spacer
home > ebr > autumn 2017 > revamping regulations
PUBLICATIONS
European Biopharmaceutical Review

Revamping Regulations

Stem cell products hold great promise for multiple clinical indications and are actively studied in clinical trials (1). However, many related safety features raise concerns and currently generate extensive and costly testing of the final product, if intended for clinical use. The regulatory expectations for stem cell testing for clinical studies, as well as marketing authorisation applications, are not entirely clear, up-to-date or detailed. Furthermore, differences between US and EU guidance, in this respect, hamper the current development of products for EU markets.

Risk Profiling

Stem cells used for therapeutic purposes include a wide variety of cells with different type differentiation capacities, spanning from restricted multipotent up to pluripotent stem cells (2). Hematopoietic stem cells have been widely used for ex vivo gene therapy of monogenic inherited diseases, and mesenchymal stem cells have been utilised eg in treatment of Graft versus Host Disease for more than a decade due to their immunomodulatory properties (3,4). Pluripotent stem cells, ie embryonic stem cells and induced pluripotent stem cells (iPSCs), are still in early clinical development, yet some clinical data are already available (5,6).

When considering possible risks of stem cells, several issues need to be considered, such as the donor (autologous versus allogeneic), characteristics of the cells, extent of manipulation, design of vectors for genetic modification, dose and the existing knowledge of the cell type/product to be used. One of the main concerns related to stem cell products is tumourigenicity. For pluripotent stem cells, teratoma formation is part of their natural characteristics, which calls for thorough control of the cell differentiation status before clinical use.

Read full article from PDF >>

Rate this article You must be a member of the site to make a vote.  
Average rating:
0
     

There are no comments in regards to this article.

spacer
Dr Paula Salmikangas, Director for Biopharmaceuticals and ATMPs, NDA Advisory Board, is a clinical biochemist by original training with a PhD in muscle cell biology. Since 2006, she has been an Adjunct Professor of Biochemistry for the University of Helsinki, Finland. Paula joined NDA in 2017, has served as a member of the EMA Committee for Advanced Therapies (CAT) from 2009 to 2017 and has been the Chair of the CAT 2014-2017. Her main areas of expertise are biological medicinal products, especially ATMPs, chemistry, manufacturing and controls aspects of biopharmaceuticals.
spacer
Dr Paula Salmikangas
spacer
spacer
Print this page
Send to a friend
Privacy statement
News and Press Releases

GE Healthcare opens its first European 3D printing and design center


More info >>

White Papers

Crunch Time: The Impact of Serialisation Requirements on Packaging Operations

PCI Pharma Services

The deadlines for including unique product identifiers on prescription drugs is putting a strain on multiple parts of the industry, much of which is ill-prepared to meet the target. Here, we look to the experience of a company that is ahead of the curve for lessons on how to manage and ease the transition to serialisation.
More info >>

 
Industry Events

ASPIRE: International EDGE Conference

28 February - 1 March 2018, The Vox Conference Centre, Birmingham

The ASPIRE international conference is taking place on the 28th February and 1st March 2018 at the Vox Conference Centre in Birmingham. The conference brings together members of the research community including research professionals from the NHS, Clinical Trial Networks and international organisations. The ASPIRE conference will allow delegates to share knowledge and best practise with the focus on enhancing the efficiency and productivity of clinical research across the UK and beyond.
More info >>

 

 

©2000-2011 Samedan Ltd.
Add to favourites

Print this page

Send to a friend
Privacy statement