rhIGF-I for the Treatment of Neuromuscular Disorders
Type I insulin-like growth factor-I (IGF-I) receptors are widely distributed throughout the nervous system, suggesting an important role for IGF-I in the growth, development, and maintenance of the central nervous system (CNS). This, in part, has been the stimulus for extensive studies on the actions of IGF-I on muscle and in the CNS. In vitro, IGF-I induces differentiation of muscle cells, prevents myoblast apoptosis and promotes survival of several neuronal types. Furthermore, IGF-I has been shown to stimulate In vitro the proliferation of neuronal progenitor cells and to increase the survival of both neurons and oligodendrocytes. In vivo, IGF-I has profound effects on neuronal and skeletal muscle development including increases in motor neuron sprouting, rate of neuronal recovery after injury, and muscle mass . This pre-clinical evidence suggests that IGF-I may have beneficial effects in pathological conditions characterized by motor neuron loss, denervation, and skeletal muscle atrophy. Recently, the efficacy of recombinant human IGF-I (rhIGF-I) has been studied in patients with amyotrophic lateral sclerosis, post-polio syndrome and myotonic dystrophy. In addition, indirect data from one clinical study suggested that IGF-I may mediate the clinical benefit provided by prednisone in the treatment of Duchenne muscular dystrophy.
KeywordsAmyotrophic Lateral Sclerosis Motor Neuron Duchenne Muscular Dystrophy Amyotrophic Lateral Sclerosis Patient Duchenne Muscular Dystrophy
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