Clinical trials for neuroprotection in ALS.
CNS Neurol Disord Drug Targets
; 9(3): 305-13, 2010 Jul.
Article
en En
| MEDLINE
| ID: mdl-20406180
ABSTRACT
Owing to uncertainty on the pathogenic mechanisms underlying motor neuron degeneration in amyotrophic lateral sclerosis (ALS) riluzole remains the only available therapy, with only marginal effects on disease survival. Here we review some of the recent advances in the search for disease-modifying drugs for ALS based on their putative neuroprotective effetcs. A number of more or less established agents have recently been investigated also in ALS for their potential role in neuroprotection and relying on antiglutamatergic, antioxidant or antiapoptotic strategies. Among them Talampanel, beta-lactam antibiotics, Coenzyme Q10, and minocycline have been investigated. Progress has also been made in exploiting growth factors for the treatment of ALS, partly due to advances in developing effective delivery systems to the central nervous system. A number of new therapies have also been identified, including a novel class of compounds, such as heat-shock protein co-inducers, which upregulate cell stress responses, and agents promoting autophagy and mitochondriogenesis, such as lithium and rapamycin. More recently, alterations of mRNA processing were described as a pathogenic mechanism in genetically defined forms of ALS, as those related to TDP-43 and FUS-TLS gene mutations. This knowledge is expected to improve our understanding of the pathogenetic mechanism in ALS and developing more effective therapies.
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Bases de datos:
MEDLINE
Asunto principal:
Neurofarmacología
/
Ensayos Clínicos como Asunto
/
Fármacos Neuroprotectores
/
Esclerosis Amiotrófica Lateral
Tipo de estudio:
Prognostic_studies
Límite:
Animals
/
Humans
Idioma:
En
Revista:
CNS Neurol Disord Drug Targets
Asunto de la revista:
NEUROLOGIA
/
TERAPIA POR MEDICAMENTOS
Año:
2010
Tipo del documento:
Article
País de afiliación:
Italia