Author(s): Emily J Culme-Seymour 1 , Natasha L Davie 2 , David A Brindley 3 , Simon Edwards-Parton 4 , Chris Mason [*] 5
cell therapy; clinical trials; ClinicalTrials.gov; commercialization; regulation; translation
The cell therapy industry (CTI) is presently a small but potentially rapidly growing new global healthcare sector. Success is totally dependent on resolving a number of factors unique to cells as therapies, including: manufacturing, enabling technologies, regulation, reimbursement and essential infrastructure. To understand how to solve these challenges in a timely and cost-effective manner, it is essential to be able to forecast the size and resource demands of the sector for a least the next decade. Due to the highly regulated nature of medicines, one predictive method is to analyze the candidate therapies that are currently undergoing clinical trials (i.e., the future pipeline). A search was performed on the website ClinicalTrials.gov  using the embedded search engine and key terms relating to 'cell therapy'. A total of 17,362 files were extracted (27 June 2010) and individually checked for relevance using the British Standard Institute (BSI) definition of 'cell therapy'  . The resulting 2724 trials were then categorized and core information collated, including: trial phase, cell source (autologous/allogeneic), current activity of the trial and responsible national regulatory agency. Key results included: near equal numbers of autologous (46%) and allogeneic (41%) trials; many of the trials are in the later stages - Phase I (49%), Phase II (40%) or Phase III (10%); and there are significantly larger numbers of transient cell therapies (50%) as opposed to permanent cell replacement (5%). This is the first time that the number and composition of all the cell therapy trials on ClinicalTrials.gov has been researched at the level of individual entries, analyzed and published. These data have important planning and resource allocation implications for translational scientists, clinicians, healthcare providers, businesses and governments.
The two questions, 'how many cell therapy clinical trials are there currently?' and 'what is their composition?' are frequently heard when discussing progress in the cell therapy sector. With a number of official databases available on the internet, such as ClinicalTrials.gov  , the answer should only be a few clicks away. Unfortunately, the reality is that obtaining accurate figures from the ClinicalTrials.gov database for a platform technology such as cell therapy requires a more sophisticated approach. The research described in the following text examined, at an individual level, every cell therapy trial in the ClinicalTrials.gov database, using a combination of an initial search with key terms to provide a coarse screen of the entire database, followed by an exceptionally high level of manual sorting of each of the listed results. The 'cleaned-up' data (i.e., those entries confirmed to be true cell therapy) were then analyzed to produce the first survey of the number and composition of the entire ClinicalTrials.gov cell therapy clinical trials listing.
The CTI is now firmly established as a billion-dollar business that is entirely distinct from the existing pillars of healthcare  : conventional pharmaceuticals, biologics and medical devices. An understanding of...