Cellular Therapy Training Course
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The Inaugural ISCT-ASBMT Cell Therapy Training Course – One Scholar’s Experience

Beth Sage MBBS, PhD
UCL Respiratory
University College London, London, UK

Having been lucky enough to be selected as an international scholar for the inaugural Cell Therapy Training Course I was looking forward to leaving behind the rather disappointing British summer and heading towards the much warmer Houston fall.  Having googled my destination and accommodation I boarded the plane with great excitement and hopes of enjoying the Texan heat whilst exploring the cosmopolitan offerings of America’s fourth largest city – oh and learning something about cell therapy! 

The course, chaired by Dave DiGiusto and John Barrett, was the first of its kind, a joint enterprise between the ISCT and the American Society of Blood and Marrow Transplantation.  Following a competitive selection procedure 12 scholars from all over the world were invited to attend a 5 day intensive workshop with the primary objective of giving junior cell therapy researchers an insight into the process of taking their research project from bench to bedside, including processing, clinical trial design, regulatory requirements, commercialization and ethical research.  Alongside didactic lecture based teaching there were tours of good manufacturing practice (GMP) facilities, both academic and commercial, and most anticipated by the scholars was the opportunity to participate in small group discussions, led by experts in the field, dissecting and improving the individual cell therapy projects.

To break the ice on the ‘light’ first day each scholar gave a short presentation on their project.  It was immediately clear that there is a great breadth of exciting, novel cell therapy projects under investigation throughout the world, from the use of modified T-cells in hematological malignancies to the development of a tissue-engineered oesophagus using amniotic fluid stem cells.  Projects ranged from early pre-clinical to those embarking on a first in man clinical trial and every stage in between, making the session interesting and varied.  Having fought the jet-lag, the session ended with an ice-breaker drinks and dinner before retiring to prepare for the days ahead.

Over the next few days we were exposed to a wealth of information with detailed talks on pre-clinical development of different cell therapies from CD34 cells to mesenchymal stromal cells, quality systems development and one of the most useful from my personal perspective, manufacturing and release testing of different products.  We were able to visit different manufacturing facilities and to understand the processes involved in the production of a clinical grade therapy.  It made us challenge the protocols we were developing in the lab as we gained an insight into how it would scale up into a commercially viable process – a 26 day culture process of autologous cells requiring purification and multiple cytokine stimulations is significantly more challenging (and expensive) than allogeneic cells cultured for 14 days with no manipulation and simple media exchanges. 

Once the process development sessions were complete, we switched gears to look at how to conduct cell therapy clinical trials, covering issues of producing products including normal donors that are used to treat multiple recipients, the challenges of pooling donor cells, how to run multicenter studies and most importantly (although I can say almost universally never thought about by the scholars) how to deal with a regulatory body audit.  This really was a really informative session that opened our eyes to the challenges and complexities of working in the field of cell therapy trials. 

Just when we were beginning to feel that our jobs over the next few years would be focused on clinical trial design, process validation and filling in an endless paper chain of regulatory documents we were brought back to where we all started – the excitement of the translational science.  This was, for me, a really interesting session on the importance of correlative studies not only to assess clinical trial performance but to provide mechanistic insights into the behavior of cells when delivered to patients with disease.  As scientists we can design and perform many experiments to predict how manipulated cells will behave but the most important data of all comes from the patients themselves.  For me, the importance of testing a novel therapy is not just to see if it works but how it works and, just as importantly, if it doesn’t - why.

To end to course, our wise leaders Dave DiGiusto and John Barrett decided to test whether we had been listening, and each scholar had to deliver a detailed presentation on how their project had developed during the course.  Each scholar had to address the potential pitfalls and specific challenges they faced in moving towards the clinic. Whilst many of us stayed up into the small hours worrying about aspects we were previously oblivious to, undoubtedly we found this one of the most rewarding moments.  Despite the potential difficulties we were now aware of, we also felt better placed to solve them and could see a clearer path ahead.

This was without a doubt one of the best courses I have attended.  The chance to talk to so many experts who have really been the trail blazers of cell therapies was invaluable, and although the road ahead is still a challenging one, it seems less scary to navigate with the new supporters we have found. As is so often the case feedback comes down to ‘how likely are you to recommend us to a friend’.  My answer? Extremely likely.  



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