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Wednesday, December 10, 2008

Commercially Available Cell Therapies?

The week of December 2, 2008 I posed the following one-question survey:

1. How many unique cell therapy* products** do you believe are currently commercially available*** in the world?

* "cell therapy" is defined as any cell-based product involving live cells 'produced' ex vivo and used as part of the therapeutic (not just to create the product) including cell-mediated gene therapies.

** "products" is meant to exclude "mere" stem cell transplantation for any and all indications thus excluding the uses of SCT in regulated markets as well as unregulated SCT services - a "unique product" is one which is distinguishable in a material, technical way from another product (e.g., not just the same product marketed under a different name in different countries).

*** "commercially available" is meant to include any "product" a consumer can purchase whether or not it is part of a clinical trial (given that in some countries these are not mutually exclusive).

I distributed the survey to members of the following groups:
  • those who follow me on Twitter (currently a group 66 individuals sufficiently interested in cell therapy to follow my postings);
  • my connections on LinkedIn (a group of 203 professionals in or interested in the cell therapy sector);
  • the 110+ 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 1-10 to 71-80 in increments of 10 (e.g., 1-10, 11-20, etc).

Data, Assumptions, & Observations
There have been 8 responses to-date. This is a small n but I can't justify spamming my audience any more than I have so we'll have to work with this for now.

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.

25% (2 responders) believe there are 10 or fewer cell therapy products commercially available per my definitions. Another 25% responded 11-20. One person thought there were 30-something and another 40-something. A final 25% thought there were 50-some cell therapy products (as I defined them) currently commercially available worldwide.

Once again the quick and easy conclusion is that there is a significant lack of clarity around the current size or maturity of the cell therapy industry even amongst those in or following it closely and who consequently are - at the very least - a lot more informed about it than most.

This is admittedly not an easy question to answer because so much hinges on the definition of "cell therapies", whether or not to include the various types of stem cell transplantation products and/or services, and what constitutes a "unique" product (e.g., whether one autologous chondrocyte transplant product for cartilage repair is truly distinguishable from another and how one would define such distinctions).

Let's look at my data. I am currently tracking 700+ companies which I define as stakeholders in the cell therapy sector. This includes ~250 therapeutic companies [i] with ~340 cell-based therapeutic products in the market or in some stage of clinical or pre-clinical development [ii].

Approximately 67 of these 'products' are commercially available in at least one country. There are a number of observations worth noting about these 'products'.
  1. Not all of these 'products' have been 'approved' in 'regulated markets'. Some are simple stem cell transplants such as we see in the kind of unregulated clinics discussed in the recent issue of Cell Stem Cell (see the ISSCR Commentary article and a Correspondence article assessing the online portrayal of commercial stem cell transplantation retail ventures providing treatments in unregulated markets). Approximately 17 of the 67 are these unregulated stem cell transplantation “products” where companies are transplanting stem cells in clinics outside the US for a wide variety of diseases with little oversight or published data. I've assumed each such "clinic" has only one product (although that's almost impossible to discern in most instances) and I am certain there are more than 17 such clinics but accurately tracking them is not really one of my goals here.
  2. Roughly 14 of the 67 are very similar skin (e.g., wound repair/regeneration) products available in various countries;
  3. Approximately 10 of the 67 are very similar cartilage repair products available in various countries – many of which are autologous chondrocyte transplant (ACT) products; and
  4. In some instances, there are multiple inclusions of the same product commercially available through different companies in different countries sometimes under different brand names.

By way of external reference, in his recent publication in Tissue Engineering, M. Lysaght cites 47 companies with at least one commercial product and Proteus Venture Partners often refers to “over 25 FDA-approved cell therapies”.

My analysis of the data leads me to conclude that there are currently ~50 cell therapy products commercially available worldwide. Approximately 26 of these are uniquely distinguishable product types. Roughly 20% (depending on your interpretations) are cell therapies available in the United States. These include extracorporeal photopheresis (ECP) therapy performed using the UVAR XTS, Fusionary, Osteocel\Trinity, Epicel, Carticel, OrCel, Prokera, AmnioGraft, Dermagraft, and others.

50% of the respondents to my admittedly-small survey believe there are less than 20 unique cell therapy products commercially available worldwide. None selected the 20-some answer (the correct answer according to my data and analysis). From the highly-informed group of respondents, answers ranged from 1-60 products with a full 50% of respondents split evenly between the extreme opposite ends of the spectrum (1-10 vs 51-60).

Regardless of whether my numbers are right, the lack of consensus among those in the sector regarding the size and maturity of the cell therapy industry - or at the very least how one defines it - is clear. For those of us in the's up to us to fix this. More on that in an upcoming post...

[i] Estimates range from ~225 to ~300 therapeutic companies among what I define as ~700 stakeholder companies in the cell therapy industry. Sources: author’s database; Cell Therapy Pages (Connexon Communications); Proteus Venture Partners; Bionest Partners. Cell therapies and tissue engineering. February 2007; Burger SR. 2004. Cell and Gene Therapy - Challenges and Strategies for an Emerging Industry. Cell and Gene Therapy 5:9-14.

[ii] “Pre-clinical development” is defined to product in development prior to initiation of a phase I trial but not including products in the early research phase.


Frog said...

Thanks for that little research project. A lot of great information. I'll admit that I am surprised by the number of commercially available products out there.

Anonymous said...

Formerly Stem Cell Biotherapy, Cellulogix International Inc. under the direction of founder Dr. Casey Nabavi is now an established leader and also announces the launch of the new Adult Stem Cell treatment procedure referred to as “Apheresis” methodology which is known for its anti-aging qualities. A universal product from and for the patient donor.

For more information and a consultation with a scientist, please phone 514-448-2199

Lee Buckler said...

Thank you "anonymous" for your marketing message.

Note to readers (from Blog author):

Cellulogix is a company marketing stem cell therapies in the US but treating patients outside the US because their procedure is not approved by the FDA. On its own this is a perfectly acceptable practice.

There are reasons, however, to carefully investigate before deciding to be treated by such clinics. This is not to say I recommend you do not - merely that you do your own research before deciding.

Recommended reading would begin with the most recent issue of the journal Cell Stem Cell in which there is an article by the International Society for Stem Cell Research (ISSCR). In the ISSCR Commentary article they outlined their proposed Guidelines for the Clinical Translation of Stem Cells. Their proposed guidelines are available in full online at The recommendations include an insistence on expert evaluation and independent oversight, a thorough informed consent process, and transparency in reporting of clinical trial results. This is aimed at all those stem cell transplant clinics selling direct to consumers with little - if any - regulatory oversight, formal clinical trials, or peer-reviewed publications.

Complementing the release of the Guidelines, in a Correspondence article in the same issue of Cell Stem Cell, stem cell law and policy professor Tim Caulfield and colleagues published an assessment of the online portrayal of commercial stem cell transplantation retail ventures providing offshore treatments. Conclusion? They reveal most such clinics exaggerate claims and omit risks.

Cellulogix lists the following as "Treatable Conditions" on the home page of its website:
Becker Muscular Dystrophy
Brain Damage
Cardiac Disease
Cerebellar Ataxia
Cerebral Palsy
Crohn's Disease
Eye Diseases
Kidney Disease
Macular Degeneration
Multiple Sclerosis
Primary Lateral Sclerosis
Radiation Myelitis
Rheumatoid Arthritis
Spinal Cord Injuries
Transverse Myelitis

It is true that there are ongoing clinical trials testing the potential of various cell-based therapies for the treatment of many of these conditions. Many, however, have yet to be proven as these trials are not yet complete and the data not yet published.

I do not intend these remarks to be disparaging of Cellulogix or Dr. Nabavi. Indeed I have on this blog (see my post on August 6) been sympathetic to clinics who provide the kinds of services Cellulogix provides and the patients who have reason to use them. I have spoken with Dr. Nabavi and believe he intends good and not harm.

There is a noted tendency, however - in the opinion of many leading scientists and clinicians - for such clinics to make claims about the effectiveness of their procedures/products to cure conditions which are as yet unproven and many - if not all - such clinics have been reticent to provide such proof other than anecdotal reports.