Cell Therapy Industry HiLites 2008-12-19

Ok so it's almost not Friday anymore even by my Pacific Time Zone but I promised to try to do this every Friday and so here I am. I will not be doing a HiLites next Friday however. I'm taking a break for the holidays.

For the week before Christmas this was one busy week!

I'll start this week's HiLites with a bit of a focus on Bioheart, Inc. (Nasdaq:BHRT). The company has been busy this week reinventing itself - just how much remains to be seen. As followers of the industry will recall, Bioheart had a disappointing IPO earlier in the year but was able to raise ~$1.8M in a private placement in October. This, I suspect, is not much more than survival money as they are financing a 330-patient phase II/III trial in US and Europe.

So the company's announcement this week came as no surprise that it intends intends o spin out or sell off its interest in several non-core technology platforms including:
- Biopace -- A biological pacemaker.
- MyoValve -- A cell seeding method for repairing and building biological heart valves.
- EndoCell -- A system for bedside preparation of adipose tissue for delivery of endothelial progenitor cells to the adventitia of coronary arteries for prevention of restenosis following
balloon angioplasty, stenting or atherectomy.
- AortaCel -- Cell compositions and delivery systems for repairing aortic aneurysms or for reinforcing the neck area in aortas repaired with an endovascular stent graft.

In addition, Bioheart is seeking a strategic partner for the development of its bi-ventricular pacemaker technology, called MyoStim, which utilizes a patented electrical stimulation software program and an additional pacemaker lead to enhance cell transplantation.

Understandably Bioheart is doing this in order to intensify its strategic focus on the treatment of patients suffering from heart failure. Interestingly, however, in their press release Bioheart made no mention of their cell therapy saying rather that Company is strategically focused on commercializing patented technologies for reducing and repairing heart muscle damage caused by heart attacks as well as marketing advanced heart failure monitoring devices including the Bioheart 3370 Heart Failure Monitor and the Bioheart-Monebo CardioBelt(tm) ECG Acquisition Device.

Then yesterday Bioheart announced it had had signed a deal with Life Technologies Corporation (Nasdaq:LIFE) (the new combined Invitrogen-Applied Biosystems entity) which provided Life the global distribution to Bioheart's myoblast cell-based research products to life science researchers. The deal is intended to "expand the number of therapeutic applications under development with this type of cell" but again no mention of Bioheart's own trial or product.

All of which leads to at least a couple questions we hope Bioheart will soon answer. Firstly, is the company positioning itself away from its lead product MyoCell®? Secondly, the deal with Life Technologies was described as a "distribution and OEM agreeement" but while there was discussion about the distribution aspects of the deal, there was no description of the OEM part. Given that neither company has any notable capacity or expertise in producing clinical-grade cells will this only encompass research-grade cells or are there other plans here not being discussed?

I'll move on to other news of the week.

In a move that will certainly enhance his legacy on his way out the door, Alan Lewis announced that Novocell, Inc. has entered into a non-exclusive drug discovery collaboration with Pfizer which gives Pfizer access to Novocell’s proprietary pancreatic progenitor cells derived from human embryonic stem (hES) cells. This appears to be an open-platform type of agreement (limited for now to 2 years). Novocell will receive an upfront payment, research funding, additional amounts payable upon the achievement of certain technical milestones, and payments relating to the sale by Pfizer of any exclusive therapeutic discovered as a result of the collaboration.

Novocell has shown it can coax human embryonic stem cells to become fully functioning pancreatic beta cells that secrete insulin in mice but about 15 percent of the animals got teratomas, a type of tumor. This, the company will have to learn to prevent or at least control before it gets approval to go to human clinical trials. For now, as Xconomy's Luke Timmerman says, "Novocell can take some comfort knowing that some of the deepest pockets in the pharmaceutical industry are shelling out at least a little spare change to help it crack daunting challenges like that."

BioLife Solutions Inc. (OTCBB: BLFS) announced that it has completed an initial series of small animal safety studies. The studies evaluated injections of HypoThermosol, CryoStor, or control phosphate buffered saline (PBS) into healthy rodents. The study was designed to mimic human clinical applications where stem cells isolated from cord or peripheral blood are re-suspended in a carrier solution and administered intravenously to treat patients suffering from a variety of diseases and disorders including leukemia, anemia, lymphoma, myeloma, and other cancers.
The results of these studies demonstrate- the Company claims - that infusion of HypoThermosol and CryoStor present no safety risk within the parameters of this two-stage evaluation in a rodent model. Data to be published and/or presented soon.

Avita Medical Ltd (ASX: AVH) says the French Ministry of Health is funding a 200 patient medico-economics study on the impact of Recell on burn wound healing. Avita said that the first patients were enrolled and had been treated in the study which was part of a French program “to advance the implementation of innovative technologies”. Avita said the primary goal was to demonstrate the medical effectiveness and economic benefits of the Recell technology to the hospital and health care system in the treatment of burns and wounds.

Recell, already commercially available in Australia, is a single-use medical device for harvesting autologous skin cells. Developed as an 'off the shelf' kit, ReCell enables a thin split thickness biopsy, taken at the time of procedure, to be processed into an immediate cell population for delivery onto the wound surface. Once processed, the cell suspension is available for immediate use and can cover a wound up to 80 times the area of the donor biopsy. ReCell enables the delivery of keratinocytes, melanocytes, fibroblasts and Langerhans cells harvested from the epidermal-dermal junction for application onto a wound surface in order to promote effective wound healing.

Pluristem Therapeutics Inc. (NasdaqCM:PSTI) (DAX:PJT) filed a European Investigational Medicinal Product Dossier (IMPD) to begin clinical trials of its placental-derived adherent stromal cell product, PLX - PAD, an allogeneic cell therapy, for critical limb ischemia. The company filed the IMPD application with the Paul Ehrlich Institute (PEI), the German competent authority in the European Union (EU). An IMPD is a harmonized procedure for the authorization to perform a clinical study in any one of the EU Member States. An IMPD is requested whenever the performance of a clinical study in any one of the EU Member States is intended, and it is similar to an Investigational New Drug (IND) Application that would be filed for the performance of a clinical study in the United States. Assuming approval of the IMPD by this authority, Pluristem said it plans to initiate the dose ranging clinical trial during the second quarter of 2009 at two sites in Berlin, Germany

Amgen lobbied this week for personalized medicine by presenting to the FDA the argument that one of its drugs should be used by just a subgroup of patients with colorectal cancer who appear to be most likely to benefit from it.

NeuralStem filed an IND to use its neural stem cell technology in a trial for Lou Gehrig’s disease in what it described as the first ALS trial using stem cells.

Progenitor Cell Therapy has brought in a new CFO with over 21 years of experience in the life sciences industry and fundraising. This will undoubtedly help them raise the funds needed for their continued US and European expansion plans.

StemCells, Inc (NASDAQ: STEM), which already has a trial underway for Batten disease, just received FDA approval to start a trial for Pelizaeus-Merzbacher Disease (PMD), also fatal brain disorder that affects mainly young children. The mechanism of the potential Batten disease therapy is to establish healthy neural cells that can help a patient’s own cells clear out some toxic garbage that builds up in the disease, the mechanism for PMD is to boost myelinization of neurons. StemCells uses cells originally derived from fetal tissue.

Although this news will be old to some I discovered this week Arteriocyte's announcement from November 10 that it had been awarded the DARPA Blood Pharming award to the tune of $1,950,000 which presumably it has to share with collaborators at Johns Hopkins University, The Ohio State University and INSERM, Paris. While this seems like a sizeably juicy award, the US Military's Defense Advanced Research Projects Agency (DARPA) has issued a tall order. DARPA wants a self-contained device small enough (≤47 ft3) to go into the military field and create at least 100 units of universal blood (i.e., can be transfused into anyone regardless of blood type) per week for eight weeks. The key ingredient to Arteriocyte's proposal and the company's Nanofiber Based System (NANEX) technology is umbilical cord blood stem cells.

While technically not a cell therapy play, both these players are notable enough in the cell-as-tools sector that we thought it worth noting that VistaGen Therapeutics has signed a deal with Wisconsin Alumni Research Foundation (WARF) to license WARF's human embryonic stem cell patents for the development and commercialization of stem cell-based research tools.

Winning this week's award for the press release with no news, is the annoucement from Advanced Cell Technology, Inc.(Other OTC: ACTC.PK) that "its technology platform, which utilizes a single cell biopsy that does not destroy the embryo, is well positioned to receive Federal funding under the new administration led by President-Elect Barrack Obama."

They made up for it the next day, however, by releasing news of new funding. Demonstrating, once again, their tenacious survivability, ACT announced it had licensed a "non-core technology" to one of its creditors, Ireland-based Transition Holdings, Inc, for $2.5 million - comprised of the extinguishment of $1.5 million of debt and an additional funding commitment of $1 million. The Company expects to apply the proceeds it receives in the future towards its retinal pigment epithelium (RPE) cells program.

Cytori has now launched a a 30-person clinical trial using adipose (fat) stem cells for chronic radiation induced injury.

Life Technologies announced its CIRM award will be used to develop human stem cell models of neurodegenerative diseases to advance drug development. The company intends to use human embryonic stem cells to develop new models of Lou Gehrig's Disease and other neurodegenerative diseases.

Inno Bio Diagnostics Sdn Bhd (IBD), which specialises in cell-based diagnostics and regenerative medicine, is investing RM4.58 million (~$1.3M) to set up a cell therapy centre at the Universiti Kebangsaan Malaysia Medical Centre (UKMMC) in Kuala Lumpur, Malaysia. IBD, a wholly-owned subsidiary of Inno Bio Ventures Sdn Bhd, aims to create and develop business opportunities in stem cell technology in the area of cell-based diagnostics and therapy. The cell therapy centre, which is expected to start operations next year, will be jointly managed by IBD and UKMMC to develop human hepatocyte-like cells from stem cells that are derived from embryonic, bone marrow, mesenchymal and adipose tissues. The human hepatocyte-like cells can be used for in vitro assays for screening and testing potential drugs for the pharmaceutical and nutraceutical industries. According to Ahmad Zaharudin, the company is projecting a revenue of RM15 million to be generated from the product, knowledge transfer and licensing of technology as outcome of the partnership.

Genzyme Corporation announced that the U.S. Food and Drug Administration has granted marketing approval for Mozobil(TM) (plerixafor injection), a drug intended to be used in combination with granulocyte-colony stimulating factor (G-CSF) to mobilize hematopoietic stem cells to the bloodstream for collection and subsequent autologous transplantation in patients with non-Hodgkin's lymphoma (NHL) and multiple myeloma (MM). The product has also been granted orphan drug designation. Genzyme has submitted an application in Europe for approval of Mozobil and expects approval of the product in the second half of 2009. Genzyme recently filed applications in Australia and Brazil, and additional global applications in up to 60 countries are planned. Mozobil has received orphan drug designation in Mexico which allows the product to be commercialized in the country upon U.S. approval. Genzyme believes that Mozobil may have broad application outside the current indication. Early preclinical and clinical investigations are already underway to explore additional therapeutic indications for Mozobil, including mobilization of hematopoietic stem cells in allogeneic stem cell transplants and tumor sensitization in oncology/hematology treatments such as adult myeloid leukemia.

And finally... it looks like there's a showdown shaping up in Colorado. As disucssed previously on this blog, in July FDA CBER's Mary Malarkey sent a warning letter to Dr. Christopher J. Centeno, Medical Director of Regenerative Sciences, Inc (RSI), doing business as Regenexx. On a recent visit to the Regenexx website, we see that they state the following:

"The Food & Drug Administration's (FDA) role is to regulate the sale and distribution of drugs, devices, biologics and combination products, but not how these products are used by physicians. The FDA does not approve surgical procedures. RSI conducts all surgical procedures within the State of Colorado. There are no interstate aspects to the procedures conducted by RSI that invoke FDA jurisdiction under the Public Health Service Act (PHSA), which requires an interstate nexus. Our surgical procedure involve the use of autologous stem cell therapies (ACTs) consisting of products or treatments incorporating the in vivo use of a patient's own stem cells, following, in certain circumstances, ex vivo manipulation or processing, for the prevention, or treatment of disease. The surgical procedures conducted by RSI including surgery and bone marrow transplants are not performed as part of any research intended for FDA approval of a product that is under the jurisdiction of the FDA. Therefore, the stem cell procedures conducted by RSI are not under the jurisdiction of the FDA. Rather, the procedures conducted by RSI are considered the "practice of medicine," and is left up to individual surgeons' judgment and experience and oversight by the Colorado Board of Medicine." [emphasis added]


And that ends the way I saw the cell therapy industry this week.... More HiLites to come in 2009! Happy holidays to one and all.

--Lee

Cell Therapy Industry HiLites 2008-12-12

Rumor is there was little in the way of cell therapy at the ASH meeting this year and the TERMIS meeting was mostly "basic research". I'd love to hear other reports. Maybe because of that, it seemed oddly slow on the cell therapy front this week. Despite that, we've got some interesting news to review.


This issue of Newseek had an article by David T. Scadden, M.D. and Anthony L. Komaroff, M.D. "Will Stem Cells Finally Deliver? Major breakthroughs are propelling the field. Science becomes medicine."

TiGenix secured grant support to deepen its pipeline. The company has been awarded a €1.81 million, or $2.41 million, grant to support its R&D efforts a meniscus repair program. The two-year program will focus on the further development and application of allogenic stem cells in combination with a biocompatible scaffold, the identification of biomarkers predictive for the quality of meniscus-like tissue, and establishing preclinical proof-of-principle for cellular repair of meniscus lesions.

The European registration procedure for its lead product, ChondroCelect®, is reportedly progressing. As this is the first cell based product submitted to the EMEA under the central registration procedure, some further documentation on the product and process validation processes was recently requested by the regulatory authorities. The company is apparently now compiling further information for submission, and expects the CHMP to issue an opinion on the marketing authorization for ChondroCelect in the first half of 2009. If approved, ChondroCelect will be the first cell-based product to receive a central marketing authorization for an advanced therapy medicinal product in Europe. The preparation of the Biologics License Application (BLA) to obtain product registration in the US is also reportedly progressing according to plan. The company intends to start the registration process with the US Food and Drug Administration (FDA) in the first half of 2009. (for a recent TiGenix update, click here).

Athersys received its third IND for MultiStem in less than a year with authorization to proceed on a phase I trial in the treatment of ischemic stroke.

CIRM grants were approved to 4 San Diego biotechs:

• $827,072 for Novocell to advance development of “an implantable device” to capture and retain pancreatic stem cells.
• $749,520 for a joint effort by Fluidigm of South San Francisco and StemGent, a Cambridge, MA, biotech with operations in San Diego, to develop a screening technology to help stem cell researchers “reverse engineer” skin cells into stem cells.
  
• $869,262 for an application by Invitrogen, now known as Life Technologies, for developing methods of modelling human neurodegenerative diseases in human embryonic stem cells.
• $906,629 for Vala Sciences to develop new and improved techniques for developing mature heart cells called cardiomyocyte cells from human embryonic stem cells.
  

Immunotherapy has certainly seen its share of failures but T-cell oriented therapies are not going away as they continue to attract funding by companies looking to avoid the difficulties that have sunk other efforts to commercialize immunotherapies. One such new company is Adaptimmune Ltd which recently spun out of MediGene AG to carry forward T cell receptor engineering with HIV as its initial target. The company's ex vivo approach sees a patients' own T cells harvested, those cells' receptors genetically enhanced for increased potency against certain infections or tumors in the manner of monoclonal antibody selection, and re-infused into the patient.

Osteotech authorized a stock buyback of up to $5 million.

Novocell announced it will collaborate with renowned stem cell researcher Shinya Yamanaka of Kyoto University to explore the creation of human islet cells from iPS cells. Common wisdom says Novocell has been in the lead in the race to a cell-based approach to diabetes therapy but they are not without competition. Earlier in the year giant Novo Nordisk did a deal with Cellartis and Lund University to create insulin-producing cells for diabetes. Novo Nordisk has been selling insulin since 1923 and knows the diabetes market well. Additionally, Geron announced a publication on its progress coaxing embryonic stem cells into what it calls islet-like clusters.

ERYtech Pharma announced the results of its phase II clinical trial. ERYtech Pharma, the specialty pharmaceuticals company developing red cell-based medicinal products, announces the results of its phase II clinical trial. GRASPALL 2005-1 was presented orally on the 8th December 2008 at the 50th Annual Meeting of the American Society of Hematology in San Francisco (USA). This study aimed to assess the efficacy and the safety of 3 doses of GRASPA®, a new formulation of L-asparaginase, given in frontline treatment for children and adults with
relapsed acute lymphoblastic leukemia (ALL).

In a December 3 press release and conference call ThermoGenesis announced it has reached an agreement with CEO William Osgood for his immediate resignation. This comes as a result of a clear disagreement between Osgood and Board regarding future directions of the company. A search for a new CEO will be launched. In the interim, the board has named Matthew T. Plavan, the Company’s Executive Vice President and Chief Financial Officer, as ThermoGenesis’ interim Chief Executive Officer. The company has announced its intention to reorganize, downsize, maximize, sideline its Vantus business, and dispose of the ThermoLine™ business yet this quarter.

BioLife Solutions was granted a European biopreservation patent expanding its IP related to technologies which minimize cell death during or resulting from cell preservation.

Chemokine Therapeutics Corp. (CTI.TO) filed Chapter 11 equivelant in Canada - a Proposal to Creditors under the federal Bankruptcy and Insolvency Act.

Stem Cell Therapy International Inc. announced the elimination of what it described as "major obstacles" previously blocking its merger with Histostem including the establishing of a relationship with a clinic in Mexico. The companies are apparently in the final stages of completing the merger to create AmStem International and begin creating revenue almost immediately presumably involving a new relationship with U.S. company, Cancun Stem Cell Clinic, Inc. (with its stem cell clinic in Cancun, Mexico) and Histostems cord blood banking operations.

MicroIslet Inc. is reorganizing its way through Chapter 11 with the help of new financing.

Pluristem received a $1.6M grant from the Office of Israel’s Chief Scientist. The non-dilutive funds will be designated to support the clinical trials of the Company as well as research and development activities.The grant covers the development costs of their PLX cells from March 2008 to February 2009. Furthermore, Pluristem plans to submit an application for a new grant to the OCS in March 2009 to cover the Phase I trial of PLX-PAD and pre-clinical studies of PLX-IBD, targeting Inflammatory Bowel Disease (IBD).

HCT/P establishments must complete annual registration by end of this month. Establishments that manufacture human cells, tissues, and cellular- and tissue-based products have until Dec. 31 to complete their annual registration with the Food and Drug Administration. Instructions for listing HCT/Ps have been revised this year, as have policies and procedures for submitting the form. FDA is encouraging facilities to submit the form thr obtained subject to court approval.ough electronic registration. Review the revised instructions or submit form electronically.

Interesting perspective on stem cells in biotech in BIO's year-end summary of the most important trends and stories of 2008 and looking forward to 2009.

In the spirit of passing along interesting resources, here are a couple I came across this week:

• How Stem Cell Research is Transforming Health Care. A panel discussion from the 2008 World Stem Cell Summit with panelists: Alan Trounson (California Institute for Regenerative Medicine), Lawrence S.B. Goldstein (University of California-San Diego), and John Wagner (University of Minnesota). Moderator: Timothy Kamp
• Steven Badylak @ Pop!Tech 2008. Last year regenerative medicine expert Dr. Badylak made news in the medical world when two of his patients re-grew severed fingertips in just six weeks. He spoke at this year's Pop!tech on how he and his colleagues are applying this breakthrough solution to help Iraqi war veterans re-grow tissue and body parts lost in battle.

Finally, it always disturbs me when I miss important news in the industry because it tells me there are holes in my systems. It also troubles me when I discover cell therapy companies that I here-to-fore did not know existed. Both happened to me this week. Nevertheless, better late to discovery than not at all.

One of the more important bits of news I'm late to the table for (announced at the end of October) is the acquisition of "certain of the assets and the continuing business of Celltran Limited (“Celltran”) from its administrators" by York Pharma plc (AIM: YRK) for what it describes - in the very undestated British way - a "compelling valuation".

This seems an apt description when one learns that despite the fact that "approximately £15 million has been invested in the Celltran portfolio of products" to-date, York bought the company's two commercial product plus "a rich pipeline of associated products and technologies" and the ongling business for - make sure you're sitting down - "£70,000 plus the payment of royalties on future sales for a period of five years (at the greater of £100,000 (£20,000 per year) or 10% of future revenues generated by the products over such period)".

The real reason this is such a compelling valuation is that Celltran had - and now York Pharma has - two cell therapies on the market (albeit only in the UK) in Myskin™ and Cryoskin™.

But the news does not end there. York also announced simultaneous to the acquisition that they had completed an institutional placing raising £510,000 (before expenses) to provide "additional working capital for the integration, operation and further development of the assets within York Pharma’s business." York, established in 2003, describes itself as a pharmaceutical group with particular focus on dermatological products which makes the two cell therapy products fit well in their existing portfolio.

So for what its worth...that's the cell therapy industry I saw this week.

Commercially Available Cell Therapies?

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

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

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

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

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

I distributed the survey to members of the following groups:

• those who follow me on Twitter (currently a group 66 individuals sufficiently interested in cell therapy to follow my postings);
• my connections on LinkedIn (a group of 203 professionals in or interested in the cell therapy sector);
  
• the 110+ or so members of the LinkedIn Cell Therapy Industry Group;
• readers of this Blog; and
  
• the roughly 55 members of ISCT's cell therapy commercialization committee.

There was no control on multiple responses and no restriction on participation. Participants were forced to choose only one answer among responses ranging from 1-10 to 71-80 in increments of 10 (e.g., 1-10, 11-20, etc).

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

If we assume that only members of the groups listed above participated in the survey, then it is reasonable to characterize the participants, at the very least, as informed participants who know more about the cell therapy industry than the average person. I will also assume that participants only voted once.

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

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

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

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

Approximately 67 of these 'products' are commercially available in at least one country. There are a number of observations worth noting about these 'products'.

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

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

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

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

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

---

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

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

Should I Stay or Should I Go? What's a Good Founder to Do?

Recently I'm come across some valuable articles speaking to the issue of Founder CEO's. We've got a lot of these in cell therapy. It's not an easy decision to stay or leave and the timing is critical. I pass these articles along without any commentary but for the benefit of the industry.

"Company founders: voices of experience". Interviews with nine leading scientists who founded startup companies reveal some common themes and lessons (Published online: 22 March 2004, doi:10.1038/bioent796)

"Moving on". As a company grows, the founding scientist sometimes discovers (and investors often hint) that it’s time to leave the CEO spot and hand it to someone with more business experience. To ease the possible shock of the process, consider this transition from day one. (nature biotechnology volume 26 number 12 december 2008)

"Rich Versus King: The Entrepreneur's Dilemna". (Academy 2006 Best Paper Proceedings). See here for a blog-post about the core concept and for a fuller article in Harvard Business Review (February 2008) see "The Founder’s Dilemma". Most entrepreneurs want to make a lot of money and to run the show. New research shows that it’s tough to do both. If you don’t figure out which matters more to you, you could end up being neither rich nor king. An interesting post-publication discussion can also be found on Wasserman's blog.

Cell Therapy HiLites 2008-12-05

While the cell therapy sector certainly has more than its fair share of companies spreading their peanut butter thinly to survive their burn rate until their next financing closes, another week of funding news and milestones means its not all bad news out there for the cell therapy industry.

Pluristem Therapeutics Inc. (NasdaqCM:PSTI) announced $750,000 of new investment funds - well actually $600,000 from a restricted share purchase and an additional $150,00 from management and BOD members by way of reduced compensation. But the investor has the option, within 10 business days following the initiation of the Company's first human clinical trial, to put in another $600,000.

Osiris completed enrollment in its first Phase III stem cell trial - this one studying Prochymal for steroid refractory acute GvHD - encompassing 244 patients at 74 centers in 8 different countries (N. America & Europe). The last patient is expected to complete the trial in May 2009. MSCs have been reported as an effective agent in the treatment of GvHD by a number of medical centers. In a recent Lancet publication, Le Blanc et al. reported a 55% complete response rate when using MSCs in the treatment of steroid-resistant GvHD. Many people are watching with keen interest to see how the big questions around Osiris' BLA/MAA submissions/approval and then (hopefully) commercial issues like IP, reimbursement and product revenue models will be addressed as Osiris gets ever-closer to market.

Osiris also received approval from Health Canada to expand access to Prochymal for pediatric patients in Canada with GvHD following a similar ruling earlier this year by the FDA. Under the expanded access program, children 2 months to 17 years in age inclusive with Grades B-D acute GvHD not responsive to steroids are eligible for treatment.

Biocompatibles' CellMed treated their first stroke patient in a phase I/II trial with gene-modified, adult, allogeneic, alginate-encapsulated, MSCs dipped into the brain for 14 days (and then removed). The trial is described as the first "authorized" trial using stem cells for stroke.

In this issue of Cell Stem Cell, an ISSCR Commentary article outlines their proposed Guidelines for the Clinical Translation of Stem Cells, available in full online at the ISSCR website. The recommendations include an insistence on expert evaluation and independent oversight, a thorough informed consent process, and transparency in reporting of clinical trial results. This is aimed at all those stem cell transplant clinics selling direct to consumers with little - if any - regulatory oversight, formal clinical trials, or peer-reviewed publications.

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

Organogenesis announced the receipt of two awards honoring the company's development, manufacturing and economic impact. It's been a long and twisty road for the company reinventing itself from Chapter 11 (filed in 2002) but it's certainly now the real deal and a somewhat unheralded beacon for regenmed.

On the not-so-bright side, Cell Genesys, Inc. (Nasdaq:CEGE) and Takeda Pharmaceutical Company Limited (TSE:4502) announced the termination of their collaboration agreement for the development and commercialization of GVAX Immunotherapy for prostate cancer. Takeda will return all commercial rights to GVAX immunotherapy for prostate cancer to Cell Genesys and make certain wind-down payments to Cell Genesys in connection with the phasing out of the remaining clinical development activities.

In keeping with their unique approach to life ACT has formed another ACT. Worcester biotech Advanced Cell Technology Inc. and Korean biotech CHA Biotech Co. Ltd. have formed a new stem cell technology development company called Allied Cell Technology, to be based - wherelse? - in Worcester. The international joint venture will use ACT’s hemangioblast cell technology to develop human blood cells. Allied Cell Technology will be majority owned by CHA, with ACT (OTC: ACTC) licensing its technology for a $500,000 license fee from CHA. Leading the new company in Worcester will be Young Chung, as well as Shi-Jiang Lu and the ACT hemangioblast team. Robert Lanza of ACT will serve as the chief scientific advisor of Allied Cell Technology.

Katarina LeBlanc of Karolinska Institute has been awarded a $1.2 million prize for her work in "immunological characteristics of mesenchymal cells and their use in mesencymal stem cell therapy". The prize, funded by the Tobias Foundation, will be awarded December 17.

Another wannabe cell therapy CMO takes the exit stage right - I think? WuXi-Apptec intends to close its US biologics manufacture operations to focus on testing. Chalk this up to another of several over the past handful of years who have entered - and shortly thereafter exited - the cell therapy CMO business. It's not an easy game and the true players are in it for the long-term. While they announced Wuxi-Apptec will no longer be doing "biologics manufacturing" in the US they said they are now going to focus on "cell therapy". There seems to be some confusion about what exactly that means. I guess we'll stay tuned.

Finally found a half-decent and relatively objective analysis of the recent European Patent Organization (EPO) ruling on the WARF patents sans spin.

On the gene therapy front, Ceregene reported clinical data from a double-blind, controlled Phase 2 trial of CERE-120 in 58 patients with advanced Parkinson’s disease. Regrettably the trial did not demonstrate an appreciable difference between patients treated with CERE-120 versus those in the control group. Continuing the theme, Introgen Therapeutics, Inc. (NASDAQ:INGN) announced that as part of its ongoing restructuring, the company has elected to file Chapter 11 petitions in the U.S. Bankruptcy Court. The EMEA has accepted, from Introgen's wholly-owned subsidiary in Ireland, Gendux Molecular Limited, a Marketing Authorization Application (MAA) for Introgen's ADVEXIN to treat recurrent, refractory head and neck cancer. However in September 2008, the FDA notified Introgen that the BLA was not sufficiently complete and would not be filed at that time. Demonstrating once again that success and failure are a hair's breadth apart.

HemoGenix announced the implementation of biorisk-free, green-source growth factors and cytokines for use in all HALO® Assay Kits and HALO® Contract Research Services

CBER has been busy these past few months. FDA released another new Guidance this week. This one: Guidance for Industry: Cooperative Manufacturing Arrangements for Licensed Biologics.

NovaRx Corporation appointed former CSO of Invitrogen - remember them? - Norrie J.W. Russell, Ph.D as President and COO this week. His pedigree should be invaluable as NovaRx manages the late-stage development of it lead product candidate, Lucanix(R) - a whole cell-based therapeutic vaccine comprised of four allogeneic cell lines, currenly in Phase III clinical trial for the treatment of progressive non-small cell lung cancer. Once fully open, the trial is expected to run in 90 centers in the US, Canada, Serbia, Hungary, UK, Netherlands, Germany, Poland, Italy, and India.

And finally, on the mere speculation front, yet another analyst has identified Genzyme as a ripe acquisition target by one of those cash-rich, pipeline-poor pharma's on a shopping spree.

That's the cell therapy industry as I saw it this week...

Cell Therapy HiLites 2008-11-28

For what is typically a slow business week in the US, the cell therapy sector had some big news. More money, more deals, and some interesting developments to watch.

Argos Therapeutics completed a second and final tranche of a $35.2 million Series C financing from new and existing investors (the first tranche was completed in April 2008) to support ongoing phase II trials of its RNA-loaded dendritic cell product in renal cell carcinoma and HIV as well as support the further pre-clinical development of its CD83 soluble protein for transplant rejection/autoimmune disorders

In a move I'd say has some elements of "perfect timing", Novocell CEO, Alan Lewis, announced he plans to leave the company after 3 years at the helm to head up the well-funded and well-connected JDRF. Alan positioned the company well and leaves it with a healthier cash balance than many in its position. Due to the nature of ESC work and the financial markets Novocell has an arduous road ahead of it but they are at the forefront and have the backing from some heavy hitter investors.

Sigma-Aldrich entered into a research collaboration with South Carolina's D-Finitive Cell Technologies, Inc. The collaboration is expected to support the development and commercial offering of 14 products over a two-year span with a planned launch of many of these products in 2009 expected to consist of serum-free media for bone marrow and umbilical cord stem cells, mesenchymal stem cells and neural stem cells; cytokine cocktails for stem cell expansion and differentiation; novel formulations for cyropreservation of stem cells; and specialized products to conduct clonogenic assays of the various stem cells. Paul Price, the man behind the science at D-Finitive, is a key component to the deal.

James Thomson's Cellular Dynamics International- focused initially on creating cells for pharma toxicity testing - merged with its two sister companies, Stem Cell Products Inc. and iPS Cells Inc. and raised $18 million (link).

t2cure acquired a patent family in in vitro potency testing patents for testing of stem cells for cardiovascular applications which it intends to use in its phase III trial starting next year.

Cytori announced it is strengthens it European Union sales, training and service operations with key hirings and expansions. The company is introducing its Celution^® 800 System in Europe into the reconstructive surgery market. The Celution® Technology has been developed as a bedside device to enable real-time, adult stem and regenerative cell processing. The technology automates and standardizes the separation and concentration of a patient’s own (autologous) adult stem and regenerative cells from body fat (adipose) for real-time redelivery to the same patient - at the moment primarily patients recovering from the effects of breast cancer treatment.

Something's up at Stem Cell Sciences. Last week they announced they had signed a Master Service Agreement of up to five years with Pfizer Limited, the UK operating unit of the global research-based pharmaceutical company Pfizer, Inc. (NYSE: PFE - News). Under the terms of the agreement, SCS will provide research services, cell lines, media and reagents to support Pfizer's R&D efforts in the field. It is no secret the company has been busily reinventing itself and signing of a major deal with a pharmaceutical has been one of their stated goals since they did a major re-org of the company. Nevertheless, on the heals of the deal the company announced that they requested a temporary suspension in the trading of its shares ahead of what it described as a review of its financial position and strategic options. It will be curious to see what transpires.

As Jon Rowley pointed out, last week I missed Aastrom's announced treatment of the first patient in its U.S. Phase II trial of their Cardiac Repair Cells (CRCs) in the treatment of dilated cardiomyopathy (DCM).

That's just the way I saw cell therapy this week...

What's in a Name? The FDA's Got Yours.

At the ISCT-FDA Somatic Cell Therapy Symposium this Fall, there was much discussion about product nomenclature for cell therapies. It was a new subject for many in the audience.

CBER observed that several organizations in the sector are now laying the groundwork for setting standards in product nomenclature of HCT/Ps as a means of supporting and facilitating reimbursement, commerce, quality control (standards), labeling, etc. By asking that the session be included in the meeting, CBER wanted to ensure that such efforts were done in compliance with the product nomenclature framework that FDA already has in place.

Obviously this was top-of-mind also because at the time they were putting the finishing touches on a draft guidance for industry entitled "Contents of a Complete Submission for the Evaluation of Proprietary Names" which was just released.

For more background information, review Kim Benton's slides from the Somatic meeting. Darrin Weber also reviewed USP's efforts in putting together a product nomenclature monograph and Fran Rabe summarized ICCBA's efforts in applying the ISBT-128 labelling to human cells and tissue products. Click here for a link to these presentations (listed at the bottom of that page).

The guidance applies to proprietary name submissions for prescription drug products, including biologics, that are the subject of a new drug application (NDA), an abbreviated new drug application (ANDA), or a biologics license application (BLA), or that are currently the subject of an investigational new drug application (IND) in anticipation of submission in a marketing application.

The FDA does not perform premarket name review or labeling review of HCT/Ps regulated under PHSA 361 .

The draft guidance is open for comment for 60 days from the date it was published in the Federal Register.

Cell Therapy HiLites 2008-11-21

This is a week in which 2 of GEN's top 7 News of the Week stories were about regenerative medicine and they weren't even the two big financing events. Not bad.

Good news for Tissue Genesis Inc and their Commercialization Consortium partners Bioheart and VetStem. Effective immediately, the Tissue Genesis Cell Isolation System will be available to the European marketplace.

In a piece about biotech survival, Luke Timmerman, now of Xconomy.com, reports that according to an October report by Eun Yang, an analyst with Jeffries & Co., "about half of the 248 unprofitable biotechs that are publicly traded have less than a year’s worth of cash on hand".

Dr. Michael D. West announced an open letter to President-Elect Obama signed by 22 scientists and economists (including 19 Nobel laureates), urging the new Administration to make regenerative medicine, specifically, the cure of human disease, a national priority, similar in scope to the Kennedy Administration’s commitment to land a man on the moon within that decade.

Aldagen announced positive phase 1/2 results for ALD-301 (an autologous, bone marrow-derived stem cell population expressing high levels of ALDH) for critical limb ischemia.

The story everyone loved this week was the new trachea built from autologous stem cells and effectively transplanted in Spain. Chalk one up for more proof-of-principle but more consternation on the "business models" side of things. Researchers and surgeons from Britain, Italy and Spain collaborated to grow tissue from Ms Castillo’s own bone marrow stem cells, using them to fashion the new bronchus – a branch of the windpipe. Details of the transplant, performed by Paolo Macchiarini, at the Hospital Clinic of Barcelona, are published online in The Lancet and discussed in accompanying commentary. First a section of trachea was taken from a donor and stripped of cells that could cause an immune reaction, leaving a grey trunk of connective tissue. Stem cells were then taken from Ms Castillo’s bone marrow and grown in Professor Birchall’s laboratory to cover the 7cm graft. It was then “seeded” with the new cells using a process developed in Milan. Finally the trachea, covered in cartilage and lined with epithelial cells, was cut to shape and fitted. The researchers said that the surgery could help some patients in Britain but admitted that the procedure was too expensive to be widely available. They are seeking EU funding and commercial sponsors for trials to create and transplant a larynx, an operation that could be more cost-effective.

The two stories I loved the most this week were finance-related.

Last week Stem Cells Inc. announced its intention to raise $20M gross and this week it closed the deal netting $18.5 million from institutional investors.

Not to be outdone, Tengion raised $21 million in a second close of its Series C adding Safeguard Scientific to its heady roster of investors.

What money does exist for investing in cell therapies appears to be increasingly focused on a handful of companies that have demonstrated a somewhat mysterious capability to ease money from investors. Stem Cells, Inc and Tengion are two of the best along with MolMed in Italy which netted around $76M in an IPO earlier this year. Osiris has also done well on the public markets but also through the undying commitment of Peter Friedli and timely deal-making. Notable mention should also go to Aldagen (despite them having to recently pull a planned ~$80M IPO), Novocell, Geron, and Dendreon who all have done well in the past with investors.

This trend for capital congregation is nothing new. It certainly supports popular notions that either investors all think the same or they are lemming-like in their movements. It certainly does not bode well for the plethora of companies out there that are on the brink of burnout despite having seemingly strong management and data but do not seem to have that same type of investor-attracting charisma as the likes of Weissman/McGlynn, Nichtberger, Bordignon, Mills, Lewis, Okarma, or Gold.

At the end of the day, a company's success almost always comes down to its rainmaker. This is not a "if-you-build-it-they-will-come" type of industry. Great science, technical prowess, manufacturing experience, or even business acumen will not get companies over the threshold without that someone with that somewhat magic prowess to relieve investors of their money. Many cell therapy companies are now learning that lesson.

In the interim, from a mere objective point-of-view I could argue it's not a bad thing that investors focus precious resources on a few companies' products, get them approved, and get some fresh upticks in the "precedent" column for cell therapies and the new therapeutics and business models they represent. Like lawyers - and perhaps its because there are so many law degrees in the halls of VC firms - nothing makes them more comfortable than precendent.

That's just the way I saw cell therapy this week...

How mature is the cell therapy industry? Do we know?

Survey
The week of November 17, 2008 I posed the following one-question survey:

How many phase III or pivotal industry-sponsored cell or gene therapy clinical trials do you believe are currently ongoing in the world?

to members of the following groups:

• those who follow me on Twitter (currently a group 43 individuals sufficiently interested in cell therapy to follow my postings);
• my connections on LinkedIn (a group of 188 professionals in or interested in the cell therapy sector);
  
• the 90 or so members of the LinkedIn Cell Therapy Industry Group;
• readers of this Blog; and
  
• the roughly 55 members of ISCT's cell therapy commercialization committee.
  

There was no control on multiple responses and no restriction on participation. Participants were forced to choose only one answer among responses ranging from 1-5 to 50+ in increments of 5 (e.g., 1-5, 5-10, etc).

Data, Assumptions, & Observations
There have been 18 responses to-date.

If we assume that only members of the groups listed above participated in the survey, then it is reasonable to characterize the participants, at the very least, as informed participants who know more about the cell therapy industry than the average person. I will also assume that participants only voted once.

Based on these assumptions, the data leads to one simple, overarching observation:

There is significant divergence of perception (and perhaps misconception) about the maturity of the cell therapy industry even among those in and/or informed about the sector.

My database tells me there are 33 industry-sponsored cell or cell-mediated gene therapy trials currently underway around the world.

45% of survey participants answered fewer than 15 to the survey question; 33% indicated more than 45. In fact, only 1 participant (6%) do-date has chosen the category of 30-35.

Analysis
Admittedly this is an unscientific and informal survey. Furthermore there is likely some margin of error to account for different interpretations of what "industry-sponsored" means and/or what types of products should be included/excluded as "cell or gene therapies" since none of these terms were defined for participants.

Certainly in my day-to-day dialogue with people in the industry, the typical opinion is that there are but a handful of late-stage cell therapy trials (far fewer than what my data tells me actually exists). Having said that, when I asked a colleague in the industry yesterday to respond verbally to the survey question, they said "350" ( and then pulled back to say "maybe 200").

There is no doubt my data likely lacks a material number of industry-sponsored, phase III or pivotal gene therapy trials - given that the focus of my database is cell therapy and thus typically only includes cell-mediated gene therapies. This exposes a weakness in comparing the survey results to my data in that the survey related to "cell or gene therapy" while my data likely only reflects a small subset of gene therapy trials.

There is also without doubt more than 33 cell therapy or cell-mediated gene therapy clinical trials currently underway in the world with many of the indicia of a "late-stage" trial if one includes what is happening in markets where such trials or products are currently unregulated. I have not included these in my data because there is no mechanism for identifying whether such trials are, indeed, phase III or pivotal as there is no mechanism as yet in those markets for formal review or approval of these trials and/or products.

Nevertheless, what conclusions can/should be drawn?

• There is much divergence of perception on the maturity of the cell/gene therapy industry even amongst those in the sector.
• Even if my data is inaccurate, compared to whatever the real number is there is a significant % of industry professionals who are suffering from significant misconceptions about the true number of late-stage industry-sponsored trials in the sector.
  
• Good data on the state of the cell/gene therapy industry is not readily accessible.
• If those in the industry lack consensus on the state of the sector, it is no wonder investors and others outside the industry have mixed opinions on the matter.
• We need to do a much better job as a sector in gathering and communicating solid data on the commercial side of the cell/gene therapy industry.
  

For those interested in the data, here's how it looks:


Industry-Sponsored Cell or Gene Therapy Phase III or Pivotal Clincial Trials (Nov 2008) - DocStoc

Coming soon...a similar discussion on the number of cell therapy products currently commercially available around the world.

In the interim, if any of you have data relevant to this discussion, please share...

Cell Therapy HiLites 2008-11-14

Here's what caught my attention this week:

Celgene received FDA clearance to proceed with its placenta-derived stem cell clinical ttudy - a phase I, multi-center trial for moderate-to-severe Crohn's disease.

MiscroIslet filed for Chapter 11 bankruptcy protection but expects to restructure as a private company, continue operations, and file its first IND by year's end for an encapsulated porcine-islet product to treat diabetes.

Celladon released promising phase I results for the first gene therapy trial for advanced heart failure.

Cytori is starts to sell some of its StemSource® cell banking systems for collecting, processing, and storing adipose-derived cells. The company received its first order from Asian distributor Green Hospital Supply. The cell bank is to be set up in Japan. Despite all the continued skepticism about the value of cell banking to the donor-potential-patient, cell banking businesses are popping up prolifically around the world on the promise of short-term revenue to fund long-term R&D while providing a service some believe to be of irreplaceable value.

Although technically not a company-related cell therapy story, one of the more interesting developments this week was the announcement out of Charite in Berlin that they have a patient who is AIDS-free following a unique stem cell transplant. The patient developed leukemia which the docs were going to treat with a stem cell transplant. But rather than do the routine transplant by finding a match and treating just his leukemia they dared to be a little different. They went looking for a donor that matched and also had a CCR5-gene mutation (a naturally-occurring mutation which makes life difficult for the HIV virus according to some studies). The Berlin team found 80 compatible bone marrow donors for their patient, an unusually high sum, two of whom had the CCR5-Gene mutation. One of these they used with surprising and heartening results. What I like about this story almost as much as the happy ending and exciting discovery is how the doctors are trying to control the hype around this single-patient data and early results. It's not likely that this kind of transplant is a future treatmtent (for reasons related to supply, demand, and other commercial realities) but perhaps there is a kernal of a cell therapy product here somewhere that someone as innovative as these doctors will develop in time.

Ok I'm late on the draw here but it just came to my attention that in August NovaRx initiated a pivotal phase III in lung cancer patients. Watch on this blog in the coming days for more on the perception-vs-reality gap related to the number of industry-sponsored cell and gene therapy trials currently active worldwide.

Introgen may disappear while on the brink of success. Despite have an MAA being reviewed in the EU for their gene therapy for head and neck cancer, their BLA submission for the same therapy was rejected by the FDA as incomplete (currently under appeal). The company is now "exploring options" after a troubled Q3.

While not for its cell therapy program, Athersys announced it had achieved a clinical development milestone in its Bristol-Myers Squibb alliance. The amount of the milestone payment has not yet been revealed but any revenue is good revenue to offset the cell therapy development burn.

Bucking recent trends, StemCells Inc. (STEM) announced it will raise $20M in a stock sale to select institutional investors. Good news for any company in these times. Even better news for the cell therapy sector!

Finally, in the most over-reported news of the day, Pfizer opens up a regenerative medicine shop. I agree this is big news. This is not a minor investment; it's not throwing some money at a portfolio of biotechs in hopes that something will stick; it's not using stem cells as tools for drug testing or discovery. This is evidence of a belief by those in the halls of some of the tallest towers in life sciences, that cell therapies are going to change the way we treat people. Here are a couple quotes:

“Pfizer Regenerative Medicine represents a great opportunity to focus world-class research in a field that holds considerable promise for biomedical science and for the treatment of many debilitating conditions such as diabetes and neurodegenerative disorders.” said Rod MacKenzie, PhD, head of Worldwide Research at Pfizer.

The unit will be led by Chief Scientific Officer, Ruth McKernan, PhD, who said “I’m very excited to lead this new research unit. While there is still a lot to understand about how stem cells can be used therapeutically, we believe it is one of the most promising areas of scientific research.”

Ok, one more. Corey Goodman, PhD, President of Pfizer’s Biotherapeutics and Bioinnovation Center, said, “The formation of this new unit represents another key step forward in Pfizer’s commitment to be at the forefront of new approaches in biotherapeutics and bioinnovation and to expand our research efforts and expertise into emerging areas of biomedical science, like regenerative medicine, that have great potential for human health.”

Apparently the unit will operate independently (in the fashion of smaller biotechs) with about 70 researchers when fully staffed - in both Cambridge, UK and Cambridge, Mass - and work collaboratively with "leading academic, biotech and pharmaceutical partners around the world.


That's the cell therapy industry as I saw it this week. Happy weekend.

one-question survey

I am trying to pull together some data on "perceptions" regarding the cell therapy industry and ask that you complete my one-question survey by clicking here: http://tinyurl.com/62brra

Thank a veteran today

We may not agree on everything...

We may not even agree on the need for war.
But we can agree that our world would not be what it is today without brave service men and women willing to fight oppression and protect freedoms.

Thank a veteran today.

Cell Therapy HiLites 2008-11-07

I'm not going to turn this blog into a news service by any means, particularly since I wouldn't want to compete with what I already created in Cell Therapy News - still the single best news source for this sector. I'm going to aim, however, to put out a brief weekly email highlighting what I believe to be some of the key content I noticed during the week (news or otherwise).

For what it's worth, here's what I noted this week.

Athersys, Progenitor Cell Therapy, NeuroNova, Stem Cell Sciences, and Amorcyte featured in GEN article on regenerative medicines. GEN Oct 15 2008 (Vol. 28, No. 18)

Onyvax publishes data supporting the notion that the intersection of biomarkers, personalized medicine, and cell therapy is just around the corner and it will turn medicine on its head. They believe they have identified a biomarker profile that could be predictive of response to cancer vaccines.

Science Direct's Top25 Hottest Articles for 2008 Q2 have a very high ratio stem cell-related publications.

It was a big week for BioLife Solutions with several new adoptions of their preservation and storage media as well as the filing of their second MasterFile with the FDA.

On the economic front, the Genzyme-Osiris deal (worth a potential $1.4B) was obviously great news for a sector so keen to see signs that investors aren't shunning the field but on the other hand is something of a confirmation that companies will have to get creative to survive what Burrill projects will be a lengthy dry-spell in terms of biotech investment.

Invitrogen & Applied Biosystems resurrect the Life Technologies brand as the new name for their merged entity.

Sartorius Stedim relieves us of the confusion of having two Wave Biotech's out there with single use bioreactor systems (SUBs) by purchasing the European Wave Biotech AG. (full story)

The FDA takes a new approach to the annual HCT/P Establishment Registration Updates. (link)

Finally, if you haven't checked out www.biobusiness.tv yet you really must. Jean-Loup Romet-Lemonne has created a valuable biotech business video content channel that promises to focus a great deal on cell-based therapies because of Jean-Loup's experience in and passion for cell therapies.

Until next week...

Don't yet feel the pain of ignoring social media? Just wait a minute...

Only a few minutes ago (relatively speaking in the passage of time) most companies didn't think they needed to have a website or a domain name strategy. This seems as foolish now as ignoring social medial will seem in a few minutes from now.

In 1995 - a mere 13 years ago - I was a young associate in a downtown law firm making a pitch to a skeptical management committee at a weekend retreat that the firm needed a website and an email "system". It was not an easy sell. They "knew" lawyers would never send their own emails and certainly not use it to communicate with clients. Similarly they were convinced a website would likely not bring in any new clients and existing clients would not likely find a website useful. In the end, the only thing that convinced them of the need for a website was that it would likely be a good recruiting tool because it would make them seem hip to all the kids coming out of school. They finally also acquiesced to email only because it was seen to be an easy way to exchange documents with fellow attorneys.

How quickly they were proven wrong.

On a similar front, not that long ago many companies were caught without the domain names they would later need - or wish they had - related to their company names, product names, IP, etc. The most coveted .com domain name turf went to those with early foresight to be proactive and/or those with deep enough pockets to buy back the domains they wanted. Many companies wished they had not ignored the importance of domain names to their overall IP and business strategy.

We are at a similar technological threshold. Most companies are ignoring the importance of "social media" to their corporate strategy. Most consider using social media an unnecessary "luxury" in exactly the same way websites, domain names, and email were considered by people in their positions only a few years ago. They do not understand how social media is already changing the way they do business, the importance of staking position/profile early, nor the pain they will feel in the very near future for having failed to be proactive in building and leveraging online communities.

This is very understandable. In times of fiscal constraint one tends to focus on that which hurt you yesterday or causes you the most pain today. Nonetheless, the companies that will succeed tomorrow are looking at tomorrow now and preparing for it. Tomorrow's mainstream business activities involve social media as much as today's involves website and email.

For those thinking that this will be true only for businesses that deal direct with the retail consumer, you're dead wrong. If you have a constituency that you need to keep informed about and actively engaged in your company, then this applies to you. Whether its business (B2B) customers, investors, media, patients, recruits, employees, or collaborators, they are online and engaging in social media. If you're not there, they're listening to and engaging with someone else.

For those thinking this is just for tech companies or small companies (with small budgets that can't afford traditional media), you're also wrong. Pieter Kim has put together an enlightening list of examples of big companies now using social media: (http://www.beingpeterkim.com/2008/09/ive-been-thinki.html).

Let's start with some basics. I'll define "social media" as the 'interactive' side of the internet. So not the 'passive' sites where there is a one-way feed on information but rather Web 2.0 - those sites that have the capacity for readers to provide content, interact with the author/each other, and/or post comments or feedback. I'm talking about blog, podcasts, online communities, discussion boards, Wikis, and social networking sites.

This year Bioinformatics LLC announced the results of a survey of 1,500+ scientists in the life sciences primarily from North America (47%), Europe (36%), and Asia (11%) (http://lifesciencesocialmedia.com). Among their top 10 findings were the following:

1. Scientists interact with various forms of social media intermittently throughout the day

• 27-32% spending1-2 hrs/day on blogs, content aggregators/portals, discussion boards, social networking sites, audiocasts, and/or wikis).

2. Social media provide scientists with a fresh perspective in their decision-making process

• 30% said it has helped them "discover more options to solve a problem
• 24% "faster access to news, research, and trend information"

3. Social media appeals to the most fundamental values of science — communicating, contributing and collaborating.

• ~50% of respondents said blogs, discussion boards, and/or social networking sites "facilitate the sharing of ideas with colleagues and/or the scientific community"
• ~30+% said blogs, content aggregators, podcasts, and wikis allowed them to "make a more educated decisions about purchasing new products and/or technologies"
• ~40% said content aggregators, podcasts, and wikis made "it easier to research new products and/or technologies"
  

4. Tried-and-true discussion boards still dominate the social media landscape (50%) but online communities/social media sites were used by 30% of respondents in their "research and/or professional activities". Only 23% said they did not use any social media of any kind in their "research and/or professional activities".

5. Over half of respondents said they participate or "visit but don't participate" in online communities for their "research and/or professional activities.

6. Scientists agree that social media has influenced their purchasing decisions — but not the purchasing process.

7. Scientists want content that helps them better do their jobs. The top objection to using online communities was that "it would take too much time to maintain". Yet among users of social media, one of the top benefits cited was that it helped them do their jobs better.


There is no question the internet is now considered a primary resource of passive information access. Web 2.0's social media is all about making the internet the place to exchange information. Just as the best part of a lecture is often the post-talk Q&A or informal networking, web 2.0 is about online discussions. Users/participants of social media know that to maximize the value of these tools, one must share. The biblical maxim, "Give and you shall receive" applies here. You get out the same kind of value you put in. It's a discussion. It only works if both sides contribute. As with any form of networking, it always comes down to exchanging value. If you're only about self-promotion and not sharing something of value, you will be ignored and the benefits of social media will elude you.

Increasingly your constituents (customers, collaborators, investors, patients, employees, etc) expect to see you making a contribution to the online community. For many, being a good corporate citizen involves contributing value and exercising transparency and accountability. Increasingly they will select to do business with those companies they see and interact with online.

J&J and other pharmas now have blogs used to inform and interact with their constituents - media, patients, investors, etc. Many now have patient-oriented and/or disease-specific websites loaded with social media features. Even the NIH is using YouTube for clinical trial patient recruitment.

It is not enough now to build a website. For example, I spoke with a potential client just this week moaning that despite having a site for over a year it simply isn't showing up on search engines. You have to position your online content so people find it, have content that is useful to people vs simple self-promotion, and find ways to engage people: make them participate and keep them coming back.

Think beyond the website. Think professional society sites (e.g., ISCT, blogs). Think content aggregators (e.g., Cell Therapy News, BioSpace). Think business utilization of social media tools (e.g., LinkedIn, Facebook). Think blogging - corporate blog, employee blogging, following and commenting on blogs, and microblogging (e.g, Twitter). Think online discussion boards - monitoring for and posting content related to your business. Think using online tools that enhance search engine optimization and profile (e.g, Wikis). Think online audio-video content (e.g., podcasts, YouTube, Flickr) for education, training, profile, patient-recruitment, and general profile among media, investors, employees/recruits, etc.

And just in case you're still not convinced that your company must have a social media strategy, consider that your employees and customers are already there and likely dropping your company name. Here's a couple examples. I read people all the time on Twitter who regularly slag their job talking about things their boss would likely cringe at knowing were not just shared with a few friends but with all of Cyberspace in a searchable, eternally-archived way. If anyone is looking to work at their firms, a few online searches will bring up these threads. Another story I heard first-hand recently was of a young employee who was simply posting events of his day online including the troubles they were encountering with the technology s/he was working on. Information of the kind that would have been very informative to the competitor and not so comforting to the customers!

One simple application with enormous and immediate potential ROI is leveraging social media for sales and customer support. Online discussion groups or user groups are the easiest way to support customers and provide an information database for CSRs and product development teams. Building and mining "contacts" on social media sites is the modern-day version of the rolodex for sales teams. Contact lists easily convert to lead sheets, newsletter subscribers, and customers. Active participants in topical groups on social media (e.g., the Cell Therapy Industry group on LinkedIn) can be used to identify thought leaders, champions, detractors, etc. Furthermore they can be used as idea generators, beta thought-testing, focus groups, recruitment tools, etc.

Finally, hearkening back to my lawfirm days...maybe you just decide to use social media to recruit the new, smart kids. Just remember, its a little different world online so make sure your participation is genuine, transparent, and of value. With recruitment becoming increasingly competitive, leveraging social media may well be the only way to find and attract top talent.

Bottom line? There are so many different kinds of social media out there that can be used to reach so many different kinds of audiences at a fraction of the cost of traditional media that it's just good business to figure out how to use it to your advantage. In fact, you may be feeling the pain of ignoring it already and just don't know it...