Many interesting and difficult questions were raised in Tuesday’s round table discussion on production and manufacture hosted by Jim Reid chairman of Sistemic U.K. The topic of this open discussion was ‘What is the role of improved product characterisation on the development and approval of cell therapy product?’.
Morrie Ruffin of the US alliance for regenerative medicine (ARM) kicked of the discussion by announcing that in collaboration with the national institute of standards in technology (NIST), ARM is leading a multinational ‘consensus building exercise’ with the aim producing a set of universally applicable product characterisation standards which all cell therapy products should adhere. But what kind of assays are applicable in defining something as complex as a cell? asks Ekkehart Steinhuber – Innovacell biotechnologies. We are not yet able to fully characterise monoclonal antibodies and even if we could, would we want to? With every technological development that improves our analytical capability, we add another layer of specifications and therefore complexity to our products. Do we even know which characteristics are fundamental to our cell therapy product’s performance? In previous panel discussions the interactive nature of a cell’s therapeutic effects has been repeatedly emphasised. In order to define a cell’s potency therefore we would first need to define the physiological milieu with which it interacts. Understanding how this varies between individuals and what effect that in turn has on the transplanted cells opens the door to a whole new set of challenges.
Another avenue is to standardise the extent to which the manufacturing process is defined; if we can’t say exactly how or why the product works, at least we can say it is always the same product. There has been a significant escalation of efforts in this regard in recent years which will be discussed in further detail throughout the conference. Another delegate put forward the suggestion that we should learn from other industries; performance-based characterisation is a methodology used often in material sciences, i.e. we should define the cells by the effect they produce in patients. Knowing what we know about the variable affects of the simple pharmaceuticals in humans, would such an approach leave us any the wiser about the safety and mechanism of action of our cell therapies?
Morrie Ruffin encourages us all to sign up to the monthly ARM newsletter at www. alliancerm.org in which updates on this standards defining exercise will be reported.
Dr. Amelia Lane for Total BioPharma at the World Stem Cell and Regenerative Medicine Congress, London.
Post Doctoral Research Scientist at UCL Institute of Ophthalmology