Global Regulatory Perspective for MSCs

Lodge, Anthony; Detela, Giulia; Barry, Jacqueline; Ginty, Patrick J.; Mount, Natalie

doi:10.1016/b978-0-12-802826-1.00010-6

Cited by 5 publications

(5 citation statements)

References 19 publications

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“…The term ATMP is an EU-specific term for cell- and gene-based therapies developed for commercial use. Regulatory frameworks for cell- and gene-based therapies also exist in other global jurisdictions, 39 and non-EU countries where these therapies are in active development include the United States, Canada, Australia, Japan, and Korea. In the ICH regions of the United States and Japan, regulatory frameworks around cell and gene therapies have been further elaborated in recent years.…”

Section: Main Textmentioning

confidence: 99%

EU Regulatory Pathways for ATMPs: Standard, Accelerated and Adaptive Pathways to Marketing Authorisation

Detela¹,

Lodge²

2019

Molecular Therapy - Methods & Clinical Development

120

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Advanced therapy medicinal products (ATMPs) require evaluation by the European Medicines Agency’s Committee for Advanced Therapies prior to being placed on the European market, subject to a Marketing Authorisation granted by the European Commission. In common with other medicinal products, various regulatory pathways are available for taking ATMPs through clinical trials to market authorisation, and the regulatory pathway taken will depend on a product’s characteristics and the target patient population. With the industry poised to deliver more late-stage clinical and commercial ATMPs for serious diseases with high unmet medical need (e.g., T cell immunotherapies for cancer), bringing medicines to patients through optimized regulatory strategies and expedited pathways is assuming greater importance. The European Medicines Agency’s priority medicines (PRIME) scheme was introduced in 2016 specifically to enable this, and eligibility has been granted to 19 ATMPs as of the fourth quarter (Q4) 2018. Furthermore, two chimeric antigen receptor (CAR) T cell therapies, Yescarta and Kymriah, have recently completed their journeys through the scheme to Marketing Authorisation. This review discusses how the regulatory pathway for any particular ATMP, with or without PRIME designation, is determined and navigated.

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Section: Main Textmentioning

confidence: 99%

EU Regulatory Pathways for ATMPs: Standard, Accelerated and Adaptive Pathways to Marketing Authorisation

Detela¹,

Lodge²

2019

Molecular Therapy - Methods & Clinical Development

120

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“…Currently, there are regulatory differences among countries, beginning with the nomenclature of cell-based products, e.g., ATMP in the European Union; Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/P’s) in the USA; Regenerative Medicinal Product in Japan; Biological Drug in Canada; and Cell Therapy Product in Korea. More importantly, discrepancies also exist in defining the criteria of quality and potency [ 18 , 19 ]. Even with more than 1400 MSC studies registered on clinicaltrials.gov and the efforts of agencies around the world to regulate these ATMPs, only 11 products have been licensed for human use.…”

Section: Introductionmentioning

confidence: 99%

Mesenchymal Stromal Cell-Based Products: Challenges and Clinical Therapeutic Options

Mello,

Mesquita,

Silva dos Santos

et al. 2024

IJMS

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Mesenchymal stromal cell (MSC)-based advanced therapy medicinal products (ATMPs) are being tried in a vast range of clinical applications. These cells can be isolated from different donor tissues by using several methods, or they can even be derived from induced pluripotent stem cells or embryonic stem cells. However, ATMP heterogeneity may impact product identity and potency, and, consequently, clinical trial outcomes. In this review, we discuss these topics and the need to establish minimal criteria regarding the manufacturing of MSCs so that these innovative therapeutics may be better positioned to contribute to the advancement of regenerative medicine.

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“…Depending on the origin of the tissue, MSCs differ functionally in their paracrine mechanisms and their secretome-derived products [12]. MSCs are considered medicines from a regulatory viewpoint [13], and hence their quality needs to be controlled like other medicinal products [14]. After only minimal manipulation, MSC administration to humans can be considered as a form of transplantation [14].…”

Section: Introductionmentioning

confidence: 99%

“…MSCs are considered medicines from a regulatory viewpoint [13], and hence their quality needs to be controlled like other medicinal products [14]. After only minimal manipulation, MSC administration to humans can be considered as a form of transplantation [14]. On the other hand, the ex vivo preconditioning has been proposed as a way of priming MSCs' immunological features [15].…”

Section: Introductionmentioning

confidence: 99%

Microfluidic Tools for Enhanced Characterization of Therapeutic Stem Cells and Prediction of Their Potential Antimicrobial Secretome

Marrazzo

Pizzuti

Zia³

et al. 2021

Antibiotics

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Antibiotic resistance is creating enormous attention on the development of new antibiotic-free therapy strategies for bacterial diseases. Mesenchymal stromal stem cells (MSCs) are the most promising candidates in current clinical trials and included in several cell-therapy protocols. Together with the well-known immunomodulatory and regenerative potential of the MSC secretome, these cells have shown direct and indirect anti-bacterial effects. However, the low reproducibility and standardization of MSCs from different sources are the current limitations prior to the purification of cell-free secreted antimicrobial peptides and exosomes. In order to improve MSC characterization, novel label-free functional tests, evaluating the biophysical properties of the cells, will be advantageous for their cell profiling, population sorting, and quality control. We discuss the potential of emerging microfluidic technologies providing new insights into density, shape, and size of live cells, starting from heterogeneous or 3D cultured samples. The prospective application of these technologies to studying MSC populations may contribute to developing new biopharmaceutical strategies with a view to naturally overcoming bacterial defense mechanisms.

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Global Regulatory Perspective for MSCs

Cited by 5 publications

References 19 publications

EU Regulatory Pathways for ATMPs: Standard, Accelerated and Adaptive Pathways to Marketing Authorisation

EU Regulatory Pathways for ATMPs: Standard, Accelerated and Adaptive Pathways to Marketing Authorisation

Mesenchymal Stromal Cell-Based Products: Challenges and Clinical Therapeutic Options

Microfluidic Tools for Enhanced Characterization of Therapeutic Stem Cells and Prediction of Their Potential Antimicrobial Secretome

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