Dexpramipexole: Difference between revisions

Dexpramipexole
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Latest revision as of 11:01, 15 February 2025

An investigational drug for neurodegenerative diseases

Dexpramipexole is a small molecule drug that has been investigated for its potential use in treating neurodegenerative diseases, particularly amyotrophic lateral sclerosis (ALS). It is a chirally pure form of the drug pramipexole, which is used in the treatment of Parkinson's disease.

Mechanism of Action[edit]

Dexpramipexole is believed to work by enhancing the function of mitochondria, the energy-producing organelles in cells. This enhancement may help protect neurons from degeneration, which is a hallmark of diseases like ALS. The exact mechanism by which dexpramipexole exerts its effects is not fully understood, but it is thought to involve the modulation of mitochondrial function and reduction of oxidative stress.

Clinical Development[edit]

Dexpramipexole has undergone several clinical trials to assess its efficacy and safety in patients with ALS. Initial studies showed promise, with some patients experiencing a slower progression of the disease. However, a large Phase III clinical trial did not demonstrate a statistically significant benefit in slowing the progression of ALS compared to placebo.

Potential Applications[edit]

While the primary focus of dexpramipexole research has been on ALS, there is interest in exploring its potential use in other neurodegenerative conditions, such as Huntington's disease and Alzheimer's disease. The drug's ability to modulate mitochondrial function makes it a candidate for diseases characterized by mitochondrial dysfunction.

Challenges and Future Directions[edit]

The failure of dexpramipexole to show significant efficacy in late-stage clinical trials for ALS has led to a reevaluation of its potential. Researchers are investigating whether different dosing regimens, combination therapies, or targeting specific patient subgroups might yield better results. Additionally, ongoing research into the drug's mechanism of action may uncover new therapeutic targets or applications.

Related pages[edit]

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-'''Dexpramipexole''' ([[IUPAC]] name: (6R)-4,5,6,7-tetrahydro-N6-propyl-2,6-benzothiazole-diamine) is a [[pharmacology|pharmacological]] compound that has been investigated for its potential therapeutic effects. Initially researched for its neuroprotective properties, dexpramipexole has been studied in the context of several [[neurodegenerative diseases]], including [[Amyotrophic Lateral Sclerosis (ALS)]] and [[Parkinson's disease]]. Despite its initial promise, the development of dexpramipexole for these indications has faced challenges.
+{{Short description|An investigational drug for neurodegenerative diseases}}
+{{Drugbox
+| verifiedfields = changed
+| verifiedrevid = 477002123
+| image = Dexpramipexole.png
+| image_size = 250px
+| image_alt = Chemical structure of Dexpramipexole
+}}
-==Pharmacodynamics and Mechanism of Action==
+'''Dexpramipexole''' is a small molecule drug that has been investigated for its potential use in treating [[neurodegenerative diseases]], particularly [[amyotrophic lateral sclerosis]] (ALS). It is a [[chirality|chirally]] pure form of the drug pramipexole, which is used in the treatment of [[Parkinson's disease]].
-Dexpramipexole is believed to exert its effects through multiple mechanisms, although its exact mode of action remains not fully elucidated. It has been suggested that the compound may have antioxidant properties, reducing oxidative stress in neurons, which is a common pathological feature in many neurodegenerative diseases. Additionally, dexpramipexole might modulate mitochondrial function, potentially improving energy production and reducing cell death in affected neural cells.
-==Clinical Trials and Research==
+==Mechanism of Action==
-Clinical trials of dexpramipexole have primarily focused on its potential as a treatment for ALS. In early-phase studies, dexpramipexole showed some promise in slowing the progression of the disease, leading to larger, phase III clinical trials. However, these later trials did not demonstrate a significant benefit in terms of disease progression or survival, leading to a discontinuation of its development for ALS.
+Dexpramipexole is believed to work by enhancing the function of [[mitochondria]], the energy-producing organelles in cells. This enhancement may help protect neurons from degeneration, which is a hallmark of diseases like ALS. The exact mechanism by which dexpramipexole exerts its effects is not fully understood, but it is thought to involve the modulation of mitochondrial function and reduction of oxidative stress.
-Research into the use of dexpramipexole for other conditions, such as Parkinson's disease, has also been conducted, but like its investigation in ALS, has not led to a marketable therapy. Despite these setbacks, the compound's underlying mechanisms continue to be of interest for potential therapeutic applications in neurodegenerative diseases and other conditions characterized by oxidative stress and mitochondrial dysfunction.
+==Clinical Development==
+Dexpramipexole has undergone several clinical trials to assess its efficacy and safety in patients with ALS. Initial studies showed promise, with some patients experiencing a slower progression of the disease. However, a large Phase III clinical trial did not demonstrate a statistically significant benefit in slowing the progression of ALS compared to placebo.
-==Safety and Tolerability==
+==Potential Applications==
-In clinical trials, dexpramipexole has generally been well-tolerated, with a safety profile that did not significantly differ from placebo. The most common adverse effects reported were mild and included nausea, headache, and dizziness. The lack of significant adverse effects has been a positive aspect of the compound's profile, although its efficacy remains a challenge.
+While the primary focus of dexpramipexole research has been on ALS, there is interest in exploring its potential use in other neurodegenerative conditions, such as [[Huntington's disease]] and [[Alzheimer's disease]]. The drug's ability to modulate mitochondrial function makes it a candidate for diseases characterized by mitochondrial dysfunction.
-==Future Directions==
+==Challenges and Future Directions==
-The development of dexpramipexole for neurodegenerative diseases has been a complex journey, with initial optimism tempered by challenges in demonstrating clear clinical benefits. However, the ongoing interest in its mechanism of action suggests that further research may uncover new applications for this compound or inform the development of related therapeutic agents. The field of neurodegeneration remains in need of effective treatments, and compounds like dexpramipexole contribute to the broader understanding necessary to achieve this goal.
+The failure of dexpramipexole to show significant efficacy in late-stage clinical trials for ALS has led to a reevaluation of its potential. Researchers are investigating whether different dosing regimens, combination therapies, or targeting specific patient subgroups might yield better results. Additionally, ongoing research into the drug's mechanism of action may uncover new therapeutic targets or applications.
-[[Category:Pharmacology]]
+==Related pages==
-[[Category:Neurodegenerative diseases]]
+* [[Neurodegenerative disease]]
-{{pharmacology-stub}}
+* [[Amyotrophic lateral sclerosis]]
+* [[Mitochondria]]
+* [[Oxidative stress]]
+[[Category:Experimental drugs]]
+[[Category:Neurodegenerative disorders]]