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 from 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 reimbursement...so 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)

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.

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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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FINANCIAL

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.


CLINICAL

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.

COMMERCIAL

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


MISCELLANIA

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 CIRM.

**

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.

**

StemCellDigest.net 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 http://reimbursement.atwiki.com. 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 http://reimbursement.atwiki.com/signup, and email me your @ID. I will then invite you through the wiki. My email address is scott@argentaadvisors.com, or you can contact me via LinkedIn.

Sign-off

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
Principal

toll-free: 1-877-760-1966
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Cell Therapy Group. Your Cell Therapy Business Consultancy.
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Cell Therapy Industry HiLites 2009-05-26

This week I'm ROTFLMAO loving this cartoon so I thought I'd share the chuckles (thanks to www.onstartups.com). I can't tell you how many times in the past years I've seen the inevitable 'hockey stick' market projections graph from companies, analysts, and investors when discussing the coming 5 years in the cell therapy sector. It's become a standing joke about how we just keep pushing the hockey stick out to the right but the curve is inevitably the same. This pokes a little fun at this much maligned tradition.

Maybe it's just because I'm Canadian that I love this so much but c'mon the beloved hockey stick has become almost more a marketing tool than the venerable piece of sports equipment every Canadian boy must own! Maybe we can adapt this new hockey stick model to give the over-used Gartner cycle a run for its money!

Down to business...

I was told last week that Senators have been inviting people to Capital Hill to speak to them about FDA regulation of adult stem cells perhaps at the instigation of Chris Centeno's lobbying through ASCTA (www.stemcelldocs.com) that the medical use of moderately expanded autologous adult stem cells should not be regulated by the FDA but rather self-regulated by physicians. This could, of course, have serious implications on business models for autologous cell therapy companies. I've heard nothing of the Senators asking for ISSCR or ISCT counsel to-date. It would, of course, likely be Centeno's position that anyone involved in those orgs are "pharma hacks" as he has accused me of being.

ASCTA which is comprised of physicians none of whom are acknowledged leaders in stem cell research recently proudly announced the release of their stem cell guidelines which they suggest should replace FDA regulation. In what appears to be simple ignorance of the years of work being done by AABB & FACT, ASCTA described their guidelines as "the world's first clinical guidelines to allow U.S. doctors to safely begin using the patient's own stem cells to treat disease." Their lab practice guidelines contain such gems as:

• Any facility used in the processing A-ASC’s must be of suitable size, construction, and location to prevent contamination.
• The facility should be in a good state of repair.
  
• Equipment used in the facility should be adequately maintained
• In humid areas, dehumidifiers should be used to control the spread of infectious agents in the lab.
• Any reagents used must be approved for human use (where practical).

In an online posting entitled "The FDA is Killing Adult Stem Cell Therapy Thus Killing Patients", ASCTA supporter, Don Margolis, states:

In a startling new interview on stem cell research and adult stem cells in the near future, Dr. Christopher Centeno warns that Big Pharma and the FDA are teaming to control the use of a person’s own stem cells thus slowing innovation and the use of Adult Stem Cells to help patients now.

FINANCIAL

ReNeuron Group plc (LSE: RENE.L) has raised the £3 million it announced it intended to raise. ReNeuron recently received regulatory approval to commence a Phase I clinical trial in the UK with its lead ReN001 stem cell therapy for disabled stroke patients. The Company is developing stem cell therapies for a number of other conditions, including peripheral arterial disease and diseases of the retina. ReNeuron has also developed a range of stem cell lines for non-therapeutic applications – its ReNcell® products for use in academic and commercial research. The Company’s ReNcell®CX and ReNcell®VM neural cell lines are marketed worldwide under license by USA-based Millipore Corporation.

**

Pfizer expects to pump $100 million into its international stem cell development program which aims to focus both on small molecules that work by modifying cells in the body and cell therapy where the cells are manipulated outside the body. The first major use of the Pfizer money is for a collaboration with University College London (UCL) for the treatment of certain forms of blindness through the London Project to Cure Blindness. The collaboration will attempt to develop stem cell-based therapies primarily for wet and dry macular degeneration (AMD), which Pfizer has the rights to progress through clinical trials and then commercialize. Pfizer’s work on this could potentially provide a boost for the cash-strapped and searching-for-a-buyer, Intercytex Group plc (AIM: ICX), whose involvement on the London Project could yield cash royalties. But that's not where the Pfizer-Intercytex connection may end, apparently. Reports indicate that Pfizer is in fact eyeing the troubled Intercytex as a potential acquisition or investment. The company is on the auction block and actively seeking suitors.

**

Cytori Therapeutics, Inc. (NASDAQ: CYTX) expected to close a private placement for $4.2 million by May 11. No word yet...

**

Cord Blood America, Inc. (OTCBB:CBAI) has received a commitment for up to $2.3 million of new funding to acquire or build its own state-of-the-art laboratory for the storage of multiple stem cell products including umbilical cord blood stem cells. Less than two years ago the company signed a deal with Progenitor Cell Therapy LLC to process and store their stem cell units on a contract basis. Now CBAI says having their own facility will over the long term reduce their costs for both the processing and storage and additionally allow CBAI the flexibility and capacity to pursue new sources of revenue such as the storage of adipose tissue and peripheral blood stem cells processing and storage much easier than under the current structure of outsourced relationships.

**

Core Dynamics announced it has recently closed on a new financing round of US$17.9 million institutional and private investors. The money will be used to further develop the company's process for freeze drying human stem cells to be stored at room temperature.

**

Bioheart, Inc. (OTCBB:BHRT) announced today that its common stock is now being quoted on the OTC Bulletin Board (OTCBB) under the trading symbol "BHRT," the company's original ticker symbol.

CLINICAL

Aastrom Biosciences, Inc. (Nasdaq:ASTM) temporarily suspended enrollment and patient treatment in its U.S. Phase II IMPACT-DCM clinical trial following a report that a patient died at home after being released from the hospital following treatment in the trial. IMPACT-DCM is a clinical trial to evaluate the surgical delivery of autologous cells directly into the human heart muscle for the treatment of congestive heart failure associated with dilated cardiomyopathy (DCM) in both ischemic and non-ischemic patients. The patient's cause of death has not yet been determined and is the subject of a pending investigation at the clinical site. An independent Data Safety Monitoring Board (DSMB) will also assess the circumstances of the event. The Company has voluntarily suspended patient enrollment and treatment in the trial and the FDA placed the trial on temporary clinical hold pending an investigation.

**

ImmunoCellular Therapeutics, Ltd. (OTC: IMUC.OB ) (IMUC) announced that clinical data from the company's Phase 1 trial of its antigen-pulsed dendritic cell immunotherapeutic cancer vaccine ICT-107 for patients with brain stem glioma and glioblastoma will be presented at this year's ASCO. Having said that, they also said that while "encouraging data" was observed from the phase I trial of ICT-107, IMUC's primary focus going forward will be on its lead product candidate, ICT-121, which is an peptide-based "off-the-shelf" cancer vaccine that targets cancer stem cells and may have applicability to multiple types of cancer. IMUC anticipates filing an Investigational New Drug (IND) application in the third quarter of 2009 for a Phase I trial of ICT 121 in the treatment of glioblastoma (brain cancer).

**

Little-known, Memgen, LLC will be presenting results from a recently completed phase I clinical trial of its novel active immunotherapy product, ISF35, in patients with high-risk, progressive chronic lymphocytic leukemia (CLL), at next week's ASCO meeting. According to the study's principle investigator, Dr. Castro, one injection with no chemotherapy resulted in durable reductions in lymph nodes and spleen size in most patients and three of fifteen patients with rapidly progressing disease did not require any further treatment for more than one year. Based on the results from this single injection study, a phase II trial evaluating multiple intranodal injections of ISF35 in CLL has begun funded by - of all things! - the Food and Drug Administration at UCSD. The company believes the product is also positioned for use in lymphomas and solid tumor cancers.

**

Aderans Research Institute Inc. (ARI) announced it has launched Phase 2 of its clinical study on cell-based hair regeneration for men and women. The company's research is focused on developing a state-of-the-art tissue engineering solution to the regeneration of hair, taking actual hair cells--fibroblasts and keratinocytes, the two primary cell types within hair follicles--and promoting their growth in controlled laboratory environments. The research teams then recombine them and these "combined hair cells" are then placed in the patient's skin, where they are expected to elicit hair growth.

**

Cytori Therapeutics, Inc. (NASDAQ:CYTX) completed enrollment in the first study to investigate adipose derived stem and regenerative cells in chronic heart disease. The trial, named the PRECISE study, was designed as a double-blind, randomized, placebo controlled, dose escalation study specifically enrolling patients suffering from an advanced form of chronic heart disease, known as chronic myocardial ischemia, for which there is no generally accepted treatment. The trial enrolled 27 patients in a study involving the patients’ own cells extracted from adipose tissue and processed for delivery at the point of care using Cytori’s Celution® System. The cells were then injected back into the patients using the NOGA® XP System (Biologics Delivery Systems, Cordis Corp., a Johnson and Johnson company). Primary endpoints were safety and feasibility of Cytori’s Celution® System as part of a novel procedure for chronic heart disease.

A major attribute of the Celution® output is that it is comprised of multiple cells types. This, the company believe, contributes to multiple mechanisms of action, and thus increases the potential to restore heart function. The Celution® System used in PRECISE was configured specifically for cardiovascular disease, including proprietary processes and enzymes needed to achieve a level of purity required for vascular delivery.

COMMERCIAL

Cytori Therapeutics (NASDAQ:CYTX) and GE Healthcare, a unit of General Electric Company (NYSE:GE) announced an agreement by which GE Healthcare will commercialize Cytori's StemSource(R) technology in the North American stem cell banking and research markets. The StemSource technology includes automated equipment to process stem and regenerative cells found in adipose tissue, cryopreserve them or use them directly for research purposes. In January 2009, Cytori and GE Healthcare formed a separate agreement to commercialize Cytori's products in ten European countries. This includes selling the Celution(R) 800/CRS System in the European cosmetic and reconstructive surgery market as well as selling StemSource products in the European cell banking and research markets. The recent partnership is similar in nature to the European agreement, but is limited to the sale of StemSource banking and research products in the U.S., Canada and Mexico for 18 months starting in the second quarter of 2009. The agreement does not include U.S. commercialization of Cytori's Celution System, which is currently under review by the FDA.

**

Celsense, Inc. has announced the formal opening of its new and expanded R&D facility said to be a key component to Celsense’s commercialization strategy for its pre-clinical and clinical-grade molecular imaging agents used to non-invasively visualize and measure cellular activity and biodistribution. The company is now actively bringing both their lead research and clinical-grade cellular and molecular imaging agents to market over the coming months and is said to be collaboration with several academic and industry parties currently evaluating our products for potential use in their discovery research or clinical protocols.

Their clinical-grade Cell Sense reagent is a fluorocarbon tracer agent used to label cells in vitro then transplanted thus enabling investigators and clinicians to non-invasively track the administration and delivery of therapeutic and diagnostic cells in vivo using MRI. Applications include tracking therapeutic cells in regenerative medicine and immunotherapy applications, and observing localized immune system response by tracking populations of immune system cells. The research-grade V-Sense is an injectable fluorocarbon MRI tracer agent that labels leukocytes in situ, enabling the direct, non-invasive observation of immune system response and localized inflammation. Applications include observing change in immune system response to therapeutics and mapping localized disease such as certain cancers and infections.

**

After securing a $12M equity financing facility and closing a $1.5M private placement, formerly on-the-rocks Melbourne-based Prima BioMed (ASX: PRR), which had less than $500,000 in the bank at the end of December, is now preparing IND filing in the US for a phase IIb / III pivotal trial of CVac for ovarian cancer. As previously reported here, the company has also commenced CVac™ ovarian cancer treatments on selected patients in Australia made possible through the Australian Government’s Australian Regulatory Control Mechanism’s Special Access Scheme under the Therapeutic Goods Administration. Meanwhile the company is looking at taking the product to other countries as a means of generating cash flows in the short-term through the sale of CVac™ treatments. Now the company's executive director, Mr Martin Rogers, is touring North America in discussions with a number of hedge funds and other biotech focused investment funds about new investment and partnering opportunities for the commercialization of CVac.

**

Progenitor Cell Therapy, LLC has done a deal with NeoStem, Inc. (NYSE Amex: NBS) as their exclusive provider of commercial adult stem cells processing and storage. Progenitor founder, Andrew L. Pecora, has also joined NeoStem's Advisory Board.

**

NeoStem, meanwhile, - the new favorite company of alternative "medical" products promoter, Suzanne Sommers - has been busy the last few weeks creating a curious empire of sundry types of bedfellows. Recently the company announced it had signed an agreement to license the exclusive worldwide rights to a technology, with pending patent applications, developed by Vincent Falanga, M.D., Chairman of the Department of Dermatology and Skin Surgery at Roger Williams Medical Center, Providence, R.I. Dr. Falanga's stem cell product under development, Primcel, is a mesenchymal stem cell (MSC) product. Early clinical studies are said to indicate that Primcel can be used to accelerate or jump start healing of chronic wounds. Dr. Falanga's work seeks to develop a prepackaged product created from a patient's own cells, ready for physician use in a clinical setting, consisting of three applications over a 12-week period.

Almost in the same breath, the company filed a patent application claiming the proprietary stem cell technology of Vincent C. Giampapa, M.D., F.A.C.S. relating to cosmetic facial rejuvenation, which NeoStem first licensed in February 2009. Dr. Giampapa is director of the Giampapa Institute for Anti-Aging Medical Therapy, a board-certified plastic reconstructive surgeon and Assistant Clinical Professor of Plastic and Reconstructive Surgery at the University of Medicine and Dentistry of New Jersey. His cosmetic stem cell face lift technology is said to comprise a "non-surgical procedure for complete facial rejuvenation that involves injecting pluripotent cells, including stem cells, into the skin of individuals whose skin has lost its firmness and texture due to age".

Days later NeoStem announced it had signed an exclusive royalty-bearing license agreement for the Asia territory for a procedure developed by Regenerative Sciences, LLC and marketed for the treatment of chronic orthopedic conditions, under the name, Regenexx. Additionally, Regenerative Sciences, through its founder and CEO, Christopher Centeno, M.D., will serve as a consultant to NeoStem, Inc. in the area of stem cell therapy in orthopedics.

Undoubtedly Centeno will also embroil and leverage NeoStem in his fight with the FDA about the marketing of Regenexx in direct flagrance of FDA regulation. In any event this is clearly a solid "plan B" for Centeno should the FDA ever get around to shutting him down in the U.S. In the interim, Centeno continues to strengthen his network of supporters and is reportedly actively working Capital Hill for support for his fight.

To mix it up a little, NeoStem then announced they had signed a deal to promote and glean royalties from the sale of a liquid, nutritional supplement - AIO Premium Cellular Health. Not kidding. They have signed an agreement for Ceres Living, Inc., a developer and direct marketer of health and wellness products. AIO Premium Cellular Health was apparently developed in conjunction with NeoStem's scientists and Advisory Board members, based on certain nutraceuticals that have been shown to "optimize stem cell functions". The promotion of AIO is said to be "part of NeoStem's corporate commitment to advancing anti-aging and regenerative medicine therapies and technologies."

**

Novocell, Inc. announced that it has received U.S. Patent # 7,534,608 with with wide-ranging method claims covering the Company’s innovative stem cell therapy for the production of functional pancreatic, insulin-producing cells from human embryonic stem cells (hES). Novocell’s therapy is being developed as a method for the use of hES cells to replace insulin-producing pancreatic cells that are destroyed in people with diabetes.

**

Neuralstem, Inc. (NYSEAmex:CUR) recently published a reaction to a press release by StemCells, Inc. (STEM) refuting many of their statements in what has become a very public dispute. Included in the reaction is the claim that "the PTO upheld the patentability of Neuralstem’s core technology in May, 2006, in response to a challenge from STEM" and that the Neuralstem "patents are not being challenged in the PTO or in any of the suits with STEM".

**

This is not a cell therapy story but it could be. Takeda Pharmaceutical Company Limited (TSE: 4502) and IDM Pharma, Inc. (Nasdaq: IDMI) today announced that Takeda America Holdings, Inc., a wholly-owned subsidiary of Takeda (Takeda America), and IDM Pharma have entered into an agreement for Takeda America to acquire IDM Pharma. Takeda America has established Jade Subsidiary Corporation as a wholly-owned subsidiary to effect that transaction. Under the agreement, Takeda America will purchase all of IDM Pharma's outstanding shares for US$2.64 per share in an all cash tender offer followed by a merger.

You may recall that on April 1 last year (omen perhaps?), Cell Genesys, Inc. (“Cell Genesys”, Nasdaq: CEGE) and Takeda announced that the companies had formed a global alliance for the development and commercialization of GVAX immunotherapy for prostate cancer, Cell Genesys' lead product candidate then in Phase 3 clinical development. Under the agreement, in exchange for exclusive worldwide commercial rights to GVAX immunotherapy for prostate cancer, Takeda was to pay Cell Genesys an upfront payment of $50 million and additional milestone payments totaling up to $270 million relating to regulatory approval and commercialization of GVAX immunotherapy for prostate cancer in the United States, European Union and Japan.

This deal quickly unraveled when they decided to mothball both phase 3 studies of GVAX on statistical likelihood of failure. This was Takeda's big venture into cell therapy and it is an ugly, recent memory. Now they have bought IDM Pharma. They 've bought it for the company's primary asset, MEPACT, (mifamurtide), a macrophage activator drug therapy indicated for the treatment of non-metastatic osteosarcoma (malignant bone cancer) following surgical removal of the tumor (resection) in children, adolescents and young adults.

Here's the cell therapy part: IDM Pharma has 3 autologous cell therapy products currently on developmental hold as a means of perserving resources to get them to the home run on MEPACT. BEXIDEM is activated macrophages (Monocyte-derived Activated Killer cells or MAK® cells) for bladder cancer. UVIDEM, for melanoma, and COLLIDEM, for colorectal cancer, are both therapies using dendritophages (specialized immune cells derived from the patient's own white blood cells). It will be very interesting to see what Takeda does with these products, if anything.

**

Veterinary stem cell company, Vet-Stem, Inc, has now expanded from its US base into Canada, providing commercially available autologous, adipose-derived stem cell tranplants for horses and dogs.

**

According to the Seattle Times which played a critical role in exposing what is now alleged to be potential fraud, CellCyte Genetics, "left a cashless zombie after the stock-touting campaign that lifted its value to $400 million faded away, says it has reached a tentative settlement in the Securities and Exchange Commission (SEC) inquiry into its activities" but it still faces a criminal probe and the "number of lawyers embroiled in the company's legal troubles have now outnumbered the company's peak work force".

MISCELLANEA

International Stem Cell Corporation (OTCBB:ISCO), the company behind human stem cells from unfertilized eggs (called “parthenogenetic stem cells”), submitted comments to NIH on their draft stem cell research guidelines issued by the National Institutes of Health (NIH) on April 17, 2009. ISCO then published its comments in a press release and then their CEO published a blog to address questions regarding their comments.

Genetics Policy Institute also submitted a letter to the National Institutes of Health (NIH) commenting on the Draft Guidelines for Human Stem Cell Research. The complete letter is available at www.genpol.org for public viewing.

**

The California Institute for Regenerative Medicine is working its way through Congress seeking $500 million in federal funds it hopes to apply toward guaranteeing the long-planned small-business loan program.

Forbes published what is actually quite an informative article on cell therapy medical tourism for a change.

Sign-off

If you've read this far you're obviously interested in the cell therapy industry. If you're on LinkedIn make sure you have joined the LinkedIn Cell Therapy Industry Group.

Thank you all for all your good wishes about the new addition to my family - my own little sample of regenerative medicine! Now I gotta go and spend some time with her before I'm enrolling her in college...

Remember. Cell Therapy Means Business.

Cell Therapy Industry HiLites 2009-05-15

I'm back.

That on the left my friends is the new squawking reason I've been quite on the blogosphere and Twitter scene for the past few weeks!

I know this issue of my Industry HiLites has some rather stale news but it also has some recent news. What I can't promise is that I've captured all the cell therapy related news from the past few weeks. I'll attempt to back fill as time permits.

"Cell Therapy is expected to be one of the most important innovation drivers of modern medicine". See below who is the source of such zeal!

____________________________________________________

If you missed the Invitrogen-sponsored breakfast session at the 2009 Phacilitate Cell & Gene Therapy Forum in Washington, DC, the session videos are now posted for your convenience.

Realizing the potential of cell therapy: new enabling platforms
Brian Newsom, Cell Therapy Business Development Leader, Invitrogen/Life Technologies

Exciting lentiviral platform technology applicable to clinical indications from leukemia to HIV
Dr. Boro Dropulic, President and Chief Scientific Officer, Lentigen, Inc.

20-year history of Organogenesis
Katie Faria, Director, Process Development, Organogenesis, Inc.

Contact them at celltherapysystems@invitrogen.com if you want to engage in a discussion about how Invitrogen™ Cell Therapy Systems can deliver proven products and expertise to help with your translational research and clinical development plans. Find more videos and information about their products and services (including immunotherapy, stem cells, and tissue engineering) go to www.invitrogen.com/celltherapy.
______________________________________________________


The race is on between Dendreon and Osiris to be the first cell-based therapeutic approved in over 10 years. Osiris Therapeutics, Inc. (NASDAQ:OSIR) has confirmed with the U.S. Food and Drug Administration, receipt of the first portions of its BLA (Biological License Application) for Prochymal as part of a rolling submission. Dendreon expects to file revisions to its already-filed BLA by Q4 of this year. If things go as Mitch Gold (Dendreon CEO) predicts, it looks like Dendreon will win the race to market but they are not competitive products and everyone wins if they are both approved regardless of the order!

Meanwhile, troubled cell therapy company Isolagen, Inc. (AMEX: ILE) received good news from FDA recently that its BLA has been accepted for full review - decision due by Jan 4.

FINANCIAL

Dendreon, Corp (NASDAQ: DNDN) raised $221 million in a public offering of nearly 12 million shares of its stock at $19.20 per share. After announcing the offering only last week, the company said the money will be used to “develop manufacturing facilities, a distribution network, an information technology platform and other infrastructure, to hire sales and marketing, manufacturing, quality and other personnel in preparation for the licensure by the FDA and commercialization of Provenge, and for general corporate purposes, including working capital.”

**

Back in April, Opexa Therapeutics, Inc. (NASDAQ:OPXA) announced the closing of an initial tranche of a private offering for gross proceeds of approximately $1.1 million. Opexa will use the proceeds from the financing to support the ongoing partnering discussions for both its Tovaxin® T-cell program for multiple sclerosis (MS) and stem cell therapy for diabetes. Participating in the financing were existing shareholders, consisting of institutional investors, private individuals and members of Opexa’s Board of Directors.

**

Cell therapy company Isolagen, Inc. (AMEX: ILE) received bridge financing to allow it to seek debtor-in-possession financing in conjunction with a possible chapter 11 filing. Meanwhile, they've filed their latest 10Q with the SEC for those interested in the gory details.

**

The California Institute for Regenerative Medicine (CIRM) has handed out 15 grants worth $67.7 million to 13 not-for-profit and two for-profit organizations, BioTime and Novocell. An additional 12 grants which were recommended are on hold and money will made available only if funds allow. They will consider those grants in June. Novocell has been awarded $5,405,397 for its embryonic stem cell-derived diabetes therapeutic program and BioTime has been granted $4,721,706 to facilitate the use of its ACTCellerate™ technology for manufacturing patient-specific therapeutic products through the expansion of over 140 highly purified primitive human embryonic progenitor cells (hEPCs) from hES or iPS cells.

Novocell will receive two funding awards totaling $6.2 million from CIRM. The grants cover development of methods for delivery and monitoring safety of human embryonic stem cell (hESC)-derived pancreatic cells for diabetes cell therapy.

**

Virxsys Corp. of Germantown, Md., and Rockville’s GlobalStem Inc. are the only two private companies among the recipients of nearly $19 million of funding for stem cell projects granted recently by the Maryland Stem Cell Research Commission to 59 projects out of 147 applications that it received. GlobalStem's project is entitled "Scale-Up Manufacturing, Authentication, and Banking of Human Pluripotent Stem Cells" and curiously Virxsys' is "Developing safer methods for induced Pluripotent Stem (iPS) cell generation using Spliceosome-mediated RNA trans-splicing (SMaRTTM) technology".

**

While functionally the company is in a deep coma for the moment, according to a recent SEC filing, Cell Genesys, Inc. (NASDAQ:CEGE) is continuing to explore financing alternatives and consider strategic alternatives, including merger with or acquisition by another company, further restructuring, other sales of non-core assets, and other potential technology outlicensing or liquidation of the company.

**

Embryome Sciences, Inc. parent co BioTime, Inc. (OTCBB:BTIM) has raised $4M with a possible $16M in total with the potential sale of additional shares and warrants.


CLINICAL

Pervasis Therapeutics received Orphan Drug Designation for Vascugel cell therapy as they
prepare for a phase III trial of the product.

**

Osiris Therapeutics, Inc. (NASDAQ:OSIR) completed patient enrollment in the second of its Prochymal Phase III trials for Graft vs. Host Disease (GvHD).

**

I have to confess that until I read this recent press release from ReNeuron I was suffering under the misconception that the regulatory framework for cell therapies in Europe was becoming clearer but listen to this nightmarish-sounding bureaucratic cluster-schmuck. ReNeuron Group plc (LSE: RENE.L) provided a regulatory update regarding its ReN001 stem cell therapy for stroke. In January of this year, the UK Medicines and Healthcare Products Regulatory Agency (MHRA) granted regulatory approval for a Phase I clinical trial with ReN001 in disabled stroke patients, to be undertaken in Scotland at Glasgow Southern General Hospital. The decision was ratified by the Commission on Human Medicines, with the support of its Clinical Trials Expert Advisory Group which was also involved in reviewing the application.The Company has since submitted the clinical trial application to the Gene Therapy Advisory Committee (GTAC) who, in addition to their existing gene therapy remit, were recently given responsibility to act as the national research ethics committee for stem cell therapy clinical trials in the UK. The Company has been notified by GTAC that a Provisional Opinion has been granted in respect of the applicatio , subject to the resolution of a small number of points raised pertaining to non-safety-related pre-clinical data and the clinical trial protocol. The Company is reportedly currently working to respond to the points raised, the majority of which involve straightforward amendments to the clinical trial protocol. Further clarification is being sought by the Company, particularly within the context of the above-mentioned regulatory approvals already received, regarding one request for further non-safety-related data by GTAC, the provision of which would potentially delay the start of the clinical trial by a few months. Meanwhile their IND in the U.S. remains on hold after well more than a year has passed since its submission.

**

Cellerant Therapeutics, Inc. announced the dosing of the first patient in a phase 1 trial of CLT-008 for hematological malignancies. CLT-008 is an allogeneic cell therapy designed to rapidly produce mature neutrophils and platelets in vivo and facilitate long-term engraftment in patients undergoing bone marrow or cord blood transplantation, or being treated for chemotherapy- or radiation-induced neutropenia among other applications. CLT-008 is said to have the potential to make cord blood transplants safer and more available for everyone regardless of age, size, or ethnic and racial background. In preclinical testing CLT-008 has reportedly shown to provide hematopoetic support to enable long-term engraftment of blood-forming stem cells, which could lead to broader use of cord blood transplants as an effective therapy for multiple life-threatening blood diseases. The company intends to study CLT-008 to addresses several significant markets and including neutropenia in the near future.

Led by Dr. John Wagner, Professor of Pediatrics and Director of the Division of Hematology-Oncology and Blood and Marrow Transplantation at the University of Minnesota, the Phase 1 study is an open-label, dose-escalation trial and will investigate the safety and tolerability of CLT-008 in 20 to 25 patients undergoing cord blood transplants.

**

ERYtech Pharma has started its phase II clinical trial of GRASPA as first-line treatment among patients with acute lymphoblastic leukaemia (ALL) aged 55 and older. A first cohort of 3 patients has already been recruited for the 30 patient study in France. GRASPA^® is an enzyme formulation of L-asparaginase which is clinically delivered entrapped in homologous red blood cells.

**

Ever-innovative Medistem Inc. (PINKSHEETS: MEDS) is bringing pre-clinical research, commercial cell therapy clinical practice outside the U.S., and veterinary stem cell protocols together to report positive multiple sclerosis data using fat stem cells. A recent paper describes the scientific rationale and preliminary results of three patients with multiple sclerosis treated with their own fat derived stem cells. The paper provides some evidence that a stem cell-rich component of adipose tissue, called the Stromal Vascular Fraction, can concurrently immune modulate, while inducing regenerative activities in multiple sclerosis patients.


Publication collaborator, Dr. Robert Harman, CEO of Vet-Stem, has treated over 3,500 horses and 1,500 dogs with fat derived stem cells for inflammatory conditions such as osteoarthritis immune-mediated polyarthritis. The article, titled "Non-expanded adipose stromal vascular fraction cell therapy for multiple sclerosis," appeared today in the Journal of Translational Medicine (http://www.translational-medicine.com), and was a collaboration between scientists and clinicians from Medistem; the University of Western Ontario; Hospital CIMA, San Jose, Costa Rica; Indiana University; Cell Medicine Institutes; University of California, San Diego; and University of Utah


COMMERCIAL

Finally, we hearing some more about David Mooney's company InCytu, Inc. which makes "smart biopolymer devices that enable in vivo cell programming". I hear a lot of great talks but I have to confess hearing Mooney speak last year topped the list for 2008. This stuff might be too intelligent (read "complicated") to be commercializable but it certainly doesn't lack for brilliance.

The first of their in situ bioreactive devices (iBDs), coined Cellarium(TM) by the company, will be an intelligent biopolymer scaffold that once implated releases a factor that selectively attracts dendritic cells (DCs) into the scaffold, presents antigenic (melanoma specific) proteins to them, and then, upon maturation, releases the activated DCs to home to the lymph nodes where they stimulate T cells to specifically attack the melanoma.

This takes the whole notion of cell therapy (traditionally involving the ex vivo manipulation of harvested cells for implantation) and turns it on its head by doing in vivo what we have believed can/must be done in cGMP facilities.

Now this is all a long way off and the Lord Almighty knows we have had more than our share of cell-based cancer vaccine failures but other applications in the pipeline include chronic ischemia, peripheral vascularization, muscle regeneration and even potentially stroke and CNS disease.

InCytu plans to develop partnerships for product sales and marketing but retain control of the cGMP production of its iBD devices. The company's product development facility is in Lincoln, RI likely for reasons associated with its CEO - serial CEO and cell therapy entrepenuer, Alfred Vasconcellos, who has been involved with CytoTherapeutics, ETEX, LCT, Sertoli Technologies. LCT Biopharma, Multicell, and cut his early teeth in Pfizer's Strategic Market Development group. Vasconcellos was most recently the CEO of LCT Biopharma Inc. which is the wholly owned subsidiary of diabetes cell therapy company Living Cell Technologies Ltd (listed on the Australian Securities Exchange (ASX:LCT) and the US-based OTCQX (OTCQX:LVCLY) from New Zealand and now conducting a trial in Russia.

**

Dendreon (NASDAQ:DNDN) CEO Mitchell Gold's ship came in when he recently sold 600,000 shares of the company at $24.92 apiece on April 29, generating gross proceeds of $15 million.

Meanwhile Dendreon is going on a hiring binge in prep for FDA approval of Provenge.

**

It is interesting to see the different approaches companies take to competitive IP. Whereas Osiris appears content for the moment to let companies develop all kinds of MSC products without interference, Stem Cells, Inc couldn't be more aggressive in litigating any IP they believe infringes on their patents, like that of Neuralstem. Cytori and Vesta had a similar battle.

StemCells, Inc. (NASDAQ:STEM) recently announced that the U.S. Patent and Trademark Office (PTO) has upheld the validity of the remaining two neural stem cell patents which were subjected to reexamination proceedings commenced by Neuralstem, Inc. The upheld patents are the subject of two related lawsuits initiated by StemCells against Neuralstem, which allege infringement of a total of six patents. These six patents collectively claim the manufacture and use of human neural stem and progenitor cells as tools for drug discovery and as therapeutic agents. The PTO’s decision to uphold the two patents is final and cannot be appealed. Consequently, StemCells is asking the federal district court in Maryland to resume the infringement lawsuits against Neuralstem.

“The PTO’s latest action is a reaffirmation of the validity of the patents asserted against Neuralstem. Each of our reexamined patents has now been twice tested and twice granted by the Patent Office,” said Martin McGlynn, President and CEO of StemCells, Inc. “We now look forward to our day in court. Litigation can be a slow and meticulous process, but we plan to ensure that those who wish to commercialize neural stem cells for drug discovery or therapeutic uses, recognize the validity and value of our patents.”

**

Lonza Group Ltd is expanding its cell therapy business with the construction of a new facility in Singapore representing a $27 million investment for the first phase which will include two manufacturing suites that are expected to come on-stream by mid-2011. The company which reportedly already operates 12 cGMP-certified cell therapy manufacturing suites in the U.S. and Europe, said the Singaporean cell therapy suites will be next to the company’s existing large-scale mammalian production plant and additional suites could be added in the future to meet increased demand.

This comes after Lonza's announcement in January that it was spending $26m to expand cell therapy manufacturing capacity at its facility in Walkersville, Maryland, US installing three class 10,000 cell production suites as well as the requisite clean room and processing space at the plant, all of which will be housed in a 44,000 sq ft, purposed designed building.

Here's my favorite part of this story. Lonza is a $2.65 billion/yr multinational company and quoted in the release is Lonza CEO, Stefan Borgas, saying:

"Cell Therapy is expected to be one of the most important innovation drivers of modern medicine".

**

Pfizer is clearly seeing ocular therapies as an important franchise for its regenerative medicine enterprise. After making a seed-like investment of $3M last year in the startup EyeCyte Inc, Pfizer has now announced a collaboration with University College London to advance development of stem cell-based therapies for age-related macular degeneration (AMD). The London Project to Cure Blindness, led by Professor Pete Coffey (UCL Institute of Ophthalmology), is involved in producing a cell replacement therapy from human embryonic cells; a therapy which it aims to introduce into clinics by 2011. The goal is to replace cells essential for “seeing” lost through disease at the back of the eye. The UCL–Pfizer collaboration will accelerate the research process by bringing together the pioneering work of UCL’s researchers in the field of cell-based therapies and Pfizer’s expertise in design and delivery of therapeutics.

Under the terms of the agreement, Pfizer will provide funding to UCL to enable research into the development of stem cell-based therapies for AMD as well as other retinal diseases. Pfizer will also contribute expertise in the design and execution of clinical studies, interaction with global regulators, and in product manufacturing techniques. After the completion of preclinical safety studies, Pfizer will have the option to conduct clinical trials to determine efficacy of treatment and commercialize any resulting product.


Pfizer also recently signed deal with WARF for rights to human embryonic stem (hES) cells for dev of new drug therapies

**

One of the topics of discussion among those of us trying to lead the charge in commercializing cell therapies is the lack of experienced executive leadership at the helm of many cell therapy companies. Novocell, Inc, as it has done before on several fronts, is leading by example by hiring a seasoned biotech executive as its new President & CEO to replace Alan Lewis who went to lead the JDRF.

Novocell, Inc. recently announced the appointment of John West as President, Chief Executive Officer

"Novocell addresses one of the largest commercial and medical opportunities in stem-cell-derived therapeutics, and its team is increasingly recognized for its scientific and IP leadership,” said Mr. West. "I look forward to working with Novocell through clinical development and market launch and to growing a major new business.”

West, former CEO of Solexa, Inc. (NASDAQ:SLXA - News), brings more than 27 years of experience building profitable businesses from transformational technologies in the life sciences industry. Mr. West served as Chief Executive Officer of Solexa from 2004 until the company’s acquisition in 2007. When Mr. West joined Solexa, it was a private venture backed company with a radically different biochemistry and molecular biology for DNA sequencing. During Mr. West’s tenure, the company became public through a reverse merger, raised almost $100M and launched its first commercial product. This product lowered DNA sequencing costs over 100-fold, achieved almost $100M revenue in its first full year and became the dominant system for genome-scale genetic research. Mr. West led sale of the company in 2007 to Illumina, Inc (ILMN) for over $600M, and the business market cap subsequently exceeded $1B. Prior to Solexa, Mr. West served as VP of DNA Platforms, approximately a $1B business, at Applied Biosystems (ABI) (now Life Technologies). Mr. West’s responsibilities included the company’s businesses in DNA sequencing, preparative and real-time PCR, DNA synthesis and arrays. Prior to joining ABI, Mr. West was President of Princeton Instruments, a private company with transformational technology for low light imaging and spectroscopy. During Mr. West’s tenure, the company grew over 10 times in revenue, with much of the growth leveraging advances in cell biology. The company was sold to Roper Industries (NYSE:ROP - News), where Mr. West continued as subsidiary President. Mr. West holds B.S. and M.S. degrees in engineering from MIT, and an M.B.A. in finance from the University of Pennsylvania’s Wharton School.

**

Advanced Cell Technology, Inc. (Advanced Cell, ACT) (OTC: ACTC) and Korea-based CHA Bio & Diostech Co., Ltd. (CHA Bio), formerly known as CHA Biotech, Co., Ltd. and whose shares are publicly traded on KOSDAQ, announced that the parties have entered into a licensing agreement under which Advanced Cell will license its proprietary “single blastomere technology,” which has the potential to generate stable cell lines, including retinal pigment epithelium (RPE) cells for the treatment of diseases of the eye, to CHA Bio for development and commercialization exclusively in Korea. ACT received an undisclosed up-front license fee.

**

At this year's ISCT conference, Invitrogen, a division of Life Technologies (NASDAQ:LIFE), announced a new technology to separate undifferentiated stem cells from those that are differentiated by utilizing magnetic beads that latch onto a common marker on embryonic and induced pluripotent stem cells, which are not considered as suitable for transplantation as those which are differentiated because of the potential of unregulated cell growth, removing them from a culture in less than 45 minutes. This leaves behind highly pure and differentiated cells that are unaffected by the process.

**

Histogen Aesthetics, a division of Histogen focused on providing unique, clinically proven, human skin cell-derived products for aesthetics medicine, has announced the launch of the ReGenica™ Rejuvenation System for anti-aging and post-resurfacing.

Histogen Aesthetics utilizes a proprietary manufacturing process in which newborn dermal fibroblast cells are grown on beads in controlled bioreactors that maintain an embryonic-like environment, including low oxygen and low gravity conditions. During this process, the cells secrete growth factors such as Keratinocyte Growth Factor and Vascular Endothelial Growth Factor, and soluble extracellular matrix proteins such as collagens, laminin and decorin, which are associated with skin renewal and scarless healing. The resulting product is ReGenica, a complex made up of the naturally produced cell signaling materials and embryonic-like proteins which support the epidermal stem cells that renew skin.

**

Stem cell therapy faces more scrutiny in China but regulations remain unclear for companies that supply treatments.

**

Hemogenix, Inc. is stepping up the heat a little in its competitive battle with Stem Cell Technologies, Inc. over the preferred assay method for stem cell quality control and potency. Hemogenix recently sent out an email and posted a statement on the homepage of its website saying "It has taken Stem Cell Technologies 6 years to acknowledge one of the basic concepts of the HALO Platform: By counting undifferentiated colonies at 7 days, they are actually counting clusters of "proliferation units" which can, in turn, be measured using ATP without any manual enumeration of colonies at 7 days. "


MISCELLANEA

FDA CBER's Cellular, Tissue and Gene Therapies Advisory Committee held a meeting over the last couple day. On May 14 in the morning, in open session, the Committee was scheduled to discuss the potential for Chlamydia trachomatis and Neisseria gonorrhea transmission by human cells, tissues, and cellular and tissue-based products (HCT/Ps) that are recovered from the reproductive system or gestational tissues (e.g., amnionic membrane and placenta, cells recovered from menstrual blood, foreskin, placental/umbilical cord blood derived cell products), or other sources. In the afternoon, in open session, the Committee will discuss animal models for porcine xenotransplantation products intended to treat Type 1 diabetes or acute liver failure. On May 15, in open session, the Committee intended to: (1) Receive an update on Guidance documents from the Office of Cellular, Tissue and Gene Therapies, Center for Biologics Evaluation and Research and the Center for Veterinary Medicine and (2) discuss clinical issues related to the FDA draft guidance ``Preparation of IDEs and INDs for Products Intended to Repair or Replace Knee
Cartilage.''

FDA intends to make background material available to the public no later than 2 business days before the meeting. If FDA is unable to post the background material on its Web site prior to the
meeting, the background material will be made publicly available at the location of the advisory committee meeting, and the background material will be posted on FDA's Web site after the meeting. Meeting material is available online here.

**

BrightTalk recently hosted a stem cell webcast summit including presentation by ISCO - check it out here.


Sign-off

So with that I'm easing back in. As mentioned, I'll try to back fill any news I missed and get back on schedule. Until then... ciao.