View from the floor 4: Risk aversion in cell therapy development

Commercialization plenary summary: part 3 of 4

On day two (May 1) of the Till and McCulloch Meetings, Dr. Emily Culme-Seymour from the London Regenerative Medicine Network introduced work that leverages previous clinical trial information to support future cell therapy efforts. In an analysis of almost 3000 clinical trials extracted from clinicaltrials.gov, Culme-Seymour focused on cell therapy related trials. She found that half of the trials were in Phase 1 (40% were Phase 2 and only 10% were Phase 3), and almost three quarters of all cell therapy trials being conducted in the United States.

With an overwhelming majority of cell therapy trials being found at
 relatively early stages, it suggests that that
 risk-aversion is partly involved in keeping cell therapy trials from
 launching Phase 3 trials. The idea of risk aversion was rearticulated with the finding that a significantly larger proportion of trials were of transient cell therapies such as Osiris’ Prochymal as opposed to permanent cell replacement. Culme-Seymour found that only 5% of trials were testing permanent therapies.

The numbers between autologous and allogeneic trials are approximately balanced.

The most basic conclusion is that future recipients of approved cell therapies will likely need repeated treatments to maintain efficacy. Permanent cell based treatments that will act as a one time “repair” will probably remain uncommon, at least for the near future.

Temporary cell based treatments may not be a bad thing overall.

I believe the bias towards transient therapies to be a symptom of two factors: relative ease of their development and caution on the part of the community.

Culme-Seymour explained that it’s less complicated to develop a technology that safely produces a temporary benefit. I would extend that to say that modifying, reversing, or even upgrading transient therapies for indications will be relatively easy while changing permanent modifications presents a whole new set of problems, namely how to undo the modification before the subsequent treatment.

I definitely don’t buy the argument that a commercial desire to sell (and re-sell, and re-sell) treatments to individuals drives the development of transient therapies versus permanent ones; it’s just a matter of what technology is currently available. Dr. Culme-Seymour’s work provides an excellent roadmap for those interested in following the prevailing trends in regenerative medicine product development.

See also:
Clinical translation summary at Stem Cell Assays
Meeting Summary 3: Anticipatory ethics and the problem of expectations
Meeting Summary 2: Commercialization plenary
Meeting Summary 1: Commercialization plenary

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Paul Krzyzanowski

Paul is a computational biologist and writer living in Toronto. He's been a contributor to Signals for three years, writing articles for the general public about how biotechnology and biomedical research can be used to solve pressing medical problems. Alongside Paul's experience in computational biology,
 bioinformatics, and molecular genetics, he's interested in how academic research develops into real world, commercial technology, and what's needed for the Canadian biotech industry needs to grow. Paul is currently a Post-doctoral Fellow at the Ontario Institute of Cancer Research. Prior to joining the OICR, he worked at the Ottawa Hospital Research 
Institute and earned a Ph.D. from the University of Ottawa, specializing in computational biology. And finally, Paul earned an H.B.Sc. from the University of Toronto a long time ago. Paul's blog can be read at www.checkmatescientist.net

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