Updated listing of late-stage industry-sponsored cell therapy trials.

We've just updated our listing of industry-sponsored pivotal, ph3 and 2/3 cell therapy trials.  See the posting here.

Late-stage industry-sponsored cell therapy trials

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Updated 22 August 2012 (originally posted December 2011)

At Cell Therapy Group, we track cell therapy industry activity globally but  particularly activity related to the clinical development and commercialization of cell based therapeutic products globally.

Below is a synopsis of the pivotal, phase 3 or 2/3 industry-sponsored cell therapy trials we are currently tracking globally.  

There is a database behind this that includes more data fields like therapeutic category, cell/tissue source, cell type, expansion, indication, expected completion date, clinical trial site locations, etc. 

Industry-sponsored pivotal, phase 3 or 2/3 trials*
    *active or expected to be active in 2012 

Aastrom                         

• Ixmyelocel-T

Argos

• Arcelis - AGS-003

Avita Medical

• ReCell

Baxter                           

• ACT34-CMI

Bone Therapeutics           

• PROEB

Cardio3 Biosciences         

• C-CURE

Cell Medica                     

• adoptive cellular therapy

CellCoTec

• INSTRUCT

CellSeed France               

• CAOMECS (Cultured Autologous Oral Mucosal Epithelial Cell-Sheet)

co.don                            

• co.don chondrosphere

Cook Myosite                  

• AMDC

Cytori                            

• ADRCs

DePuy Mitek

• CAIS

ERYtech Pharma              

• GRASPA

GamidaCell - Teva           

• StemEx

Harvest Technologies       

• SmartPReP 2 BMAC

Healthpoint                     

• HP802-247

Histogenics                     

• NeoCart

Innocell

• Immunecell-LC

Innovacell                        

• IES13 (Urocell?)

ISTO Technologies            

• DeNovo ET

JW Creagene

• CreaVax-RCC

Kiaidis Pharma                

• ATIR

MacroCure                       

• CureXcell

Mesoblast

• Revascor    

Miltenyi                          

• CliniMACS CD34 Selection System

MolMed                          

• TK

Newlink Genetics             

• HyperAcute Pancreas

NKBio

• Biocell Natural Killer Mixture

Northwest Biotherapeutics

• DCVax-L

NovaRx                          

• Lucanix

NuVasive                        

• Osteocel Plus

Osiris                            

• Prochymal

Pharmicell

• Heartcellgram-AMI

Prima Biomed                  

• Cvac

Sanofi (Genzyme)

• MACI

SOTIO

• DVAC/Pca

Tigenix (Cellerix)

• Cx601

The Hawkins Foundation (in collaboration with Depuy Mitek

• CAIS

TissueTec

• amniotic membrane

Some of the products listed above are in more than one clinical trial - different regions and/or different indications.

In total we are tracking 48 industry sponsored, active (or expected to be active 2012) cell therapy trials in pivotal, ph III or II/III expected to involve over 11,000 patients.  Some of the products listed above are the subject of multiple trials.

The 50 trials are sponsored by 40 companies.  The trials roughly break down as follows:

• 59% are autologous
• 45% are allogeneic
• Two are gene-modified allogeneic
• One involves autologous and allogeneic cells (we counted it for both)

• 30% for oncology or related indications
• 13% for cardiac-related indications
• 13% for non-cardiovascular (PAD, CLI, VLU, etc)  & wound 
• 34% (6) for cartilage and bone repair

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I encourage readers to comment below or email direct with any errors and/or omissions.

How mature is the cell therapy industry? Do we know?

Survey
The week of November 17, 2008 I posed the following one-question survey:

How many phase III or pivotal industry-sponsored cell or gene therapy clinical trials do you believe are currently ongoing in the world?

to members of the following groups:

• those who follow me on Twitter (currently a group 43 individuals sufficiently interested in cell therapy to follow my postings);
• my connections on LinkedIn (a group of 188 professionals in or interested in the cell therapy sector);
  
• the 90 or so members of the LinkedIn Cell Therapy Industry Group;
• readers of this Blog; and
  
• the roughly 55 members of ISCT's cell therapy commercialization committee.
  

There was no control on multiple responses and no restriction on participation. Participants were forced to choose only one answer among responses ranging from 1-5 to 50+ in increments of 5 (e.g., 1-5, 5-10, etc).

Data, Assumptions, & Observations
There have been 18 responses to-date.

If we assume that only members of the groups listed above participated in the survey, then it is reasonable to characterize the participants, at the very least, as informed participants who know more about the cell therapy industry than the average person. I will also assume that participants only voted once.

Based on these assumptions, the data leads to one simple, overarching observation:

There is significant divergence of perception (and perhaps misconception) about the maturity of the cell therapy industry even among those in and/or informed about the sector.

My database tells me there are 33 industry-sponsored cell or cell-mediated gene therapy trials currently underway around the world.

45% of survey participants answered fewer than 15 to the survey question; 33% indicated more than 45. In fact, only 1 participant (6%) do-date has chosen the category of 30-35.

Analysis
Admittedly this is an unscientific and informal survey. Furthermore there is likely some margin of error to account for different interpretations of what "industry-sponsored" means and/or what types of products should be included/excluded as "cell or gene therapies" since none of these terms were defined for participants.

Certainly in my day-to-day dialogue with people in the industry, the typical opinion is that there are but a handful of late-stage cell therapy trials (far fewer than what my data tells me actually exists). Having said that, when I asked a colleague in the industry yesterday to respond verbally to the survey question, they said "350" ( and then pulled back to say "maybe 200").

There is no doubt my data likely lacks a material number of industry-sponsored, phase III or pivotal gene therapy trials - given that the focus of my database is cell therapy and thus typically only includes cell-mediated gene therapies. This exposes a weakness in comparing the survey results to my data in that the survey related to "cell or gene therapy" while my data likely only reflects a small subset of gene therapy trials.

There is also without doubt more than 33 cell therapy or cell-mediated gene therapy clinical trials currently underway in the world with many of the indicia of a "late-stage" trial if one includes what is happening in markets where such trials or products are currently unregulated. I have not included these in my data because there is no mechanism for identifying whether such trials are, indeed, phase III or pivotal as there is no mechanism as yet in those markets for formal review or approval of these trials and/or products.

Nevertheless, what conclusions can/should be drawn?

• There is much divergence of perception on the maturity of the cell/gene therapy industry even amongst those in the sector.
• Even if my data is inaccurate, compared to whatever the real number is there is a significant % of industry professionals who are suffering from significant misconceptions about the true number of late-stage industry-sponsored trials in the sector.
  
• Good data on the state of the cell/gene therapy industry is not readily accessible.
• If those in the industry lack consensus on the state of the sector, it is no wonder investors and others outside the industry have mixed opinions on the matter.
• We need to do a much better job as a sector in gathering and communicating solid data on the commercial side of the cell/gene therapy industry.
  

For those interested in the data, here's how it looks:


Industry-Sponsored Cell or Gene Therapy Phase III or Pivotal Clincial Trials (Nov 2008) - DocStoc

Coming soon...a similar discussion on the number of cell therapy products currently commercially available around the world.

In the interim, if any of you have data relevant to this discussion, please share...