Development of an advanced cell therapy product indicated for the treatment of gonarthrosis

Vives, Joaquim; Blanco, Miguel; Caminal, Marta; Coca, María Isabel; Codinach, Margarita; Coll, Ruth; Doral, Manuel; Lloret, M.; Oliver-Vila, Irene; Ortega, Isabel; Reales, Laura; Requena-Montero, Míriam; Rodríguez, Laura; Torrents, Sílvia; Garcı́a, Joan

doi:10.1186/1753-6561-9-s9-o9

Cited by 5 publications

(2 citation statements)

References 16 publications

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“…The present study covers pharmacodynamics (PD), pharmacokinetics (PK) and toxicology (Tox) aspects of intrathecal administration of MSC,WJ, in the context of a broader non-clinical development package (described in Table 1 ) included in the IMPD submitted to the competent regulatory authority with the aim of clinical testing a candidate advanced therapy for SCI [ 14 , 15 ]. All animal procedures were conducted in accordance with local, national and European legislation (Decret 214 de 1997, Real Decreto 53 de 2013, European Directive 2010/63/EU, respectively) and were approved by the Universitat Autònoma de Barcelona’s Ethical Committee on Human and Animal Experimentation.…”

Section: Methodsmentioning

confidence: 99%

Preclinical Development of a Therapy for Chronic Traumatic Spinal Cord Injury in Rats Using Human Wharton’s Jelly Mesenchymal Stromal Cells: Proof of Concept and Regulatory Compliance

Vives

Hernández

Mirabel

et al. 2022

Cells

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(1) Background: the use of Mesenchymal Stromal Cells (MSC) in emerging therapies for spinal cord injury (SCI) hold the potential to improve functional recovery. However, the development of cell-based medicines is challenging and preclinical studies addressing quality, safety and efficacy must be conducted prior to clinical testing; (2) Methods: herein we present (i) the characterization of the quality attributes of MSC from the Wharton’s jelly (WJ) of the umbilical cord, (ii) safety of intrathecal infusion in a 3-month subchronic toxicity assessment study, and (iii) efficacy in a rat SCI model by controlled impaction (100 kdynes) after single (day 7 post-injury) and repeated dose of 1 × 106 MSC,WJ (days 7 and 14 post-injury) with 70-day monitoring by electrophysiological testing, motor function assessment and histology evaluation; (3) Results: no toxicity associated to MSC,WJ infusion was observed. Regarding efficacy, recovery of locomotion was promoted at early time points. Persistence of MSC,WJ was detected early after administration (day 2 post-injection) but not at days 14 and 63 post-injection. (4) Conclusions: the safety profile and signs of efficacy substantiate the suitability of the presented data for inclusion in the Investigational Medicinal Product Dossier for further consideration by the competent Regulatory Authority to proceed with clinical trials.

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Section: Methodsmentioning

confidence: 99%

Preclinical Development of a Therapy for Chronic Traumatic Spinal Cord Injury in Rats Using Human Wharton’s Jelly Mesenchymal Stromal Cells: Proof of Concept and Regulatory Compliance

Vives

Hernández

Mirabel

et al. 2022

Cells

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“…Importantly, this information is required for the regulatory submission, especially when animal-derived components are used. Any raw materials and excipients must be suitable for clinical use, and some may have to be substituted for others with similar characteristics upon appropriate risk analysis and experimental testing (e.g., xeno-free, clinical grade ingredients) ( 19 ), which may be more expensive. Additionally, sometimes extensive ex vivo expansion is required to yield sufficient cell numbers for clinical use, and scale-up processes should be defined and incorporated within the manufacturing procedure to adapt doses and/or final product dimensions to human scale ( 20 ).…”

Section: Sections On Assessment Of Policy/guidelines Options and Implmentioning

confidence: 99%

Transitioning From Preclinical Evidence to Advanced Therapy Medicinal Product: A Spanish Experience

Gastelurrutia

Vives

Coll

et al. 2021

Front. Cardiovasc. Med.

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A systematic and ordered product development program, in compliance with current quality and regulatory standards, increases the likelihood of yielding a successful advanced therapy medicinal product (ATMP) for clinical use as safe and effective therapy. As this is a novel field, little accurate information is available regarding the steps to be followed, and the information to be produced to support the development and use of an ATMP. Notably, successful clinical translation can be somewhat cumbersome for academic researchers. In this article, we have provided a summary of the available information, supported by our experience in Spain throughout the development of an ATMP for myocardial infarction, from the pre-clinical stage to phase I clinical trial approval.

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Design and validation of a consistent and reproducible manufacture process for the production of clinical-grade bone marrow–derived multipotent mesenchymal stromal cells

et al. 2016

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Development of an advanced cell therapy product indicated for the treatment of gonarthrosis

Cited by 5 publications

References 16 publications

Preclinical Development of a Therapy for Chronic Traumatic Spinal Cord Injury in Rats Using Human Wharton’s Jelly Mesenchymal Stromal Cells: Proof of Concept and Regulatory Compliance

Preclinical Development of a Therapy for Chronic Traumatic Spinal Cord Injury in Rats Using Human Wharton’s Jelly Mesenchymal Stromal Cells: Proof of Concept and Regulatory Compliance

Transitioning From Preclinical Evidence to Advanced Therapy Medicinal Product: A Spanish Experience

Design and validation of a consistent and reproducible manufacture process for the production of clinical-grade bone marrow–derived multipotent mesenchymal stromal cells

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