Tweet 



The meeting “Commercialization of Your Regenerative Medicine Research: Lessons from Spin Out Successes” was hosted by the Oxbridge Biotech Roundtable (OBR) (Oxford, UK) at the University of Oxford in February, 2013, and attracted a multi-stakeholder audience spanning academia and industry. 


The event featured case studies from Gregg Sando, CEO, Cell Medica (London, UK), John Sinden, CSO, Reneuron (Guilford, UK), and Paul Kemp, CEO and CSO, Intercytex (Manchester, UK). 


OBR is a student-led initiative with over 7000 members across eight different UK and US locations with a mission to foster a conversation about the healthcare and life sciences industry. 


Anna French and David A. Brindley, along with some of my assistance, captured and have now published the main themes of the meeting and the major questions facing the regenerative medicine industry and its rapidly emerging subsets of cellular and gene therapies. 


Notably, we discuss the compatibility of regenerative therapies to the existing healthcare infrastructure, biomanufacturing challenges (including scalability and comparability), and the amenability of regenerative therapies to existing reimbursement and investment models. Furthermore, we reiterate key words of advice from seasoned industry leaders intended to accelerate the translation path from lab bench to the marketplace.


To read the review see: Commercialization of Regenerative Medicine: Learning from Spin-Outs


Anna French, R. Lee Buckler, and David A. Brindley. Rejuvenation Research. April 2013, 16(2): 164-170. doi:10.1089/rej.2013.1423.

Source:
http://feedproxy.google.com/~r/CellTherapyBlog/~3/4Uv2o54_hWQ/commercialization-of-regenerative.html

Tweet 


The Alliance for Regenerative Medicine announced today the release of the 2013 annual regenerative medicine industry report.  Here is the announcement in the Wall Street Journal online.

I'm proud to have been a part of putting it together and hope people find it useful.  It is available for download on the ARM website here.  


In addition to the complete download, ARM will make many of the figures, charts,  tables and sections available for members to download and use in their own publications and presentations. Watch for these resources to be announced soon.

Source:
http://feedproxy.google.com/~r/CellTherapyBlog/~3/yFBYKblnudk/2013-annual-regenerative-medicine.html

Source:
http://feedproxy.google.com/~r/CellTherapyBlog/~3/ABphTtPOrwo/cell-therapy-blog-welcomes-2013.html

Source:
http://feedproxy.google.com/~r/CellTherapyBlog/~3/z30UCNTkzIQ/the-accuracy-of-adipose-stem-cell-doses.html

I welcome comments and feedback. 


1. In addition to helping patients distinguish between compliant and non-compliant treatments (and providers) we must do more to help patients distinguish between non-compliant cell therapy treatments (and providers) which are more or less risky. 


2. Whatever we do in response to this issue should be done with an eye to being practical and helpful to patients in the real-life context of their decision about whether or not to buy a non-complaint cell therapy.


3. Our response to this issue should be based on a risk-based approach recognizing that not all non-compliance is created equal.  We should create a framework for risk-based analysis (both for us and our audiences) and focus initiatives around those which present the highest risk.


4. We recognize the problem of non-compliant cell therapies is not just a problem that exists in jurisdictions with little, no, or poor regulation but that is a growing problem even in the most highly regulated jurisdictions meaning the solution cannot be regulated it depends on education and enforcement.


5. We recognize regulatory agencies cannot enforce non-compliance on their own.  We as an industry need to complement their efforts through our own standards and enforcement.


6. Stakeholder groups should support the formation of a multi-organizational  initiative to, based on a risk-based assessment, spotlight the categories or signs of highest-risk offenders for use by patients and/or their physicians in identifying  whether or not treatments (and providers) they may be considering fall into the that category associated with the highest level of risk.

What do you think?

http://www.celltherapyblog.com hosted by http://www.celltherapygroup.com

Source:
http://feedproxy.google.com/~r/CellTherapyBlog/~3/wkSMxAV9938/a-proposed-6-step-platform-for-cell.html

For other things, however,  the people around you have such high expectations you'll do them that you only lose points if you don't but gain very little if you do.  For you, this may be true of the dishes.  Certainly I've always maintained this is true for Valentine's Day.  Get flowers and you simply maintain the relationship's status quo; fail to do anything and you lose big points fast.  

The result is "Why the stem cell sector must engage with social media".  What I attempted to succinctly outline are the ways social media primarily benefit one's career and organization or company.


"I can tell you without the slightest hesitation of conviction – having experienced it myself and seen it repeated countless times – is that active and successful social media engagement translates into:

• Unparalleled learning: accessing more information relevant to your discipline, specialty and company than you otherwise will. 
• Enhanced profile: higher profile within your industry, profession, specialty and community. Social media is not the only way to build a profile but it can be very effective.
• Wider network: more touch points and meaningful relationships with people than you otherwise will accomplish by any other means combined."

"In order to create the kinds of perceptions and solicit the kinds of actions we want from the world around us, we must engage the world around us. The world around us is engaging online. 

For all kinds of selfish and selfless reasons you, your company or organization and your career will benefit from you engaging there too."

and this prediction:

http://www.celltherapyblog.com hosted by http://www.celltherapygroup.com

Source:
http://feedproxy.google.com/~r/CellTherapyBlog/~3/k7ANTnLXNjc/the-roi-on-pant-wearing-and-other.html

The panelists emphasized that more accurate information should be provided to the public regarding stem cell treatments.

1.  50 shades of grey. Let's admit that this issue is not black-and-white but, as is almost always, involves a broad spectrum of grey in the middle.  


In addition to helping patients distinguish between compliant and non-compliant treatments (and providers) there are a lot of ways to help patients distinguish between non-compliant cell therapy  treatments (and providers) which are more or less risky.  

In between - in my opinion - are clinics like Stem Cell Institute and StemCellMD.

For some, helping patients distinguish between the better and worst non-compliant clinics might involve a fair amount of nose-pinching but this is the equivalent of the methadone clinic for heroine addicts.  By supporting the better of two evils we are not endorsing it as 'good', we are simply recognizing it is better.

This is a recognition that we cannot just abandon people because they made (or are going to make) decisions with which we ultimately disapprove.   It is a recognition that sometimes the most righteous thing to do is not only to help people do what we would ideally want them to do but to help them do the best they can in their circumstances and on their terms - even terms with which we may ultimately disagree.

6.  Let's grow a pair and call a spade a spade.  If a non-compliant clinic is providing treatments that we believe represent a high-risk to patient safety and the industry's credibility, let's have the b*lls to call them on it.  They can't sue everyone.  

http://www.celltherapyblog.com hosted by http://www.celltherapygroup.com

Source:
http://feedproxy.google.com/~r/CellTherapyBlog/~3/2b8Ffw4b1pU/six-steps-to-fighting-non-compliant.html

I'm pleased to point out that today the LinkedIn Cell Therapy Industry Group welcomed its 4,000th member today. 


The Cell Therapy Industry group was created to serve as a network of those in the cell therapy industry. The group acts as a vehicle for referrals, networking, information, and facilitating collaboration.  The group's focus is on the activities of companies in and serving the space.


The group began in July 2008. It took 2.5 years to reach the first 1000 members, 9 mos to reach 2,0000, 6 months to reach 3,000, and 6 months to meet today's 4,000 member mark. 


As is typical, there is a very high percentage of passive participants but the group benefits from an avid group of participants who post, share, exchange, and debate on a range of topics ranging from regulatory, clinical, commercial, scientific, manufacturing, financial, and other topics of interest to the group.


As the group has grown I've noted two trends pertaining to the composition of the membership:  (a) having tapped out the c-level suite, growth is increasingly coming from down the hierarchy of the corporate food chain and including those in the operational trenches, and (b) a much higher ratio of new members of late is from outside the US, presumably as LinkedIn increasingly penetrates OUS markets.


We strive hard to maintain the quality of the participation by screening each applicant, deleting off-topic posts, moving promotional posts to the "promotions" tab" and encouraging a balance of news-sharing with useful discussion threads.


I'm proud to say the group has become a vibrant and valuable part of the sector due to the hard work and contributions of all involved.    


As these kind of virtual networks become exponentially larger and provide different value than the professional societies representing the sector, I will be fascinated to watch if and how this affects how sectors like our interact and how this will impact the traditional value proposition of member-based professional societies.


If you are not a member of the LinkedIn Cell Therapy Industry Group, check it out.


--Lee

http://www.celltherapyblog.com hosted by http://www.celltherapygroup.com

Source:
http://feedproxy.google.com/~r/CellTherapyBlog/~3/EOlUEgJGsTQ/cell-therapy-industry-group-welcomes.html

http://www.celltherapyblog.com hosted by http://www.celltherapygroup.com

Source:
http://feedproxy.google.com/~r/CellTherapyBlog/~3/ctijFXeY01M/gens-cellular-therapy-wave-finally.html

CIRM: “To support and advance stem cell research and regenerative medicine under the highest ethical and medical standards for the discovery and development of cures, therapies, diagnostics and research technologies to relieve human suffering from chronic disease and injury.”

CIRM: For-profit entities have been and currently are eligible for CIRM funding covering stages of research which range from basic biology programs (in which industry has shown little interest) through Phase II clinical trials. Of these programs, 13% have been awarded to companies thus far. Having built 12 state of the art stem cell facilities and having seeded  the field with training and other types of grants of similar purpose, CIRM is now focusing on funding translational and clinical programs.  

This is where companies' primary interests are and we expect greater company participation in our translation and clinical Request for Application. The translation and clinical awards programs provide for much larger awards as compared to the basic research and the overall amount of later stage funding is significantly larger than the earlier basic research awards. The number of awards made in the translational and clinical development funding rounds is much less than in the basic science area. 

CIRM’s Strategic Partnership Funding Program is a cornerstone of our efforts to fund industry.   We expect to make awards through this program approximately every six months to assist companies whose financing demands is frequently at shorter intervals than academic institutions. These awards will be made following a robust peer review process ensuring that awards are made to projects that are based on sound scientific data and have a reasonable chance of success.

CIRM: Four clinical trials that were fostered by CIRM funds are already in clinical trials for cancer and blood disorders. We expect one or more CIRM-funded projects to join that list in the next year. This includes projects that are in clinical trial already for which we have funded and are funding the follow on studies.

CIRM: Yes, we have recently held the first round of applications for our Strategic Partnership Awards that are designed specifically to attract applications from industry and include significant leveraged funding from multinational biopharmaceutical companies and/or venture capital. The first of these awards will be announced at an upcoming meeting of our governing board, the Independent Citizens Oversight Committee. Industry also accesses CIRM funding through the Disease Team awards, which include teams comprised of both academic researchers and industry as partners, consultants and advisors. 

CIRM: No. We have awarded more basic research grants in numbers, but those grants are much smaller in dollars than those in our translational portfolio. That translational portfolio includes 75 projects that have been awarded nearly $600 million, well over half of the research dollars committed.

When CIRM funding was initiated in late 2006, there was a need to build intellectual and facility capacity because doubts about support from federal sources had limited the entry of scientists into the field and there was a need for “safe harbor facilities. “ Research into stem cells was also at an early stage and so it made sense for us to focus on the discovery phase of basic biology and pre-clinical work to enable more effective utilization of the potential that was evident.

Increasingly however we are moving towards clinical science, to enable a proper assessment of the value of cell therapies and related approaches for advancement of human medicine.

Our focus has always included all stem and progenitor cells. Pluripotential stem cells are immortal and develop into all cells of the body, so the potential is large and the available funding outside CIRM has been modest. We have concentrated on human rather than animal model cells because this is where the need has been greatest. Our goal is to fund transformational research with the highest potential benefit to patients, regardless of the stem cell type they utilize.

As for infrastructure, we spent $271 million in major facilities grants to help create new, state-of-the-art safe harbor research facilities in California which are essential for  delivering  the goals of CIRM. That investment was used to leverage almost $900 million in additional funds from private donors and institutions to help pay for those facilities. Each facility  attracted new researchers to the state,  employed local construction workers  and created expanded research facilities that will now be able to offer long-term employment for the high tech innovators in stem cell research, transformative new medicines  for intractable disease and deliver economic benefit for Californians.

CIRM: Yes, our focus in our new Strategic Plan does just that, emphasizing the increased focus on translation and clinical trials. As described above, we are investing strongly in this sector. But we firmly believe that advancement in medicine is dependent on the science that underpins the medical strategies. We will also  continue to support high quality basic science that can transform medical opportunities.  

CIRM: We are required by our statute to fund in those areas that are under-invested. Otherwise we are agnostic to cell type. We expect a mixture of embryonic (induced pluripotent stem cells as well when they are ready for clinical studies), fetal, adult, cancer stem and progenitor cells, as well as small molecules, biologics and other approaches, evolving from stem cell assays and research. We are most concerned with the ability to produce results for patients.

Yes, we have appointed a Vice President with business development responsibilities and are further strengthening this capacity with key staff. We are actively working with industry to develop sustainable partnerships in research, we hold webinars and face to face meetings with the FDA to better equip industry with the tools that can aid in their investigational new drug (IND) submissions . We also assist industry to better understand what they need to do to successfully apply for CIRM funding.

We have also made changes to our intellectual property regulations and loan regulations to make it even more attractive for companies  to partner with us in research.

Our focus has been in moving promising research through the "Valley of Death" phase, from the lab through Phase 1 and 2 clinical trials. We are working with major industry and financial institutions to inform them of our developing portfolio with the belief that they will be interested in taking many of these products to the market place. We are probably unable to afford to do these late stage clinical trials alone and feel it is likely that commercial interests will provide the follow on funding. 

CIRM: We are always interested in proposals that will enhance our mission. While this hypothetical has not been put to us we would have to assess the proposal on its merits and our available finances. 

CIRM:  In our RFA’s we have provided guidance as to what entities qualify for CIRM funding.  Future requirments  are presently under review by our General Counsel. Certainly, companies will need to show genuine steps at the time of application  towards relocation of a significant component of their research activities to California in addition to establishing a California operation with California employees. CIRM funding would be largely limited to in-state  activities.

http://www.celltherapyblog.com hosted by http://www.celltherapygroup.com

Source:
http://feedproxy.google.com/~r/CellTherapyBlog/~3/wzhx7dkP3vk/cirm-addresses-some-tough-questions-is.html

CT model portfolio compared to 3 major indices YTD

• Cell Therapy Portfolio:  +24.44%
• Dow Jones:  +4.5%
• S+P 500:  +6.78%
• Nasdaq:  +10.26%

http://www.celltherapyblog.com hosted by http://www.celltherapygroup.com

Source:
http://feedproxy.google.com/~r/CellTherapyBlog/~3/ediPNE1NBDw/cell-therapy-portfolio-outperforms.html

The scandal of clinical trial data loss is eroding the fundamentals of evidence-based research and clinical medicine.


Before you right this post off as the stuff of conspiracy theories, fear-mongering, and 'alternative world views' consider that this view is shared by the likes of the FDA, the International Committee of Medical Journal Editors, the Cochrane Collaboration, and researchers at institutions like Johns Hopkins School of Medicine.


Here's the underlying premise as succinctly described by author Ben Goldacre:

"Drugs are tested by the people who manufacture them, in poorly designed trials, on hopelessly small numbers of weird, unrepresentative patients, and analysed using techniques that are flawed by design, in such a way that they exaggerate the benefits of treatments. Unsurprisingly, these trials tend to produce results that favour the manufacturer.

When trials throw up results that companies don't like, they are perfectly entitled to hide them from doctors and patients, so we only ever see a distorted picture of any drug's true effects. Regulators see most of the trial data, but only from early on in a drug's life, and even then they don't give this data to doctors or patients, or even to other parts of government. This distorted evidence is then communicated and applied in a distorted fashion."

Authors M. Todwin and J. Abramson summarize it thusly:

"Trials with positive results generally are published more frequently than studies that conclude that a new drug poses greater risks or is no more effective than standard therapy or a placebo. Furthermore, some articles may distort trial findings by omitting important data or by modifying prespecified outcome measures. Lack of access to detailed information about clinical trials can undermine the integrity of medical knowledge."

Here is a great list of very recent resources that may convince you of the merits of this concern:

• What doctors don't know about the drugs they prescribe (2012: TED video)
• The drugs don't work: a modern medical scandal (2012: The Guardian)
• Clinical Trial Data as a Public Good (2012: JAMA [subscription req'd])
• Registering Clinical Trial Results. The Next Step (2010: JAMA [subscription req'd])
• The Imperative to Share Clinical Study Reports: Recommendations from the Tamiflu Experience (2012: Plos Medicine)

http://www.celltherapyblog.com hosted by http://www.celltherapygroup.com

Source:
http://feeds.feedburner.com/CellTherapyBlog

http://www.celltherapyblog.com hosted by http://www.celltherapygroup.com

Source:
http://feeds.feedburner.com/CellTherapyBlog

I always read WIRED magazine.  Aside from GEN it's the only magazine I read.  Just reading something outside of cell therapy or biotech often infuses me with an idea that otherwise would have never occurred to me like the need for a cell therapy X Prize or cellular aggregates as microcarriers or tissue-engineered memory and processing devices or even just the conviction to better represent cell therapy to the broader world out there of scientists, engineers, journalists, policy-makers, or perhaps people with too much money looking to be inspired and wanting to make a difference.

http://www.celltherapyblog.com hosted by http://www.celltherapygroup.com

Source:
http://feeds.feedburner.com/CellTherapyBlog

Cell therapies present unique challenges when complying with this paradigm for several reasons only two of which I will mention here.  Firstly, it is not possible to achieve the level of product purification as one might with other therapeutic products.  Secondly, the product characterization is at a cellular rather than molecular level.

Autologous cell therapies present another set of unique challenge in this paradigm because of the notable patient-to-patient variability where the patient is also the donor of the raw material.  This often means there is a wider tolerance of heterogeneity in the product but it still must be within what has been proven to the regulatory agency as a safe and effective range.  


In cases where an autologous cell therapy is centrally manufactured, they are most often subjected to product release testing similar to that described above.  One notable difference, particularly for fresh products, is that the products may be shipped to the clinic and even administered before the full panel of test results are obtained.  This wold be considered highly unusual (if ever acceptable) with other types of products but is tolerated because of the time-sensitivity of these products and their high safety profile.


In the case of autologous cell therapy products produced at the bedside there is often not the same kind of product release discipline.  Often the regulatory agencies deal with the product consistency and specification compliance issue by ensuring that the cell processing device used point-of-care is validated to ensure the cellular product output is always within a specified range shown to be clinically safe and effective.


The Varying Degree of Product Characterization/Specification of Autologous GTP Cell Therapy Products


However - and now I get to the point of this blog post - for cell-based products, procedures and/or devices/kits which are not mandated to be formally approved by a regulatory agency before they can be commercially marketed, there is no product specification rigor.  Compliance with the Good Tissue Practice regulations and guidance is deemed to ensure safety.  In the United States, cell-based products which are deemed to be "minimally manipulated" and intended for "homologous use" are typically allowed to go straight to market with no formal approval.  Safety and clinical data is not required but is practically necessary to support physician adoption and, where applicable, reimbursement.  


This means that for these products there is a great deal of variability in terms of how much rigor companies apply in characterizing their product and then ensuring that each batch complies with the specifications they themselves have determined to be safe and effective. Again, where such products are manufactured in a centralized facility the likelihood of some release testing is greater.  However, those companies relying on a point-of-care processing kit or device business model that has not been deemed to require formal market approval, rarely (if ever) include product release testing.


The common criticism of these companies is that they simply do not know what they are injecting into patients because of the combination of the patient-to-patient donor variability, the lack of any disciplined product characterization or dosing studies, and the absence of any product release testing.  


This criticism is not equally levied at all autologous GTP products or companies - even those relying on point-of-care processing.  Of course some companies care and do a lot to try to ensure their product is well-characterized and that each batch complies with product specifications. This may involve the use of product release tests but can also involve the combination of pre-market research into the product characterization, safety, and dosing along with validation of the device/kit output.  In this way a company can say that within a very small margin, the output will be within the product specifications the company knows is safe and efficacious.


However, in a rush to get their device/kit to market some companies appear to care very little about the cell product characterization, validation of the output of their device/kit, or tying this data to optimal dose.


More concerning are those companies that appear to provide such data but it is wrong or meaningless.  What follows appears to potentially be a case study of precisely this problem. 


The INCELL Study 


This week I came across a fascinating white paper from Incell Corporation analyzing the output of adipose tissue processing kits of MediVet-America apparently demonstrating the inaccuracy of their cell counts (a common type of cell therapy product characterization) and calling into the question the cell count claims of Intellicell Biosciences (New York, NY) and Adistem (Hong Kong).


At the heart of the critique is the claim that the cell counting (product characterization) techniques employed by these companies counts as cells things (namely acellular micelles) which are not cells.

I encourage you to read the white paper in its entirety.  They corresponding author told me to watch for one or more papers which they are preparing for submission to peer-reviewed publications shortly.  Presumably these will rely on a larger data set and perhaps test other methodologies or technologies.


For the purposes of this blog, I've pulled what I believe are the most salient excerpts below:

Intrigued by the high cell numbers  (5 to 20 million cells/gram)  reported by kit/device manufacturers such as MediVet-America (Lexington, KY), Intellicell  Biosciences (New York, NY), and Adistem, Ltd. (Hong Kong) in adipose stem cell therapy compared to other methods (e.g., 
Chung,Vidal, and Yoshimura), INCELL staff conducted a research study to  investigate the high apparent yield of stem cells.  This initial work was focused  on SVF cells from the MediVet Kit, which is marketed to isolate adiposederived canine SVF and stem cells.

The cell yields reported for the Medivet Kits are five to more than ten times higher than the yields routinely obtained by INCELL from freshly harvested human or animal adipose tissue using our adipose tissue processing methods.  These yields are also tenfold or higher than those reported in the literature by most academic researchers (Chung-canine, Vidal–equine, Yoshimura–human).  Since these  cell counts are used to support stem cell dosing recommendations and cell banking, it is important to better understand why the cell numbers are higher.

...

A comparative analytical study of three dog donors of adipose tissue was designed to evaluate the cell yields using the MediVet Kit as an example of this class of isolation system. All  kit procedures were followed as per the instructions provided.  A brief overview of the different cell counting methods used, and the resultant cell counts, observations and explanations of the results observed, are described below

....

This study shows that incorrect counting of adipose derived SVF cells and the subset of regenerative stem cells can subsequently result in inaccurate dosing, both in direct therapeutic applications and in cryostorage of cells for future use.  The DAPI-hemocytometer cell count (manual) was considered the most accurate, but there are various sources of technical difficulties that  can lead to incorrect  cell numbers. The nature of adipose tissue itself with variability in dissociation by enzymatic digestion can all contribute to the outcomes. Fat tissue has a propensity to form acellular micelles and oils upon tissue disruption. Processing methods or reagents (e.g., Solution E or lecithins) can generate micelles that may be  erroneously  counted as cells. Autofluorescence and dye trapping or uptake by the micelles can lead to very high inaccurate cell counts when automated cell counting is used. 

In this study the most inaccurate counting came from the Cellometer. When used according to kitrecommended guidelines and on-site training provided by Nexelcom for counting  cells by the MediVet procedure, the Cellometer overstated the DAPI-hemocytometer cell count by up to 20X or more. The Coulter Counter protocols also led to incorrect, high cell numbers. Although the cell counts were still a bit high, the authors recommend the NucleoCounter, or similar equipment, as more acceptable for automated counting.  The manual hemocytometer-DAPI method is the most accurate, but requires a highly experienced cell biologist or technician to make accurate counts and  is not suitable for routine clinical use. 

...

Other companies also have claims of very high cell numbers when their processes are used. Adistem, like MediVet, states they add an emulsifying agent to their kits to assist in cell release, and they also use a light activation system. Their kits were not tested in this study but it is possible that the high cell numbers reported by Adistem are also incorrect and result from the same problems highlighted in this paper for the MediVet procedure. Ultrasonic energy, which is commonly used to manufacture micellular  liposome  structures and to disrupt and lyse cells, is  another potentially problematic procedure for counting and verifying viable, regenerative cells.  Intellicell 3uses ultrasonic energy to release cells from adipose tissue, and it is possible that resultant micelles or cell fragments contribute to the higher than expected cell numbers.  This assumption could be verified with additional studies.  

In summary, the authors caution that great care must be taken when using kits and automated cell counting for stem cell dosing and cryobanking of cells intended for clinical use. Overestimated  cell numbers would be a major confounding source of variation when efficacy of stem cells injected are compared as doses based on cell number and when cryostored cells are aliquoted for use based on 

specific cell numbers as a treatment dose.  Hopefully, this study will lead to more  reproducible counting and processing methods being reported in the literature, more inter-study comparability of cell doses to clinical outcomes,  more industry diligence to support claims, and more accurate counting for dosing stem cell therapies to patients.

...

Chung D, Hayashi K, Toupadakis A, et al.  Osteogenic proliferation and differentiation of canine bone marrow and adipose tissue derived mesenchymal stromal cells and the influence of hypoxia.  Res Vet Sci, 2010; 92(1):66-75. Vidal MA, Kilroy GE, Lopez MJ, Johnson JR, Moore RM, Gimble JM. Characterization of equine adipose tissue-derived stromal cells: adipogenic and osteogenic capacity and comparison with bone marrow-derived mesenchymal stromal cells. Vet Surg, 2007; 36:613–622.  Yoshimura K, Shigeura T, Matsumoto D, et al:  Characterization of freshly isolated and cultured cells derived from the fatty and fluid portions of liposuction aspirate.  J Cell Phys, 2006; 205:64-76.

http://www.celltherapyblog.com hosted by http://www.celltherapygroup.com

Source:
http://feeds.feedburner.com/CellTherapyBlog

http://www.celltherapyblog.com hosted by http://www.celltherapygroup.com

Source:
http://feeds.feedburner.com/CellTherapyBlog

As followers of this blog will know I've been blogging about Regenerative Sciences and predicting their eventual run-in with the FDA since my first post in September 2008 (Cell Therapy is Not the Practice of Medicine) and again in February 2009 (Regenexx vs the FDA 2009).  When the FDA finally proceeded with an injunction against RSI in August 2010,I helped spread the news (here).

I've watched the development of the fight between RSI and the FDA with interest.  In September 2001 I posted a rather lengthy commentary about the potential impact of the case (Potential far-reaching implications of the ongoing fight over point-of-care autologous cell therapy.

Since then I have welcomed other bloggers and commentators who are now following and commenting on the case much more closely and frequently than I including @LeighGTurner (on Twitter) and Paul Knoepfler (@PKnoepfler on Twitter and his Knoeplfer Lab Stem Cell Blog).  Recently I enjoyed being interviewed by Paul on the issue of unregulated stem cell activity and touched on the case for his blog.

Consequently I read with interest yesterday's federal court ruling upholding the FDA's injunction against RSI and the immediate commentary from the New Scientist, Stanford's Scope Blog and Knopfler's multiple posts (here and here). As a long-term follower of this case, I've been asked to comment.  Here is my brief reaction:

This is a case that was always destined for the appellate courts regardless of which way the initial court ruled.    The fact the federal court ruled in the FDA's favor certainly now sets the onus on RSI and what is anticipated to be a gamut of intervenors but taking this case to the appellate courts is what the legal team have anticipated and legal arguments designed for all along.

This is just the beginning of what will be a long and interesting battle.  The ruling was nothing more than the granting of an injunction in response to the government's motion for summary judgement.  In granting the injunction the court  agreed with the government's position that it was acting under the authority given it under the Federal Food, Drug, and Cosmetic Act, 21 U.S.C. § 321(g) but it provided little-to-no rationale for its ruling.

The court chose, in its wisdom, not to address the bulk of the RSI's legal arguments which are largely jurisdictional in nature. These are the kinds of arguments which the lower courts prefer be dealt with by appellate courts and frankly the judge did us all a favor by ruling quickly, succinctly and punting the case where we all knew it was inevitably headed.

In my opinion, other than chalking one up in the government's win column there is little to be gleaned from this ruling in terms of how RSI's arguments will be received in appellate court.  The interesting day is yet to come.

In terms of a short-term practical impact, frankly I see very little.  RSI has already ceased distributing Regenexx within the US so there will be little-to-no impact there.  As for the potential impact on other companies or clinics who might be operating on the fringes of FDA regulation within the US, I suspect it will be business as usual.

Most of the clinics/companies offering cell-based treatments/products which are arguably in contravention of FDA regulation are operating under the clear knowledge of what they are doing and where the FDA stands with respect to the treatments/products they offer and yet they persist and continue.


 For the truly fraudulent there is the risk of criminal charges and/or litigation but for those companies or practitioners who are operating in this shade of grey which are not shady (and they do exist), the  risks associated with this practice are barely higher than in the routine practice of medicine. 


In reality, with the exception of the most fraudulent examples, it takes a fair long-time for the FDA to catch up with these folks and there is good money to be made in the interim.  When they get caught, they will stop. If they've recouped their initial investment (which is nominal and the margins are high) there is very little penalty to this course of action.  Perhaps they set up shot elsewhere or simply enjoy the proceeds.  I doubt we will see much of a slow-down of this kind of activity.  Indeed it may strengthen the resolve of those committed to the cause.

In my opinion yesterday's ruling was in interesting and important milestone in a continuing evolution in the debate of how best to regulate the use of cells in treating people but I'm not sure it's the seminal pivot point that some believe.  I suspect we will not see any radical shift in terms of FDA or industry activity until (if then) the appellate courts rule.

Just my two cents....

--Lee

http://www.celltherapyblog.com hosted by http://www.celltherapygroup.com

Source:
http://feeds.feedburner.com/CellTherapyBlog

Tweet




A short and sweet note to point you to a great article on bioreactor technologies related to cell therapy bioprocessing by CTG consultant and Director of Stem Cell-based Drug Discovery, John E. Hambor, who you can now follow on Twitter @StemCellonDrugs.


"Bioreactor Design and Bioprocess Controls for Industrialized Cell Processing" was published in the June issue of BioProcess International.  


The BPI team has made a real and meaningful commitment to representing cell therapy bioprocessing and we applaud them for their contribution to this emerging discipline.




If this is a topic of interest to you, I recommend you also check out a conference being held this Fall by BPI's sister company, IBC LifeSciences, entitled "Cell Therapy BioProcessing" to be held September 11-12 in Arlington, Virginia.

http://www.celltherapyblog.com hosted by http://www.celltherapygroup.com

Source:
http://feeds.feedburner.com/CellTherapyBlog

Tweet


Cell therapies for cardiovascular-related conditions is a closely watched, much studied, oft-discussed, and hotly contested segment of the cell therapy industry.


The data to-date are admittedly confusing.  From a clinical perspective, the studies for which we have data have been relatively small involving a mish-mash of indications, endpoints, eligibility criterion, methods and/or route of administration, as well as the time of administration relative to event or disease progression.


Further compounding any interpretation of the data, from a technical perspective, is the fact the products have been widely varied in terms of being autologous vs allogeneic, expanded and not, genetically modified and not, from a plethora of different sources, and utilizing a wide variety of cell types from skelatal myoblasts, cardiomyocytes, mesenchymal stromal cells, mononuclear cells, etc. 


All this makes it extremely difficult to draw any conclusions with respect to what's working and what's not.  We will not attempt to do so.


All we do below is attempt to give a snapshot of the industry-sponsored cell therapy trials currently ongoing for cardiovascular-related conditions.  So here it is:


Commercial:
Pharmicell's Heartcelligram is the only cell therapy to have received regulatory approval for commercial distribution for the treatment of a cardiac-related indication.  Heartcelligram is an autologous cell therapy approved in 2011 by the Korean Food and Drug Administration (KFDA) for the treatment of Acute Mycardial Infarction (AMI).  The price is reportedly $19,000 and the trial data behind the approval has not yet been published in a peer-reviewed journal.


Phase III or II/III:
There are currently only 3 active and recruiting cardiac-related, industry-sponsored cell therapy trials.  Interestingly they all involve autologous products, two involve devices, two involve centralized manufacturing, two involve bone marrow cells as a source, two are only in European clinical sites, and two are targeting ischemic-related conditions.

• Baxter Therapeutics' Auto-CD34+ cells
• phase III trial actively recruiting
• Indication:  refractory angina and chronic myocardial ischemia
• Estimated enrollment:  444
• Estimated primary completion: June 2016 
• Cytori
• phase II/III trial actively recruiting
• Indication: ST-elevation acute myocardial infarction
• Estimated enrollment: 360
• Estimated primary completion: July 2014

• Miltenyi Biotec
• phase III trial actively recruiting
• Indication: myocardial ischemia or coronary artery disease
• Estimated enrollment: 142
• Estimated primary completion: July 2012

http://www.celltherapyblog.com hosted by http://www.celltherapygroup.com

Source:
http://feeds.feedburner.com/CellTherapyBlog

Tweet 

http://www.celltherapyblog.com hosted by http://www.celltherapygroup.com

Source:
http://feeds.feedburner.com/CellTherapyBlog