Advanced Therapy Medicinal Products in type I diabetes mellitus: technological and regulatory challenges

• Autor(a) principal: Leal-Lopes, Camila
• Data de Publicação: 2018
• Outros Autores: Mantovani, Marluce da Cunha, Sogayar, Mari Cleide
• 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
• 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

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.