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Monday, February 23, 2009

Cell therapy products vs cell therapy products; stem cell clinics vs stem cell clinics

I will find a new favorite topic in due course but for now this drum needs a little more beating. The feedback I've been getting is helping me get to the heart of the matter.

Here on these very ephemeral 'pages', I have suggested that the cell therapy industry is not well informed about how many cell therapy products/treatments are currently commercially available worldwide. Some in the industry argue that many of the products I include are not 'real' cell therapy products/treatments (more on that in a minute...) so they shouldn't really be counted.

I have also been sympathetic to unregulated stem cell clinics around the world and the patients that access them while advocating for and supporting the implementation of more stringent ethics, business practices, and therapeutic regulation.

Both sides of this debate have provided useful feedback and perspectives.

Ardent supporters of unregulated stem cell clinics, accuse me of criticizing their pioneering efforts to treat patients that the medical industry can't or won't treat. They believe I tow the party line on the need for rigorous scientific evidence, regulated clinical trials, peer-reviewed publications and a therapeutics review/approval framework. They accuse this view as archaic and not permissive of allowing patients the right to determine their own treatment pathway particularly when there is no harm in the cell-based products being sold.

On the other hand, some of my peers in the cell therapy industry believe I am doing a disservice to the industry by acknowledging that these unregulated stem cell clinics are part of this sector and are doing a robust business with an increasingly large number of American patients.

I happen to believe that just because a company is operating in an unregulated market does not necessarily mean they are unethical, that it may not be fair to characterize all 'stem cell clinics' in unrelated markets as 'the same', and there may be some of these clinics which are conducting business is ways which should be considered acceptable.

In the spirit of informed and informative discussion, I have 4 comments - not in an attempt to be defensive or argumentative but to keep what I believe to be a valuable discussion flowing.

1. There are those that want to believe they know the products available in the market and if there is any gap between their number and mine it is entirely comprised of unregulated products in unregulated markets.
Wrong. A large percentage of this gap is comprised of regulated products.

According to my data there continues to be a wide gap between the perception of how many cell therapy products are commercially available in markets around the world and how many there actually are.

For example.

At the 2009 Phacilitate Cell & Gene Therapy Forum the very first speaker of the conference inferred (by mistake) that there was only one cell or gene therapy product approved in the world. He meant gene therapy products. Based on that presentation, however, there were delegates at the conference - people who work in the cell therapy industry and should clearly know better - who were tempted to believe there was only one approved cell or gene therapy product in the world. These are people who would know better if they thought about it for a minute. That's an egregious example but watch the interviews of delegates by and you will see most experts in the field believe there is but a handful of approved cell therapy products worldwide.

According to data in a recent TERMIS newsletter, for example, there are 10 cell therapy products in Korea alone that have been approved by the Korean FDA (KFDA) for treatment of various conditions. This is a regulated market and these are regulated products owned and sold by companies. According to my data there are at least another ten approved products between Australia and Germany. Again both regulated markets. That is already more approved products than most people believe are available in the world.

So yes... I certainly have included unregulated products/treatments in previous data.
Nonetheless, but even when I exclude these, the number is larger than most believe.

This is the gap I want to close the most. The more I interact with people now as they react to my data and perspective, I believe I am starting to get a clearer picture about what is behind some of this gap.

2. There may be some who disagree with my definition of 'product' and/or use of the adjective 'approved'.

I have to-date considered it a 'product' whether it is cGMP or cGTP and whether or not it is the subject of an IND/BLA (or equivalent) . I'm certain the FDA CBER shares that view. 'Approval' of a product does not only come by way of BLA/MAA (or equivalent), it also comes from tacit approval in regulated markets where a product falls within a category which is allowed without formal application for review and approval by the regulatory authority.

So, for example, the Xcell-Center clinic in Germany currently provides bone-marrow derived, minimally-manipulated stem cell 'products' under the authority of the German regulatory authorities. They do so on a commercial basis. The products ('treatment' some might prefer) are currently being sold to treat patients with the following (degenerative) diseases: ALS, alzheimer's disease, cardiovascular diseases, cerebral palsy, diabetes mellitus (type 1 & type 2), erectile dysfunction, macular degeneration, multiple sclerosis, osteoarthritis, Parkinson's disease, spinal cord injuries, and stroke.

It's not clear to me whether there is any unique IP around these treatments and the business model here is clearly more of service model than product model but the products/treatment may each be somewhat different and may be administered differently depending on the indication. By at least some people's definition, that is at least 12 different 'products' - all being sold with complete regulatory approval (though not an MAA because of the nature of the products/treatments) - with prices starting at around 12,000 euros depending on the procedures required. Some might argue it's only 1 product but isn't it at least that?

In regulated markets stem cell transplantation is a regulated cell therapy in that there is a regulatory framework within which the service falls and one cannot simply transplant stem cells for any indication one wants (let's say pump them into the brain) even if one simply takes them from the bone marrow, isolates the cells of interest and puts them straight back in.

Stem cell transplantation is commonly perceived to be a medical procedure rather than a product but this does not make it any less a cell therapy. If the cells are more than minimally manipulated they become defined as a cGMP product (in the US). At what point in between does it cross the line and become a 'product'?

Is it whether or not there is IP around the product or process used to make it? Is it whether or not it is characterized primarilyy as a 'service' rather than a 'product'? Certainly some cGMP products will require an extensive clinical service model. It certainly seems to me that at least some cGTP products will be no less cell therapy products than a cGMP product in that they may be the subject of IP and may involve no more of a service element than some cGMP cell therapies.

3. There must be a way to have an informed discussion in which we distinguish between cell therapy products and cell therapy products; stem cell clinics and stem cell clinics.

The Xcell-Center, for instance, operating under complete regulated authority in a regulated market providing treatments for a limited number of conditions and conducting at least some type of long-term follow-up must be considered in a substantially different light than a clinic providing stem cell injections for almost any condition in an unregulated market, without any long-term follow-up, and with no transparency as to their product or process.

I believe the ISSCR and many others share my desire to create the foundation for what will become for patients - in an informed and global medical market - a very important discussion upon which they can discriminate between legit and shoddy clinics and make informed decisions.

4. As for those clinics arguing they should not be not be subjected to standards requiring
rigorous scientific evidence, regulated clinical trials, external audits, and peer-reviewed publications within a therapeutics review/approval framework... please stop. Stop now.

Cell therapy is not inherently safe regardless of what kind of cells you use, how you treat them, and what you do with them. As recent reports suggest, this can be as dangerous and deadly as any drug.


As to point #1, I am still working on my list of products which I hope to publish soon for all to see and review.

As to point #2, I hope some of you will comment on how to best define 'product' for this discussion. There is a similar and very informative discussion thread in the LinkedIn Cell Therapy Industry Group focused around different therapeutic business models. Although the question is framed differently, the discussion hinges on similar distinctions and issues.

As always... I reserve the right to be wrong. I welcome any and all feedback. My only goal here is an informed and informative exchange of ideas and information.


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