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

Leal-Lopes, Camila; Mantovani, Marta Sílvia Maria; Sogayar, Mari Cleide

doi:10.22239/2317-269x.01056

Cited by 1 publication

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“…Detailed standard operating procedures (SOP) and MBRs for manufacturing of stem cell-derived β-cells lots suitable for clinical transplantation are key to ensure that a viable mass of insulin-producing cells can be safely infused into the recipients. These SOPs detail each procedural step, from stem cell expansion and differentiation in vitro to pre-transplantation, quality controls and product release criteria for transplantation [49] ensuring that the reproducibility of the final product is in accordance with established specifications [50]. Also, operation in a closed system and automation of the manual steps enable sterility, processing robustness and reproducibility [48].…”

Section: Therapeutic Alternatives For T1dmentioning

confidence: 99%

Stem cells differentiation into insulin-producing cells (IPCs): recent advances and current challenges

et al. 2022

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Type 1 diabetes mellitus (T1D) is a chronic disease characterized by an autoimmune destruction of insulin-producing β-pancreatic cells. Although many advances have been achieved in T1D treatment, current therapy strategies are often unable to maintain perfect control of glycemic levels. Several studies are searching for new and improved methodologies for expansion of β-cell cultures in vitro to increase the supply of these cells for pancreatic islets replacement therapy. A promising approach consists of differentiation of stem cells into insulin-producing cells (IPCs) in sufficient number and functional status to be transplanted. Differentiation protocols have been designed using consecutive cytokines or signaling modulator treatments, at specific dosages, to activate or inhibit the main signaling pathways that control the differentiation of induced pluripotent stem cells (iPSCs) into pancreatic β-cells. Here, we provide an overview of the current approaches and achievements in obtaining stem cell-derived β-cells and the numerous challenges, which still need to be overcome to achieve this goal. Clinical translation of stem cells-derived β-cells for efficient maintenance of long-term euglycemia remains a major issue. Therefore, research efforts have been directed to the final steps of in vitro differentiation, aiming at production of functional and mature β-cells and integration of interdisciplinary fields to generate efficient cell therapy strategies capable of reversing the clinical outcome of T1D.

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Section: Therapeutic Alternatives For T1dmentioning

confidence: 99%