Advanced Therapy Medicinal Products in type I diabetes mellitus: technological and regulatory challenges
Autor(a) principal: | |
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Data de Publicação: | 2018 |
Outros Autores: | , |
Tipo de documento: | Artigo |
Idioma: | eng por |
Título da fonte: | Vigilância Sanitária em Debate |
Texto Completo: | https://visaemdebate.incqs.fiocruz.br/index.php/visaemdebate/article/view/1056 |
Resumo: | Introduction: Type 1 Diabetes mellitus (T1DM) is an autoimmune disorder which arises from the destruction of insulin-producing pancreatic β-cells. Currently, Brazil’s advanced therapy medicinal products (ATMP), developed for clinical research and therapeutic purposes, take place in the so-called Cellular Technology Centers (CTC), according to the Resolution nº. 9/2011 of the Collegiate Board of Directors (RDC), enacted by the National Health Surveillance Agency (Anvisa). Objective: This study was conducted with the main objective of describing and discussing the development of ATMP for T1DM treatment. Method: A qualitative research, narrative review and critical discussion of the literature were under taken. Results: ATMP promote new therapeutic approaches for Diabetes, holding great potential to restore the patients’ endogenous insulin secretion, improving their life quality, overcoming the chronic complications of Diabetes and reducing the socioeconomic burden. Nowadays, ATMP in T1DM comprise: a) cell therapy; b) gene therapy products; c) tissue engineering and d) ATMPassociated to biopharmaceutical products. Conclusions: Further research should contribute to stimulate public and private organizations to effectively act towards reducing the impact of Diabetes on individuals and the society as a whole. It is essential that Brazilian legislation closely follows the biotechnological developments, supporting the scientific progress and benefiting T1DM patients with modern and cutting-edge therapies. |
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Advanced Therapy Medicinal Products in type I diabetes mellitus: technological and regulatory challengesAdvanced Therapy Medicinal Products in type I diabetes mellitus: technological and regulatory challengesAdvanced Therapy Medicinal Products (ATMP)Type 1 Diabetes Mellitus (T1DM)Pancreatic Islet Transplantation and EncapsulationRegulatory Legislation for Cell and Gene TherapyBiopharmaceuticalsProdutos Medicinais de Terapia AvançadaDiabetes Mellitus do Tipo 1Transplante e Encapsulamento de Ilhotas PancreáticasLegislação Regulatória de Terapias Celulares e GenéticasBiofármacosIntroduction: Type 1 Diabetes mellitus (T1DM) is an autoimmune disorder which arises from the destruction of insulin-producing pancreatic β-cells. Currently, Brazil’s advanced therapy medicinal products (ATMP), developed for clinical research and therapeutic purposes, take place in the so-called Cellular Technology Centers (CTC), according to the Resolution nº. 9/2011 of the Collegiate Board of Directors (RDC), enacted by the National Health Surveillance Agency (Anvisa). Objective: This study was conducted with the main objective of describing and discussing the development of ATMP for T1DM treatment. Method: A qualitative research, narrative review and critical discussion of the literature were under taken. Results: ATMP promote new therapeutic approaches for Diabetes, holding great potential to restore the patients’ endogenous insulin secretion, improving their life quality, overcoming the chronic complications of Diabetes and reducing the socioeconomic burden. Nowadays, ATMP in T1DM comprise: a) cell therapy; b) gene therapy products; c) tissue engineering and d) ATMPassociated to biopharmaceutical products. Conclusions: Further research should contribute to stimulate public and private organizations to effectively act towards reducing the impact of Diabetes on individuals and the society as a whole. It is essential that Brazilian legislation closely follows the biotechnological developments, supporting the scientific progress and benefiting T1DM patients with modern and cutting-edge therapies.TÍTULO PT: Produtos Medicinais de Terapia Avançada no diabetes mellitus tipo I: desafios tecnológicos e regulatóriosIntrodução: O diabetes mellitus do tipo 1 (T1DM) é uma desordem autoimune que culmina na destruição das células β-pancreáticas produtoras de insulina. Atualmente, Produtos Medicinais de Terapia Avançada (ATMP) são desenvolvidos no Brasil, para fins de pesquisa clínica e terapia celular, pelos chamados Centros de Tecnologia Celular (CTC), de acordo com a Resolução da Diretoria Colegiada nº 9/2011, emitida pela Agência Nacional de Vigilância Sanitária (Anvisa). Objetivos: Esse estudo foi conduzido com o objetivo principal de descrever e discutir o desenvolvimento de produtos medicinais em terapia avançada (ATMP) para o tratamento de T1DM. Método: Esse estudo foi realizado através de pesquisa qualitativa, revisão narrativa e discussão critica. Resultados: Os ATMP promovem abordagens terapêuticas para o diabetes demonstrando grande potencial para restaurar a secreção endógena de insulina dos pacientes, aumentando sua qualidade de vida, superando as complicações crônicas do Diabetes e reduzindo o impacto socioeconômico desta doença. Atualmente, os ATMP para T1DM compreendem: a) terapia celular; b) terapia gênica; c) engenharia tecidual e d) ATMP associados a produtos biofarmacêuticos. Conclusões: Pesquisas adicionais deverão contribuir para mobilizar governos e organizações para ações que promovam, de forma eficiente, a redução do impacto do diabetes nos indivíduos e na sociedade. Para isso, é essencial que a legislação brasileira acompanhe de perto os desenvolvimentos biotecnológicos, dando suporte para o progresso científico e beneficiando pacientes de T1DM com terapias modernas.Instituto Nacional de Controle de Qualidade em Saúde2018-02-28info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersion"Peer-reviewed article""Artículo revisado por pares""Artigo avaliado pelos pares"application/pdfapplication/pdfhttps://visaemdebate.incqs.fiocruz.br/index.php/visaemdebate/article/view/105610.22239/2317-269X.01056Health Surveillance under Debate: Society, Science & Technology ; Vol. 6 No. 1 (2018): February - Tecnologias celulares avançadas: desafios biotecnológicos e regulatórios; 41-55Vigilancia en Salud en Debate: Sociedad, Ciencia y Tecnología; Vol. 6 Núm. 1 (2018): Febrero - Tecnologias celulares avançadas: desafios biotecnológicos e regulatórios; 41-55Vigil Sanit Debate, Rio de Janeiro; v. 6 n. 1 (2018): Fevereiro - Tecnologias celulares avançadas: desafios biotecnológicos e regulatórios; 41-552317-269Xreponame:Vigilância Sanitária em Debateinstname:Fundação Oswaldo Cruz (FIOCRUZ)instacron:FIOCRUZengporhttps://visaemdebate.incqs.fiocruz.br/index.php/visaemdebate/article/view/1056/428https://visaemdebate.incqs.fiocruz.br/index.php/visaemdebate/article/view/1056/522Copyright (c) 2018 Vigilância Sanitária em Debate: Sociedade, Ciência & Tecnologia (Health Surveillance under Debate: Society, Science & Technology) – Visa em Debatehttps://creativecommons.org/licenses/by-nc-nd/4.0info:eu-repo/semantics/openAccessLeal-Lopes, CamilaMantovani, Marluce da CunhaSogayar, Mari Cleide2023-06-27T15:07:41Zoai:ojs.visaemdebate.incqs.fiocruz.br:article/1056Revistahttps://visaemdebate.incqs.fiocruz.br/index.php/visaemdebatePUBhttps://visaemdebate.incqs.fiocruz.br/index.php/visaemdebate/oaiincqs.visaemdebate@fiocruz.br || gisele.neves@fiocruz.br2317-269X2317-269Xopendoar:2023-06-27T15:07:41Vigilância Sanitária em Debate - Fundação Oswaldo Cruz (FIOCRUZ)false |
dc.title.none.fl_str_mv |
Advanced Therapy Medicinal Products in type I diabetes mellitus: technological and regulatory challenges Advanced Therapy Medicinal Products in type I diabetes mellitus: technological and regulatory challenges |
title |
Advanced Therapy Medicinal Products in type I diabetes mellitus: technological and regulatory challenges |
spellingShingle |
Advanced Therapy Medicinal Products in type I diabetes mellitus: technological and regulatory challenges Leal-Lopes, Camila Advanced Therapy Medicinal Products (ATMP) Type 1 Diabetes Mellitus (T1DM) Pancreatic Islet Transplantation and Encapsulation Regulatory Legislation for Cell and Gene Therapy Biopharmaceuticals Produtos Medicinais de Terapia Avançada Diabetes Mellitus do Tipo 1 Transplante e Encapsulamento de Ilhotas Pancreáticas Legislação Regulatória de Terapias Celulares e Genéticas Biofármacos |
title_short |
Advanced Therapy Medicinal Products in type I diabetes mellitus: technological and regulatory challenges |
title_full |
Advanced Therapy Medicinal Products in type I diabetes mellitus: technological and regulatory challenges |
title_fullStr |
Advanced Therapy Medicinal Products in type I diabetes mellitus: technological and regulatory challenges |
title_full_unstemmed |
Advanced Therapy Medicinal Products in type I diabetes mellitus: technological and regulatory challenges |
title_sort |
Advanced Therapy Medicinal Products in type I diabetes mellitus: technological and regulatory challenges |
author |
Leal-Lopes, Camila |
author_facet |
Leal-Lopes, Camila Mantovani, Marluce da Cunha Sogayar, Mari Cleide |
author_role |
author |
author2 |
Mantovani, Marluce da Cunha Sogayar, Mari Cleide |
author2_role |
author author |
dc.contributor.author.fl_str_mv |
Leal-Lopes, Camila Mantovani, Marluce da Cunha Sogayar, Mari Cleide |
dc.subject.por.fl_str_mv |
Advanced Therapy Medicinal Products (ATMP) Type 1 Diabetes Mellitus (T1DM) Pancreatic Islet Transplantation and Encapsulation Regulatory Legislation for Cell and Gene Therapy Biopharmaceuticals Produtos Medicinais de Terapia Avançada Diabetes Mellitus do Tipo 1 Transplante e Encapsulamento de Ilhotas Pancreáticas Legislação Regulatória de Terapias Celulares e Genéticas Biofármacos |
topic |
Advanced Therapy Medicinal Products (ATMP) Type 1 Diabetes Mellitus (T1DM) Pancreatic Islet Transplantation and Encapsulation Regulatory Legislation for Cell and Gene Therapy Biopharmaceuticals Produtos Medicinais de Terapia Avançada Diabetes Mellitus do Tipo 1 Transplante e Encapsulamento de Ilhotas Pancreáticas Legislação Regulatória de Terapias Celulares e Genéticas Biofármacos |
description |
Introduction: Type 1 Diabetes mellitus (T1DM) is an autoimmune disorder which arises from the destruction of insulin-producing pancreatic β-cells. Currently, Brazil’s advanced therapy medicinal products (ATMP), developed for clinical research and therapeutic purposes, take place in the so-called Cellular Technology Centers (CTC), according to the Resolution nº. 9/2011 of the Collegiate Board of Directors (RDC), enacted by the National Health Surveillance Agency (Anvisa). Objective: This study was conducted with the main objective of describing and discussing the development of ATMP for T1DM treatment. Method: A qualitative research, narrative review and critical discussion of the literature were under taken. Results: ATMP promote new therapeutic approaches for Diabetes, holding great potential to restore the patients’ endogenous insulin secretion, improving their life quality, overcoming the chronic complications of Diabetes and reducing the socioeconomic burden. Nowadays, ATMP in T1DM comprise: a) cell therapy; b) gene therapy products; c) tissue engineering and d) ATMPassociated to biopharmaceutical products. Conclusions: Further research should contribute to stimulate public and private organizations to effectively act towards reducing the impact of Diabetes on individuals and the society as a whole. It is essential that Brazilian legislation closely follows the biotechnological developments, supporting the scientific progress and benefiting T1DM patients with modern and cutting-edge therapies. |
publishDate |
2018 |
dc.date.none.fl_str_mv |
2018-02-28 |
dc.type.driver.fl_str_mv |
info:eu-repo/semantics/article info:eu-repo/semantics/publishedVersion "Peer-reviewed article" "Artículo revisado por pares" "Artigo avaliado pelos pares" |
format |
article |
status_str |
publishedVersion |
dc.identifier.uri.fl_str_mv |
https://visaemdebate.incqs.fiocruz.br/index.php/visaemdebate/article/view/1056 10.22239/2317-269X.01056 |
url |
https://visaemdebate.incqs.fiocruz.br/index.php/visaemdebate/article/view/1056 |
identifier_str_mv |
10.22239/2317-269X.01056 |
dc.language.iso.fl_str_mv |
eng por |
language |
eng por |
dc.relation.none.fl_str_mv |
https://visaemdebate.incqs.fiocruz.br/index.php/visaemdebate/article/view/1056/428 https://visaemdebate.incqs.fiocruz.br/index.php/visaemdebate/article/view/1056/522 |
dc.rights.driver.fl_str_mv |
https://creativecommons.org/licenses/by-nc-nd/4.0 info:eu-repo/semantics/openAccess |
rights_invalid_str_mv |
https://creativecommons.org/licenses/by-nc-nd/4.0 |
eu_rights_str_mv |
openAccess |
dc.format.none.fl_str_mv |
application/pdf application/pdf |
dc.publisher.none.fl_str_mv |
Instituto Nacional de Controle de Qualidade em Saúde |
publisher.none.fl_str_mv |
Instituto Nacional de Controle de Qualidade em Saúde |
dc.source.none.fl_str_mv |
Health Surveillance under Debate: Society, Science & Technology ; Vol. 6 No. 1 (2018): February - Tecnologias celulares avançadas: desafios biotecnológicos e regulatórios; 41-55 Vigilancia en Salud en Debate: Sociedad, Ciencia y Tecnología; Vol. 6 Núm. 1 (2018): Febrero - Tecnologias celulares avançadas: desafios biotecnológicos e regulatórios; 41-55 Vigil Sanit Debate, Rio de Janeiro; v. 6 n. 1 (2018): Fevereiro - Tecnologias celulares avançadas: desafios biotecnológicos e regulatórios; 41-55 2317-269X reponame:Vigilância Sanitária em Debate instname:Fundação Oswaldo Cruz (FIOCRUZ) instacron:FIOCRUZ |
instname_str |
Fundação Oswaldo Cruz (FIOCRUZ) |
instacron_str |
FIOCRUZ |
institution |
FIOCRUZ |
reponame_str |
Vigilância Sanitária em Debate |
collection |
Vigilância Sanitária em Debate |
repository.name.fl_str_mv |
Vigilância Sanitária em Debate - Fundação Oswaldo Cruz (FIOCRUZ) |
repository.mail.fl_str_mv |
incqs.visaemdebate@fiocruz.br || gisele.neves@fiocruz.br |
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1797042045200629760 |