Esclerosis Lateral Amiotrófica: Enfermedad, tratamiento actual y nuevas líneas de investigación. Masitinib
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2017
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Abstract
La Esclerosis Lateral Amiotrófica (ELA), es una enfermedad degenerativa de las neuronas motoras que afecta tanto a hombres como a mujeres, sin importar la raza, de edades comprendidas entre los 40 y 70 años. Actualmente carece de una terapia efectiva para su tratamiento. El único fármaco autorizado hasta el momento es el riluzol (Rilutek®) del que se han demostrado unos escasos e insuficientes beneficios que alargan la vida del paciente de dos a tres meses. Es por ello una labor importante fomentar la investigación y los ensayos clínicos en pacientes, con fármacos en los que se haya demostrado la eficacia y seguridad. La ELA es una enfermedad neurodegenerativa en la que, entre otros muchos factores, la interacción entre la microglía, mastocitos y astrocitos puede causar la destrucción de las neuronas motoras. El fármaco en estudio, Masitinib, afecta tanto a los mastocitos como a la microglía y se demostró que es capaz de reducir la muerte y la atrofia de las motoneuronas. El objetivo de esta revisión bibliográfica es proporcionar una visión general a cerca de los tratamientos actuales para la ELA y nuevas terapias con fármacos como Masitinib.
Amyotrophic Lateral Sclerosis (ALS) is a degenerative disease of motor neurons that affects both men and women, regardless of race, between the ages of 40 and 70. It currently lacks effective therapy for its treatment. The only currently authorized drug is riluzole (Rilutek®) which has shown poor and insufficient elongation of the patient's life, only for two or three months. It is therefore important to promote research and clinical trials in patients with drugs that have shown efficacy and safety. ALS is a neurodegenerative disease in which, among many other factors, the interaction between microglia, mast cells and astrocytes can cause the destruction of motor neurons. The study drug, Masitinib, affects both mast cells and microglía, and has shown to be able to reduce motor neuron death and atrophy. The objective of this literature review is to provide an overview of current treatments for ALS and new therapies with drugs such as Masitinib.
Amyotrophic Lateral Sclerosis (ALS) is a degenerative disease of motor neurons that affects both men and women, regardless of race, between the ages of 40 and 70. It currently lacks effective therapy for its treatment. The only currently authorized drug is riluzole (Rilutek®) which has shown poor and insufficient elongation of the patient's life, only for two or three months. It is therefore important to promote research and clinical trials in patients with drugs that have shown efficacy and safety. ALS is a neurodegenerative disease in which, among many other factors, the interaction between microglia, mast cells and astrocytes can cause the destruction of motor neurons. The study drug, Masitinib, affects both mast cells and microglía, and has shown to be able to reduce motor neuron death and atrophy. The objective of this literature review is to provide an overview of current treatments for ALS and new therapies with drugs such as Masitinib.