Volume 12.04 | Jan 31

Cell Therapy News 12.04, January 31, 2011.
In this issue:  Science  |  Policy  |  Business  |  NIH  |  CBER  |  Regulatory  |  Events Subscribe  


Adult Skin Cells Converted Directly to Beating Heart Cells 
Scripps Research Institute scientists have converted adult skin cells directly into beating heart cells efficiently without having to first go through the laborious process of generating embryonic-like stem cells. The powerful general technology platform could lead to new treatments for a range of diseases and injuries involving cell loss or damage, such as heart disease, Parkinson's, and Alzheimer's disease. [Press release from ScienceDaily discussing online prepublication in Nature Cell Biology]



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Adult Kidney Stem Cells Found in Fish
It has long been a given that adult humans — and mammals in general — lack the capacity to grow new nephrons. Researchers have now identified adult kidney stem cells in the zebrafish that can generate new nephrons. These cells can be transplanted from one fish to another fish, whereby they grow into functional nephrons in the transplanted recipient. [Press release from Harvard University discussing online prepublication in Nature]

Uncovering the Trail Behind Growing Too Old, Too Soon
Scientists have produced the world's first human cell model of progeria. The team used a novel technique of deriving induced pluripotent stem (iPS) cells from cells of human progeria patients. The researchers used their iPS cells to identify two types of cells — mesenchymal stem cells and vascular smooth muscle cells — that were particularly adversely affected by progeria. [Press release from the Agency for Science, Technology and Research (A*STAR) discussing online prepublication in Cell Stem Cell]

Red Blood Cell Hormone Modulates the Immune System
New research reveals that a hormone best known for stimulating the production of red blood cells can modulate the immune response. The study finds that erythropoietin has contrasting influences on infectious and inflammatory diseases and may be useful in the design of new therapeutic strategies. [Press release from ScienceDaily discussing online prepublication in Immunity]

NIH Researchers Extend Use of Gene Therapy to Treat a Soft Tissue Tumor
This study is the first to use genetically modified immune cells, in a technique known as adoptive therapy, to cause cancer regression in patients with a solid cancer as opposed to melanoma. This approach represents a method for obtaining immune cells from any cancer patient and converting them into ones that can recognize cancer cells expressing the target antigen, NY-ESO-1, according to researchers. [Press release from the National Institutes of Health discussing online prepublication in the Journal of Clinical Oncology]

Study Raises Safety Concerns About Experimental Cancer Approach
A study has raised safety concerns about an investigational approach to treating cancer. The strategy takes aim at a key signaling pathway, called Notch, involved in forming new blood vessels that feed tumor growth. When researchers targeted the Notch1 signaling pathway in mice, the animals developed vascular tumors, primarily in the liver, which led to massive hemorrhages that caused their death. [Press release from Washington University School of Medicine in St. Louis discussing online prepublication in the Journal of Clinical Investigation]

At Last, a Function at the Junction–Researchers Discover That Stem Cell Marker Regulates Synapse Formation
Among stem cell biologists there are few better-known proteins than nestin, whose very presence in an immature cell identifies it as a stem cell. As helpful as this is to researchers, until now no one knew which purpose nestin serves in a cell. Investigators show that nestin has reason for being in a completely different cell type–muscle tissue. There, it regulates formation of the so-called neuromuscular junction, the contact point between muscle cells and “their” motor neurons. [Press release from the Salk Institute of Biological Studies discussing online prepublication in Nature Neuroscience]

Roundworm Unlocks Pancreatic Cancer Pathway
A team of researchers took a step back to a simpler organism — a common roundworm — and made a discovery about how the Ras oncogene chooses a signaling pathway and how the consequences of that choice play out in cellular development — a key issue in cancer, which is characterized by uncontrolled cell growth. [Press release from University of North Carolina Lineberger Comprehensive Cancer Center discussing online prepublication in Developmental Cell]

Growth-Factor-Containing Nanoparticles Accelerate Healing of Chronic Wounds
Investigators have developed a novel system for delivery of growth factors to chronic wounds such as pressure sores and diabetic foot ulcers. In their work, the team reports fabricating nanospheres containing keratinocyte growth factor, a protein known to play an important role in wound healing, fused with elastin-like peptides. [Press release from Massachusetts General Hospital discussing online prepublication in the Proceedings of the National Academy of Sciences USA]

Research into Synthetic Antibodies Offers Hope for New Diagnostics
In a pair of new papers, researchers demonstrated a simple means of improving the binding affinity of synbodies, which are composed of 20 unit chains of amino acids, strung together in random order. They also used random peptide sequences spotted onto glass microarray slides to mine information concerning the active regions or epitopes of naturally occurring antibodies. [Press release from the Biodesign Institute at Arizona State University discussing online prepublication in Molecular and Cellular Proteomics and PLoS One]

Novel Effort to Fight Cancer with Cancer Cells
Researchers are taking tumor cells from mice, encapsulating them in agarose beads, and implanting them in the abdomen of cancer patients. There, cells in the beads secrete proteins researchers believe could signal a patient's cancer to stop growing, shrink or even die. [Press release from The Rogosin Institute discussing online prepublication in Cancer Research]

With Chemical Modification, Stable RNA Nanoparticles Go 3D
By replacing a chemical group in RNA, researchers have found a way to bypass RNase and create stable three dimensional configurations of RNA, greatly expanding the possibilities for RNA in nanotechnology. [Press release from the University of Cincinnati discussing online prepublication in ACS Nano]

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]

New Anti-HIV Gene Therapy Makes T-Cells Resistant to HIV Infection
An innovative genetic strategy for rendering T-cells resistant to HIV infection without affecting their normal growth and activity is described. A team of researchers from Japan, Korea, and the U.S. developed an anti-HIV gene therapy method in which a bacterial gene called mazF is transferred into CD4+ T-cells. [Press release from ScienceDaily discussing online prepublication in Human Gene Therapy]

Caffeine Energizes Cells, Boosting Virus Production for Gene Therapy Applications
Give caffeine to cells engineered to produce viruses used for gene therapy and the cells can generate 3- to 8-times more virus, according to a paper. This simple and inexpensive strategy for increasing lentivirus production was developed by researchers from the University of Texas Southwestern Medical Center, Dallas. [Press release from ScienceDaily discussing online prepublication in Human Gene Therapy]

Researchers Eliminate Major Roadblock in Regenerative Medicine
A team of stem cell biologists and engineers has identified an optimal combination and concentration of small-molecule inhibitors to support the long-term quality and maintenance of human embryonic stem cells in feeder-free and serum-free conditions. [Press release from the University of California, Los Angeles discussing online prepublication in Nature Communications]

Chinese Academy of Sciences Initiates “Trailblazing” Stem Cell Research Project
The Chinese Academy of Sciences (CAS) announced it has initiated a “strategic, trailblazing” research project on stem cells and regenerative medicine. The project mainly aims to remove the bottlenecks China is confronted with in stem cell research, the CAS said in its 2011 work meeting in Beijing. [Chinese Academy of Sciences Press Release]

CURRENT PUBLICATIONS (Ranked by Impact Factor of the Journal)

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]

A Human iPSC Model of Hutchinson Gilford Progeria Reveals Vascular Smooth Muscle and Mesenchymal Stem Cell Defects
Because mesenchymal stem cells (MSCs) reside in low oxygen niches in vivo, researchers propose that, in Hutchinson-Gilford Progeria syndrome, this causes additional depletion of the MSC pool responsible for replacing differentiated cells lost to progerin toxicity. [Cell Stem Cell]

Erythropoietin Contrastingly Affects Bacterial Infection and Experimental Colitis by Inhibiting Nuclear Factor-KappaB-Inducible Immune Pathways
Researchers show that erythropoietin (EPO) inhibits the induction of proinflammatory genes including tumor necrosis factor-alpha and inducible nitric oxide synthase in activated macrophages, which is mechanistically attributable to blockage of nuclear factor-B p65 activation by EPO. [Immunity]

Conversion of Mouse Fibroblasts into Cardiomyocytes Using a Direct Reprogramming Strategy
Researchers show that conventional reprogramming towards pluripotency through overexpression of Oct4, Sox2, Klf4 and c-Myc can be shortcut and directed towards cardiogenesis in a fast and efficient manner. [Nat Cell Biol]

Tumor Regression in Patients with Metastatic Synovial Sarcoma and Melanoma Using Engineered Lymphocytes Reactive with NY-ESO-1
Observations indicate that T-cell receptor-based gene therapies directed against NY-ESO-1 represent a new and effective therapeutic approach for patients with melanoma and synovial cell sarcoma. [J Clin Oncol]

Notch1 Loss of Heterozygosity Causes Vascular Tumors and Lethal Hemorrhage in Mice
Researchers report that the loss of Notch1 caused widespread vascular tumors and organism lethality secondary to massive hemorrhage. These findings reflected a cell-autonomous role for Notch1 in suppressing neoplasia in the vascular system and provide a model by which to explore the mechanism of neoplastic transformation of endothelial cells. [J Clin Invest]

Nestin Negatively Regulates Postsynaptic Differentiation of the Neuromuscular Synapse
Results suggest that nestin is required for acetylcholine (Ach)-induced, Cdk5-dependent dispersion of ACh receptor clusters during neuromuscular junction development. [Nat Neurosci]

Ras Effector Switching Promotes Divergent Cell Fates in C. elegans Vulval Patterning
Observations define the utility of Ras effector switching during normal development and may provide a possible mechanistic basis for cell and cancer-type differences in effector dependency and activation. [Dev Cell]

Self-Assembling Elastin-Like Peptides Growth Factor Chimeric Nanoparticles for the Treatment of Chronic Wounds
Researchers demonstrate the fabrication of a fusion protein comprising of elastin-like peptides and keratinocyte growth factor. [Proc Natl Acad Sci U S A]

An Optimized Small Molecule Inhibitor Cocktail Supports Long-Term Maintenance of Human Embryonic Stem Cells
Researchers identify, using a feedback system control scheme, a unique combination of three small molecule inhibitors that enables the maintenance of human embryonic stem cells on a fibronectin-coated surface through single cell passaging. [Nat Commun]



Consensus Statement: Statement on Policies and Practices Governing Data and Materials Sharing and Intellectual Property in Stem Cell Science
Tension is increasing between fairly new and pervasive policies and practices governing data and materials sharing and intellectual property in science, and norms of openness and free exchange. While intellectual property rights can bring private investment into areas underfunded by governments and help bridge gaps between scientific invention or discovery and useful technologies, some new and emerging policies and practices risk slowing innovation in research and development and skewing attention toward large markets, to the disadvantage of small markets, such as those for rare diseases and in some emerging economies. [Hinxton Group]



Stanford Joins First Embryonic Stem Cell Therapy Clinical Trial
The first clinical trial of cells derived from human embryonic stem cells began in October 2010 in a paralyzed patient at the Shepherd Center in Atlanta. Stanford University School of Medicine and Santa Clara Valley Medical Center became the third site to participate in the trial, which will enroll up to 10 patients with spinal cord injuries at up to seven institutions nationwide. [Stanford University School of Medicine Press Release]

Amgen to Acquire BioVex, a Privately Held Biotechnology Company Headquartered in Woburn, Mass
Amgen and BioVex Group, Inc. announced that the companies have entered into a definitive acquisition agreement under which Amgen has agreed to acquire BioVex Group, Inc., a privately held, venture-funded, biotechnology company headquartered in Woburn, Mass. BioVex is developing OncoVEX(GM-CSF), a novel oncolytic vaccine in Phase III clinical development, that may represent a new approach to treating melanoma and head and neck cancer. [Amgen Inc. Press Release]

Radient Pharma Wholly-Owned Subsidiary NuVax Therapeutics Signs Exclusive License Agreement For Immune-Gene Therapeutics Technology from University of Florida Research Center
Radient Pharmaceuticals Corporation announced its wholly owned subsidiary NuVax Therapeutics, Inc. has signed an exclusive license agreement with the University of Florida College of Medicine in Gainesville, Florida for the use and commercialization of four innovative and patented immune­gene therapy tools and systems developed by Dr. Lung­Ji Chang, Professor of Molecular Genetics and Microbiology at University of Florida’s Research Center. [Radient Pharmaceuticals Corporation Press Release]

Wake Forest Institute and Virginia Tech form Center for Veterinary Regenerative Medicine
The Institute for Regenerative Medicine at Wake Forest University Baptist Medical Center has joined with the Virginia-Maryland Regional College of Veterinary Medicine to form the Virginia Tech/Wake Forest Center for Veterinary Regenerative Medicine. Researchers from both organizations will work collaboratively to develop new regenerative medicine treatments for animals and human patients. [Wake Forest University Baptist Medical Center Press Release]

NYU Langone Medical Center Joins Pfizer’s Centers for Therapeutic Innovation (CTI)
NYU Langone Medical Center announced a new partnership with Pfizer, Inc. to accelerate the translation of biomedical research into effective new medications and therapies for patients. [NYU Langone Medical Center Press Release]

AMT Receives EUR1.1 Million Funding for Acute Intermittent Porphyria Gene Therapy as Part of EU Consortium Grant
Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced that the European Union (EU) has finalized a EUR3.3 million grant to the AIPGENE consortium, of which AMT is a member, for the development of a gene therapy product for Acute Intermittent Porphyria. [Amsterdam Molecular Therapeutics Press Release]

CIRM Approves Funding $32 Million to Remove Hurdles to New Stem Cell Therapies
The Governing Board of the California Institute for Regenerative Medicine (CIRM), the state stem cell agency created by proposition 71, approved funding $32 million to 19 research projects intending to create new tools and technologies that overcome barriers to moving stem cell research into clinical trials. [California Institute for Regenerative Medicine Press Release]

CIRM Generating 25,000 Jobs and $200 Million in Taxes for California with Grants Awarded So Far — With More to Come
The California Institute for Regenerative Medicine (CIRM), the state stem cell agency created by voters with Proposition 71, released an independent economic impact study that shows CIRM has already delivered significant financial benefit for the state with its first $1.1 billion in grants creating 25,000 job years and $200 million in new tax revenue through 2014. [California Institute for Regenerative Medicine Press Release]

Cancer Research Institute Announces Second Investment from Its Cancer Vaccine Acceleration Fund
Cancer Research Institute, Inc. announced that its Cancer Vaccine Acceleration Fund, a joint initiative with the Ludwig Institute for Cancer Research, has completed a new investment agreement with Oncovir, Inc., a biotechnology company based in Washington, D.C., to enable the production of the immunological stimulant Hiltonol® (Poly-ICLC). [Cancer Research Institute, Inc. Press Release]

UCSF Receives Second Major Gift from Dolbys for Stem Cell Building
Ray and Dagmar Dolby donated $20 million to the University of California, San Francisco (UCSF) to provide funding for a stem cell building on the Parnassus Campus. [University of California, San Francisco Press Release]

H3 Biomedicine Launches with $200 Million from Eisai
Eisai Inc. launched a wholly owned research and development subsidiary in Cambridge called H3 Biomedicine Inc. that will focus on personalized cancer medicines based on cancer genetics and chemistry advances. [Mass High Tech]

Athersys Announces Registered Direct Offering of $13.1 Million
Athersys, Inc. announced that it has entered into definitive agreements with certain investors to sell 4,366,667 shares of its common stock and warrants to purchase up to an additional 1,310,000 shares of common stock in a registered direct offering for gross proceeds of approximately $13.1 million, before deducting fees and offering expenses. [Athersys, Inc. Press Release]

Pluristem Announces Pricing of Public Offering of Common Stock and Warrants to Purchase Common Stock
Pluristem Therapeutics Inc. announced it has priced a firm commitment underwritten public offering of 11,000,000 units, with each unit consisting of one share of the company's common stock and one warrant to purchase 0.4 of a share of common stock, at a purchase price of $3.25 per unit for an aggregate gross offering amount of $35.8 million. [Pluristem Therapeutics Inc. Press Release]

Life Sciences Center Backs UMass Boston Cancer Center with $2 Million
The Massachusetts Life Sciences Center today awarded a $2 million grant to help launch a joint Center for Personalized Cancer Therapy on the University of Massachusetts (UMass) Boston campus. [Mass High Tech]

Arteriocyte Receives FDA Approval to Move Forward with Critical Limb Ischemia Tria
Arteriocyte®, a leading clinical stage biotechnology company with offices in Cleveland, Ohio and Hopkinton, Massachusetts that develops proprietary stem cell and tissue engineering based therapies, announced approval from the Food and Drug Administration (FDA) to initiate a Phase I clinical trial using its Magellan MAR01™ technology in the treatment of Critical Limb Ischemia. [Arteriocyte, Inc. Press Release]

BioSante Announces FDA Orphan Drug Designation for its Melanoma Cancer Vaccine
BioSante Pharmaceuticals, Inc. announced the receipt of Orphan Drug designation for its Melanoma Cancer Vaccine in the treatment of stage IIb to IV melanoma, from the FDA's Office of Orphan Products Development. [BioSante Pharmaceuticals Inc. Press Release]

Stem Cell Agency Governing Board Will Swear in Two New Board Members January 27 Along with Nine Reappointed Members
The Governing Board of the California Institute for Regenerative Medicine (CIRM), the state stem cell agency created by Proposition 71, swore in two new members and nine members reappointed to second six-year terms at its meeting in Burlingame January 27. [California Institute for Regenerative Medicine Press Release]

NYU Langone Medical Center Establishes The Rita J. and Stanley H. Kaplan Stem Cell/Bone Marrow Transplant Center
NYU Langone Medical Center announced the opening of The Rita J. and Stanley H. Kaplan Stem Cell/Bone Marrow Transplant Center (Kaplan Center), dedicated to providing stem cell transplants and comprehensive in-patient and out-patient care for adult and pediatric patients with hematologic cancers. [NYU Langone Medical Center Press Release]

Ecron Acunova Adds on Stem Cell Therapy and Diabetes Center to Conduct Studies for Global and Indian Sponsors
Ecron Acunova, a full-fledged clinical research organization, has added an additional focus area which is the stem cell therapy and diabetes centre. The center provides the marketing authorization for allogenic or autologous stem cell products. [PharmaBiz]


NIH Reorganization Draws Scrutiny from Congress
Biomedical scientists aren't alone in questioning a plan by National Institutes of Health (NIH) Director Francis Collins to create a new center to spur drug development. Last week, a staffer for a key congressional committee asked NIH and the Department of Health and Human Services for a long list of details about the reorganization, which also entails dissolving an existing center.

Office of Biotechnology Activities, Office of Science Policy, Office of the Director (FR Doc. 2011-2022)

National Human Genome Research Institute; Notice of Closed Meeting (FR Doc. 2011-2024)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-2026)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-1681)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-1834)

Center for Scientific Review; Notice of Closed Meeting (FR Doc. 2011-1832)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-1874)

National Institute of Biomedical Imaging and Bioengineering; Amended Notice of Meeting (FR Doc. 2011-1875)

Stem Cells 645x110B


OCTGT Learn 
OCTGT Learn, the Center for Biologics, Evaluation and Research's Office of Cellular, Tissue and Gene Therapies (OCTGT) is a web page for industry education.

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.

Draft Guidance for Industry: Pre-Storage Leukocyte Reduction of Whole Blood and Blood Components Intended for Transfusion

List of Establishments Granted Approval for a Variance to 21CFR640.3(d) and 21CFR640.3(f)




Fourth FDA Orphan Drug Designation Workshop Scheduled for February 28-March 1, 2011
The U.S. Food and Drug Administration has scheduled its fourth orphan drug designation workshop for academics, biotechnology companies, and those unfamiliar with the process for Feb. 28 – March 1, 2011, in Claremont, Calif. in collaboration with Keck Graduate Institute.

Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; Testing Communications on Biological Products [Docket No. FDA-2010-N-0464]

Draft Guidance for Industry: Pre-Storage Leukocyte Reduction of Whole Blood and Blood Components Intended for Transfusion; Availability [Docket No. FDA-2001-D-0254; formerly Docket No. 2001D-0037]


European Medicines Agency Publishes Final ‘Road Map to 2015’
The European Medicines Agency has published its final 'Road map to 2015', coinciding with the 16th anniversary of its inauguration on 26 January 2011.


NEW Biointerface Symposium 2011: Proteins and Cells at the Biointerface
February 7-8, 2011
Seattle, United States

NEW Fourth FDA Orphan Drug Designation Workshop
February 28-March 1, 2011
Claremont, United States

NEW IBC's 3rd Annual Asia Tides 2011
March 2-4, 2011
Tokyo, Japan

NEW Prostate of the Art: 10th Annual MBiotech Symposium
March 3, 2011
Toronto, Canada

NEW 20th Partnerships in Clinical Trials
March 30-April 1, 2011
Phoenix, United States

NEW Biotech Clinical Forum West: Clinical Development Strategies for Biotech
March 31, 2011
Phoenix, United States

NEW 3rd Meeting of the Forum of Italian Researchers on Mesenchymal and Stromal Stem Cells (FIRST)
April 18, 2011
Milan, Italy

NEW 2011 PDA Europe Workshop on Advanced Therapy Medicinal Products
June 7-8, 2011
Helsinki, Finland

NEW DNA Vaccines 2011
July 12-14, 2011
San Diego, United States

NEW 17th Annual Meeting of the Japan Society of Gene Therapy (JSGT)
July 15-17, 2011
Fukuoka, Japan

NEW Inaugural BioProcessing, Biologics & Biotherapeutics Congress
July 20-21, 2011
Edinburgh, Scotland

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)

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

Cell Therapy Manufacturing Manager (Opexa Therapeutics)

Post-Doctoral Positions (University of Texas MD Anderson Cancer Center)

Faculty Position Openings (Shanghai Advanced Research Institute, Chinese Academy of Sciences)
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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