Volume 12.05 | Feb 7

Cell Therapy News 12.05, February 7, 2011.
In this issue:  Science  |  Policy  |  Business  |  NIH  |  CBER  |  Regulatory  |  Events Subscribe  


Save Messengers — Modified mRNAs Open Up New Therapeutic Possibilities
Researchers show that, in gene therapy, chemical modification of mRNAs provides a promising alternative to DNA-based procedures. In contrast to the latter, the modified RNAs do not increase the risk of cancer, and do not induce frequently observed severe immune reactions seen with DNA or unmodified mRNA. [Press release from Ludwig-Maximilians-Universität München discussing online prepublication in Nature Biotechnology]



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Progress on Stem Cells: A Meandering March
On the occasion of a recent finding about abnormal gene expression in reprogrammed stem cell lines , Ed Yong built a handy timeline on the science of cell reprogramming. [ScienceInsider]

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Researchers Identify Genetic Cause of New Vascular Disease
Clinical researchers have identified the genetic cause of a rare and debilitating vascular disorder not previously explained in the medical literature. [Press release from the National Institutes of Health discussing online prepublication in the New England Journal of Medicine]

Researchers Unlock the Potential for Exploring Kidney Regeneration
Researchers have identified a cell in zebrafish that can be transplanted from one fish to another to regenerate nephrons, providing the potential to improve kidney function. [Press release from Brigham and Women’s Hospital discussing online prepublication in Nature]

Cell Reprogramming Leaves a “Footprint” Behind
Reprogramming adult cells to recapture their youthful “can-do-it-all” attitude appears to leave an indelible mark, found researchers. When the team scoured the epigenomes of so-called induced pluripotent stem cells base by base, they found a consistent pattern of reprogramming errors. [Press release from the Salk Institute for Biological Studies discussing online prepublication in Nature]

Some iPS Cells Indistinguishable from Embryonic Stem Cells
Scientific studies comparing the similarities and differences between the inducible stem (iPS) and embryonic stem (ES) cells have been conflicting and inconclusive. Seeking to settle this debate, researchers devised a tool incorporating a genomics-based analytical approach. [Press release from the Broad Institute of MIT and Harvard discussing online prepublication in Cell]

Scientists Unlock One Mystery of Tissue Regeneration
Researchers have identified a genetic switch that controls oxidative stress in stem cells and thus governs stem cell function. [Press release from the University of Rochester discussing online prepublication in Cell Stem Cell]

Study Finds a Stem Cell Origin of Skin Cancer and the Genetic Lesions That Promote Its Malignancy
Researchers have unmasked a long sought stem cell origin of carcinoma and identified the genetic lesions occurring within these cells that spur them on to malignancy. [Press release from Cold Spring Harbor Laboratory discussing online prepublication in Cell Stem Cell]

Stem Cells from Different Sources Are Not Equivalent
A team of researchers at Stanford University School of Medicine found that human induced pluripotent stem cells (hiPSCs) exhibited substantially more heterogeneity in gene expression than did human embryonic stem cells (hESCs), leading them to suggest that hiPSCs have a less stable pluripotent state than hESCs, information that is important to consider when thinking about using these cells for the purpose of regenerative medicine. [Press release from EurekAlert! discussing online prepublication in the Journal of Clinical Investigation]

Study Shows Promise That Heart Damage in Children Could Be Repaired by Using Stem Cells from Patient's Own Heart
Visionaries in the field of cardiac therapeutics have long looked to the future when a damaged heart could be rebuilt or repaired by using one’s own heart cells. A study shows that heart stem cells from children with congenital heart disease were able to rebuild the damaged heart in the laboratory. [Press release from Children's Memorial Hospital discussing online prepublication in Circulation]

Cells' Energy Factories Linked to Damaging Inflammation
Scientists have discovered that molecules called reactive oxygen species produced by the mitochondria, in cells, may play a role in a rare inherited disorder in which uncontrolled inflammation damages the body’s tissues. Their research in human and mouse cells suggests that blocking these molecules could reduce inflammation in TNF receptor-associated periodic syndrome and possibly other inflammatory diseases. [Press release from the National Institutes of Health discussing online prepublication in the Journal of Experimental Medicine]

New Induced Stem Cells May Unmask Cancer at Earliest Stage
By coaxing healthy and diseased human bone marrow to become embryonic-like stem cells, a team of scientists has laid the groundwork for observing the onset of the blood cancer leukemia in the laboratory dish. [Press release from the University of Wisconsin-Madison discussing online prepublication in Blood]

Researchers Discover Way to Reverse Immune System Aging
Researchers have discovered a way to reverse the aging process by removing old B lymphocytes from old mice, and forcing the production of young, potent cells to replace them. [Press release from the American Technion Society discussing online prepublication in Blood]

New Probiotic Combats Inflammatory Bowel Disease
A genetically tweaked version of a common probiotic found in yogurt and cheese appears to be an effective therapy for inflammatory bowel diseases such as Crohn’s disease and ulcerative colitis. It may also prove to be useful in colon cancer, another disease triggered by inflammation. [Press release from Northwestern University Feinberg School of Medicine discussing online prepublication in the Proceedings of the National Academy of Sciences USA]

MicroRNA Cocktail Helps Turn Skin Cells into Stem Cells
Researchers identified several specific microRNAs that are important during reprogramming and exploited them to make the transition from skin cell to induced pluripotent stem cell more efficient. [Press release from Sanford-Burnham Medical Research Institute discussing online prepublication in The EMBO Journal]

Researchers Develop Safer Way to Make Induced Pluripotent Stem Cells
Researchers have found a better way to create induced pluripotent stem cells from a small blood sample. This new method avoids creating DNA changes that could lead to tumor formation. [Press release from Johns Hopkins Medicine discussing online prepublication in Cell Research]

Engineered Cells Could Usher in Programmable Cell Therapies
In work that could jumpstart the promising field of cell therapy, researchers have engineered cells that could solve one of the key challenges associated with the procedure: control of the cells and their microenvironment following transplantation. [Press release from Brigham and Women's Hospital discussing online prepublication in Biomaterials]

New 3D Nanoscaffold Could Revolutionize Human Tissue Engineering
Researchers have created a three-dimensional electrospun scaffold on the nano scale that more effectively and efficiently facilitates cell and tissue growth in the laboratory. [Press release from the University of Alabama at Birmingham's discussing online prepublication in Biomaterials]

Bioengineered Veins Could Help Patients Needing Bypass Surgery, Dialysis
New research demonstrates the capability of tissue-engineered vascular grafts that are immediately available at the time of surgery and are less likely to become infected or obstructed. The bioengineering method of producing veins shows promise in large- and small-diameter applications, such as for coronary artery bypass surgery and for vascular access in hemodialysis. [Press release from East Carolina University discussing online prepublication in Science Translational Medicine]

CURRENT PUBLICATIONS (Ranked by Impact Factor of the Journal)

NT5E Mutations and Arterial Calcifications
Researchers identified mutations in NT5E in members of three families with symptomatic arterial and joint calcifications. This gene encodes CD73, which converts AMP to adenosine, supporting a role for this metabolic pathway in inhibiting ectopic tissue calcification. [N Engl J Med]

Identification of Adult Nephron Progenitors Capable of Kidney Regeneration in Zebrafish
The data demonstrate that the zebrafish kidney probably contains self-renewing nephron stem/progenitor cells. The identification of these cells paves the way to isolating or engineering the equivalent cells in mammals and developing novel renal regenerative therapies. [Nature]

Hotspots of Aberrant Epigenomic Reprogramming in Human Induced Pluripotent Stem Cells
Researchers report the first whole-genome profiles of DNA methylation at single-base resolution in five human Induced pluripotent stem cell (iPSC) lines, along with methylomes of embryonic stem (ES) cells, somatic cells, and differentiated iPSCs and ES cells. [Nature]

Reference Maps of Human ES and iPS Cell Variation Enable High-Throughput Characterization of Pluripotent Cell Lines
Researchers have established genome-wide reference maps of DNA methylation and gene expression for 20 previously derived human embryonic stem (ES) lines and 12 human induced pluripotent stem (iPS) cell lines, and have measured the in vitro differentiation propensity of these cell lines. This resource enabled researchers to assess the epigenetic and transcriptional similarity of ES and iPS cells and to predict the differentiation efficiency of individual cell lines. [Cell]

Expression of Therapeutic Proteins After Delivery of Chemically Modified mRNA in Mice
Researchers investigated the therapeutic utility of chemically modified mRNA as an alternative to DNA-based gene therapy. [Nat Biotechnol]

Redox Regulation by Keap1 and Nrf2 Controls Intestinal Stem Cell Proliferation in Drosophila
Findings establish Keap1 and Nrf2 as a critical redox management system that regulates stem cell function in high-turnover tissues. [Cell Stem Cell]

(Delta)Np63(Alpha) Is an Oncogene that Targets Chromatin Remodeler Lsh to Drive Skin Stem Cell Proliferation and Tumorigenesis
Findings indicate that (delta)Np63(alpha) is an oncogene that cooperates with Ras to promote tumor-initiating stem-like proliferation and suggest that Lsh-mediated chromatin-remodeling events are critical to this process. [Cell Stem Cell]

Policy Uncertainty and the Conduct of Stem Cell Research
A survey of U.S. stem cell scientists shows that uncertainty following the legal challenge to the Obama Administration's human embryonic stem cell (hESC) research policy has negative scientific and economic impacts and affects a range of stem cell scientists, not just those working with hESCs. The international implications of these results are also discussed. [Cell Stem Cell]

Single Cell Transcriptional Profiling Reveals Heterogeneity of Human Induced Pluripotent Stem Cells
Researchers used single cell analysis to resolve the gene expression profiles of 362 human induced pluripotent stem cells (hiPSCs) and human embryonic stem cells (hESCs) for an array of 42 genes that characterize the pluripotent and differentiated states. Comparison between single hESCs and single hiPSCs revealed markedly more heterogeneity in gene expression levels in the hiPSCs, suggesting that hiPSCs occupy an alternate, less stable pluripotent state. [J Clin Invest]

Characterization and Functionality of Cardiac Progenitor Cells in Congenital Heart Patients
Resident human cardiac progenitor cells (hCPCs) are most abundant in the neonatal period and rapidly decrease over time. Human cardiosphere-derived cells can be reproducibly isolated and expanded from young human myocardial samples regardless of age or diagnosis. hCPCs are functional and have potential in congenital cardiac repair. [Circulation]

Mitochondrial Reactive Oxygen Species Promote Production of Proinflammatory Cytokines and Are Elevated in TNFR1-Associated Periodic Syndrome (TRAPS)
Findings suggest that mitochondrial reactive oxygen species may be a novel therapeutic target for TRAPS and other inflammatory diseases. [J Exp Med]

Efficient Generation of Transgene-Free Induced Pluripotent Stem Cells from Normal and Neoplastic Bone Marrow and Cord Blood Mononuclear Cells
Scientists demonstrated that induced pluripotent stem cells free of transgene and vector sequences could be efficiently generated from human bone marrow and cord blood mononuclear cells using non-integrating episomal vectors. [Blood]

B Cell Depletion Reactivates B Lymphopoiesis in the BM and Rejuvenates the B Lineage in Aging
Results suggest that immunosenescence in the B-lineage is not irreversible, and that depletion of the long-lived B cells in old mice rejuvenates the B-lineage and enhances immune competence. [Blood]



Ongoing Policy Uncertainty Is Detrimental for Stem Cell Scientists
While there is no doubt that the ethical controversy surrounding human embryonic stem cell (hESC) research has given rise to an uncertain policy environment, the true impact of years of frequent policy changes has not been fully assessed. Now, an article reports on a recent survey of several hundred stem cell scientists in the United States and begins to reveal the substantial negative impact that this uncertainty has had on them, including both those who work directly with hESCs and those who work with less contentious types of stem cells. [Press release from EurekAlert! discussing online prepublication in Cell Stem Cell]

Science Accounts Hit Hard by Planned House Budget Cuts
House of Representatives Republicans unveiled a long-awaited plan to reduce federal spending this year that includes double-digit cuts in the panels that fund most of civilian basic research. It is $74 billion lower than President Barack Obama's 2011 request, submitted 1 year ago and never enacted, and is divided between $56 billion in civilian spending and $18 billion for security expenditures, including $9 billion from the military. [House of Representatives, United States]

Interior Department Inks Scientific Integrity Policy
The Department of the Interior has finalized its policy on scientific integrity, creating code of conduct and procedures for investigation, as well as designating an official in charge. [Department of the Interior, United States]

Solicitation for Nominations for the National Vaccine Advisory Committee (FR Doc. 2011-2372) [Department of Health and Human Services, United States]



Kyoto University, iPS Academia Japan, and iPierian Announce Global Licensing and Patent Assignment Agreement
Kyoto University, iPS Academia Japan Inc., and iPierian Inc., announced they have entered into a series of intellectual property agreements creating a unified, worldwide patent estate covering induced pluripotent stem cell technology. [iPierian Inc. Press Release]

Genzyme Will Allow Sanofi-Aventis to Conduct Due Diligence
Genzyme Corporation announced that ongoing discussions with Sanofi-Aventis have progressed to the point where Genzyme’s board has authorized the company to enter into a confidentiality agreement with Sanofi-Aventis in order to allow Sanofi to conduct due diligence. [Genzyme Corporation Press Release]

Genzyme Completes Sale of Diagnostics Business to Sekisui Chemical Co.
Genzyme Corporation announced that it has completed the sale of its diagnostic products business to Sekisui Chemical Co., Ltd. for $265 million in cash. [Genzyme Corporation Press Release]

Apeiron Acquires Rights for a Phase II Biologic Against Neuroblastoma and Other Cancers from Merck KGaA, Germany
Vienna-based biotech company Apeiron Biologics AG strengthened its cancer immunotherapy portfolio by acquiring rights to further develop and commercialize the immunocytokine hu14.18-IL2 from Merck KGaA, Germany. [Apeiron Biologics AG Press Release]

Alexion Acquires Taligen Therapeutics and Creates Translational Medicine Group to Accelerate Development of Expanded Portfolio
Alexion Pharmaceuticals, Inc. announced that it has acquired Taligen Therapeutics, Inc., a privately held development-stage biotechnology company based in Cambridge, Mass. [Alexion Pharmaceuticals, Inc. Press Release]

The Debiopharm Group and Marina Biotech Partner to Develop and Commercialize RNAi-based Therapies for the Treatment of Bladder Cancer
The Debiopharm Group and Marina Biotech, Inc. announced that they entered into an exclusive agreement concerning the development and commercialization of Marina Biotech’s pre-clinical program in bladder cancer. [Debiopharm Group Press Release]

Scientists Look to Stem Cells to Mend Broken Hearts
Britain's leading heart charity launched a 50 million pound ($80 million) research project into the potential of stem cells to regenerate heart tissue and “mend broken hearts”. [Reuters]

Cedars-Sinai Research Team Awarded $1.9 Million from State Stem Cell Agency to Develop New Tools and Technologies
A team of scientists from the Cedars-Sinai Regenerative Medicine Institute has been awarded a $1.9 million grant  from the California Institute for Regenerative Medicine to fund development of a new technique to aid pharmaceutical discoveries for specific diseases. [Cedars-Sinai Medical Center Press Release]

Digna Biotech and CIMA Receive EUR 1.3 Million Funding for Acute Intermittent Porphyria Gene Therapy as Part of an EU FP7 Grant for Clinical Development of Orphan Drugs
Digna Biotech and the Centro de Investigación Médica Aplicada (CIMA) de la Universidad de Navarra announced that the European Union has finalized a EUR 3.3 million grant to AIPGENE consortium. Digna Biotech and CIMA will receive, as members of the consortium, EUR 0.5 and EUR 0.8 million respectively for the clinical development of a gene therapy product for Acute Intermittent Porphyria. [PR Newswire]

Pfizer's Plan to Cut R&D Spending Rattles Some Cages
Pfizer, the world's largest drug company, and one that's proud of its scientific acumen, announced yesterday that it will cut R&D spending in 2012 by about 20%, from $8.5 billion to $7 billion. [ScienceInsider]

Antisense Pharma Receives Approval for Clinical Studies in South Korea and Taiwan in Malignant Brain Tumors
The biopharmaceutical company Antisense Pharma announced that the South Korean health agency KFDA as well as the Taiwanese health agency TFDA have granted their approvals for the implementation of clinical studies using the anti-cancer drug trabedersen for patients with high-grade brain tumors. Trabedersen is a gene-silencing substance inhibiting the tumor factor Transforming Growth Factor beta 2 at its translational level. [Antisense Pharma Press Release]

RXi Pharmaceuticals Initiates Development Program for Dermal Anti-Scarring Product Candidate
RXi Pharmaceuticals Corporation announced further details regarding RXI-109, the company's dermal anti-scarring candidate. [RXi Pharmaceuticals Corporation Press Release]

Dr. Yvonne Greenstreet Joins Pfizer as Senior Vice President and Head of Medicines Development, Specialty Care Business Unit
Pfizer Inc. announced that Dr. Yvonne Greenstreet has been appointed Senior Vice President and Head of Medicines Development for the Specialty Care Business Unit, effective February 1, 2011. [Pfizer Inc. Press Release]

Genome Canada Announces New Competition: Entrepreneurship Education in Genomics
Genome Canada announced the launch of a new pilot program called Entrepreneurship Education in Genomics (EEG). This program aims to support initiatives to educate the Canadian genomics research community with respect to how to create and capture value from their research and translate their discoveries into marketable applications, products, technologies, systems and processes. [Genome Canada Press Release]

Enabling the Next Biomedical Revolution
In his State of the Union address President Obama singled out biomedical innovation as a key driver of America's future prosperity. This sector has indeed generated tens of thousands of jobs, attracted billions in investment and created advanced medical treatments for Americans. [Forbes]

Generics Companies Weigh In on Biological Drugs
Generic drug makers are expecting copies of complex biological drugs to become a multibillion dollar market in the near future. But a lack of clear regulation, intense scrutiny from makers of the original drugs and the high cost of research may damp their prospects. [The Wall Street Journal]


Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-2202)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-2208)

National Cancer Institute; Notice of Closed Meeting (FR Doc. 2011-2189)

National Cancer Institute; Notice of Closed Meetings (FR Doc. 2011-2205)

National Cancer Institute; Notice of Closed Meetings (FR Doc. 2011-2206)

National Heart, Lung, and Blood Institute; Notice of Closed Meeting (FR Doc. 2011-2210)

National Institute of Biomedical Imaging and Bioengineering; Notice of Closed Meeting (FR Doc. 2011-2185)

National Institute of Biomedical Imaging and Bioengineering; Notice of Closed Meeting (FR Doc. 2011-2186)

Submission for OMB Review; Comment Request; Pretesting of NIAID's Biomedical HIV Prevention Research Communication Messages (FR Doc. 2011-2546)
Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-2494)



Pluripotent Stem Cells in Translation: Early Decisions
The National Institutes of Health and the Food and Drug Administration are collaborating on a series of workshops on moving pluripotent stem cell therapies into the clinic.

January 28, 2011 Approval Letter – Menevo 

January 25, 2011 Approval Letter – Prevnar 13  

February 25, 2011: Vaccines and Related Biological Products Advisory Committee Meeting Waivers 

January 25, 2011 Approval Letter – Pediarix 

February 25, 2011: Vaccines and Related Biological Products Advisory Committee Meeting Announcement 

January 21, 2011 Approval Letter – Pneumococcal, Polyvalent 



Potential Signals of Serious Risks/New Safety Information Identified by the Adverse Event Reporting System (AERS) Between July – September 2010
The names of products and potential signals of serious risks/new safety information were identified for these products during the period July – September 2010 in the AERS database.

Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; Guidance for Industry on Pharmacogenomic Data Submissions; Extension [Docket No. FDA-2010-N-0536]

Guidance: PET Drug Applications — Content and Format for NDAs and ANDAs


Agency Releases New Guidance on Medicines Based on Stem Cells
The Agency has released new guidance on medicines based on stem cells. The guidance covers the different types of stem cells used in medicines, and the considerations that companies need to take into account when developing medicines including stem cells or developed from stem cells.

New Version of EudraGMP Allows Access to Information from all Member States
The European Medicines Agency has launched a new version of its EudraGMP database giving the general public access to information on manufacturing inspections performed by regulatory authorities from all European Economic Area countries.

Expressions of Interest Invited for Transatlantic Workshop on Drug-Related PML
The Agency is inviting experts and stakeholders to express their interest in attending a two-day workshop on drug-related progressive multifocal leukoencephalopathy (PML) in London on 25-26 July 2011.


Information Sent to Healthcare Professionals in January About the Safety of Medicines


NEW Neurodegenerative Diseases: The Molecular and Cellular Basis for Neurodegeneration
February 21-26, 2011
Taos, United States

NEW The 14th U.S.-Japan Cellular and Gene Therapy Conference 
February 24, 2011
Bethesda, United States

Visit our events page to stay up to date with the latest events in the cell, gene and immunotherapy community.


Lab Technologist – Cell Separation (STEMCELL Technologies)

Lab Technologist – Human Embryonic and Induced Pluripotent Stem Cells (STEMCELL Technologies)

Lab Technologist – Tissue Culture (STEMCELL Technologies)

Field Applications Specialist – Cell Therapy (Medical) (Pall Corporation)
Postdoctoral Positions – Stem Cell Biology (Joslin Diabetes Center/Harvard Stem Cell Institute)

Cell Therapy Manufacturing Manager (Opexa Therapeutics)

Assistant Professor (University of Pittsburgh School of Medicine, Center for Cellular and Molecular Engineering)

Post-Doctoral Positions (University of Texas MD Anderson Cancer Center)
PhD Students – Stem Cell Differentiation and Cellular Reprogramming (Ulm University Hospital)

Senior Scientist – Embryonic Stem Cells – Hepatocytes (Hays Pharma)

Research Technician Position in Human Stem Cells (University of Pittsburgh)

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