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Thursday, November 29, 2012

Six steps to fighting non-compliant cell therapy treatments. --- The stuff of grey shades, spades, ivory towers and (ahem) balls.


Today an article entitled "Professors Critique Stem Cell Medical Tourism" appeared in the online version of The Harvard Crimson summarizing a recent panel discussion hosted in least in part by Harvard Law School assistant professor I. Glenn Cohen and University of Alberta law professor Timothy Caulfield.  The article concludes thusly:
The panelists emphasized that more accurate information should be provided to the public regarding stem cell treatments.
Certainly what Cohen and Caulfield concluded is true.  It has now been true for several years.  We keep saying it.  But are we listening to ourselves?  Are we doing anything meaningful to address this?  If so, is it enough?

Sadly (in my opinion, of course) the answer is 'not nearly enough'.*

For several years now, experts and organizations in the cell therapy sector have been saying that more must be done to educate and assist patients who are seeking stem cell or other cell-based treatments which do not comply with existing regulation and/or widely accepted medical or clinical research practices (hereafter called "non-compliant cell therapies").  

In my opinion, attempts to address this need by the sector's professional organizations, while important, have been unnecessarily feeble, not gone nearly far enough, and legitimately appear by many to be high-minded and pedantic. 

Almost all efforts to-date to address this issue by ISSCR, CIRM, ISCT and others including authors such as Caufield - as laudable and needed as they are - have been focused on helping distinguish between compliant and non-compliant treatments (and providers).  This is certainly much needed.  But what is left, I submit, is an even greater unmet need.

What almost all efforts to-date have failed to recognize or address is that where real help is needed is in helping patients distinguish between the many shades of grey among non-compliant treatments (and providers).

Emerging organizations like ICMS (now in partnership with AABB) have recognized and attempted to address this unmet need through a commitment to create some level of certification, accreditation or standardization of clinics participating in this business of selling non-compliant cell therapies.

While their intentions appear on-target as one meaningful way to address this unmet need and certainly their willingness to tackle this issue in a bold way is to be lauded, the ICMS is inexperienced and underfunded.  I remain hopeful that now through their new partnership with AABB they will be able to provide something that really addresses this unmet need but the jury remains out on whether they will succeed.

Anyone who has followed this blog and/or my threads on LinkedIn know I have been thinking about and discussing this issue for some time. In a desire to move to very concrete suggestions, I want to recommend the following 6 steps to my industry colleagues and professional organizations:

1.  50 shades of grey. Let's admit that this issue is not black-and-white but, as is almost always, involves a broad spectrum of grey in the middle.  

In addition to helping patients distinguish between compliant and non-compliant treatments (and providers) there are a lot of ways to help patients distinguish between non-compliant cell therapy  treatments (and providers) which are more or less risky.  

Let me use examples.  

On the one end of the non-compliant spectrum I would put forward a clinical like Okyanos Heart Institute which (as I understand it) intends to provide cell therapy treatments in the Bahamas to US patients using the Cytori system for cardiac conditions as soon as such treatments are perfectly legal and available to European patients but years before such treatments will be available in the US.  

Non-compliant?  Yes.  But certainly no evidence I'm aware of to support a belief that seeking treatment from them would be any more risky than travelling to Europe to receive the same treatment in a manner perfectly compliant with European regulations.

On the other end of the spectrum are the kinds of clinics highlighted recently by 60 minutes or which are the subject of ongoing lawsuits.

In between - in my opinion - are clinics like Stem Cell Institute and StemCellMD.

2.  Step out of the ivory tower.  Let's recognize that in certain circumstances patients are going to go pay for non-compliant cell therapies and we must do more to help these patients than simply shake our finger and tell them they mustn't.

For some, helping patients distinguish between the better and worst non-compliant clinics might involve a fair amount of nose-pinching but this is the equivalent of the methadone clinic for heroine addicts.  By supporting the better of two evils we are not endorsing it as 'good', we are simply recognizing it is better.

This is a recognition that we cannot just abandon people because they made (or are going to make) decisions with which we ultimately disapprove.   It is a recognition that sometimes the most righteous thing to do is not only to help people do what we would ideally want them to do but to help them do the best they can in their circumstances and on their terms - even terms with which we may ultimately disagree.

3.  A risk-based strategy.  Let's recognize that even the FDA triages their response to non-compliance and we would do well to do the same.  As a regulated industry we are perfectly comfortable with risk-based assessments and it should be applied here.  

Rather than treating all non-compliance as equally evil, let's apply some risk-based analysis to the situation and develop a strategy to root out the worst (highest-risk) offenders.  

4.  This is not just about tourism anymore - the problem has come home to roost. Let's recognize that this is no longer just a problem of patients leaving a regulated jurisdiction seeking a non-compliant treatment in a jurisdiction with no or more permissive regulation.  

Non-compliant treatments are growing rapidly even in the most highly regulated jurisdictions.  No where is this more true than in the United States.

5. Take responsibility.  Let's recognize that we cannot expect our regulatory enforcement agencies to do it all.  They are under-staffed and under-funded.  They - and the people we all serve - need our active participation in dealing with offenders and those risking patient safety.  

From a self-interested perspective, we owe it to our industry to help crack down on those who put the credibility and legitimacy of cell therapies at highest risk.

6.  Let's grow a pair and call a spade a spade.  If a non-compliant clinic is providing treatments that we believe represent a high-risk to patient safety and the industry's credibility, let's have the b*lls to call them on it.  They can't sue everyone.  

ISSCR backed down on their stem cell tourism initiative after being threatened by lawsuits. Who has stepped up in their absence?  Individual bloggers and authors like Paul Knoeplfer, Alexey Bresenev, Leigh Turner, and myself all who have been threatened with litigation several times for having the audacity to call certain non-compliant clinics out for what we deem - in our own risk-based analysis - to be the worst offenders.  

By way of example, several of my colleagues have recently committed to doing all they can do to call out David Steenblock and his non-compliant cell therapy treatments, many of which are provided at his clinic in California for a plethora of conditions.  In their opinion, many of his treatments represent some of worst examples of non-compliance in the United States right now.  There are many faces or fronts to his practice including,,, etc.

If, as an industry, we act with more cohesion (collaboratively applying a risk-based assessment of non-compliant clinics) and speak with a more cohesive voice in terms of calling out those clinics and treatments which we conclude pose the greatest risk based on an objective set of criterion, this will present a multi-pronged, formidable and existential threat to clinics that they can't ignore or threaten away.


I will be taking these 6 recommendations to any organization who will listen.  I hope you will consider doing the same.

In the meantime - as always - I welcome your comments.


* This is my opinion not necessarily the opinion of any clients I represent or organizations I serve. Judge me - not them - accordingly.

Monday, November 19, 2012

Updated cell therapy index portfolio



This an update of a post originally released October 18.  What has changed is we've added four companies (shown below in red).


There are certainly analysts, traders, and brokers who cover the public segment of the cell therapy industry with much more expertise and experience than I -- among them is Henry W. McCusker of Scimitar Equity (@henryw09 on Twitter).

As part of our tracking of the sector's financial data and trends, we have created a portfolio of public cell therapy companies comprised of the 34 companies listed below (linked to their page on Google Finance).

I've been watching this portfolio since the beginning of the calendar year (January 2012) and have been commenting on its performance as against some of the major indices.  (Oct 8 post and Aug 10 post).  

In addition to these occasional reports I will occasionally report on Twitter (@celltherapyblog) on the stocks in this portfolio which are enjoying the largest gains/losses and which have the largest/smallest trading volumes.  From time-to-time I will aggregate this content into a blog post.     

Symbol         Name
ASTM        Aastrom Biosciences, Inc.
ACTC  Advanced Cell Technology, Inc.
ABH          Angel Biotechnology Holdings Plc
ATHX  Athersys, Inc.
AVMXY Avita Medical Ltd
BHRT  Bioheart, Inc.
BLFS BioLife Solutions, Inc.
BTX          BioTime, Inc.
CBAI         Cord Blood America Inc.
CNDO        Coronado Biosciences Inc.**
CCEL CRYO-CELL International, Inc.
CMXI Cytomedix, Inc.
CYTX Cytori Therapeutics Inc. (USA)
DNDN Dendreon Corporation
FCSC  Fibrocell Science Inc
IMUC  ImmunoCellular Therapeutics Ltd
ISCO International Stem Cell Corp
ISLT          Islet Sciences Inc
LVCLY       Living Cell Technologies **
MEDS Medistem Inc.
MSB          Mesoblast limited
MLM          Molecular Medicine SpA **
NBS          Neostem Inc.
CUR          Neuralstem, Inc.
NLNK  NewLink Genetics Corp
NWBO       Northwest Biotherapeutics, Inc.
OPXA  Opexa Therapeutics Inc.
OSIR Osiris Therapeutics, Inc.
PSTI          Pluristem Therapeutics Inc.
RENE ReNeuron Group Plc
REPCF       RepliCel Life Sciences Inc. **
STEM  StemCells, Inc.
TNGN Tengion, Inc.
KOOL ThermoGenesis Corp.
TIG           Tigenix NV

** added after original index portfolio was created October 18

Thursday, November 8, 2012

Cell Therapy Industry Group Welcomes its 4,000th member


I'm pleased to point out that today the LinkedIn Cell Therapy Industry Group welcomed its 4,000th member today. 

The Cell Therapy Industry group was created to serve as a network of those in the cell therapy industry. The group acts as a vehicle for referrals, networking, information, and facilitating collaboration.  The group's focus is on the activities of companies in and serving the space.

The group began in July 2008. It took 2.5 years to reach the first 1000 members, 9 mos to reach 2,0000, 6 months to reach 3,000, and 6 months to meet today's 4,000 member mark. 

As is typical, there is a very high percentage of passive participants but the group benefits from an avid group of participants who post, share, exchange, and debate on a range of topics ranging from regulatory, clinical, commercial, scientific, manufacturing, financial, and other topics of interest to the group.

As the group has grown I've noted two trends pertaining to the composition of the membership:  (a) having tapped out the c-level suite, growth is increasingly coming from down the hierarchy of the corporate food chain and including those in the operational trenches, and (b) a much higher ratio of new members of late is from outside the US, presumably as LinkedIn increasingly penetrates OUS markets.

We strive hard to maintain the quality of the participation by screening each applicant, deleting off-topic posts, moving promotional posts to the "promotions" tab" and encouraging a balance of news-sharing with useful discussion threads.

I'm proud to say the group has become a vibrant and valuable part of the sector due to the hard work and contributions of all involved.    

As these kind of virtual networks become exponentially larger and provide different value than the professional societies representing the sector, I will be fascinated to watch if and how this affects how sectors like our interact and how this will impact the traditional value proposition of member-based professional societies.

If you are not a member of the LinkedIn Cell Therapy Industry Group, check it out.


Thursday, November 1, 2012

GEN's "Cellular Therapy Wave Finally Cresting". An overview and data set.


We first provided a listing (with very few details) of industry-sponsored late-stage (pivotal, phase 3 and 2/3) cell therapy clinical trials on this blog late last year (see the posting here).

We are now pleased to we have worked with Genetic Engineering and Biotechnology News and Enal Razvi of Select Biosciences to provide an updated (as of June 2012) and more detailed listing of industry-sponsored late-stage (pivotal, phase 3 and 2/3) cell therapy clinical trials (excluding cell-based immunotherapies which we intend to cover in a follow-up article).

A link to the listing can be found in an article published today entitled "Cellular Therapy Wave Finally Cresting" found in the November 1, 2012 issue of GEN.  

While not my favorite title, the article is a brief - but we hope useful - overview of the sector and its pipeline.  It also provides a snapshot of the cell therapy products already in commercial distribution. 

Some will quibble about the numbers. Certainly others have published larger revenue numbers, for instance, but in our view these have almost always included revenue from cord blood banking which we have excluded.

We encourage you to read the article but for convenience here is a direct link to the spreadsheet.  Of course it's already out-dated but we'll do an update again soon here on this blog.

Hope this is useful.


[post-publication clarification]: Regarding the last sentence of the article, this should read:

"No cell therapy products approved 2002-8 vs 12 cell therapy products approved 2009-12."

2009-12 saw 9 regulatory approvals of industry-backed products : 

  • ChondroCelect from TiGenix (EU),
  • Provenge from Dendreon (US), 
  • laViv from Fibrocell Sciences (US), 
  • GINTUIT from Organogenesis (US), 
  • HeartcelliGram-AMI from Pharmicell (S. Korea), 
  • Cupistem from Anterogen (S. Korea), 
  • Cartistem from Medipost (S. Korea),
  • Prochymal from Osiris (Canada) and
  • Prochymal from Osiris (NZ). 

This year also saw 3 regulatory (FDA) approval of two products from non-profits:

  • HemaCord from New York Blood Center (US), 
  • HPC, Cord Blood* from Clinimmune Labs, University of Colorado Cord Blood Bank (US)
  • DUCORD from Duke University

* please also note the correction of "HPC, Cord Blood" which is the correct product name incorrectly stated as "Clinimmune" in the article.