Elk Run Biotechnology Center is a Slow Process – KAALtv.com
By Dr. Matthew Watson
 KAALtv.com | Elk Run Biotechnology Center is a Slow Process KAALtv.com (ABC 6 News) -- It's a project years in the making, but many are now wondering what is going on with the Biotechnology Center in Pine Island. It's a sign that carries much more than a name of a yet to be developed technology campus. Promises of high ... |
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September 2nd, 2012
Are some cell counts too good to be true? Why some companies’ product data may mislead.
By Dr. Matthew Watson
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 onspecific 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.
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September 2nd, 2012
California Stem Cell Agency: A New Board Member and a New Vacancy
By Dr. Matthew Watson
The chairs are shifting a tad on the
governing board of the $3 billion California stem cell agency as a
French immigrant is added, a Latino leaves and a veteran patient
advocate is reappointed.
Coming on board for next week's meeting
is Anne-Marie Duliege, chief medical officer of Affymax Inc., of
Palo Alto, a publicly traded biopharmaceutical company that deals
with kidney disease. Leaving is David Serrano Sewell, who has been
named to the state Medical Board by Gov. Jerry Brown. Reappointed is
Jeff Sheehy, an HIV/AIDs patient advocate who may be the most public face
of patient advocates on the stem cell agency.
 |
| Anne-Marie Duliege Affymax Photo |
State Controller John Chiang appointed
Duliege to the CIRM post, saying,
“Dr. Duliege brings
first-hand knowledge of what is required to take a drug from research
phase through FDA approval.”
the Bioscience Forum in South San Francisco called “Beating the
Odds,” a discussion of Affymax's first commercial product.
According to information posted by the group, Duliege led the way by
shepherding it through a 10-month gauntlet at the FDA.
2007. Her prior positions included time at Chiron and Genentech. She
is a practicing physician, working part-time, and received her
medical degree from Paris Medical School.
stem cell agency. Ted Love, one of the initial members of the CIRM board, also sits on the Affymax board of directors. Indeed, Duliege fills the seat
vacated by Love when he resigned from the CIRM board. The position must be
filled by an officer of a California life science company.
 |
| David Serrano Sewell CIRM Photo |
Serrano Sewell, who has also served on
the CIRM board since its inception, is apparently resigning to accept
an appointment to the board that regulates
California physicians. Apparently – because the stem cell agency
has not confirmed that he is leaving, although this morning it placed a resolution honoring him on the agenda for next week's meeting. That almost invariably means a board member is departing.
city of San Francisco, was one of 10 patient advocate members on the
29-member board. Sewell was apppointed by the California lieutenant
governor. His seat will remain vacant until the current lieutenant
governor, Gavin Newsom, makes an appointment, who must also be a patient advocate.
 |
| Jeff Sheehy CIRM Photo |
Sheehy was reappointed recently by
state Senate President Pro Tem Darrell Steinberg. Sheehy is a
communications manager at UC San Francisco and a nationally known
HIV/AIDS advocate. He is co-chairman of CIRM's Science Subcommittee
and vice chairman of the grants review group. Sheehy leads the
discussion of grant applications when they come before the full board
in public session.
Eugene Washington, dean of the UCLA medical school, is a member of
the board but never attends the meetings. Instead he sends a
surrogate. Serrano Sewell's departure brings the number of Hispanics
to three, co-vice chairman Art Torres, Francisco Prieto and Marcy
Feit. No Asians sit on the board.
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September 2nd, 2012
Bob Klein, "Lobbying" and Reader Reaction
By Dr. Matthew Watson
A robust discussion has arisen
concerning Bob Klein and his appearance last month before the
governing board of the $3 billion California stem cell agency, a body
that he once chaired and an enterprise that he once oversaw.
was manipulated and whether he was engaged in so-called “revolving
door” activity – the practice of former government officials,
such as Klein, becoming paid representatives of enterprises that were
involved with their former agency.
interesting questions that we will discuss on the California Stem
Cell Report during the next few days.
of those who commented for the delay in posting their remarks.)
http://californiastemcellreport.blogspot.com/feeds/posts/default?alt=rss
Nearly $6 Million Sought: Four Scientists Seek to Overturn Rejection by CIRM Reviewers
By Dr. Matthew Watson
Four researchers are appealing
rejection of their proposals to win millions of dollars from the
California stem cell agency just as the agency is moving to curb such
reconsideration efforts by scientists.
agency calls its basic biology round. The agency's governing board
meets next Wednesday and Thursday to hand out as much as $35 million
to as many as 25 scientists competing for the research dollars.
round. One upshot has been a proposal that would tighten the review process. That plan also comes before directors next week.
applications were given scientific scores that exceeded those of some
proposals that were approved by reviewers. The lower scoring
proposals were given the go-ahead on the basis of “programmatic
review,” which one CIRM document says is designed to allow
“consideration of issues beyond scientific merit, such as disease
representation and societal impact.”
as extraordinary petitions – were filed by Michael Teitell of UCLA,
Deborah Lieu of UC Davis, Tony Hunter of Salk and Hanna Mikkola, also
of UCLA. In all, their applications seek nearly $6 million from CIRM.
said she was “relatively inexperienced.” Lieu's appeal said she
has “24 publications with over 6 years of experience in the
differentiation of cardiac muscle cells from human pluripotent stem
cells, 12 publications (3 co-corresponding author) on human
pluripotent stem cells and their cardiac derivatives, and 3
publications on the engineering of pacemaker cells” in addition to
other related professional experience.
application received a score of 68, ranking it above two other grants
approved by reviewers and equal to a third also approved by
reviewers.
reviewers did not have access to. Mikkola's application for $1.4 million
received a score of 65, which ranks it above one grant approved by
reviewers.
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September 2nd, 2012
Stem Cell Agency Moving to Curb Free-Wheeling Appeals by Researchers
By Dr. Matthew Watson
The $3 billion California stem cell
agency on Tuesday released details of proposed, major changes in how
scientists are allowed to appeal decisions when their
applications for millions of dollars are rejected by grant reviewers.
would formalize ad hoc procedures that have emerged over the last
several years. The plan would also make it clearer exactly what can
and cannot be done or expected under the agency's appeal process,
which is poorly understood by at least some researchers.
on at the CIRM board meeting next Wednesday and Tuesday, is heavily
nuanced, dealing with such matters as “supplemental information,”
an “additional analysis option,” “criteria for material dispute
of fact,” “criteria for material new information” – not to
mention the old standby – “extraordinary petition.”
distinction between an “appeal” and an “extraordinary
petition.” However, it is a distinction without a difference except
to those in thrall of bureaucratic jargon. Both are appeals. Their
purpose is to provide a method for overturning reviewers' decision under certain conditions.
only four business days prior to next week's governing board meeting
– a little late to generate thoughtful comment and constructive
suggestions from those most likely to be affected by the changes –
the 500 or so recipients of $1.6 billion in CIRM funding. Before final action on the changes, the board may well want to send out the proposal to all of its grant recipients and ask them for written comment that could then be considered at a public meeting of its Science Subcommittee.
the appeal process for more than four years, including the
presentations at its public meetings by scientists. Ironically, the
first such public appearance was made by Bert Lubin, who is now a member of the CIRM
board and CEO of Childrens Hospital in Oakland, Ca..
wrote at the time, the pitch by Lubin, who was unsuccessful,
disturbed some board members. Gerald Levey, then dean of the UCLA
medical school and a member of the board, said,
"I don't think we can run a board
this way. If we do, it would be chaos."
Nature as saying that his rejected application did not come from “the
in crowd” of stem cell researchers or organization.
“So a project that was really going
to go into patients was essentially triaged.”
the appeal process also uses language that obfuscates exactly what
researchers can do under state law. The document says that scientists
“may” make oral and written comments to the board, which is a
state government entity. In fact, state law makes it clear that
researchers as well as any member of the public have the “right”
to comment. The board legally cannot prevent them from speaking or
making comments. And the board, to its credit, has always allowed
ample public comment even when it slows the board's work.
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September 2nd, 2012
USC Researchers Appeal Rejection of $20 Million Proposal
By Dr. Matthew Watson
Researchers from the University of
Southern California are making a pitch to overturn rejection of their
$20 million grant application by reviewers in one of the signature
commercialization rounds of the California stem cell agency.
Lon Schneider will be taken up one week from tomorrow by the
governing board of the $3 billion state enterprise.
million disease team round that was considered last month during a
record-breaking outpouring of appeals and a day of emotion-filled
appearances by patients. CIRM directors adjourned their meeting
without completing action on a number of items, leaving open the possibility of additional appeals such as the one from USC.
received a score of 63 from reviewers. They said in a letter to
the board,
“We are submitting the petition at
this time as we are new to the CIRM ICOC(governing board) process and after listening
to the July 26 ICOC meeting deliberations now understand that the
petition process allows the ICOC to further consider our proposal.
We noted that the proposal scored one point above ours and another
two points below ours, each utilized the extraordinary petition
strategy to gain ICOC review which resulted in funding approval in
the former, and reconsideration in the latter instance.”
expressed by CIRM Director Oswald Steward, director of the Reeve Center at UC Irvine, at last month's board
meeting about fairness in the grant process. He said,
“I'm not really quire sure that all
of the applicants clearly understood that they could come back to us
to address the criticisms(of reviewers).”
Concerns about whether all applicants fully understand the appeal process have surfaced on a number of occasions over the last several years. The CIRM board, however, is generally reluctant to overturn negative recommendations by reviewers. It also almost never reverses positive recommendations.
make unspecified changes in the appeal process. No further details on
those changes have yet been released by the agency although the
meeting is just four business days away.
CIRM board, the scientists defended their scientific approach and
responded to criticism by reviewers, especially those related to
sedation. Reviewers expressed reservations about over-sedation, which
the researchers said were erroneous.
be making appeals during next week's board meeting.
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September 2nd, 2012
Researcher Alert: Troubling CIRM Grant Appeal Process Up for Revision
By Dr. Matthew Watson
Directors of the California stem cell
agency next week are expected to make unspecified changes in how
scientists can appeal denials of their applications for millions of
dollars in research grants.
emotional meeting last month in which the CIRM governing board faced a record outpouring of appeals of negative decisions by grant
reviewers. The board is the ultimate arbiter on applications. While it almost never overturns positive decisions by reviewers, it sometimes
approves applications that they have rejected.
the appeal process are yet available for the meeting Sept. 5-6 in
Burlingame, Ca. All that is known at this point is the following item
from the board agenda: “consideration of modifications to the
extraordinary petition policy and adoption of additional
information policy.” Extraordinary petitions are the key vehicle
for appeals.
month's high stakes, $243 million round posed new challenges and
consumed so much time that the board was unable to complete action on
several items.
board sent five applications back for re-review. (See here, here and
here.) Some of those are expected to come up next week and others at
the end of October. The board agenda, however, did not specify which
applications would be considered next week. Nor did it specify how many additional appeals have been filed in the round that was up for
approval in July.
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September 2nd, 2012
UCLA Researchers Discover "Missing Link" Between Stem Cells and the Immune System
By Dr. Matthew Watson
Newswise UCLA researchers have discovered a type of cell that is the missing link between bone marrow stem cells and all the cells of the human immune system, a finding that will lead to a greater understanding of how a healthy immune system is produced and how disease can lead to poor immune function.
The studies were done using human bone marrow, which contains all the stem cells that produce blood during postnatal life.
We felt it was especially important to do these studies using human bone marrow as most research into the development of the immune system has used mouse bone marrow, said study senior author Dr. Gay Crooks, co-director of the Eli and Edythe Broad Center of Regenerative Medicine and a co-director of the Cancer and Stem Cell Biology program at UCLAs Jonsson Comprehensive Cancer Center. The few studies with human tissue have mostly used umbilical cord blood, which does not reflect the immune system of postnatal life.
The research team was intrigued to find this particular bone marrow cell because it opens up a lot of new possibilities in terms of understanding how human immunity is produced from stem cells throughout life, said Crooks, a professor of pathology and pediatrics.
Understanding the process of normal blood formation in human adults is a crucial step in shedding light on what goes wrong during the process that results in leukemias, or cancers of the blood.
The study appears Sept. 2 in the early online edition of Nature Immunology.
Before this study, researchers had a fairly good idea of how to find and study the blood stem cells of the bone marrow. The stem cells live forever, reproduce themselves and give rise to all the cells of the blood. In the process, the stem cells divide and produce intermediate stages of development called progenitors, which make various blood lineages like red blood cells or platelets. Crooks was most interested in the creation of the progenitors that form the entire immune system, which consists of many different cells called lymphocytes, each with a specialized function to fight infection.
Like the stem cells, the progenitor cells are also very rare, so before we can study them we needed to find the needle in the haystack. said Lisa Kohn, a member of the UCLA Medical Scientist Training Program and first author in the paper.
Previous work had found a fairly mature type of lymphocyte progenitor with a limited ability to differentiate, but the new work describes a more primitive type of progenitor primed to produce the entire immune system, Kohn said
Once the lymphoid primed progenitor had been identified, Crooks and her team studied how gene expression changed during the earliest stages of its production from stem cells.
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UCLA Researchers Discover "Missing Link" Between Stem Cells and the Immune System
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September 1st, 2012
Press Release
By NEVAGiles23
Research and MarketsPosted on:31 Aug 12
Research and Markets (http://www.researchandmarkets.com/research/9fkkzb/cell_therapy_tec) has announced the addition of Jain PharmaBiotech's new report "Cell Therapy - Technologies, Markets and Companies" to their offering.
This report describes and evaluates cell therapy technologies and methods, which have already started to play an important role in the practice of medicine. Hematopoietic stem cell transplantation is replacing the old fashioned bone marrow transplants. Role of cells in drug discovery is also described. Cell therapy is bound to become a part of medical practice.
Stem cells are discussed in detail in one chapter. Some light is thrown on the current controversy of embryonic sources of stem cells and comparison with adult sources. Other sources of stem cells such as the placenta, cord blood and fat removed by liposuction are also discussed. Stem cells can also be genetically modified prior to transplantation.
Cell therapy technologies overlap with those of gene therapy, cancer vaccines, drug delivery, tissue engineering and regenerative medicine. Pharmaceutical applications of stem cells including those in drug discovery are also described. Various types of cells used, methods of preparation and culture, encapsulation and genetic engineering of cells are discussed. Sources of cells, both human and animal (xenotransplantation) are discussed. Methods of delivery of cell therapy range from injections to surgical implantation using special devices.
Cell therapy has applications in a large number of disorders. The most important are diseases of the nervous system and cancer which are the topics for separate chapters. Other applications include cardiac disorders (myocardial infarction and heart failure), diabetes mellitus, diseases of bones and joints, genetic disorders, and wounds of the skin and soft tissues.
Regulatory and ethical issues involving cell therapy are important and are discussed. Current political debate on the use of stem cells from embryonic sources (hESCs) is also presented. Safety is an essential consideration of any new therapy and regulations for cell therapy are those for biological preparations.
The cell-based markets was analyzed for 2011, and projected to 2021. The markets are analyzed according to therapeutic categories, technologies and geographical areas. The largest expansion will be in diseases of the central nervous system, cancer and cardiovascular disorders. Skin and soft tissue repair as well as diabetes mellitus will be other major markets.
The number of companies involved in cell therapy has increased remarkably during the past few years. More than 500 companies have been identified to be involved in cell therapy and 284 of these are profiled in part II of the report along with tabulation of 274 alliances. Of these companies, 154 are involved in stem cells. Profiles of 70 academic institutions in the US involved in cell therapy are also included in part II along with their commercial collaborations. The text is supplemented with 55 Tables and 11 Figures. The bibliography contains 1,050 selected references, which are cited in the text.
Key Topics Covered:
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September 1st, 2012
Research and Markets: Cell Therapy – Technologies, Markets and Companies – Updated 2012 Report
By raymumme
DUBLIN--(BUSINESS WIRE)--
Research and Markets (http://www.researchandmarkets.com/research/9fkkzb/cell_therapy_tec) has announced the addition of Jain PharmaBiotech's new report "Cell Therapy - Technologies, Markets and Companies" to their offering.
This report describes and evaluates cell therapy technologies and methods, which have already started to play an important role in the practice of medicine. Hematopoietic stem cell transplantation is replacing the old fashioned bone marrow transplants. Role of cells in drug discovery is also described. Cell therapy is bound to become a part of medical practice.
Stem cells are discussed in detail in one chapter. Some light is thrown on the current controversy of embryonic sources of stem cells and comparison with adult sources. Other sources of stem cells such as the placenta, cord blood and fat removed by liposuction are also discussed. Stem cells can also be genetically modified prior to transplantation.
Cell therapy technologies overlap with those of gene therapy, cancer vaccines, drug delivery, tissue engineering and regenerative medicine. Pharmaceutical applications of stem cells including those in drug discovery are also described. Various types of cells used, methods of preparation and culture, encapsulation and genetic engineering of cells are discussed. Sources of cells, both human and animal (xenotransplantation) are discussed. Methods of delivery of cell therapy range from injections to surgical implantation using special devices.
Cell therapy has applications in a large number of disorders. The most important are diseases of the nervous system and cancer which are the topics for separate chapters. Other applications include cardiac disorders (myocardial infarction and heart failure), diabetes mellitus, diseases of bones and joints, genetic disorders, and wounds of the skin and soft tissues.
Regulatory and ethical issues involving cell therapy are important and are discussed. Current political debate on the use of stem cells from embryonic sources (hESCs) is also presented. Safety is an essential consideration of any new therapy and regulations for cell therapy are those for biological preparations.
The cell-based markets was analyzed for 2011, and projected to 2021. The markets are analyzed according to therapeutic categories, technologies and geographical areas. The largest expansion will be in diseases of the central nervous system, cancer and cardiovascular disorders. Skin and soft tissue repair as well as diabetes mellitus will be other major markets.
The number of companies involved in cell therapy has increased remarkably during the past few years. More than 500 companies have been identified to be involved in cell therapy and 284 of these are profiled in part II of the report along with tabulation of 274 alliances. Of these companies, 154 are involved in stem cells. Profiles of 70 academic institutions in the US involved in cell therapy are also included in part II along with their commercial collaborations. The text is supplemented with 55 Tables and 11 Figures. The bibliography contains 1,050 selected references, which are cited in the text.
Key Topics Covered:
The rest is here:
Research and Markets: Cell Therapy - Technologies, Markets and Companies - Updated 2012 Report
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September 1st, 2012
Genetic Engineering Turns Bacteria Into Car Fuel – Business Insider
By Dr. Matthew Watson
 Business Insider | Genetic Engineering Turns Bacteria Into Car Fuel Business Insider A humble soil bacteria has become a genetically engineered factory capable of making fuel for cars. But the project still has to get out of the lab and scale up to industrial-size production. The MIT project aims to make transportation fuels 10 times ... |
Source:
http://news.google.com/news?q=genetic-engineering&output=rss
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August 26th, 2012
Alt Text: Cleaning Up the Olympics, Genetic-Engineering Style – Wired News
By Dr. Matthew Watson
Wired News | Alt Text: Cleaning Up the Olympics, Genetic-Engineering Style Wired News Here's the plan: We use genetic engineering to create a human being who is genetically average in every way, clone him — or her, we can flip a coin — and issue one Average Athlete Baby to each country to raise as they choose. Then, 18 years later ... |
Source:
http://news.google.com/news?q=genetic-engineering&output=rss
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August 26th, 2012
WWF: We’re all eating GM-based meat – The Local.de
By Dr. Matthew Watson
 The Local.de | WWF: We're all eating GM-based meat The Local.de "Genetic engineering in meat, eggs or cheese is landing on our plates and we don't know it," warned WWF consultant Birgit Wilhelm. "Until now, food that comes from animals that have been fed with genetically modified feed does not have to be labelled." ... |
Source:
http://news.google.com/news?q=genetic-engineering&output=rss
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August 26th, 2012
Biotechnology is neat, kids! Monsanto sponsors coloring book – Examiner.com
By Dr. Matthew Watson
 Examiner.com | Biotechnology is neat, kids! Monsanto sponsors coloring book Examiner.com “As you work through the puzzles in the book,” promises a new Monsanto sponsored coloring book, “you will learn about biotechnology and how it can help people live better lives in a healthier world.” The kid's book, created by Monsanto supported ... Trade Group Funded by Monsanto Creates Children's BookTruth-Out |
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August 26th, 2012
CIRM Board Member Prieto Defends Klein’s Right to Appear Before Board
By Dr. Matthew Watson
California stem cell agency, Francisco Prieto, has commented
in an email about the “unseemly performance” item concerning the
agency's former chairman, Robert Klein. Prieto is a
Sacramento physician who serves as a patient advocate member of the
board. He has been on the board since its inception. Here are his
remarks.
“I wanted to comment on this piece
from the perspective of another patient advocate. While I think
you know that I did not always agree with Bob Klein during his tenure
on the ICOC(the agency's governing board), I would strongly defend
his right to appear and give his opinions to the Board. He is a
private citizen now, albeit one with considerable experience and
expertise, and I think his greatest vested interest in this case
stems (you should pardon the expression) from being the child of a
parent with Alzheimers. As you point out, some eyebrows may be
raised, and I can imagine that some board members might be swayed in
either direction by his testimony, but he is a passionate and
committed advocate, and he has the right to advocate before us.”
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August 26th, 2012
An Unseemly Performance: Former Chair of Stem Cell Agency Promotes $20 Million Research Proposal
By Dr. Matthew Watson
Bob Klein is nearly an icon in the
history of the $3 billion California stem cell. And when he appeared
before its governing board last month and aggressively touted a $20
million grant proposal already rejected by agency reviewers, his
actions raised eyebrows.
 |
| Robert Klein Elie Dolgin/Nature photo |
weight, given that they were supported by his unique and influential
relationship with the California Institute for Regenerative
Medicine(CIRM). He and his associates wrote the 10,000-word ballot
initiative that created the stem cell agency in 2004. He ran the $35
million electoral campaign that convinced voters to buy into the
idea. Klein raised millions on behalf of the effort. He personally
provided the campaign $3 million. And he was the first chairman of
the agency, leaving that office only 13 months ago, when he was
designated chairman emeritus.
first public appearance before the board on behalf of a specific
application.. He heralded the applicant, StemCells, Inc., as unique
and the “best” in United States with a “huge body of
experience.”
(The full text of his testimony can be found here.)
 |
| Irv Weissman Stanford Photo |
scientist Irv Weissman, who sits on its board. Weissman also played
an important role in the Prop. 71 ballot campaign that created the
stem cell agency. StemCells, Inc.'s application was turned down by
CIRM reviewers who gave it a score of 61, but the company appealed the action to the agency's governing board. Following the appearance by Klein, Weissman and others, the CIRM board sent the application back for more review.
The board will reconsider it next month or in October.
who requested anonymity, said it is “highly inappropriate for Bob
Klein to be advocating for any grant application from a public
company.”
“He has
considerable influence with the ICOC(the CIRM governing board), and
is closely associated with biotech in the Bay Area. Even if he
doesn't make a lot of money himself from this, then he certainly has
friends who will. Irv Weissman would be one of those friends."
California Stem Cell Report, Klein today defended his actions. He was asked if he had “any sort of
financial ties” to firms or individuals that would benefit from
approval of the award. Klein, who is a real estate investment banker and also an attorney, said he has
“no financial interest” in the firm or individuals that might
benefit.
entirely appropriate. He defined his role as a patient advocate –
not as a lobbyist who is paid for advocating on behalf of a company.
Klein said he had “a particular responsibility to contribute my
background knowledge and experience.”
members would follow his example. He said,
“(I)t would be a tragedy if the
expertise of board members built up over six or more years is lost.”
time for financially strapped StemCells, Inc. The company's
financial information shows that it is losing $5.4 million a quarter
as of the end of June and had only $9 million in cash on hand. It
also had liabilities of $11.6 million, up substantially from $8.5
million in September of last year.
"StemCells Inc has been on the
stock market for 20 years, without producing anything of value for
the investors. The stock price has been sinking fast: it
was 60 cents this June; last year at this time, it was around $5 a
share.
“On July 17, when the CIRM Disease
Team Award review results became available, the stock rose from 87
cents to $1.80 – a person who could anticipate the outcome of the
CIRM applications could have made considerable money in that 24 hour
period.”
with the StemCells, Inc., is more than scientific. According to the
company's financial statements, he holds 88,612 shares. His wife,
Ann Tsukamoto, is executive vice president of the firm. She holds
185,209 shares in the firm.
the Prop. 71 campaign. He did the “billionaire circuit,” raising
money for the initiative, according to an article by Diana Kapp in
San Francisco magazine. Among other things, Weissman worked the
exclusive Bohemian Grove in Northern California and “pitched”
Bill Bowes, a co-founder of Amgen, who, along with his wife, gave
$1.3 million to the campaign. Weissman was the key to securing a
$400,000 contribution from Microsoft's Bill Gates. Weissman also plumped for Prop. 71 in a TV campaign ad.
and Weissman supported a successful attempt last month to overturn
reviewers' rejection of another $20 million application by Judith Shizuru
of Stanford. The application received a score of 53 from reviewers.
by reviewers was the availability of antibodies for the study. The
antibodies were developed by Systemix, a company founded by Weissman.
Systemix was acquired by Novartis in 1997 for about $70 million.
Weissman said he has “negotiated back” rights to key antibodies,
which he said are now held by Stanford.
research was “a showstopper” but did not think the documentation
was adequate. He told the CIRM directors that they now have a letter
with proprietary information that supports the grant application.
labored under the perception that it is something of an insiders'
club. Even the prestigious journal Nature warned in 2008 about what
it called “cronyism” at CIRM. If anything, the situation is worse today, four years later. Enterprises associated with persons on the CIRM board of directors have received more than 90 percent of the funds handed out by the agency. Klein's efforts last month
reinforce the not-so-pleasant image of the stem cell agency as an
old boy's club and create an impression – at the very least – of
unseemly insider influence.
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August 26th, 2012
Text of Klein’s Response to Questions Concerning His Advocacy on Rejected Grant Applications
By Dr. Matthew Watson
Here is the text of Robert Klein's response today to the California Stem Cell Report concerning his appearance before the governing board of the California stem cell agency July 26, 2012. Klein, former chairman of the agency, real estate investment banker and attorney, promoted two applications seeking $20 million each from the agency. Both applications had been rejected by the agency's reviewers. Here is a link to an item on the subject.
"Dear David,
"You have posed two
questions related to my continuing role as a Patient Advocate in
contributing information to the Board of the California Institute for
Regenerative Medicine, in an effort to optimize decisions on medical
and scientific grants and loans for research that could mitigate
and/or cure chronic diseases or injuries.
"Q: Do you have any sort
of financial ties to StemCells Inc. or any of the individuals or
firms that would benefit from approval of those awards by the ICOC(the CIRM governing board)?
"A: I have no financial
interest in StemCells Inc. or any of the individuals or firms that
would benefit from approval of those awards by the ICOC. In fact, I
have no financial interest in any biomedical research company."Q: Do you think it is
appropriate for the former chairman of the ICOC to lobby that body on
behalf of awards to specific companies or individuals?
"A: First, it is
fundamental that the terms be defined to properly respond to your
question. A “Patient Advocate” is a member of a patient family or
a medical/scientific care /support group who advocates for medical
and scientific advances that might potentially mitigate and/or cure a
patient’s chronic disease or injury. A “Patient Advocate” is
not paid for his/her advocacy, unless they are staff members of a
non-profit institution dedicated to a specific disease or group of
diseases or injuries.
"Second, a “lobbyist”
is a paid representative of a company or a for-profit institution(s)
with a financial interest in the outcome of a governmental decision.
"I am serving as a
Patient Advocate in my presentations to the Board of the California
Institute for Regenerative Medicine. As the former Chairman of the
Board, I have a particular responsibility to contribute my background
knowledge and experience for the Board to consider, along with all
new information, in reaching their best decision. I hope other former
Board members, who possess a wealth of scientific, medical, and
institutional knowledge that can benefit the Board, would consider
the value they can contribute to future decisions. As Board terms
expire, it will be important not to lose that institutional knowledge
and medical/scientific expertise that has been built up over the last
seven plus years of the Agency’s existence.
"In an outline format,
I would suggest the following areas where the knowledge of former
Board members can be especially valuable in optimizing the input for
Board decisions in the future.
"A number of Board
members have participated in up to 20 or more Peer Review meetings,
some of which cover multiple days. Current grant or loan requests
represent the result of scientific and medical advancement that has
been intensely vetted in prior peer reviews; the information gained
in those peer reviews should not be lost, when a subsequent grant or
loan request – built on the earlier research outcomes – is
considered. Each peer review session has the benefit of different
specialists and scientists and/or biotech representatives with
unique backgrounds and areas of expertise. The value of the prior
contributions may be pivotal, in considering a later application,
developed from the earlier medical or research advances funded
through CIRM’s grants or loans. The current peer review,
scientific staff presentation, and Board expertise, is not the limit
of the Board’s information, in reaching the best current decision.
To the extent the Board can draw from prior peer reviews (unique
insights), prior scientific staff presentations, and prior Board
expertise, additional information that can enhance a potential
decision, the Board has the opportunity to optimize its decision
making process. This is particularly valuable, when there is a high
standard deviation – a substantial split – in the scoring
positions from the current peer review.
"Beyond peer review
participation, Board members have intensely engaged in another 35
plus Working Group sessions on Facilities and Standards, in addition
to more than 70 Board meetings and over 125 Subcommittee meetings,
as of August 2012. Retiring Board members possess a treasury of
information on policy development, process, federal and state laws
and regulations, and the regulations of the agency, as well as in
depth information on research facilities and capabilities throughout
California, the nation, and the world. It takes a substantial length
of time for a new Board member to gain a comprehensive knowledge in
all of these areas and each Board member will develop unique
insights, which it would be a tragedy to lose. As Chairman, I
frequently reached back to consult with former Board members on
areas of their special expertise and I would hope that all current
and future Board members utilize the significant asset in developed
knowledge of the prior Board members. To the extent prior members
can be available for public meetings, this would be a substantial
benefit to the agency to broadly inform the Board, the scientific
staff, and the public.
"The Board has a
unique contribution to make on programmatic resource allocations and
risk management of the research and clinical investments in each
disease area. The opportunities in some disease areas for major
advancement are numerous, whereas there are major diseases and/or
critical research areas where the potential, high-value advancement
options are relatively limited. For Board members who have
participated in over 20 peer reviews and 70 Board meetings, the
programmatic perspective on the opportunities in each disease area
has been highly developed. Concurrently, those Board members or
former Board members have substantial knowledge that is of critical
value in reaching programmatic decisions on the number of
opportunities for advancement in any specific disease area and the
relative risk that needs to be taken to accomplish meaningful
breakthroughs in advancing the research and clinical opportunities
in a disease and/or injury area.
"I hope these examples
of how former Board members can contribute to the current Board’s
information in reaching decisions on the best medical/scientific
grants and loans are helpful. As I stated earlier, it would be a
tragedy if the expertise of Board members built up over six or more
years is lost. The field is extremely complicated and the Board needs
the opportunity to consider all of the information available. The
Board can choose to accept or reject any past advice or opinions
gained from prior peer review sessions or Board meetings, but the
Board should have access to the full spectrum of information and the
treasury of scientific and medical advice the agency has received
since its inception.
"There are areas that I
have not addresses in this short response, such as the institutional
value of applicants being able to rely upon prior scientific and/or
policy direction, in their current applications. From a historical
perspective, prior Board members and/or the Chairman can have
significant information that is relevant to these evaluations,
especially if the individual Board member served on a special Task
Force , Subcommittee or peer review. These more complicated areas of
individual contribution by former Board members I can address in a
future communication; but, this specific subject – alone – could
comprise several pages and I would like to obtain critical advice and
perspective from other former Board members and the scientific
community before discussing this area in greater detail.
"Bob Klein
"Chair Emeritus
"California Institute
for Regenerative Medicine"
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August 26th, 2012
Text of Klein’s Comments Supporting Rejected Applications
By Dr. Matthew Watson
Robert Klein, who served 6 ½ years as the first chairman
of the $3 billion California stem cell agency, testified before the agency's board for the first time on July 26, 2012. Klein, a real
estate investment banker and attorney, spoke on behalf of two applicants whose
grants had been rejected by the agency's reviewers. The appearance
has raised questions about the propriety of Klein's actions.
Here is a link to an item on his appearance. Here is the text of
his comments as reported in the transcript of the meeting.
“As the board knows, I've never addressed any grant from the
floor. It is critical here to understand that we have here
StemCells, Inc., which is the only company in North America
and, for that matter, maybe in the world, that has had two stem cell
therapies in the brain with these specific neural stem cells. They
have a huge body of experience here.
“Secondly, one of the fundamental issues here that it (the
company's grant application) was downgraded on was the issue of the
fundamental concept, the platform concept, of injecting two focal
injections in the brain, in the hippocampus of the brain. It's
important to note that I've sat on three (CIRM)peer reviews where the
scientists really affirmed this specific approach with extremely high
scores, three different views. All right.
“So it's very important to realize we have a standard deviation
here of 12 (on the review scores). These scientists were completely
split. With some recusals on that panel, if you have 12 or 13 that
can really vote, three or four very low scores can bring it out of
the funding category all the way down. It is in the region where
this board is looking where the other three peer reviews, right,
early translation, the one before that was the planning grant review,
that the hippocampus was a good platform.
“Then they said the key weakness was you can't show migration.
Dr. Laferla (a co-PI on the application) has told me that
today the Journal of Neuroscience accepted the publication of
the data demonstrating migration. It was stated previously in the
application, but it wasn't accepted for publication. It now is.
That is the fundamental weakness that they identified in this
approach.
“So we have a reaffirmed approach to the hippocampus by three
different peer review groups and a substantial portion of these
reviewers along with data dealing with the weak point. I'm sorry it
happened today. The data was out there, accepted for publication
today, means that it should definitely fall into this category. And,
of course, Dr. (Alan) Trounson (president of CIRM) wouldn't
have been able to review that in process because he was recused from
this grant by his own voluntary recusal. So the progress of this
data being accepted for publication is new information today.
“If I look at the entire history of CIRM, as Leeza (Gibbons,
a CIRM director) says, building up to this point, we have reaffirmed
this approach from the very beginning with Dr. Laferla, with multiple
scientific approvals, and board approval, and we have the best
company in North America with the greatest experience with these
neural stem cells, with the best researcher we have for the potential
to address this disease, and we have brand-new data that demonstrates
and totally contradicts the key weakness on which it was downgraded.”
behalf of a second application, also rejected by CIRM reviewers.
“This is the only other disease team grant I will address. Very
specifically, this was a disease team grant that I was on the peer
review in the planning grant stage. There are some fundamental
issues here. Is the international company on which the one antibody
that's not coming from Stanford, the two for sorting are
coming from Stanford, is the other antibody coming from this
international company a commitment that you can rely on?
“The reviewers said this was a showstopper. That's the word they
used. They made a decision this was a showstopper because they did
not believe the company because they thought that the documentation
was inadequate. You now have a letter that goes into great
proprietary depth about the depth of this company's commitments
written by the head of development and translation internationally
for the company.
“If we cannot depend on company commitments of this type, and
you will review the letter in executive session, if you have one, I
will not understand how we'll be able to collaborate with companies
with proprietary products and processes where they're making
commitments to academic institutions of the highest standard. I
believe this company is going to perform. I was on an hour call to
confirm with eight members of that company their level of commitment,
and I am completely convinced by that point.
“The review is completely factually wrong on this issue about
the other two antibodies for sorting this. Dr. (Irv) Weissman
has just said they have not only been developed, they have been used
in clinical trials. There's data on them. And they are, in fact,
being thawed under FDA direction to reuse in this trial.
“So I believe there's a major factual difference. Remember with
Karen Aboody there was a major factual error that was pivotal
in elevating that, and we found tremendous performance on that grant
by Karen Aboody of City of Hope.
“So you have a decision to make. As a risk issue, do we believe
this company? Finally, this is broader than SCID.
“Donald Kohn has written a letter that's in the public
domain that I suggest you read. It makes it very clear that opening
the niche for repopulating the immune system without chemotherapy and
radiation is a key contribution to every form of genetically modified
stem cells for an entire range of childhood diseases and other
genetic diseases in addition to therapies like sickle cell or aids.
“I suggest that that profound contribution that can be made to
the field is a risk that is worth taking early on because of his
contribution to so many other areas. You have 12 other letters from
North America's leading pediatric geneticists that fundamentally
provide extraordinary support for this position and this approach.”
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August 26th, 2012
Prop 37: California’s high-stakes food fight – Daily Democrat
By Dr. Matthew Watson
Los Angeles Times | Prop 37: California's high-stakes food fight Daily Democrat Genetic engineering is the process of changing the DNA of living organisms; it is often used to improve a plant's resistance to pests. According to some estimates, 40 to 70 percent of food products sold in grocery stores in California contain ... Growing concernsPasadena Weekly Yes on Prop 37 March in Santa Cruz to Label Genetically Engineered FoodsBay Area Indymedia Top 10 Lies Told by Monsanto on GMO Labeling in CaliforniaHuffington Post City Watch -Christian Science Monitor all 81 news articles » |
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August 26th, 2012