trials, indicating a promising avenue for future ALS treatments.

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Heat shock proteins and protection of the nervous system.

London, UK

2 hours ago

1 Received

  • * Overexpression of Hsp70 has been shown to reduce brain damage from ischemic events, while substances like Arimoclomol and Celastrol enhance Hsp expression, offering potential treatments for conditions like ALS.
  • * Stress tolerance in neurons benefits from not just their own Hsps but also from those produced by nearby glial cells, indicating that introducing external Hsps could effectively support neuronal health during injury.

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Arimoclomol at dosages up to 300 mg/day is well tolerated and safe in amyotrophic lateral sclerosis.

London, UK

2 hours ago

1 Received

  • The study involved 84 ALS participants who received arimoclomol in different doses or a placebo, focusing on assessing safety, tolerability, and how the drug is processed in the body.
  • Results showed that arimoclomol was well-tolerated at doses up to 300 mg/day, effectively crosses the blood-brain barrier, and supports a dosing schedule of three times a day, paving the way for future efficacy studies.

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Late stage treatment with arimoclomol delays disease progression and prevents protein aggregation in the SOD1 mouse model of ALS.

London, UK

2 hours ago

1 Received

  • - Amyotrophic lateral sclerosis (ALS) is a serious neurodegenerative disease that leads to muscle paralysis and usually death within 1-5 years after diagnosis, with unclear causes but possible links to proteasome dysfunction and heat shock proteins.
  • - A previous study showed that arimoclomol, a co-inducer of the heat shock response, can delay progression and extend lifespan in an ALS mouse model when treatment starts early in the disease.
  • - This study found that starting arimoclomol treatment during the early (75 days) or late (90 days) symptomatic stages improved muscle function, with early treatment also extending lifespan, possibly by enhancing the heat shock response and reducing harmful protein aggregates in the spinal

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Treatment for familial amyotrophic lateral sclerosis/motor neuron disease.

London, UK

2 hours ago

1 Received

  • Amyotrophic lateral sclerosis (ALS) is a rare neurodegenerative disease with a small percentage of patients reporting a family history. The cause of sporadic ALS is still unknown, whereas mutations in the superoxide dismutase-1 gene account for about 20% of familial cases.
  • This study aimed to evaluate whether there is a difference in treatment response between patients with sporadic and familial ALS by reviewing randomized controlled trials (RCTs) published until May 2006.
  • The authors successfully collected data from four significant trials involving 822 sporadic and 41 familial ALS patients, but found no statistical evidence to suggest a difference in treatment response between the two groups, despite some data remaining uncollected

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Activation of the heat shock response in a primary cellular model of motoneuron neurodegeneration-evidence for neuroprotective and neurotoxic effects.

London, UK

2 hours ago

1 Received

  • Pharmacological agents that induce heat shock proteins (hsps) can help motoneurons survive cell death, but their effects may vary.
  • In a study, arimoclomol promoted motoneuron survival against stress while celastrol not only failed to protect them but also caused cell death.
  • The findings highlight that activating the heat shock response doesn't guarantee neuroprotection, as seen with celastrol triggering both hsp70 expression and apoptosis.

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Arimoclomol: a potential therapy under development for ALS.

London, UK

2 hours ago

1 Received

  • Arimoclomol is being studied as a potential treatment for amyotrophic lateral sclerosis (ALS) due to its ability to enhance heat shock protein expression, which helps cells respond to stress.
  • Research indicates that arimoclomol can improve survival and muscle function in mouse models of ALS, and initial human safety studies support its use at doses up to 300 mg/day.
  • Ongoing studies are exploring its effects in patients with familial ALS linked to specific genetic mutations, highlighting its promise as a therapeutic option for both sporadic and familial forms of the disease.

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Arimoclomol, a coinducer of heat shock proteins for the potential treatment of amyotrophic lateral sclerosis.

London, UK

2 hours ago

1 Received

  • - Recent studies focus on neurodegenerative diseases, highlighting Arimoclomol (BRX-220) as a potential oral treatment for ALS, a severe and currently untreatable motor neuron disease.
  • - ALS is primarily sporadic, with a small percentage linked to genetic factors, particularly mutations in the SOD1 gene, which contribute to about 20% of familial cases.
  • - Arimoclomol works by enhancing the body's protective heat shock response to help motor neurons survive, showing promising results in both lab studies and early clinical trials, suggesting potential benefits for ALS and related neurodegenerative conditions.

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A heat-shock protein co-inducer treatment improves behavioral performance in rats exposed to hypoxia.

London, UK

2 hours ago

1 Received

  • The study explored how arimoclomol, a heat-shock protein co-inducer, affects responses to low oxygen levels in a rat model that simulates high altitude.
  • Rats treated with arimoclomol showed improved cognitive and motor functions during hypoxia, as assessed by various behavioral tests.
  • Treatment with arimoclomol also decreased stress markers in the brain, indicating it may help protect brain tissue during low oxygen exposure and enhance overall performance.

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Treatment with a coinducer of the heat shock response delays muscle denervation in the SOD1-G93A mouse model of amyotrophic lateral sclerosis.

London, UK

2 hours ago

1 Received

  • A longitudinal study examined changes in the neuromuscular junction of SOD1-G93A mice, focusing on histological and biochemical markers of muscle function and the effects of the heat shock protein inducer, arimoclomol.
  • Findings revealed that denervation and nerve sprouting began as symptoms appeared, with a decline in cholinergic enzyme activities and alterations in muscle fiber characteristics, particularly in fast muscles.
  • Treatment with arimoclomol not only delayed these harmful changes but also improved muscle innervation and enzyme activities, along with increased Hsp70 expression, suggesting a potential therapeutic strategy for ALS.

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Plasma neurofilament heavy chain levels correlate to markers of late stage disease progression and treatment response in SOD1(G93A) mice that model ALS.

London, UK

2 hours ago

1 Received

  • ALS is a serious neurodegenerative disease characterized by the gradual loss of motor neurons, leading to death usually within 3-5 years after symptoms appear, and currently lacks reliable diagnostic tools or biomarkers.
  • This study on SOD1(G93A) mice found that plasma levels of phosphorylated neurofilament heavy chain protein (NfH) increased as the disease progressed, correlating with declines in muscle strength and motor neuron survival.
  • The findings suggest that measuring plasma NfH levels could serve as a useful biomarker for evaluating disease progression and response to treatments in ALS research.

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Co-induction of the heat shock response ameliorates disease progression in a mouse model of human spinal and bulbar muscular atrophy: implications for therapy.

London, UK

2 hours ago

1 Received

  • Spinal and bulbar muscular atrophy (Kennedy's disease) is a hereditary neurodegenerative disorder characterized by the loss of spinal and bulbar motor neurons, leading to weakness in facial, bulbar, and limb muscles due to a genetic mutation in the androgen receptor gene.
  • Research using a mouse model of the disease shows that those affected develop motor deficits, including reduced muscle force and degeneration of motor neurons.
  • Treatment with the drug arimoclomol improved muscle function, rescued motor units, and enhanced motor neuron survival in mice, suggesting it could be a promising therapeutic option for managing spinal and bulbar muscular atrophy.

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The role of heat shock proteins in Amyotrophic Lateral Sclerosis: The therapeutic potential of Arimoclomol.

London, UK

2 hours ago

1 Received

  • - Arimoclomol is a unique compound that enhances the expression of heat shock proteins, providing neuroprotective effects under stress conditions, particularly in neurodegenerative diseases like Amyotrophic Lateral Sclerosis (ALS).
  • - In studies with SOD1 mutant mice, Arimoclomol has shown potential benefits by rescuing motor neurons, enhancing neuromuscular function, and potentially extending lifespan.
  • - Currently, Arimoclomol is being evaluated in Phase II clinical trials for ALS, as it targets multiple pathological mechanisms, including protein aggregation and oxidative stress, making it a promising candidate for developing effective therapies against ALS and other neurodegenerative diseases.

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