Cell Therapy Industry HiLites 2009-02-20

Before we get started, here's some good advice:

• renew your membership or join ISCT (www.celltherapy.org)
• if you're on LinkedIn, join the Cell Therapy Industry Group
• register to go this year's ISCT CellTherapy 2009 conference in San Diego
• get on Twitter, and follow my stream (@celltherapy) for timely news and random thoughts
• tell someone you love them.
  
  
  
  

Ok. Another week, another conference, another controversy, another clinical hold...

This was the week of RRY Publications' 4th Annual Stem Cell Summit, organized by investor and analyst Robin Young. Once again, Monya Baker, of Nature's stem cell blog "The Niche" does an outstanding job of summarizing the conference content and tenor in a post called "Investors ♥ stem cells". My only humble comment is that it might have been more aptly named "Entrepeneurs ♥ stem cells" because I don't think the plethora of companies and technologies represented at the conference was due to a flood of investment but rather the irrational (thank God!) enthusiasm of biotech entrepreneurs that keep sectors like this afloat whilst risk-averse investors await the obvious to float to the surface.

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This issue is sponsored by the company setting a new standard for what you can expect in fresh cell and tissue storage.

You owe it to your cells to find out what they know.

www.biolifesolutions.com

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The big pile to hit the fan this week was the PLoS Medicine article published online February 17 describing a patient who developed “brain tumors” (glioneuronal neoplasms) after undergoing repeated transplants of “fetal neural stem cells” in Russia starting in 2001. Fellow bloggers Monya Baker @ the Niche (click here >>) and Alexey at Hematopoiesis.info (click here>>) both provide useful summaries and commentary .

Naturally it is a concerning report for the industry and the regulators. People jumped all over it touting their various perspectives and generating a veritable miasma of opinion. Indeed, so much so that Stem Cells, Inc (NASDAQ: STEM) was forced to publish a commentary on the report in an attempt to distinguish their product from what was apparently injected into the boy in Russia. STEM's commentary is worth the read.

What I will say here - apropos to my ongoing discussion about stem cell transplant clinics around the world enticing people to pay for a shot of stem cells because 'why not? these products are safe' - is that there are stem cells and then there are stem cells and then there are stem cell products. Products are well studied, well understood, well defined, well characterized, well controled, and well tested.

Ex-FDA, regulatory consultant Dr. Darin Weber (blogging at RegenmedGuru.com) has a great post on this whole issue that is well worth the read. He sums up his commentary by saying:

The bottom line is that if you wish to test stem cells in humans in the U.S., the FDA is going to require robust evidence of efficacy and safety in relevant preclinical animal models, regardless of the source.

Now the FDA has an clear example it can point to, for better or for worse, to justify the extensive preclinical studies it requires. The good news is that many developers of stem cell derived therapies, including Geron, have in fact been able to generate the preclinical efficacy and safety data needed to enter human clinical studies.

So why is the FDA "holding back" stem cell treatments that are available in other countries? Why should people think very carefully about their decision to go to a clinic offering stem cell injections for sale? Why is 'industry' critical of what some of these clinics are doing? Why should we be concerned about whether these 'treatments' are as safe as advertised? We now have an example of why.

This is no justification for being over-reactionary about the potential for cell therapy. This is not even a good reason to paint all unregulated stem cell treatments with the same brush. It is a reason to be ever-vigilant in ensuring we are doing our utmost to produce effective AND safe therapies. It is also reason support the dialogue ISSCR is attempting to stimulate to help people discriminate between those clinics following best practices and those which are not.

In the meantime, people are getting shot up with sheep stem cells in Mexico (check it out) ...

FINANCIAL

No news is not really good news here.

CLINICAL

Neuralstem, Inc. (AMEX: CUR) announced today its spinal cord stem cell trial to treat ALS has been put on clinical hold. This seems to be pretty par for the course at this stage. The question will be how long it takes to address the issues. Geron was able to get through their hold in under 9 months; ReNeuron submitted its IND to FDA in December 2006 and has yet to get it through despite being approved to proceed in the UK last month. See Monya Baker's commentary on Neuralstem's hold on Nature's stem cell blog, the Niche - click here >>.

Neuralstem was more forthcoming with details about their hold than Geron was when in the same position months ago. The FDA has reportedly provided the Company with specific comments, questions and recommendations for modifications to its protocol including requests for additional information regarding their product manufacturing process and pre-clinical studies, as well as their clinical delivery injection device and technique. The Company believes that it can "provide this information in an expeditious manner."

Apparently the Agency has also requested various modifications to the protocol and eligibility criteria for patients in the trial, as well as slight changes to the timing of the surgeries. Perhaps more interesting was the comments by President & CEO, Richard Garr that the "Agency had extensive 'non hold' comments, requests for information, and recommendations" which he characterized as issues which will need to be "addressed for final product manufacturing and testing". By that, I'm assuming he means the FDA raised several issues which wouldn't hold up phase I testing but certainly would need to be addressed in future stages.

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HepaLife Technologies, Inc. (OTCBB: HPLF) (FWB: HL1) (WKN: 500625) announced plans for a phase III clinical trial of its HepaMate™ Bioartificial Liver System. The HepaMate™ technology has previously been tested in clinical Phase I and pivotal Phase II/III studies involving more than 200 patients. However the last phase II/III study failed to achieve its primary endpoint. In October 2008, HepaLife announced completion of its acquisition of a liver support technology previously known as HepatAssist from Arbios Systems. It's not clear how, if at all, this is playing into HepaLife's current strategy.

As an extracorporeal cell-based bioartificial liver system, HepaMate™ is designed to combine blood detoxification with liver cell therapy to provide whole liver function in patients with the most severe forms of liver failure. HepaMate™ is comprised of a blood plasma separation cartridge, a hollow-fiber bioreactor filled with proprietary porcine liver cells, a charcoal column, an oxygenator, circuit tubing and a plasma reservoir. These components are assembled into a patented blood/plasma circulation system, which is placed on the HepaDrive™ perfusion platform.

A patented liver cell cryopreservation process provides for safe and easy storing and distribution of the liver cells which detoxify existing toxins, produce albumin and other liver-specific proteins when, during therapy, the patient’s plasma is separated from whole blood, exposed to the HepaMate™ bioartificial liver and returned to the patient.

The company claims only 90 minutes are required to assemble the system, prime the blood and plasma circuits and to process the liver cells due to the logistical advantages of its proprietary cryopreserved pig hepatocyte technology over the use of fresh cells from cell lines or liver tissue.
Thawing, loading and washing of the cryopreserved cells is reported performed at the patient’s bedside using a what the company describes as a proprietary, "FDA-approved" technique.

Apparently a retrospective statistical analysis of the previous pivotal Phase II/III clinical trial data, adjusted for the impact of liver transplantation on patient survival, revealed a statistically significant survival advantage for patients with fulminant and subfulminant hepatic failure when treated with HepaMate™ compared to controls receiving standard medical care alone. The company blames the previous trial's failure on the inclusion of a subset of 24 patients who had undergone a prior, failed liver transplant. It was these patients, the company postulates, that led the the phase II/III trial failing to achieve its primary 30-day survival endpoint in the overall study population. Based on the retrospective statistical analysis of the clinical trial data, HepaLife expects a new Phase III clinical trial without the inclusion of failed liver transplant patients to be successful.

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It's now clear why J&J is closing its Sydney, Australia operation which was investigating OZ1 as its lead product against HIV. Earlier this week a report came out entitled "AIDS: 'Major advance' seen in revolutionary gene therapy'. It was an interesting spin. What was learned from the study in question was a 'major advance' only in the minds of scientists who can chalk this study up to 'lessons learned' from less than positive results. We're no closer to a therapy based on this product.

The report indicated that the world's largest experiment using gene therapy to combat the AIDS virus yielded "a major advance," in demonstrating that the technique is valid, beneficial and safe. "It shows the potential of the gene therapy approach for the treatment of HIV and represents a major advance in the field... [it] can be developed as a conventional therapeutic product," the report stated. Yet later the report admited that while the treatment is safe efficacy was modest. Finally, the report states that in an interview, the principal investigator, Mitsuyasu, said they would not be proceeding past this Phase II trial to the final, third phase of the process. Instead, the team would learn from its experience, modify the technique and start again with tests on a smaller group of volunteers.

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This week NeuroGeneration put out a press release that boldly states it published long-term results of a successful phase I trial using autologous neural stem cells to treat Parkinson's disease. Despite the over-reaching headline, this is not the data from a completed phase I trial but rather the results from the trials' first (and only?) patient. Really more of a case report.

The paper was published in the February issue of the Bentham Open Stem Cell Journal. It outlines the long term results of the treatment of a patient for the treatment of Parkinson’s disease using autologous neural stem cells. According to lead author and NeuroGeneraton owner/founder, Michel F. Levesque, MD, FRCS(C), FACS, "We have documented the first successful adult neural stem cell transplantation to reverse the effects of Parkinson’s disease and demonstrated the long term safety and therapeutic effects of this approach."

The paper described a process by which tissue is taken from the patient's cerebral cortex and expanded for 6 months (yes, 6 month!) before injection. The paper's conclusions:

• Neural stem cells can be isolated from the human adult cerebral cortex, expanded in vitro using epigenetic factors, induced to differentiate into dopaminergic, GABAergic and other types of mature neurons, and selectively delivered back to striatal targets without immunosuppressant.
• Because of their biocompatibility, safety and potential integration into the host striatum, autologous neural stem cell-derived differentiatedneurons represent an alternative to current cell therapy aimed at the restoration of the nigro-striatal circuitry inParkinson’s disease.
  

COMMERCIAL

MaxCyte, Inc announced the introduction of its GT Flow Transfection System at the 4th Annual Stem Cell Summit held in New York, NY this week. The MaxCyte GT Flow Transfection System is marketed as a a validated, scalable technology for customizing the biological activity of cells for therapeutic use. The system, the subject of a US FDA Master File, is designed to enable rapid and efficient transfection of primary cell or cell line with reported >90% cell viability following loading with a broad range of molecules (proteins, drugs, plasmids, mRNA, miRNA, and siRNA) or combinations thereof, at volumes up to tens of billion cells.

What's not clear to me is how this system is different from the clinical scale, non-viral cell loading systems MaxCyte has been selling now for some time and a quick spin through their website has not helped shed any light on that question. Indeed there is nothing about a product launch on their website so this product "introduction" might just be clever spin around a talk about a product that has been on the market for some time. I'll try to find out...

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MedCell Bioscience Ltd (merged last year with NovaTherapy) announced they won the Hong Kong Grand Award for their NovaPod - a compact, manual feed, disposable, batch culture bioreactor that allows researchers and scientists working on stem cells to culture cells in three dimensions. The Hong Kong Grand Award for Industries, Machinery and Machine Tools was presented on 21 January 2009 at the Hong Kong convention and exhibition centre.

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Ok this isn't cell therapy but it's about the so-sexy, so-'now' iPS cells so I couldn't resist. The Japanese unit of German chemical giant Bayer A.G., Bayer Yakuhin Ltd, has announced it is selling a set of patents on producing iPS cells to a U.S. venture firm. The three patents to be sold to iZumi Bio Inc include Bayer Yakuhin’s main method of producing iPS, or induced pluripotent stem cells, which have the potential to grow into any type of human body tissue. Bayer Yakuhin filed for Japanese and international patents on the method in June 2007. The two other international patents are supportive ones for the mainstay one. Bayer Yakuhin’s move has been closely watched since it made patent applications months ahead of Kyoto University.

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StemCellDigest.net did a nice job this week of detailing the historic and rather complicated but much closer licensing relationship than I realized existed between Advanced Cell Technology Inc. (OCTBB: ACTC) , Biotime, Inc, (AMEX: BTX) and International Stem Cell Corp (OTCBB: ISCO). To read more, click here>> .

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ProtoKinetix, Inc (OTCBB: PTKX) issues a press release this week that their synthetic anti-aging glycopeptide, AAGP, is "Critical for Stem Cell Therapy" saying that tests of the compound in conjunction with stem cell preservation studies at all temperatures has shown an "impressive increase in survivability and viability". Reportedly, these results have been consistent regardless of the source of the stem cells. As an example, AAGP(TM) has been independently tested as a protective agent in the cryopreservation and subsequent thawing of umbilical cord blood in low concentrations of AAGP(TM) (4mg/ml). The resultant analysis reportedly revealed a more than double average increase over the control group (cells not protected with AAGP(TM)) in the yield, following thawing. Sounds impressive enough but they provide no links to publications and despite the headline that AAGP is 'critical' for stem cell therapy, I've not seen or heard them at any stem cell conference nor have I seen any of the data published in journals. Curious...

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Stem Cell Therapy International, Inc. (OTC BB:SCII.OB) - another of these companies marketing stem cell transplant treatments in clinics located in countries where they are allowed to do so - has filed its quarterly report. It is a long and twisted tale of corporate re-orgs, reverse mergers, subsidiaries, debt, financings, payments in salaries and consultanting fees, zero revenues, and a pending deal with the Korean company, Histostem Co., Ltd., that was first announced early last year and has yet to close because of some litigation that won't go away. As best I can tell from a quick scan of the SEC filings they did not treat a single patient in 2008 - perhaps that's the good news.

RESOURCES & EVENTS

MIT Enterprise Forum of Cambridge announced an upoming session (March 3rd) on the Future of Biotech with a panel of experts who will discuss Challenges and Opportunities in Stem Cell Therapy and Regenerative Medicine.

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The FDA has posted what could be a very useful presentation if the right (wrong?) circumstances: Writing An Effective 483 Response.

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Curt I. Civin and E. Albert Reece put out a piece this week in the Baltimore Sun entitled "The Stem Cell Opportunity" outlining some of their thoughts on the state, need, and potential for the sector.

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A summary of the most recent ISCT Cellular Therapy Liaison Meeting with the FDA has been posted on the AABB Web site, along with corresponding speaker presentations. Agenda items for the meeting, which was held in October 2008 at the AABB National Office, included "Adoptive T-Cell Therapy for Melanoma: Production Steps and Associated Costs," "Autologous Expanded T-Cell Therapy in Post-Transplant Setting," and "HESC-Derived Cell Therapies: Safety in Translation." View summary


Sign-off...

Well, that's a wrap to an a wee bit of an odd but not entirely bad week in the cell therapy industry. For those of you with any doubts, remember you heard it here first: cell therapy means business.

Cell Therapy Industry HiLites 2009-01-16

Ok, seriously, this week's HiLites blog almost killed me. Hope you find it useful. This was the week for cell therapy I thought last week would be. I'm going to try and recover now...



Dendreon's (NASDAQ: DNDN) Mitch Gold announced at this week's JP Morgan conference (click here to hear the presentation) that the IMPACT study of 500 men with terminal prostate cancer will offer a final answer by April on whether its experimental immune-boosting drug helps patients live longer. When the company went to the FDA with its BLA for PROVENGE in late 2006 and - despite the advisory committee's 13-4 vote recommending approval (March 2007) - the FDA voted against approving it at that point, it was this trial the FDA said they would prefer to see the data from before deciding. There is, as always, a much longer checkered history of ever-changing FDA reviewers, missed clinical endpoints, retrospective data analysis to find a patient subset for which the therapy worked, etc, etc all of which are partial drivers I'm sure for deciding to wait for the data from this second phase III study --- but it all comes down to this. In a mid-point preliminary review of the data, the endpoints were not met - but they were almost met. Mitch Gold is, of course, confident. As was the case in early 2007 there will be much speculation and the stock will be traded wildly and the cell therapy industry will wait with bated breath - though perhaps not quite as bated as 2 years ago when it seemed more rode on the decision than it does now.

The company has built its own manufacturing facility on (perhaps in due course only for) the US East Coast (New Jersey). They still own the worldwide rights to market the drug. They intend to go to the US market with a ~120-person sales force and plan to seek a commercialization partner for marketing outside the U.S.

In other late-stage cell therapy news, Osiris had a successful pre-BLA meeting with the FDA in which they reportedly reached agreement on the timing and content of their anticipated BLA submission. This will be the first US marketing application for a stem cell product making it much-anticipated, globally monitored, and precedent-setting. The entire industry has been waiting for this moment for several years (the last cell therapy approved being 10 years ago!) and will be watching this in much the same way as we were all watching Dendreon's BLA submission a couple year's ago.

Reported key results of the pre-BLA meeting were:
• FDA agreed with the proposed content and structure of the BLA for Prochymal for the treatment of GvHD.
• The primary endpoint of the Phase III trial that will be reviewed by FDA for approval was confirmed to be GvHD Complete Response (CR). A CR is complete remission of the disease.
• FDA indicated that the statistical analysis plan was appropriate.
• The overall safety database (Integrated Summary of Safety) of Prochymal is sufficient in scope for the indication. No additional clinical data beyond what was presented is anticipated to be necessary for the BLA.
• Agreement was reached on BLA requirements for Chemistry, Manufacturing and Controls (CMC) data.
• The FDA concurred with the proposed product stability and process validation plan for submission and provided guidance on how to present the data for ease of review.
• Agreement was reached on the timing and content of the BLA in rolling submission format. The rolling submission is an FDA provision available to drug candidates that have received Fast Track designation, which allows for completed sections of a BLA to be submitted on an ongoing basis. It can facilitate the process by allowing FDA to complete review of sections as soon as they are available. It is anticipated that the nonclinical sections, such as toxicology, will be the first submitted for review.
• The BLA will be in the electronic Common Technical Document (eCTD) format. The eCTD format facilitates the review of the BLA and allows parallel submission of the dossier in other territories.
• At the time of submission, Osiris will also submit the request for Priority Review of the Prochymal BLA. Investigational drugs with Fast Track designation are eligible for consideration for Priority Review, which provides for an accelerated six month application review by FDA

In other news not to be overshadowed by the news about the pre-BLA meeting, Osiris received FDA clearance to broaden the Prochymal Expanded Access Program (EAP) to patients of all ages suffering from life-threatening GvHD. In May of 2008, FDA approved the first Prochymal EAP for the treatment of pediatric GvHD patients. Congress and the FDA created the expanded access program to facilitate the availability of promising new drugs to desperately ill patients before general marketing begins.

If you're interested, click here for an interview of Osiris CEO, Randal Mills, done by PharmaExec.com this week.

Invitrogen launched a xenogeneic-free media for human embryonic stem cell culture.

After now at least 4 years of GE Healthcare's campaign to build a 9-figure cell therapy division, a rocky relationship with Thermogenesis, and the splashy acquisition of Wave Biotech, GE has made another entree into the sector with its announcement of a partnership with Cytori (NASDAQ: CYTX) to commercialize Cytori’s Celution^® System in select European countries - exclusive for 18 mos in select countries. While GE is admittedly not lighting any fires with the pace in which they are building this division it is presumably (hopefully) strategic, synergistic, supportive of its overall business and...please let this be true...something Jeff Immelt will continue to wholeheartedly support despite the perpetual and perpetual re-orgs within the company.

Celprogen claims to have successfully de-differentiated adult somatic cells (primary skin fibroblast, keratinocytes, cardiac, liver and neuronal cells) to their embryonic pluripotent cells (iPCs) using a proprietary non-viral method involving their de-differentation media and matrix system. The iPCs were then reportedly differentiated into cardiac, neuronal and hepatic tissue with Celprogen’s Stem Cell Differentiation system.

The PerkinElmer ViaCord Research Institute and M. D. Anderson Cancer Center have agreed to extend collaboration on a study of cord blood stem expansion systems in adult transplantation. The study seeks to discover methods of treating more adults using cord blood stem cells using 'co-culture' expansion technology. Based on the results of this trial, ViaCord, PerkinElmer's cord blood banking business, and M.D. Anderson will determine the feasibility of using Unrestricted Somatic Stem Cells (USSCs) derived from cord blood in the co-culture of cord blood units for transplantation. USSCs, proprietary to ViaCord (patent pending #09/985,335), are a type of stem cell found in umbilical cord blood, which have the ability to differentiate into many cell types, including endothelial cells, fat, bone, cartilage and neuronal cells.

ERYtech Pharma, French biotech company developing a pipeline of products involving the encapsulation of therapeutic molecules inside red blood cells for a variety of orphan indications, has signed an agreement with Penn Jersey Region of American Red Cross for the "GMP" clinical batches production of ERYtech Pharma’s products in the United States.

I'll seque momentarily to the regretably inevitable 'bad' news ...

On the verge of filing a BLA, Isolagen (Amex: ILE) is forced to downsize its operations to minimize burn-rate and postpone bankruptcy while exploring its 'options'. On November 6, 2008, the Company disclosed in an SEC Form 10-Q that there existed substantial doubt about its ability to continue as a going concern, and that its ability to continue as a going concern was contingent, among other things, upon its ability to secure additional adequate financing or capital prior to January 15, 2009 under its current operating plan and condition. The company announced it was pursuing dual paths, including pursuing potential financing alternatives as well as continuing potential strategic partnership discussion. As of this week neither event has happened according to an SEC filing.

The Company has now deviated from its normal operating plan and is focusing its remaining cash resources on specific clinical programs, namely efforts to complete its Biologics License Application related to the Company’s Phase III Nasolabial Fold study, and continuing its IT-R-008 Phase II/III Acne Scar trial. All other clinical programs have been suspended in order to preserve the Company’s remaining cash resources. The company believes it can survive for awhile but not sure how long. Its largest debt is a $90M liability which can be called by one of the noteholders as early as November.

Arbios Systems, Inc. (OTC: ABOS) has filed for chapter 11 bankruptcy protection while they continue to seek bids for the sale of the Company and/or its assets - namely the SEPET™ Liver Assist Device (a blood purification therapy designed for use with a standard blood dialysis system). The company shuttered its operation in August to focus "day-to-day operations exclusively on obtaining financing or consummating a strategic transaction'. In October the company sold all its rights and interest in its bioartificial liver system, HepatAssist, to HepaLife Technologies, Inc. (OTCBB: HPLF). This was really the cell therapy part of the company. The HepatAssist™ Cell-Based Liver Support System - a hollow fiber cartridge housing living porcine liver cells - purportedly functions as a bioartificial liver that not only detoxifies the blood, but, uniquely, provides whole liver function - intended to treat acute and acute-on-chronic liver failure patients.

This is a buyers market and the reality is many potential investors or purchasers are not motivated to buy a going concern for $x if they can scoop up the assets and/or business at a significant discount under distress. Regretable for those companies that have created and nurtured good technologies but allowed their business to fail; good news for those with cash; perhaps also good news - say some - for a much-needed reorg of an industry with a much more disciplined commercial focus.

Last month we talked a little about Bioheart (Nasdaq:BHRT) and their IPO less than a year ago raising just under $6 million. Now the stem cell company Bioheart has alerted the SEC that it missed a scheduled $180,000 loan payment and is seeking new money to keep the company going.

The Miami Herald reports that the Sunrise, FL-based developer has warned investors that it faces some tough choices including potential layoffs, bankruptcy and closing the business. But, reports Jim Carroll from Fierce Biotech reports, "the company's CEO, Howard Leonhardt, says the lender--BlueCrest Venture Finance Master Fund--has agreed to provide an extra 90 days for the company to make the payment. Bioheart, he adds, had to deal with an unexpected delay in receiving a $2.5 million loan and he insisted that the company is not in danger of closing."

According to Leonhardt, ''The last 24 months in a row we've paid on time and it was an unforeseen event. We're not thinking of bankruptcy at all; it's not even close to consideration -- maybe laying off employees.'' But all is not as serene as a pic of the debonair Leonhardt, accompanying the Herald story, would seem to suggest. A group of board directors and key executives have departed the company in the last two weeks, including CFO William Kline.

Nonetheless, there is reason to be optimistic despite the alarm bells. Bioheart - which is not just a one-trick cell therapy pony - recently announced the approval or release of four new products. In October, it launched in the United States and Europe a heart failure monitor that patients can use at home. Several major sales contracts are pending and the company has set a $20 million goal for 2009, Leonhardt said. It has also won FDA approval for the Bioheart Monebo CardioBelt, which heart patients wear around their waist, as well as commercial approval for two other technologies in Europe. Also, while I still have unanswered questions about who is supplying who with what, they also recently signed a cell supply agreement with Life Technologies to sell myoblast cell-based research products to life science researchers. By March, Leonhardt said, Bioheart should be on sound footing cash-wise and would try to fund operations solely with revenue and profits. Until then, however, ''we have to bring some more capital in now to bridge the gap,'' Leonhardt said.

On now to more optimistic fronts...

In a recent interview, CIRM President, Alan Trounson, said he (CIRM) is lobbying for the creation of an 'investment' fund - sponsored by government and health insurers - to drive commercialization of early regenerative medicines. He fears that without such a 'new paradigm', the lack of funding on the scale typically provided by the pharmaceutical industry, could well be a roadblock in regenerative medicines being brought to market. Trounson predicts within 10 years we'll have new drugs based on stem cell discoveries and clinical products being tested for spinal repair, retinal repair, cardiac function repair, multiple sclerosis, etc.

BioE recieved FDA 510(K) clearance for its cord blood processing system. PrepaCyte-CB is available immediately to public and private banks separating potentially therapeutic cells and stem cells from cord blood.

Pluristem Therapeutics (NasdaqCM: PSTI; DAX: PJT) filed an IND with the FDA to initiate a two-center Phase I clinical trial in the U.S. utilizing PLX-PAD - placental adherent stromal cells (ASCs) expanded in the Company's proprietary PluriX TM 3D bioreactor - for critical limb ischemia. The company filed a IMPD in Europe for a similar trial there.

The Swedish investment company Karolinska Development AB has made what it calls a "significant investment" in EvoStem Finland Oy - a company that develops stem cell and other tissue engineering based treatments for veterinary use (and intends to go human at some point). In 2007 EvoStem Finland Oy introduced to the Scandinavian market its first product TendoStem(R) - a stem cell based treatment service for tendon and ligament injuries in horses.

Kiadis Pharma has recently come out with a bunch of news (some of which I'm a little late reporting). Most recently they Dr. Maarten Egeler, MD PhD as Chief Medical Officer and in December they reported the appointment of Mark Skaletsky to its Supervisory Board and, most significantly, announced they have received two orphan drug designations for Reviroc™ from the FDA - one for diffuse large B-cell lymphoma and the other one for the treatment of follicular lymphoma. Reviroc™ is under development for the elimination of cancer cells from an autologous graft in bone marrow transplantations for end-stage blood cancer patients.

Skaletscky, for those of you who don't know him, is very well credentialed. He served as president and CEO of GelTex Pharmaceuticals, Inc. (acquired by Genzyme for more than $1 billion in December 2000), was chairman and CEO of Enzytech, Inc., and president and Chief Operating Officer of Biogen, Inc. He is also a former chairman of the board of directors of the Biotechnology Industry Organization (BIO). Currently he is chairman, CEO and president of Fenway Pharmaceuticals, Inc.

Let me digress momentarily with a little history and speculation...

In 2001, Theratechnologies (TSX:TH) reorganized its business activities through the transfer of its ex vivo photodynamic cell therapy program to its newly created subsidiary, Celmed BioSciences, the acquisition of NeuroGeneration and NeuroGenomics, two California corporations which were developing technologies for the treatment of neurodegenerative diseases, and investments totaling $60 million by Societe generale de financement du Quebec (SGF) and the Solidarity Fund QFL. Michel F. Levesque, founder of NeuroGeneration became Vice President, Medical Affairs with Celmed BioSciences.

In 2006 Kiadis B.V and Celmed BioSciences Inc. merged the two oncology-focused companies. At that time, CelMed Biosciences was a wholly-owned subsidiary of Theratechnoloies (another Montreal company). The new combined company was named Kiadis Pharma B.V. Investors include Alta Partners, LSP, and DFJ Esprit. At or around that time, Levesque took back NeurGeneration which he is now attempting to re-launch from his practice in Hollywood, CA.

Kiadis still describes itself as a "Dutch-Canadian enterprise" which is "endorsed by our investors, academic partners and a cross-Atlantic management team". There is much cross-pollination between Montreal and Amerstdam. For instance, Pierre Caudrelier, was until this week, CMO at Theratechnologies, was previously CMO at Kiadis Pharm, VP of Clinical Research at Celmed, and VP of Clinical Research and General Manager the the Canadian subsidiary of IDM (another company that had cell therapy products with US-EU connections). Research collaborations also exist with Italy, Germany and the US (including NIH's John Barrett).

Here comes the complete and utter speculation. On the one hand we have an unnamed fairly large multi-national company (not GE) looking for acquisitions in the cell therapy space. Links have been established with personnel at Theratechnologies - a company which I believe no longer has any cell-based therapy research. On the other hand, totally unrelated to this, I am led to believe that Kiadis is looking for a partner with money. Other than some personnel and history, there would not appear to be anything still linking Theratechnologies and Kiadis. However, just to make things a little curious Theratechnologies announced this week that Dr. Pierre Caudrelier was leaving his position as chief medical officer to pursue other career opportunities. Despite the complete lack of any evidence other than this tenuous string of bits and bytes, something tickles my senses here. I'm betting there is more to this puzzle than I have yet been able to put together (or am willing to say out loud at this point). Stay tuned.

On the regulatory front...

FDA CBER has announced their Cellular, Tissue and Gene Therapies Advisory Committee 2009 meeting schedule: May 14-15 and November 5-6

For those of you using genetically engineered pigs for to grow cells, tissues, or organs that can be transplanted into humans with a reduced risk of immune rejection, you will know the FDA this week issued its Final Guidance on Regulation of Genetically Engineered Animals. For the rest of you, I can't think of better night time reading...

The FDA issued a Draft Guidance for Industry entitled: "Current Good Tissue Practice (CGTP) and Additional Requirements for Manufacturers of Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps)" [pdf version]. Although you can comment on any guidance at any time (see 21 CFR 10.115(g)(5)), to ensure that the agency considers your comment on this draft guidance before it begins work on the final version of the guidance, submit written or electronic comments on the draft guidance by April 16, 2009.

Let me mop up with a few final lighter entries.

Rurther to my blog on the subject back in October, BioTechniques just released interesting results on a survey of the extent to which scientists are using social media tools and which one for what. See more details in the comments section of my original blog post of social media use.

If you're planning to be at the Phacilitate Cell & Gene Therapy Forum in Washington, D.C. January 26-28 (and you should plan to be there if you have any interest in this field), here's a couple things to watch for:

- DCi Biotech consultant, Dawn Driscoll is moderating a lunchtime session on Tuesday the 27th hosted by Progenitor Cell Therapy in which yours truly, Robert Preti, and RTI Health Solutions, Eric Faulkner will be speaking. The session is entitled"Select Issues in Commercializing Cell Therapies" and will include a talk by Faulkner on positioning cell therapies for reimbursement.
For more details, click here.

- I will be there with the founders of BioBusiness.TV who will be there to capture some interviews from cell therapy industry executives for a cell therapy channel we are opening for the site.

- Anyone interested in potentially incorporating in vivo cell tracking/imaging in their cell therapy clinical trial, come find me.

Ok...one more thing just for kicks. For a chuckle and then a shake of the head in dismay check out the Niche blog on ESC-derivative facials! The hype is really getting out of control.

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That's it. That's all I've got for you. That, my friends, is how the cell therapy industry hit me me this week.

--Lee

p.s. Quick question: Do you mind/like this rather rambling format or should I get more organized somehow or another? Comment below...