Dr Maria Cristima Galli @CAT discussed the regulatory hurdles for stem cell therapies

The second day of the World Stem Cells Regenerative Medicine Congress got under steam with a vivid discussion of the European regulatory framework and ways of negotiating it successfully.

Before introducing his “dream team” of regulatory experts, Christopher Bravery, doyen of stem cell regulation and Director of Consulting on Advanced Biologicals, opened the discussion with a short overview of the sector.

Between 2004 and 2010, there had been 318 clinical trials across the European Union  involving 250 advanced therapy products.  40% had been commercially funded, the remaining majority falling to public sector organisations,- typically academic centres.  The non-EU funding had, unsurprisingly, been commercial, while EU domestic funding was largely academic,- typically focused on early storage clinical trials.  About 50% of the advanced therapies under trial had been gene therapy products.  Since the last Congress meeting, the European Medicines Agency (“EMA”) had provided access, via a webpage, to “Medicines under Evaluation”.  A snapshot taken on 14 May 2012 showed 4 relevant medicines,- all involving autologous cells.

The Congress then heard from the “dream team”, starting with Dr. Maria Cristina Galli and member of the EMA’s Committee for Advanced Therapies CAT, who outlined the European regulatory environment under Directive 2001/20, Regulation 726/2004 and, of course, the ATMP Regulation (Regulation 1394/2007), as well as the EMA’s recent reflection paper on stem cell based products, which is pitched at the marketing authorisation level.

Dr Galli noted the key issues included processing, cell plasticity, the definition of critical manufacturing steps, the identification of relevant markers and steps to control undifferentiated cells. Identity is key: defining, at every stage, the cells that pass through the process to clinical implementation and follow-through.  The non-clinical assessment turns out to be pretty important too.  Did you choose the right animal model? How about biodistribution and microenvironment?  What is the risk of ectopic grafts, differentiation in vivo, immunogenicity and, of course, tumourgenicity?  The latter is an inherent risk for pluripotent and somatic cell derived products: the regulatory assessment is unsurprisingly, exacting.  Add to this the direct clinical issues: safety and efficacy.

Dr Galli wound up with a few indicators of ATMP translation.  Notably, all sides are learning from experience.  For example, requests for scientific advice, such as questions to CAT on tumourgenicity and toxicity increase overall understanding of issues.

Check back here in a couple of days for the presentation. Excellent presentation Maria Cristina!

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