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Friday, June 26, 2009

Cell Therapy Industry HiLites 2009-06-26

Let's get the public services announcement out of the way right up front.
  • Curiously, the Regenerative Medicine Foundation (I know. I hadn't heard of it either until now.) has announced that its "Translational Issues in Regenerative Medicine" conference is the "premiere event for leaders in regenerative medicine translation, representing academic research, clinical, healthcare policy, regulatory, reimbursement, investment and biotechnology industry interests." Curious because it has yet to ever occur. That being said, if they do what they promise, it may just well be. I'm trying to learn more and when I do I'll let you know. Meantime, this is about all I know:
Spring 2010
Twin City Quarter
Winston-Salem, NC

" facilitate business development opportunities, the conference will feature a venture forum including 15 regenerative medicine companies, as well as partnering sessions."

Do yourself a favor. Stop monkeying around with home-made cryopreservation brew or, for than matter, any media not built to minimize cellular damage from freezing and thawing.

Do your cells a favor. Try CryoStore and HypoThermosol.

I'm dedicating this issue of Cell Therapy Industry HiLites to
TiGenix NV (NYSE EURONEXT: TIG). TiGenix wins the Cell Therapy Group "kick-ass cell therapy company of the week" award which is, of course, something I just made up but they had have a great week raising several million dollars, announcing plans for a new European facility, and receiving a positive opinion from the EMEA CHMP which all but assures them of an MAA within 90 days applicable in 30 European countries. See details below.


has secured the necessary financing to fund the set-up of a GMP manufacturing facility in Europe through a private placement for a total amount of EUR 5,4 million. The investment agreement was subscribed by NV Industriebank LIOF, Particon BV, Limburg Ventures BV and LRM NV. The funds will be used to set up a new cell expansion facility in Europe for the commercial production of its cell based products in the Netherlands. The financing for the facility has been secured through a private placement. After carefully evaluating a number of options throughout Europe, taking into consideration technical, logistical, regulatory and financial criteria, TiGenix has selected a building on the Chemelot Campus, near to Maastricht, the Netherlands, to locate its new cell expansion facility. The site is centrally located in TiGenix’ key European markets, in a region that is strong in distribution and (bio)logistics and that is highly committed to develop as a transnational knowledge centre in life sciences and regenerative medicine.

This financing has now been completed following the execution of a long term lease agreement for the facility in question.
After raising $40+ million last year and over $14 million so far this year, Cytori Therapeutics Inc (NASDAQ: CYTX) has announced it has entered into an agreement to raise up to another $20 million over the next year. The company announced recently that it had raised $850,000 in what is described as an agreement by Orlando, FL-based fund, Seaside 88, to buy 7.15 million shares over the next year by buying a specified allotment every two weeks at an agreed discount of the then current stock price. The agreement could being in roughly $20 million for the company depending on share price. Cytori intends to use the proceeds to "accelerate marketing efforts and continue our investment in clinical trials to broaden the number of potential applications for which the Celution® System may be applied".
A bankruptcy court hearing is scheduled for July 6 in which Isolagen Inc. (AMEX:ILE) will request final approval of its debtor-in-possession financing plan under Chapter 11 bankruptcy reorganization protection.
Always a master at the art of spinning good profile, NeoStem, Inc. (NYSE: NBS) (AMEX: NBS), in the business of collection, processing and long-term storage of adult stem cells for future medical need, garnered a feature in Gene Marcial's prominent Business Week column, "Inside Wall Street," on June 18, 2009. The same week saw the company profiled in a piece on the investment potential for adult stem cell businesses in a Fortune Magazine article distributed by The company has been on a deal-making, press release and publications tear the past few months. "Big plans by tiny NeoStem (NBS)," Mr. Marcial observed, "have helped catapult its stock to $2.27 a share from 50 cents on March 9." It must be the plans because the company which has been doing what it does since 2006, generated a mere $45,100 in total revenuefor the three months ended March 31, 2009. The good news is that is up from the $700 generated in the three months ended March 31, 2008.

The company also announced another deal saying it has signed an exclusive 10-year agreement with Enhance BioMedical Holdings Limited, a Shanghai corporation, to develop a Stem Cell Collection and Treatment Network using NeoStem's "technologies" Taiwan, Shanghai and the Chinese provinces of Jiangsu, Zhejiang, Fujian, Anhui and Jiangxi. Enhance BioMedical Holdings is a subsidiary of Enhance Holding Corporation, a multinational conglomerate whose CEO, Jackson Ling, recently invested $5 million in an $11-million private placement financing for NeoStem.


Osiris Therapeutics, Inc. (NASDAQ:OSIR) announced six-month interim data from a Phase II clinical trial evaluating Prochymal, the Company's formulation of adult mesenchymal stem cells, for the treatment of chronic obstructive pulmonary disease (COPD). The trial failed to demonstrate an improvement in pulmonary function in COPD patients. The company, quickly becoming practised at spinning what might be considered bad news, said they were pleased the data did provide additional evidence of safety for the product in addition to demonstrating significantly decreased inflammation in the COPD patients. Sixty-two patients were enrolled and are being followed for two years in the placebo-controlled study.
Pluristem Therapeutics Inc. (NasdaqCM:PSTI) (DAX: PJT) received approval from the Paul Ehrlich Institute to begin clinical trials evaluating the company’s placental-derived adherent stromal cell product, PLX-PAD, for the treatment of critical limb ischemia.
Aastrom Biosciences’ (NASDAQ:ASTM) trial of its cell therapy for severe chronic heart failure was released from a clinical hold by the FDA. The the agency determined the therapy did not cause the death of a participant which resulted in the hold in May.
The University of Pittsburgh Cancer Institute began enrolling patients in a Phase III study evaluating the safety and efficacy of Gamida Cell Ltd's StemEx cord blood stem cell therapy for the treatment of blood cancers.
Having received regulatory approval late last year to proceed with its clinical trial in New Zealand, Living Cell Technologies Limited (ASX:LCT; OTCQX:LVCLY) CEO Paul Tan said enrolment of patients should be complete in about two months to begin testing their pig islet cells implanted in humans to treat diabetes.
Neuralstem, Inc. (NYSE Amex: CUR) has entered into a sponsored research agreement with the China Medical University & Hospital of Taiwan, in Taichung, Taiwan, to prepare for a human clinical trial using Neuralstem’s human spinal cord neural stem cells to treat stroke patients. The therapy will focus on patients whose post-stroke symptoms, including complete or partial paralysis, have stopped improving more than six months after an ischemic stroke.


Not to be outdone by finance and operational teams, the regulatory team at TiGenix also announced reason to celebrate this week. TiGenix (NYSE EURONEXT: TIG) announced that the company received a positive opinion from the Committee for Advanced Therapies (CAT) and the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMEA) on the European Marketing Authorisation application (MAA) for
its lead product ChondroCelect.

CHMP opinions are forwarded to the European Commission for adoption of the final community Marketing Authorisation, which is typically issued between 60 and 90 days from adoption of the CHMP opinion. I'm told that European Commission generally follows the advice of the CHMP but, as Dendreon, there's little reason to celebrate until the formal approval is formalized. For the EMEA take and related info on the decision, click here.

Upon approval, ChondroCelect will be, I believe, the first product approved under the new ATMP regulatory framework. It will certainly be the first cell based product to be centrally approved in the 27 member states of the European Union as well as in Iceland, Lichtenstein and Norway under the new Advanced-Therapy Medicinal Products (ATMP) legislation.
Further to my March 31 speculation about who might be the buyer of York Pharma plc (and its two cell therapy products on the market) and my April 13 post that ULURU Inc. (NYSE Amex: ULU) had signed a non-binding offer letter to acquire the company, ULURU Inc. (NYSE Amex: ULU) has announced that at this time discussions to acquire York Pharma, plc have been terminated. ULURU has notified York that the $1 million USD line of credit it had extended York are due plus accrud interest. The revolving credit facility is secured by substantially all of the assets of York and its subsidiaries. York is now actively pursuing alternative funding facilities in order to repay the line of credit within 30 days and to meet its ongoing working capital requirements.

Meanwhile, rumor has it the company is already in talks with another potential suitor.
Here are a couple hints for your next press release. It's rarely newsworthy to have as the primary point of your release an announcement about what you are going to do, rather than what you've done; secondly, don't announce seemingly contradictory information. I'm just saying....

Stem Cell Therapy International, Inc. (OTCBB: SCII), through its wholly owned subsidiary AmStem International, Inc., announced that it has "started the process" to enable it distribute Histostem's products in anticipation of the finalization of the merger between Stem Cell Therapy International, Inc. and Histostem. This is intented, according to the SCTI President and CEO, to "enable AmStem International, Inc. to begin US distribution and immediately bolster the company's cash flow". However, the product has yet to be the subject of a clinical study in the U.S. for which they are seeking "investigators...of the highest caliber, to compare its Stem Cell Facial Cream with others on the market."

The companies reportedly continue "to work on the necessary steps to finalize the merger, and both sides are working diligently to manage its many details."
Community Blood Services has entered into a licensing agreement with AuxoCell Laboratories, Inc. to use AuxoCell’s exclusive patent rights and propriety process to extract fetal mesenchymal stem cells from the Wharton’s Jelly of donated umbilical cords, allowing Community Blood Services to operate the first Wharton’s Jelly derived stem cell bank in the United States.
After netting $221 million last month by nearly 12 million shares at $19.20 apiece, Dendreon Therapeutics Inc (NASDAQ:DNDN) expects to spend up to $50 million in a two-phase expansion of its manufacturing facility in Morris Plains, New Jersey. Monday in a regulatory filing. Additional quality control laboratories, data center, training areas, infrastructure and offices are scheduled to by done by mid-December. The final phase, with additional manufacturing clean-room work stations, production support areas, warehouse, infrastructure and offices, is to be substantially complete by April 23 of next year. Dendreon finished the initial build-out of its 158,000-square-foot facility in July 2006, before its FDA application was tabled to await additional clinical results.
Apparently yet another way has been invented to derive stem cells from placenta - reportedly a source much richer in stem cells than umbilical cord blood. The inventors are looking for commercial partners.
Cell Genesys, Inc. (NASDAQ:CEGE) - the company that just won't go away - is still pursuing "strategic alternatives". Meanwhile, it expects its cash balance will be approximately $36 million upon imminent completetion of its stock exchange offer.


This week's issue of Science has a special focus on stem cells including a piece on "" by Donald W. Fink, Jr. of FDA, CBER, an article on stem cell tourism and 3 reviews.
26 June 2009. Vol 324, Issue 5935, Pages 1603-1754
The Production Assistance for Cellular Therapies Group (PACT) Educational Web Seminar: Topic: "Deviation Management of Type 351 & 361 Cell Products" Date: Thursday, July 16, 2009 Time: 12:00pm-1:00pm (Eastern US Time) Register is now open for this Web Seminar at


Thanks again to BioLife Solutons. Congratulations to Tigenix. May the force be with all of you and for those going to ISSCR later this travels and please try not to enjoy Barcelona too much without me!

Tuesday, June 23, 2009

A Therapeutic Long Tail. Does Cell Therapy Fit the Model? (Part II)

...this is a continuation of Part I...

So are Personalized Medicines the Long Tail of the Therapeutics Industry?

In 2006, Chris Anderson, Editor-in-Chief of Wired magazine, released "The Long Tail" – a book describing the Web 2.0-induced business-to-consumer economics (r)evolution enabling businesses to now deliver product at outrageously affordable prices to global micro-markets.[i] The book quickly became one of the most influential business books of the ‘new economy’.

Indeed this flat new world – as Thomas L. Friedman describes it[ii] – is all about micro-trends and micro-markets, empowering small companies and niche products to participate successfully in the marketplace along with Fortune 100 companies and blockbuster products. More than that, this paradigm shift is forcing product and service companies to revolutionize their business and pricing models, the latest and rabidly successful of which is the pricing model of “Free!”. The blockbuster-based business model that depends on the economics of a ‘hit’ (whether it’s a platinum-selling CD or a ‘blockbuster’ drug) is no longer seen to be the only way to make money and, indeed, may not have any future at all.

To date, this internet-driven Long Tail (r)evolution has had its largest impact on – if not been largely restricted to – the retail sector. Business-to-consumer (B2C) companies are now enabled like never before to profitably supply unique global products to meet customer demands. In this respect, the (r)evolution has had a visible impact on the retail side of the pharmaceutical and biotech industries. However the ongoing realization of “personalized products” as the future of medicine for consumers seems a perfect fit for the Long Tail economic model.

But does this apply to therapeutics?

To the extent that the Long Tail economy is less about the mass market and more about businesses succeeding in serving niche markets with unique and/or customized products, the analogy fits.

Certainly, it would appear the sun is setting on the "blockbuster hit" days of the therapeutics industry. In June 2008 Genentech's Susan Desmond-Hellmann, president of product development, when outlining the biotech giant's development strategy to a group at the Goldman Sachs healthcare conference, told the group that the blockbuster drugs of the past may never be duplicated. “Future drugs,” she stated, “will cater to a smaller, better defined group of patients” and “the days of big cholesterol-lowering drugs for millions of people may be behind us.” (link).

In a June 8, 2009 post, authors of the IN VIVO Blog stated that pharma "managers these days are learning to leverage only very modest top-line growth into faster bottom-line growth through cost-savings, bolt-on acquisitions, and rapid introductions of incrementally-improved new products. The traditional, high-risk, high-growth R&D-based model is gone, in case you hadn't noticed."

The second indication that personalized medicine fits with the Long Tail supply-demand model is that therapeutics can now be customized taking advantage of personalizing elements such as HLA-typing and advances in genetic, diagnostic, and discovery testing that will permit us to more effectively both pre-select which medicines are best suited for which patient sub-groups and develop therapies that are better suited to particular genetic groups. On the clinical end of the spectrum, advances in theragnostic tools and surrogate biomarkers (e.g., imaging) are allowing clinicians to much more rapidly assess personal responses to treatment and adjust their therapeutic regimines accordingly before any symptomtic response can be observed.

The ability to match therapeutics to genetic make-up, disease stage/presentation, or predisposition will allow the provision of treatments with more confidence in their expected efficacy, fewer patient-specific adverse reactions, and result in cost savings to the health care industry and a better return to fund the premium for the development of such products.

In a recent interview with Xconomy's Luke Timmerman, Alan Frazier, founder and managing partner of Seattle-based Frazier Healthcare Ventures, one of the world’s biggest life sciences venture capital funds, said,

The trend is toward smart pharmaceuticals that address smaller populations, narrower indications, with better results. It’s based on a better understanding of disease from genomics and proteomics. You’ll see more targeted drug delivery. You’ll have incredibly focused pharmaceuticals, which will take care of some of the safety issues you read about.

Does the model apply to cell therapies?

At the intersection of the personalized and regenerative medicine sectors, lies the therapeutic industry’s response to (or reflection of) both the demand for and the ability to deliver a more personalized therapeutic to the consumer (the patient).

If the tail of the therapeutics market is being stretched longer to include products better targeted to smaller markets, this therapeutics Long Tail will almost certainly include regenerative medicine (regen 2.0) products and many of these will be cell-based therapies.

The cell therapy sector now encompasses the convergence of three distinct technologies, i.e. cell transplantation, tissue engineering, and biomaterials. Often also drawn in are elements of gene and molecular (e.g., proteins and antibodies) therapies.

Autologous cell therapies are the ultimate personalized medicine and while such medicines are often not included in definitions of personalized medicine, any approach to regenerative medicine – whether using tissue engineered products, cell-based therapies, small-molecule drugs, scaffolds, nano or biobots, etc – intended to trigger in vivo regeneration or repair... is personal!

Again, to the extent the Long Tail economy is about “selling less of more” then medicines such as cell therapies will certainly be part of the long tail of the therapeutics industry giving access to highly-personalized, tailored products to meet the unique needs of the consumer.

But does the Long Tail economic model apply?

While there may well be blockbuster cell therapies in the future, concern around the scarcity of potential blockbusters in cell therapy are seated in the notion that the blockbuster economy is the only successful model.

Who thought you could make money by giving free access to internet searches and email? There are a lot of ways the analogy breaks down but what the Long Tail economy has shown us is modern corporate ingenuity in finding profitable business models in niche markets despite the long-standing dominance of blockbuster products.

Where the paradigm falls short, in my opinion, is when one realizes that a fundamental pillar to the Long Tail economy is the commoditization of products and production processes as well as the standardization of delivery systems. The Long Tail economic model is enabled by the fact that the cost of goods sold has been brought sufficiently low enough - by new technologies which allow for small batches to be produced and shipped - that the products in question are affordable to most. Highly unique and/or customized goods were always available to those with sufficient ability to pay. The Long Tail brings these kinds of products to micro-market at the same price or lower than their mass-market counterparts.

We are a long way from this kind commoditization of personalized medicines and, in particular, of cell-based therapies. There is little-to-no standardization of production systems at any scale. The next generation of these therapies will still be highly priced goods which many people, insurers, and/or healthcare systems will be pressed to afford.

In once sense, this is not surprising. Even in the world of electronic or digial products, Moore's principle only applies because the cost of goods for new technologies when they first enter the market are prohibiitively expensive. If the model applies in the therapeutics industry, technological advances in production will bring down the cost of goods and therfore the price.

On the other hand, what drives the price of therapeutics has not been so much the cost of goods (production) but the need to recoup massive upfront R&D costs which must be recovered (with a reasonable profit) during the life of a patent.

Will this change with personlized medicines and cell therapies? One could argue it might if personlized medicines and cell therapies can deliver on the promise to have a much higher effective rate among those given the therapies. Pharmaceuticals have a notoriously low effective rate among those prescribed any given drug. New research methodologies employing surrogate biomarkers, imaging, cell-based assays, etc promise the potential to significantly lower R&D costs by (a) getting to a much quicker "go/no-go" decision point in therapeutic development, and (b) identifiying patient sub-groups for which the therapeutic will have a high rate of effectiveness. One the one hand, this has the potential to lower the cost of goods (driven by R&D costs) and simultaneously justify a higher price point because of a high rate of therapeutic success.

However, even if we were able to materially lower upfront R&D costs, this still does not change the fact that cell therapies are costly to produce on a per-unit basis and the opportunity to "scale-up" is limited particlarly with autologous cell therapies which require lot sizes to be an individual batch. While this is a seperate discussion, I am confident technological and/or commercial innovation will find a way to economically bring therapeutics to market that are proven effective. I am not alone. Recenly Howard Liang, MBA, Ph.D., Analyst and Biotechnology Managing Direct at Leerink Swan as quoted as saying that “Autologous manufacturing is not the deal breaker it used to be. If these [cell-based] vaccines work for really difficult-to-treat diseases, the manufacturing issues will be worked out.” ("Special Report: Customized Cancer Vaccines Finally (Maybe) Arrive". GEN News Highlights. 12 June 2009)

There are other reasons the Long Tail business model as it is currently defined does not fit cookie-cutter with the therapeutics industry not the least of which is that the consumer demand does not solely regulate supply. In highly regulated industries, the economies are different. Some would argue this should not be the case particularly if personalized medicines, genomics,theragnostics, etc advance to point where the consumer has an abundance of therapeutic information and options but that is the subject of another discussion.

In the end, it is certainly clear to me that the emerging technologies are enabling new therapeutic paradigms that will introduce elements of the Long Tail business model into the therapeutics industry.

How much Impact will Cell Therapies Have on New Therapeutic Paradigms and Systems?

Despite the exponential escalation of cell therapy innovation, is cell therapy ready to be a viable business that will make any material impact on the therapeutic industry? For reasons which I will continue to explore in this blog in the months to come, I believe the answer is 'yes'.

The cell therapy industry has more products in late-stage development than many believe. While the existing commercial products are far from blockbusters and questions remain regarding the business models and potential margins for cell therapy products, ground-breaking research and market enthusiasm is driving a renewed interest among an early second-round of investors including participation by mature industry players making cautious plays in the sector. Regulatory issues in the primary markets of the US and EU are rapidly becoming less problematic though harmonization remains a significant challenge.

In blogs to come I will outline the commercial developments I see transpiring that make me believe the primary issues around the 'business models' question are being addressed. Regenerative and personalized medicines are enough of a force that creative business people will (a) invent ways to lower the cost of goods and (b) figure out how to price and reimburse appropriately to ensure effective therapies get to people that need them and incentivize doctors to prescribe them.

Most indicia point to this sector being poised for a phase that will start demonstrating real answers to those challenges which have to-date prevented many from participating in a sector that they believe presents too many unanswered risks.


As with so many of the paradigm-shifting economic revolutions in the past, once the underlying technologies exist, business and economic models radically adjust to reflect and accommodate that change. The latest of these is the Long Tail economy (r)evolution with it ability to deliver niche (if not personalized) products to niche markets. Pesonalized medicines - regenerative medicine and cell therapies in particular – are the therapeutic industry’s answer for the demand for personalized products. For a host of reasons, however, the economic model is not yet one which makes for profitable delivery of such products to niche markets.

Nonetheless, while questions about the viability of the business and economic models for these paradigm-shifting therapies are reasonable, the next 5 years will show that today’s questions about the business models for cell therapy were rooted in limited imagination about the potential for technological and commercial innovation as well as outdated concepts of the therapeutics industry and its economies.


[i] Anderson C: The Long Tail: Why the Future of Business is Selling Less of More. Hyperion (2006).

[ii] Friedman TL: The World is Flat. A Brief History of the Twenty-First Century. Farrar, Straus and Giroux (2005).

A Therapeutic Long Tail. Does Cell Therapy Fit the Model? (Part I)

I'm always skeptical of companies or products or technologies that depend on a 'paradigm-shift' or 'sea-change' in the long-entrenched habits of people before they will be widely adopted and/or successful.

It's why, for example alternative fuel autos might succeed in the U.S. where public transit has not - because they don't require as big a change. People can stay in their car and change their habits less.

Disruptive Technologies Lead to Paradigm Shifts

Nevertheless there are from time-to-time disruptive technologies that do arise which are paradigm-shifting. These are readily (though perhaps not quickly) adopted because the benefits are seen to outweigh the trade-offs and/or effort required to make the change. In many instances there are entire industries built around the pre-existing technologies that foolishly resist such change rather than learn to adopt and adapt. It's the classic mistake of railway companies believing they were in the business of trains rather than transporting people and goods and thus missing the opportunity to dominate transport by road and plane when these industries started to compete for the transport customer.

Will Cell Therapies Disrupt Sufficiently to Shift the Therapeutics Paradigm?

There are a lot of people talking about how regenerative medicine, personalized medicine, and cell therapies are going to revolutionize health care. I'm one of them. There are, however, many good reasons to be skeptical - not the least of which is the sheer size of the industries built around the pharmaceutical model and the power they can exert in resisting change. This might be comparable to Detroit's resistance to alternative transport systems or Houston's resistance to the use of fossil-fuels. It's massive.

But just as Detroit is now embracing the need for change and Houston is touting alternative energies, could it be that New Jersey is having its own 'come-to-Jesus' moment and realizing there may be - perhaps must be - a future for pharma beyond the blockbuster pill model? The other big 'industry' to convince is the healthcare inudstry. How readily will hospitals and doctors adapt to a therapeutics paradigm shift?

Last week, I had an interesting conversation with someone fro
m a struggling cell therapy company that has completed a semi-successful phase II trial and is very quickly running out of money despite having been on a road show for over a year. They are engaged in a "number" of "partnering" discussions with pharma companies who understand the science, the manufacturing, and the cost-of-goods data but say they are still uncomfortable with the "business model for cell therapy."

The know the market, have good data on the COGs and can assume a certain product price and what's left? I'm told that "business model" is code - at least in this instance - for two concerns:

(1) what will clinical adoption rates be like, i.e., will doctors adopt these new therapies and, if so, at what rate? and
(2) the model they are being presented does not offer them the type of margins they've come to expect.

Let's look at each of these in turn.

Health 2.0

Doctors have traditionally been driven by clinical results and reimbursement. But there's a new driver in town - it's the informed patient and their participation in Health 2.0. Patients no longer rely solely on their physician for healthcare information.

Deputy Editor of Wired magazine, Thomas Goetz, author of
"The Decision Tree" (blog and upcoming book), describes this new era of healthcare as follows:

"We are entering a Third Phase of medicine - one that spots disease and illness based on risks rather than one that responds to symptoms (phase 1, from pre-history to the mid 19th c), or one that seeks out the causes (phase 2, from circa-1850 to just about now).

These three phases closely parallel our technological capacity to spot disease: Phase 1 was primed to spot symptoms because humans relied on our senses - and nothing more - to recognize illness. If we couldn’t see it (or smell it), it wasn’t yet evident. Phase 2 emerged with the perfection of the microscope and the discovery of the germ theory of disease: finally we could see disease in its pathogenic form - bacteria, mostly - and thus began a race to find the little buggers that were causing disease. The late 19th century saw a flurry of diagnosis, as the cause of one disease after another was identified, and the Pasteurs and Kochs went about devising antibodies or vaccines to vanquish them... So now this third phase dovetails with the bevy of new diagnostics emerging - devices and techniques that look for certain molecules and DNA strands to not only detect the presence of disease, but predispositions towards certain conditions as well...

This third phase contains elements of what many are calling “personalized medicine,” but I hate that term - it sounds too servicey and implies that this is simply regular medicine/clinical practice that’s been tailored for you, as if we have a computer watching our health (it’s “personalized!”).

To my mind, what’s happening now is much more than simply a move to personalization; it’s an entirely new way of relating to disease and illness, one that’s as impactful and as new as the realization that germs - not bad air or bad luck - caused disease. What’s happening now makes disease/illness an optional state, one that can be opted out of (ideally) or engineered against (more likely)."

This is, he says, "...a new phase of health and medical care, where more decisions are being made by individuals on their own behalf, rather than by physicians, and that, furthermore, these decisions are being informed by new tools based on statistics, data, and predictions. This is a good thing - it will let us, the general public, live better, happier, and even longer lives. But it will require us to be stewards of our health in ways we may not be prepared for. We will act on the basis of risk factors and predictive scores, rather than on conventional wisdom and doctors recommendations.

We will act in collaboration with others, drawing on collective experience with health and disease, rather than in the isolation and ignorance that can come with “privacy” concerns. And we will act early, well before symptoms appear, opting to tap the science of genomics and proteomics in order to mitigate our risks down the road.

Together, these tools will create a new opportunity and a new responsibility for people to act - to make health decisions well before they become patients. This can be characterized as a decision tree, a series of informed choices we will make to minimize uncertainty and optimize our outcomes. Indeed, we will use decision trees to navigate most of our health decisions, sometimes in overt ways - new decision support tools will both inform us and guide us, and they’ll be steeped in statistics, prediction, and the power of collective experience.”
For better or for worse, patients have an increasing multitude of sources and options. Not only are patients now much more informed of their diagnoses and conditions but also of their treatment alternatives. More each year are demonstrating a willingness to access cell-based treatments out-of-country. Others are lobbying for the loosening of the regulatory sphincter around cell-based therapies such as stem cell transplantation, Dendreon's PROVENGE, etc.

Increasingly patients' demands are influencing doctors as much or more than anything else. Pricewaterhouse Coopers recently listed "the internet and social network" as the Health Industry's "top nine issues for 2009" citing web2.0 as "
changing how healthcare is navigated and experienced by consumers".

It remains to be seen how doctors will adopt cell therapies and this will depend in large part on the adequacy of reimbursement but certainly patients are driving their doctor's decisions like never before and patients seem enthused - perhaps even overly so - about cell-based therapies.

Therapeutic Profit Margins

In terms of margins, even pharma executives are now realizing - in the face of diminishing pipelines and massive R&D costs per drug - that the models that have worked so well to-date financed by the kinds of margins they have received to-date may not be possible going forward. M&A, the creation of decentralized divisions, increased academic collaborations, etc.... they are all measures to try and address the same problem of diminishing margins.

Decreasing product margins are being tackled like it's the disease when at least some would suggest it's the symptom. The disease may not be lower margins at all but the model which demands such massive margins to succeed. But more on that in just a minute...

Personalized Medicine

I believe there is a third challenge to pharma's "business model" concerns.
From one direction we have pharma's increasing desperation for new therapeutics; from another direction comes the public's increasing demand for access to cell-based therapies, and - to complete the trifecta - we have the fast-growing sector(s) of personal genomics, theragnostics, and/or personalized medicine.

The ability to analyze the effects of therapeutics on patient sub-populations and use that to target such groups and/or tailor therapeutics accordingly opens up so many promises therapeutically but also puts incredible additional pressures on the blockbuster model with its low-rate of success per overall prescription rate. Some insurers are now catching on to the notion that reimbursement can be - perhaps should be - tied to therapeutic success and are willing to pay a higher price tag for success but nothing at all for failure.

Others before me have described the increasingly hard-to-ignore crack in the blockbuster model much better than I. One of the better analyses I've read recently can be read here.

To predict that cell-based therapies will contribute to and succeed because of a shift in the healthcare paradigm may not be that much of a stretch at all. The stars do appear to be aligning for a rather wholesale change to be readily adopted by all involved - or at least the major players.


(click here to continue to Part II)

Friday, June 12, 2009

Cell Therapy Industry HiLites 2009-06-12

Autologous manufacturing is not the deal breaker it used to be. If these [cell-based] vaccines work for really difficult-to-treat diseases, the manufacturing issues will be worked out.” Howard Liang, MBA, Ph.D., Analyst and Biotechnology Managing Direct at Leerink Swan. "Special Report: Customized Cancer Vaccines Finally (Maybe) Arrive". GEN News Highlights. 12 June 2009.

Amen. Glad to hear someone else saying it.


Ok, well, if no one else wants to advertise here...

This issue of Cell Therapy Industry HiLites is brought to you by the charming, witty and occasionally useful folks at the cell therapy group.

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Neuralstem, Inc (NYSE AMEX: CUR) received notice from the NYSE Amex Exchange that its shareholder’s equity has dropped below the $2,000,000 threshold required by the NYSE Amex for continued listing. The Company intends to submit a plan to the Exchange. CEO and President Richard Garr said, “We are confident that the company will be in compliance.”

National Institutes of Health has awarded a $5 million grant to Q Therapeutics (co-founded by former NIH researcher Mahendra Rao), the University of Utah's Cell Therapy Facility and Johns Hopkins University School of Medicine to start human clinical trials of Q-Cells for myotrophic lateral sclerosis, or ALS -- better known as Lou Gehrig's disease.

Opexa Therapeutics, Inc. (NASDAQ: OPXA) received a favorable ruling from NASDAQ allowing an extension to regain compliance and continue the listing of the Company’s securities on The NASDAQ Capital Market.


StemCells, Inc. (NASDAQ: STEM) announced positive data from its Phase I clinical trial of its proprietary HuCNS-SC® product candidate (fetal-derived purified human neural stem cells). The six-patient Phase I trial was designed primarily to assess the safety associated with transplanting HuCNS-SC cells as a treatment for infantile and late infantile NCL, often referred to as Batten disease

Celsense, Inc. is one of the recipients in a 4-year $1.95M grant from the National Institutes of Health to test its in vivo cell trafficking MRI reagent in human clinical studies. The funded studies will be the first effort to evaluate Cell Sense, the Company’s fluorocarbon-based magnetic resonance imaging (MRI) cell tracking reagent, for clinical use. Additionally, the studies will garner preliminary data critically important for improving immunotherapeutic delivery strategies in metastatic colorectal (CRC) and other forms of cancer.

The Cell Sense technology makes transplanted cells visible by MRI. Therapeutic cells are labeled ex vivo with the Cell Sense reagent prior to administration to the patient. Cell migration and biodistribution are subsequently monitored using fluorine-19 (19F) MRI or magnetic resonance spectroscopy (MRS).

The key advantage of Cell Sense is purported to be that the 19F images are extremely selective for the labeled cells, with no background signal from the host’s tissues. Furthermore, accurate cell quantification in regions of interest is possible. Existing data from pre-clinical studies using human cells is said to show that the Cell Sense reagent is highly biocompatible inside cells and does not affect their innate function.

A clinical-grade version of the Cell Sense reagent has been formulated by Celsense, Inc. and is currently being subjected to mandatory in vitro and animal safety studies in preparation for human trials.

Never a company to shy away from making bold and early claims, Advanced Cell Technology, Inc. (OTC:ACTC.PK) Chairman and CEO William M. Caldwell IV said earlier this week that they are completing the "finishing touches" on a submission to seek FDA approval to conduct human clinical trials with a cell therapy that may be able to address many of the 200 plus known retinal diseases.


It's hard to put this story under any one category but since the financial terms weren't released and it's not yet clinical, I'll call it "commercial". J&J's
Ortho Biotech Oncology Research & Development, a unit of Centocor Research & Development, Inc., has announced that it has entered into a five-year Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI), with Steven A. Rosenberg, M.D., Ph.D., chief, Surgery Branch, serving as the NCI principal investigator, to research and develop novel cell therapy technologies as potential treatments for a variety of cancers.

The deal bring together Ortho's and Rosenberg's adoptive immunotherapy technologies designed to work by helping the immune system fight cancer.

Rosenberg has been a pioneer in the field of adoptive immunotherapy of cancer developing Tumor Infiltrating Lymphocytes (TILs) - T cells obtained from a patient's tumor, expanded and then re-administered to actively seek and destroy cancer cells. In recent years, Dr. Rosenberg's team developed a new technology in which T cells obtained from a patient's blood are genetically engineered to express receptors that give them specific immunity against cancer cells and then re-administered.

Meanwhile, researchers at Ortho Biotech Oncology Research & Development independently developed a different and proprietary adoptive immunotherapeutic approach that uses tumor antigens and other materials to stimulate T cells from a patient's blood to become Cytotoxic T Lymphocytes (CTLs), which recognize and attack tumor cells.

Both technologies show postive early clinical results in melanoma,
a type of skin cancer that ranks sixth among U.S. men and seventh in U.S. women for the most commonly diagnosed cancer, according to the NCI.

Under the CRADA, Dr. Rosenberg's lab will conduct a clinical trial in melanoma patients using Ortho Biotech Oncology Research & Development's proprietary technology. It is hoped that the technology will be effective in other types of cancer, as well.

They expect to begin clinical testing of the immunotherapy technology in melanoma patients by the end of 2009, with the possibility of additional studies for other types of cancer and other technologies in years to come.

Cellular Dynamics International, Inc. has secured an exclusive license on a patent portfolio surrounding cardiovascular progenitor cells. The license covers differentiation of stem cells into all cell types of the human heart. The patent portfolio was generated by and obtained from Gordon Keller, Ph.D. and Mount Sinai School of Medicine.

Cardiovascular progenitor cells are capable of further differentiation into the multiple cell types of the heart, including cardiomyocytes, endothelial cells, and vascular smooth muscle cells, both in vitro and in vivo. CDI intends to commercialize the technology to the pharma and biotech industry for predictive toxicology and screening purposes. Iit's not clear whether the license also includes clinical applications but if so they may be looking for licensing partners
given their current lack of interest in developing these cells into therapeutics.

In a not-so-surprising move, StemCells Inc (NASDAQ: STEM) has decided to close StemCell Sciences in Melbourne, Australia after its acquisition of the company in March. British staff will now be holding their breath to see if the same will happen to them. STEM says it has offered relocation packages to employees should they wish to move to sunny California. This leaves one "stem cell" firm in Australia where previously there were four (SCS, Bresagen, Embryonic Stemcells Inc and Mesoblast). If there is good news it is that, if I recall correctly, the three others were acquired and not just shuttered.

After many months of actively looking to offload its contract manufacturing subsidiary, Fresenius Biotech, has finally sold EUFETS AG to BioNTech AG. BioNTech is looking ot leverage the acquisition to further its quest to develop innovative molecular immunotherapies and biomarker based diagnostic approaches for individualized treatment of cancer and other severe human diseases.

Having used a contract manufacturer for what and when they needed, BioLife Solutions has now reverted back to internal manfucturing with the completion of construction and validation of their internal GMP manufacturing facility. Recently they gleefully released the first production lot for sale of their biopreservation media products made in the new Bothell, WA plant.

StemCyte Therapeutics is collaborating with Chennai-based Apollo Hospitals in joint venture with Ahmedabad-based Cadila Pharmaceuticals to set up what reports to be a first-of-its-kind umbilical cord blood (UCB) public bank for India.

Ok this news is a little soft but its a product that continues to be reinvented and a company we don't hear from much so I'll give them the space. HepaLife Technologies, Inc. (OTCBB: HPLF) (FWB: HL1) (WKN: 500625) has expanded their Advisory Board to support planning (e.g., refine the clinical strategy and patient treatment protocols) for their expected bioartificial liver clinical trial. HepaLife is developing a cell-based bioartificial liver system, HepaMate, as a potentially lifesaving treatment for liver failure patients. HepaMate is comprised of a blood plasma separation cartridge, a hollow-fiber bioreactor filled with proprietary porcine liver cells, a charcoal column, an oxygenator, circuit tubing and a plasma reservoir. These components are assembled into a patented blood/plasma circulation system, which is placed on the HepaDrive perfusion platform. The HepaMate technology has previously been tested in clinical studies involving more than 200 patients and has been the subject of over 50 scientific papers. It is anticipated that the upcoming trial would be a pivotal trial of the technology.

ThermoGenesis Corp. (Nasdaq: KOOL) mentioned in a recent press release that their Res-Q system - a point-of-care system designed to collect, process, and reinfuse bone marrow stem cells for therapeutic use, is anticipated to be launched on the market this month. Presumably in markets outside the US? They also discussed that they had received $500,000 in an SBIR grant from the NIH to study and develop biomaterials that can be used to deploy placental stem cells in bone repair and regenerative medicine applications. Now free money is free money but this seems a bit of a stretch to me for a company not without its struggles in its penetrating the market with its current cord blood storage and cord blood/bone marrow process systems. Apparently the the Res-Q platform is also being studied for potential use in the preparation of platelet rich plasma (PRP) from peripheral blood. Give them credit for continuing to think long-term and cross-platform despite struggles with their existing products.

"No more pads or tampons, period." Lifecell International is set to introduce in India next month menstrual cups as a cost-effective option to control menstrual flow in women and as a preparatory move for the company's menstrual-blood banking project called Femme, which is scheduled to begin in July


Xconomy's Luke Timmerman does a nicewriting up an informative piece on Denderon's recent AGM and the thinking around the company of what will be involved in taking the company "all the way".

Ok its another pre-clinical item (which I rarely cover) but again this one's just too cool to ignore. The New Scientists did a nice job of looking at Doris Taylor's work at University of Minnesota. Her team is looking to expand on their success creating rat hearts using stem cells. The ultimate goal is to some day create a limitless supply of transplant organs for humans. The team took stripped-down scaffolding of one rat heart and coated it with tissue produced from another rat's stem cells. They now want to repeat the procedure using hearts and other organs from human cadavers or larger animals.


The NIH received 49,015 comments in response to its draft guidelines for human stem cell research. One of those was submitted by

President Barack Obama should appoint a commission on stem cell research to help the therapy fulfill its promise to save lives and reduce medical costs, says Geron Corp. Chief Executive Officer Tom Okarma.

The California Institute for Regenerative Medicine (CIRM) received 73 preapplications for the CIRM Disease Team Awards RFA 09-01. The principal investigator for 15 of those applications is based with a for-profit entity. All of the teams proposed a wide variety of collaborators with several of the not-for-profit principal investigators citing collaborators in the for-profit sector. After review by a panel of outside experts and the CIRM in-house science team, a subset of these teams have been asked to prepare a full application, which will be due to CIRM July 16. Word on the street is that to-date just over 20 of the 73 have been given the green light to the next step.

** has posted a handy chart covering the public companies they follow in the stem cell sector showing the types of stem cells and business from which theyy hope to derive income as evidenced by the company's own statements, owned intellectual property, and/or areas of research. The one critically useful but missing column is the phase in which each of these products in.

One of my contacts, Scott Shields, Reimbursement and Health Policy Consultant at Argenta Reimbursement Advisors and owner of Blue Train Consulting Group, LLC has launched "The Reimbursement Wiki".

It promises to be a valuable online resource for reimbursement information particularly for those of us who do not work in that field full time. Below is his announcement of the site's launch:

I am announcing the public availability of my Reimbursement Wiki. I have compiled links to coding, coverage, and payment information, as well as information such as the following: - Medicare (URLs for rules, topics such as surgeries, compounding, CED, MS-DRGs, DME, inpatient list, laboratory and diagnostic testing, etc.) - Managed Care (URLs for medical/coverage policy sites at various payers, technology assessment organizations, literature on coverage decision making, etc.) - Medicaid (URLs for CPT/HCPCS code lookup by state for payment info, state fee schedules, drug product data, etc.) - Payment (URLs for Medicare payment basics, top 200 CPT/HCPCS codes, top 100 lab procedures, literature on drug pricing, etc.) There is plenty more -- basically, reimbursement and health resources I have collected in one place for reference purposes. The URL is The Reimbursement Wiki is ongoing project of mine, and I add material based on topics of current work, so not all topics are represented equally. Nevertheless, I find it an invaluable resource, and I hope you do, too. Access to the Reimbursement Wiki is free. Please register at, and email me your @ID. I will then invite you through the wiki. My email address is, or you can contact me via LinkedIn.


Organogenesis, Inc has established an annual scholarship program with Canton High School to honor a graduating senior pursuing a career in life sciences research or biomedical engineering. Kudos for them. We need more of that! Me?
I sponsored a child this week. No big deal but it reminds me to remind you...

...find a way to make a difference in your world today.

One more thing. You want to make sure you hear cell therapy-related news faster? Get on Twitter and follow me. I'm @celltherapy.

Ok. Hope you found that useful.
I'm trying to get back to my every Friday schedule here. I'll do my best. Happy weekend.

Lee Buckler, BEd, LLB
Cell Therapy Group

toll-free: 1-877-760-1966
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Skype: lee.buckler | Twitter: @celltherapy

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