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Enzimoterapia - Passado, Presente e Futuro
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As enzimas desempenham um papel importante em várias áreas da terapêutica humana. Nalguns casos, particularmente nas doenças lisossomais, constituem o principal ou mesmo o único agente terapêutico disponível. No cancro, também têm mostrado o seu potencial e, para além da asparaginase, muitas enzimas têm vindo a ser alvo de estudo. Nestas duas áreas, em especial, as enzimas ganham destaque, e por isso são os principais focos deste trabalho. Recorrendo às mais atuais informações, procurou-se perceber qual o contributo das enzimas para a terapêutica; quer no passado, quer no presente, quer no futuro. Nas doenças lisossomais de sobrecarga, as enzimas permitem melhorar consideravelmente a qualidade de vida dos doentes. Estão aprovadas para as doenças de Gaucher, Fabry e Pompe e algumas mucopolissacaridoses mas espera-se que em breve estejam disponíveis para outras doenças raras. No cancro, mais especificamente na leucemia linfoblástica aguda, a L-asparaginase revelou-se muito efetiva, integrando há várias décadas os protocolos convencionais de quimioterapia. Outras enzimas como a arginina deiminase revelaram bons resultados iniciais e estão presentemente em estudos clínicos, podendo, em breve, revolucionar a terapêutica de outros tipos de tumores. Não há, pois, dúvidas de que as enzimas têm um grande contributo a dar para o futuro da terapêutica.
Enzymes play an important role in several areas of human therapy. In some cases, particularly in lysosomal storage diseases (LSD’s), they are the main or even the only therapeutic agent available. In cancer, they have also shown their potential, and besides asparaginase, many other enzymes are being object of study. In these two fields, in particular, enzymes gain prominence, and so they are the main focus of this work. Relying on the most up-to-date information available, this review tried to understand enzyme’s contribution to therapy, in the past, present and future. In LDSs, enzymes allow to improve considerably patient’s quality of life. There are approved for the treatment of Gaucher’s, Fabry’s and Pompe’s disease and some mucopolysaccharidosis but hopefully they will soon be available for other rare diseases. In cancer, more specifically in pediatric acute linfoblastic leukemia, L-asparaginase as revealed itself very effective, being part of conventional chemotherapy protocols for decades. Other enzymes such as arginine deiminase have shown good initial results and are currently in clinical studies; they may, soon, be revolutionizing the therapy of other sorts of tumors. There are, therefore, no doubts that enzymes have a major contribution to make to the future of therapy.
Enzymes play an important role in several areas of human therapy. In some cases, particularly in lysosomal storage diseases (LSD’s), they are the main or even the only therapeutic agent available. In cancer, they have also shown their potential, and besides asparaginase, many other enzymes are being object of study. In these two fields, in particular, enzymes gain prominence, and so they are the main focus of this work. Relying on the most up-to-date information available, this review tried to understand enzyme’s contribution to therapy, in the past, present and future. In LDSs, enzymes allow to improve considerably patient’s quality of life. There are approved for the treatment of Gaucher’s, Fabry’s and Pompe’s disease and some mucopolysaccharidosis but hopefully they will soon be available for other rare diseases. In cancer, more specifically in pediatric acute linfoblastic leukemia, L-asparaginase as revealed itself very effective, being part of conventional chemotherapy protocols for decades. Other enzymes such as arginine deiminase have shown good initial results and are currently in clinical studies; they may, soon, be revolutionizing the therapy of other sorts of tumors. There are, therefore, no doubts that enzymes have a major contribution to make to the future of therapy.
Descrição
Trabalho Final de Mestrado Integrado, Ciências Farmacêuticas, Universidade de Lisboa, Faculdade de Farmácia, 2013
Palavras-chave
Enzimoterapia Terapêutica de substituição enzimática Doenças lisossomais de sobrecarga L-asparaginase Cancro Mestrado Integrado - 2013