title: Cell-based therapies for cardiac repair: a meeting report on scientific observations and European regulatory viewpoints.
abstract: In the past decade, novel cell-based products have been studied in patients with acute and chronic cardiac disease to assess whether these therapies are efficacious in improving heart function and preventing the development of end-stage heart failure. Cardiac indications studied include acute myocardial infarction (AMI), refractory angina, and chronic heart failure (CHF). Increased clinical activity, experience, and multiple challenges faced by developers have been recognized at the regulatory level. In May 2014, the Committee for Advanced Therapies (CAT) discussed in an expert meeting various cell-based medicinal products developed for cardiac repair, with a focus on non-manipulated bone marrow cells, sorted bone marrow or apheresis, and expanded cells, applied to patients with AMI or CHF. The intention was to share information, both scientific and regulatory, and to examine the challenges and opportunities in this field. These aspects were considered from the quality, and non-clinical and clinical perspectives, including current imaging techniques, with a focus on AMI and CHF. The scope of this overview is to present the European regulatory viewpoint on cell-based therapies for cardiac repair in the context of scientific observations.
authors.affiliation: Committee for Advanced Therapies (CAT), European Medicines Agency (EMA), Langen, Germany.|Paul-Ehrlich-Institut, Federal Institute for Vaccines and Biomedicines, Langen, Germany.
authors.forename: Martina
authors.initials: M
authors.lastname: Schüssler-Lenz
authors.affiliation: Committee for Advanced Therapies (CAT), European Medicines Agency (EMA), Langen, Germany.|Federal Agency for Medicines and Health Products, Brussels, Belgium.
authors.forename: Claire
authors.initials: C
authors.lastname: Beuneu
authors.affiliation: Committee for Advanced Therapies (CAT), European Medicines Agency (EMA), Langen, Germany.|Infarmed-National Authority of Medicines and Health Products, Lisbon, Portugal.
authors.forename: Margarida
authors.initials: M
authors.lastname: Menezes-Ferreira
authors.affiliation: European Medicines Agency (EMA), London, UK.
authors.forename: Veronika
authors.initials: V
authors.lastname: Jekerle
authors.affiliation: Cardiovascular Center, OLV Hospital, Aalst, Belgium.
authors.forename: Jozef
authors.initials: J
authors.lastname: Bartunek
authors.affiliation: UMC Utrecht, The Netherlands.
authors.forename: Steven
authors.initials: S
authors.lastname: Chamuleau
authors.affiliation: Committee for Advanced Therapies (CAT), European Medicines Agency (EMA), Langen, Germany.|European Medicines Agency (EMA), London, UK.
authors.forename: Patrick
authors.initials: P
authors.lastname: Celis
authors.affiliation: Committee for Advanced Therapies (CAT), European Medicines Agency (EMA), Langen, Germany.|European Society of Cardiology and University Medical Center Utrecht, The Netherlands.
authors.forename: Pieter
authors.initials: P
authors.lastname: Doevendans
authors.affiliation: Committee for Advanced Therapies (CAT), European Medicines Agency (EMA), Langen, Germany.|Health Products Regulatory Authority, Dublin, Ireland.
authors.forename: Maura
authors.initials: M
authors.lastname: O'Donovan
authors.affiliation: NHS, London, UK.
authors.forename: Jonathan
authors.initials: J
authors.lastname: Hill
authors.affiliation: Committee for Advanced Therapies (CAT), European Medicines Agency (EMA), Langen, Germany.|Norwegian Medicines Agency, Oslo, Norway.
authors.forename: Marit
authors.initials: M
authors.lastname: Hystad
authors.affiliation: The DeVos Cardiovascular Research Program, Grand Rapids, MI, USA.
authors.forename: Stefan
authors.initials: S
authors.lastname: Jovinge
authors.affiliation: Committee for Advanced Therapies (CAT), European Medicines Agency (EMA), Langen, Germany.|Department of Pharmacology and Toxicology, Comenius University, Bratislava, Slovakia.
authors.forename: Ján
authors.initials: J
authors.lastname: Kyselovič
authors.affiliation: Committee for Advanced Therapies (CAT), European Medicines Agency (EMA), Langen, Germany.|University of Ljubljana, Faculty of Medicine, Ljubljana, Slovenia.
authors.forename: Metoda
authors.initials: M
authors.lastname: Lipnik-Stangelj
authors.affiliation: Committee for Advanced Therapies (CAT), European Medicines Agency (EMA), Langen, Germany.|Institute of Physiology and Pharmacology, Lithuanian University of Health Sciences, and State Medicines Control Agency, Kaunas, Lithuania.
authors.forename: Romaldas
authors.initials: R
authors.lastname: Maciulaitis
authors.affiliation: MHRA, London, UK.|Cardiovascular Working Party, European Medicines Agency (EMA).
authors.forename: Krishna
authors.initials: K
authors.lastname: Prasad
authors.affiliation: Committee for Advanced Therapies (CAT), European Medicines Agency (EMA), Langen, Germany.|Mater Dei Hospital, Malta.
authors.forename: Anthony
authors.initials: A
authors.lastname: Samuel
authors.affiliation: Committee for Advanced Therapies (CAT), European Medicines Agency (EMA), Langen, Germany.|Finnish Medicines Agency (Fimea), Helsinki, Finland.
authors.forename: Olli
authors.initials: O
authors.lastname: Tenhunen
authors.affiliation: Dresden University, Dresden, Germany.
authors.forename: Torsten
authors.initials: T
authors.lastname: Tonn
authors.affiliation: Cardiovascular Working Party, European Medicines Agency (EMA).|St George's University of London, IRCCS San Raffaele Roma†
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Rosano
authors.affiliation: University of Frankfurt, Frankfurt, Germany.
authors.forename: Andreas
authors.initials: A
authors.lastname: Zeiher
authors.affiliation: Committee for Advanced Therapies (CAT), European Medicines Agency (EMA), Langen, Germany.|Finnish Medicines Agency (Fimea), Helsinki, Finland.
authors.forename: Paula
authors.initials: P
authors.lastname: Salmikangas
mesh_terms: D000818:Animals; D017690:Cell Transplantation / Q000379:methods*; D004195:Disease Models, Animal; D005060:Europe; D006321:Heart / Q000502:physiology*; D006333:Heart Failure / Q000175:diagnosis / Q000628:therapy*; D006801:Humans; D009203:Myocardial Infarction / Q000175:diagnosis / Q000628:therapy*; D012038:Regeneration
keywords: Advanced therapy medicinal products; Cell-based therapies for cardiac repair; Committee for Advanced Therapies; European regulatory aspects