Cell Therapy Industry HiLites 2009-07-03

Last week I mentioned the Regenerative Medicine Foundation and the conference they have announced for Spring 2010. I promised to find out more and bring you what I learned.

I've discovered that the driver behind the RMF is Anthony Atala and Case Western. It is a 501(c)(3) charity, non-profit organization. It was formed in 2005 by Dr. Atala to host a meeting with the U.S. Food and Drug Administration (FDA) around the topic of regenerative medicine. In 2006, the Foundation was instrumental in the formation of STRAC, the Soldier Treatment and Rengeneration Consortium, a national partnership of leading military and academic research centers and industry. STRAC formed the basis for the Armed Forces Institute of Regenerative Medicine (AFIRM).

Now the Foundation announced the first annual "Translational Issues in Regenerative Medicine" Conference, to be held in Winston-Salem, NC, December 8-10. The conference promises to focus on best practices in clinical trials and GMP facilities, as well providing a forum to discuss regulatory and reimbursement challenges. In addition, the conference will feature a venture forum to introduce early-stage and later-stage companies to accredited and institutional investors.

As its name suggests, the organization functions like a "foundation" rather than a member-based organization. This is reinforced by the fact the "Participate" page on the website has no content.

All of this is interesting but nothing more than you would learn from the RMF's website. What I DID discover this week that isn't obvious from anything yet online is that Atala has also been a primary driver behind the creation of a newly announced organization, this one a 501(c)(4) organization intended to be much more about lobbying.

This week the Alliance for Regenerative Medicine was formerly launched. (no website yet). Headquartered in Washington, DC, the Alliance is "dedicated to promoting regulatory, research, and reimbursement policies that will foster innovation in regenerative medicine" in addition to serving as a "source of information about regenerative medicine for policy makers, the media, and the general public".

With Atala's 'encouragement', Michael Wener of DC law firm Holland & Knight and Morrie Ruffin of Adjuvant Global Advisors signed up to be the driving force in the creation and evelopment of the Alliance intented to bring together academic institutions, non-profit organziations, and companies that commit to supporting and participating in a lobbying organization to promote much-needed policies in support of the regenerative medicine sector.

The organization intends to announce its final list of charter members later this month. Thus far its a respectable list including Wake Forest Institute for Regenerative Medicine, Stanford University, the University of Washington, Georgia Tech University, the Genetics Policy Institute, Geron, Johnson & Johnson, Aldagen, iZumi, Fate Therapeutics, Maxcyte, Kleiner, Perkins, Caufield and Byers, and Proteus Ventures.

Werner and Ruffin both have a great deal of experience with Capitol Hill and the biotechnology sector. I had a great conversation with Werner this week and am very encouraged by the plans for the Alliance and their intended inclusiveness. In my opinion, the Alliance promises to fill a much-needed gap unmet by any existing organizations. I will dedicate a post to more information about the Alliance in the days to come.

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More stakeholders united in one place at one time than any other.

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FINANCIAL

Surprisingly, the past week or so - here in the beginning of summer with July 4th weekend being most everyone's primary focus in America - was a very big week for closing financing deals in the cell therapy sector.

In this financial environment the ability to raise any money is a marked accomplishment. Nevertheless four companies in this emerging field did just that: a technology company, a service-based one, a company developing a therapeutic, and one that sort of does all three.

Celsense, Inc. announced they recently raised just over $1.76 million in additional funds in their Series B.

The funds will reportedly be used to commence human clinical testing of its lead compound, Cell Sense which is a fluorocarbon tracer agent used to label cells ex vivo without the use of transfection agents. The technology reportedly enables researchers and clinicians to non-invasively track the administration and migration of the labeled and transplanted therapeutic and/or diagnostic cells using 19F MRI or MRS. Applications include tracking cells in immunotherapy or regenerative medicine as well as diagnosis of inflammatory sites by tracking selected populations of immune cells.

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NeoStem, Inc. (NYSE Amex: NBS) has also bucked the trend recently by tapping into Asian investors and deal-making. NBS announced that it now has raised additional gross funds of over $4 million from institutional and private investors. These funds, together with the $11 million private placement from three Asia-based investors announced April 13, 2009, bring the total investment obtained by NeoStem in the past two and half months to over $15 million.

The funds are said to be tagged for sundry purposes including support of the company's continuing initiatives with their VSEL (very small embryonic-like) stem cell technology licensed from the University of Louisville, advance NeoStem's expansion activities in China, expand U.S.-based operations, add seasoned management to the NeoStem team with experience in clinical drug development in pharmaceutical and biotechnology industry, fund the administration of overseas based clinical trials, and other general corporate purposes. Suddenly that $15 million doesn't seem like nearly enough for a focus spread that broadly

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Neuralstem, Inc. (NYSE Amex:CUR) announced that an investor fund has purchased $1 million worth of stock and purchased $3 million worth of warrants to be exercised in $1 million allotments to be exercised within a year, three years, five years from the date of issuance. Neuralstem plans to use these proceeds for "working capital" much of which - according to SEC-filings - is currently being spent on legal fees.

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International Stem Cell Corporation (OTCBB:ISCO) entered into a definitive agreement for a $5 million financing investment commitment (the “Investment”) with a biotechnology-focused fund (the “Investor”) that has offices in New York and California. The Company may draw down funds from the Investor as it finds a need, but is not obligated to do so. Funds will be drawn down through the issuance of redeemable Series E Preferred Stock.

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I usually don't track or include reports of grants being received by companies but this one is a good size and - I admit - more importantly I wanted to point you to their unique usage of social media.

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Pluristem Therapeutics Inc. (NasdaqCM:PSTI) (DAX:PJT) has been awarded a $1.9 million government grant from the Office of the Chief Scientist (OCS) at the Ministry of Industry, Trade and Labor of Israel, as a government participation in R&D expenses for the period March 2009 to February 2010. The OCS awards grants to industry in Israel to foster technological innovations. This is the fourth consecutive year that Pluristem has received this grant. The funds will be designated and used by Pluristem to support the clinical trials of the allogeneic placental-derived adherent stromal cell product, termed PLX-PAD, for the treatment of critical limb ischemia (CLI), the end-stage of peripheral artery disease (PAD), as well as for other research and development activities of the Company.

Here's the cool part: Check out their product animation on YouTube.

CLINICAL

Ok, this isn't exactly a clinical story but it's the closest thing I've got this week and I hate leaving sections bare.

Geron Corporation (Nasdaq: GERN) has published in advance of print in the journal Regenerative Medicine a paper that reports that dendritic cells (DCs) scalably manufactured from human embryonic stem cells (hESCs) exhibit normal functions of naturally occurring human DCs found in the bloodstream. This, they say, supports their planned use of hESC-derived DCs in their therapeutic vaccine currently in Phased II for AML but employing autologous dendritic cells. (see GEN's summary here)

COMMERCIAL

The big deal of the week, of course, was between GE Healthcare, a unit of General Electric Company (NYSE: GE), and Geron Corporation (Nasdaq: GERN).

The companies announced an agreement to co-develop human embryonic stem cell (hESC)-derived assay products for in vitro drug screening with the aim of having products on the market in by "early 2010".

Under the worldwide, exclusive license and collaboration agreement, GE Healthcare obtained an exclusive license to Geron’s hESC IP and a sublicense under Geron’s rights to foundation hESC patients held by the Wisconsin Alumni Research Foundation. IP arising from the alliance will be shared between the companies, with GE Healthcare retaining rights to the resulting technologies for drug discovery applications and Geron for cell therapy applications.

When Geron announced that its IND had been accepted by the FDA earlier this year, Okarma made quite a bit about how they had figured out how to scale the production of their product " unlike adult stem cell therapies" and that their product was "scalable in the same way as a monoclonal antibody".

This theme continues in this announcement with Geron's David Earp saying "“Geron is intensely focused on developing hESC-based cell therapies, and the expertise that we have developed in scalable manufacturing and differentiation of hESCs to specific cell types is directly applicable to the production of these dells for drug discovery.”

Interestingly, GE claims it is bringing "expertise in cell manufacturing" to the deal. GE Healthcare will fund the R&D program and will be responsible for manufacturing, sales and distribution of products developed under the agreement.

Click here for more background information

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As has been discussed here several times, ever since Cell Genesys Inc (NASDAQ: CEGE) pulled the plug on its late-stage trials of its GVAX immunotherapy triggering Takeda Pharmaceuticals to pull out of its co-development deal for the product, Cell Genesys has been busy dealing with downsizing, lawsuits, and shopping itself around to potential suitors looking primarily to take it on for its cash.

This week BioSante Pharmaceuticals, Inc. (NASDAQ: BPAX) anted up to announce it is merging with Cell Genesys in an all-stock transaction valued at about $38 million - pretty much the exact amount of cash CEGE has in its bank account. the surviving entity will be BioSante.

Cash value is actually a generous purchase price for biotech companies which usually come with a burn rate that makes then worth less than their cash. CEGE, however has no clinical program, had sold most of its assets, and had downsized to 9 staff in a rented office.

There are, of course, persistent rumors of back alley shenanigans that took place in CEGE's demise but we'll leave these on the street. What is interesting to me is that BioSante has suggested they will examine options for the future of Cell Genesys' GVAX Immunotherapies, including potential combination with BioVant, BioSante's vaccine adjuvant, as well as possible external collaborations. The combined entity is also on record saying they will try to outlicense other Cell Genesys technologies.

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Pantheon China Acquisition Corp. ("Pantheon") (OTCBB:PCQC) announced that its shareholders approved its plan to purchase China Cord Blood Services Corporation ("CCBS"). Pantheon has changed its name to China Cord Blood Corporation and will continue to trade on the OTCBB under the symbol PCQC until its expected transition to a larger U.S. exchange is complete. Pantheon China Acquisition Corp. is a "special purpose acquisition company" formed for the purpose of acquiring businesses in the People's Republic of China. Pantheon China consummated its initial public offering on December 20, 2006 generating an aggregate gross proceeds of $34,500,000. China Cord Blood Corporation is currently the largest cord blood banking operator in China in terms of geographical coverage and is the only market player with two (i.e., Beijing and Guangdong) out of six exclusive cord blood bank licenses issued by the PRC government authorities to date. Under the current PRC government regulations, only one licensed cord blood bank operator is permitted to operate in each licensed region.

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Former CEO, Ed Scott, retired late last year from Lifeblood Biological Services, LLC with a plan to buy it out. Scott has now successfully closed that transaction and has rebranded as Key Biologics, LLC. The beauty here is that the company has a steadily growing book of business since its 2005 insception and Scott bought the assets of LBS for $188,000 on previous 12-month revenues of close to $1.6 million.

Key Biologics is being positioned to service the burgeoning cell therapy market. For insight into just some of the market need the company is poised to address, click here to read an article by Scott, previously published a year ago.

The company produces nearly 300 products for researchers involved in cellular-based therapies. Among other types of products and custom services, Key Biologics provides researchers with products made from human blood collected at its office which it converts into customer-specific products like leukocytes, plasma or T-cells shippint them directly to its customers per their specification.

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Living Cell Technologies Limited ASX:LCT; OTCQX:LVCLY) announced that it has now officially opened its new designated pathogen free pig breeding facility in New Zealand to support the imminent launch of its clinical trial in New Zealand of DIABECELL in subjects with type 1 diabes. DIABECELL is an encapsulated porcine insulin-producing cells which can be administered without the need to use immunosuppressive drugs.

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GEN this week carries an interesting article on a company applying its existing technology to cell therapy bioprocessing scale-up. The article sites "...large-scale culturing of embryonic and induced pluripotent stem (ES, iPS) cell lines for use in regenerative medicine and drug discovery applications" as still a "major challenge" for the industry. Cyntellect, Inc. reports that its Stem Cell Manager "is a series of stem cell management processes including automated physical passage of stem cells, automated embryoid body generation, and purification of specialized cell types derived from stem cells" which can help alleviate some of the major challenges in large-scale automation of the processes required to manipulate sensitive therapeutic cells.

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Bio-Matrix Scientific Group, Inc. (OTCBB:BMSN) announced the launch of a national marketing program aimed at hospitals "in need of storing umbilical cord blood specimens for future transplantation".

The business model is not entirely clear to me but it would appear BMSN believes hospital clients will outsource the processing and storage of cord blood units which parents donate to the hospital-owned public cord blood "bank" (or registry). I'm told BMSN may not have the appropriate license for processing of this sort but I'm sure they will be taking care of that. More importantly, I am keenly aware this hospital-based model for selling back-end cord blood processing and storage to hospitals is a very tough sell and not one that hospitals understand easily. For what it's worth, that didn't stop the WallStreetCorner.com from naming the company its “Stock Pick” for the second time since February of this year.

We will watch BMSN's future with interest.

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Toronto-based bio-commercialization center, MaRS Innovation, has selected an umbilical cord stem cell technology as its first commercialization opportunity. The technology offers a proprietary method to create multi-potent stem cells (MPSCs) from human umbilical cord blood with several promising therapeutic applications but which they will initially focus on diabetes. Pre-clinical data has reportedly demonstrated that these cells uniquely secrete insulin in response to glucose, thereby mimicking the “normal” physiological state.

MISCELLANIA

ProChon Biotech, Ltd., a company working with tissue regenerative technologies announced that it recently received the Genzyme Award for Excellence in Cartilage Research for its poster presentation entitled, “Comparative Analysis of Cell Sources for Cartilage Cell Therapy.” The company reports that the research recognized by the award is incorporated into their development pipeline and will compliment its BioCart™ Cartilage Regeneration System.

Prochon's BioCart^™ Cartilage Regeneration System is an autologous chondrocyte implant system that - and here's why I love this story because this is just another example of a commercially-available cell therapy that people in the US never talk about - is commercially available in Italy, Greece and Israel and is undergoing an FDA Phase II multicenter clinical study in the United States. The company reports that to-date over 70 patients have received the BioCart^TM implant with some patients over five years post-implantation.

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I came across a nice piece this week discussing Arteriocyte Medical Systems' work on its DARPA-funded project to create human blood from stem cells at a rate that could allow the military and hospitals to produce units of blood on site. The company claims that with a technology developed by a company called Nanex, they are able to grow stem cells at a rate 10 to 100 times faster than with more traditional methods. The Nanex technology mimics a bone marrow environment in which stem cells can grow quickly. The trick now is to automate and scale-up the system to meet the specs DARPA required in its project paramaters.

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Luke Timmerman, a recent Twitter convert (@ldtimmerman), and one of the most prodigious reporters on the biotech scene, has posted another great article on Dendreon with this great opening line: "Dendreon, as part of its plan to morph into a full-fledged company that discovers, develops, and markets cancer drugs, has worked behind the scenes to transform its shareholder base from a Wild West gallery into a roster of backers you might expect from a blue-chip biotech." The article, "Dendreon Tames Short Sellers, Transforms Itself into Old-School Buy-and-Hold Stock", is - as usual - a good read.

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The LA Times carried a very thorough and informative piece on the state (promise and challenges) of therapeutic cancer vaccines that is also well worth the read.

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Another interesting article I uncovered this week was a rather poorly written but informative review of some the scientific, clinical and regulatory progress in the field of stem cell research/therapy in India.

Sign-off

The latest issue of the journal Regenerative Medicine has been released and it looks well worth the read. Here the table of contents.

Hope this was useful.

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