Volume 12.14 | Apr 11

Cell Therapy News 12.14, April 11, 2011
     In this issue: Science | Policy | Business | NIH | CBER | Regulatory | Events

The Self-Made Eye: Formation of Optic Cup from ES Cells
A breakthrough new report describes how mouse embryonic stem (ES) cells are able to differentiate and assemble into an optic cup, capable of giving rise to a tissue exhibiting the stratified structure characteristic of the retina in vivo. [Press release from the RIKEN Center for Developmental Biology discussing online prepublication in Nature]



Helping the Heart Help Itself
Some stem cells in the bone marrow, called c-kit+ cells, appear capable of stimulating adult stem cells already present in the heart to repair the damaged tissue. [Press release from Harvard University discussing online prepublication in Cell Stem Cell]

A New Way to Make Reprogrammed Stem Cells
Researchers have devised a totally new and far more efficient way of generating induced pluripotent stem cells. The study eliminates the use of transcription factors and increases efficiency 100-fold. [Press release from the University of Pennsylvania discussing online prepublication in Cell Stem Cell]

Scientists Identify Key Trigger for Pancreatic Beta Cell Regeneration
Researchers have identified a key signal that prompts existing insulin-producing beta cells in the pancreas to form new beta cells in mice, a breakthrough discovery that may ultimately help researchers find ways to restore or increase beta cell function in people with type 1 diabetes. [Press release from the Juvenile Diabetes Research Foundation International discussing online prepublication in Cell Metabolism]

Study Shows Patient’s Own Cells May Hold Therapeutic Promise After Reprogramming, Gene Correction
Scientists moved gene therapy one step closer to clinical reality by determining that the process of correcting a genetic defect does not substantially increase the number of potentially cancer-causing mutations in induced pluripotent stem cells. [Press release from the University of Wisconsin-Madison discussing online prepublication in the Proceedings of the National Academy of Sciences USA]

Vision Loss in Eye Disease Slowed Using Novel Encapsulated Cell Therapy
A Phase II clinical trial for the treatment of a severe form of age-related macular degeneration called geographic atrophy has become the first study to show the benefit of a therapy to slow the progression of vision loss for this disease. [Press release from University of California, San Diego discussing online prepublication in the Proceedings of the National Academy of Sciences USA]

Scientists Make Skin Repair Discovery
Researchers have identified specific bone marrow cells that can transform into skin cells to repair damaged skin tissue. [Press release from King’s College London discussing online prepublication in the Proceedings of the National Academy of Sciences USA]

Researchers Mimic Body’s Own Healing Potential to Create Personalized Therapies for Inflammation
Scientists at Barts and The London School of Medicine and Dentistry and Harvard Medical School, Boston have found a way of mimicking the body’s natural mechanism of fighting inflammation. [Press release from EurekAlert! discussing online prepublication in the Journal of Immunology]

‘Good Cholesterol’ Nanoparticles Seek and Destroy Cancer Cells
Synthetic HDL nanoparticles loaded with small interfering RNA to silence cancer-promoting genes selectively shrunk or destroyed ovarian cancer tumors in mice. [Press release from the University of Texas M. D. Anderson Cancer Center discussing online prepublication in Neoplasia]

GenVec Technology Used to Restore Lost Balance Function
The article describes research in which the use of a vector targeted to and limited in expression to supporting cells can be used to restore balance function in an animal model. [Press release from GenVec, Inc. discussing online prepublication in Gene Therapy]

‘Universal’ Virus-Free Method Turns Blood Cells to Beating Heart Cells
Scientists have developed a simplified, cheaper, all-purpose method they say can be used by scientists around the globe to more safely turn blood cells into heart cells. [Press release from The Johns Hopkins University discussing online prepublication in PLoS ONE]

Progress Toward the Clinical Application of Autologous Induced Pluripotent Stem Cells and Gene Repair Therapy for Treatment of Familial Hypercholesterolemia
A study shows, for the first time, the successful reprogramming of diseased human hepatocytes into induced pluripotent stem cells. [Press release from EurekAlert! discussing research resented at the 46th Annual Meeting of the European Association for the Study of the Liver]

Pervasis Therapeutics Announces Positive Preclinical Study Data of Novel Cell Therapy for Treatment of Solid Tumors
Pervasis Therapeutics, Inc. announced preclinical data showing positive effects of PVS-30200, the company’s investigational cell therapy for the treatment of solid tumors, on controlling tumor growth and inhibiting metastases in an animal model of prostate cancer. [Press release from Pervasis Therapeutics, Inc. discussing research presented at the American Association for Cancer Research 102nd Annual Meeting 2011]

GOLFIG Increased Progression-Free Survival in Colorectal Cancer Patients
Oncologists can use colorectal cancer patients’ own immune system to boost the effects of chemotherapy and increase progression-free survival, according to Phase III study results. [Press release from the American Association for Cancer Research (AACR) discussing research presented at the AACR 102nd Annual Meeting 2011]

VitaKine® Platelet Cell Therapy from BioParadox Improves Cardiac Function after Heart Attack in Preclinical Studies
BioParadox, Inc. announced data supporting the use of VitaKine® Platelet Cell Therapy as a promising treatment for heart attack patients. [Press release from PR Newswire discussing research presented at the 2nd Annual Translational Regenerative Medicine Forum]

Tolerx Presents Preclinical Data on Novel Anti-GITR Cancer Immunotherapeutic Demonstrating Enhanced Anti-Tumor Responses
Tolerx, Inc. presented results from preclinical studies with 2F8, a murine analog of TRX518, a first-in-class immunomodulatory agent currently in a Phase I clinical study for the treatment of cancer. [Press release from Tolerx, Inc. discussing research presented at Keystone Symposia: Immunoregulatory Networks]

Scientists in Donor Organ Breakthrough
Scottish scientists have succeeded in growing kidneys in a laboratory in a breakthrough that could help tackle the tragic shortage of organs for transplant. [Scotland on Sunday]

(Ranked by Impact Factor of the Journal)

Self-Organizing Optic-Cup Morphogenesis in Three-Dimensional Culture
Researchers report the dynamic, autonomous formation of the optic cup (retinal primordium) structure from a three-dimensional culture of mouse embryonic stem cell aggregates. [Nature]

Bone Marrow-Derived Cell Therapy Stimulates Endogenous Cardiomyocyte Progenitors and Promotes Cardiac Repair
Findings suggest that stimulation of endogenous cardiogenic progenitor activity is a critical mechanism of cardiac cell therapy. [Cell Stem Cell]

Highly Efficient miRNA-Mediated Reprogramming of Mouse and Human Somatic Cells to Pluripotency
Data show that miRNA and Hdac-mediated pathways can cooperate in a powerful way to reprogram somatic cells to pluripotency. [Cell Stem Cell]

Control of Pancreatic β Cell Regeneration by Glucose Metabolism
Using islet transplantation experiments, researchers show that β cell mass is controlled systemically rather than by local factors such as tissue damage. [Cell Metab]

Genetic Correction and Analysis of Induced Pluripotent Stem Cells from a Patient with Gyrate Atrophy
Findings confirm that induced pluripotent stem cells themselves may carry a significant mutational load at initial isolation, but that the clonal events and prolonged cultured required for correction of a genetic defect can be accomplished without a substantial increase in mutational burden. [Proc Natl Acad Sci U S A]

Ciliary Neurotrophic Factor Delivered by Encapsulated Cell Intraocular Implants for Treatment of Geographic Atrophy in Age-Related Macular Degeneration
Findings suggest that ciliary neurotrophic factor delivered by the encapsulated cell technology implant appears to slow the progression of vision loss in geographic atrophy, especially in eyes with 20/63 or better vision at baseline. [Proc Natl Acad Sci U S A]

PDGFRα-Positive Cells in Bone Marrow Are Mobilized by High Mobility Group Box 1 (HMGB1) to Regenerate Injured Epithelia
Data provide unique insight into how skin grafts facilitate tissue repair and identify strategies germane to regenerative medicine for skin and, perhaps, other ectodermal defects or diseases. [Proc Natl Acad Sci U S A]

Cutting Edge: Humanized Nano-Proresolving Medicines Mimic Inflammation-Resolution and Enhance Wound Healing
Findings indicate that humanized nanoparticles, termed nano-proresolving medicines, are mimetics of endogenous resolving mechanisms, possess potent beneficial bioactions, can reduce nanotoxicity, and offer new therapeutic approaches. [J Immunol]

Selective Atonal Gene Delivery Improves Balance Function in a Mouse Model of Vestibular Disease
Delivery of Ad28 adenovector to 3,3′-iminodipropionitrile-damaged vestibular organs resulted in a significant recovery of vestibular hair cells and restoration of balance, as measured by time on rotarod compared with untreated controls. [Gene Therapy]



French Senate Backs Embryonic Stem Cell and Embryo Research
The French Senate voted to explicitly allow research on embryonic stem cells and embryos, amending part of a bill revising the country’s bioethics law. [Senate, France]

U.S. Court Puts Gene Patents Under a ‘Magic Microscope’
The Court of Appeals for the Federal Circuit in Washington, D.C., heard oral arguments in a case that could rewrite or invalidate much of biotechnology patent law in the United States. [Court of Appeals, United States]

Advisory Council on Blood Stem Cell Transplantation; Notice of Meeting (FR Doc. 2011-8146) [Health Resources and Services Administration, United States]



Cellular Dynamics Raises $30 Million to Expand Its Product Line of Human Stem Cells and Tissue Cells
Cellular Dynamics International, Inc., the world’s only industrial manufacturer of human induced pluripotent stem cells and tissue cells, has closed on a $30 million private equity round. [Cellular Dynamics International, Inc. Press Release]

Fred Hutchinson Cancer Research Center to Lead Immunotherapy Clinical Trials Network
Research institutions at 27 sites in the U.S. and Canada have been selected to participate in the Cancer Immunotherapy Trials Network, a new initiative in immunotherapy funded by the National Cancer Institute and headquartered at Fred Hutchinson Cancer Research Center. [Fred Hutchinson Cancer Research Center Press Release]

MRC-CRM and iCeMS Sign Agreement on Academic Collaboration
The University of Edinburgh’s Medical Research Council Centre for Regenerative Medicine (MRC-CRM) and Kyoto University Institute for Integrated Cell-Material Sciences (iCeMS) signed a memorandum of understanding covering future collaborative research. [MRC Centre for Regenerative Medicine Press Release]

SuperGen and Astex Therapeutics Enter Definitive Merger Agreement
SuperGen, Inc. and Astex Therapeutics Limited jointly announced that they have entered into a definitive agreement to merge the two companies, subject to customary closing conditions, including regulatory and shareholder approvals. [SuperGen, Inc. Press Release]

Sanofi-Aventis Completes Acquisition of Genzyme Corporation
Sanofi-aventis announced that it has completed its acquisition of Genzyme Corporation, which is now a wholly-owned subsidiary of sanofi-aventis. [Sanofi-aventis Press Release]

Mesoblast Update on Valeant Proposal to Cephalon 
Mesoblast wishes to inform the market that the Board of its strategic partner Cephalon Inc. has rejected the unsolicited proposal from Valeant Pharmaceuticals International Inc. to acquire its issued share capital or certain assets. [Mesoblast Limited Press Release]

American Cancer Society Awards New Research and Training Grants
The American Cancer Society has awarded 132 national research and training grants totaling $51,473,000 to 85 institutions nationwide in the second of two grants cycles for 2011. [American Cancer Society Press Release]

UCSD Scientists Receive Prestigious Hartwell Biomedical Research Awards
Three scientists at the University of California, San Diego (UCSD) School of Medicine are among this year’s 12 winners of Hartwell Individual Biomedical Research Awards, honoring researchers whose work advances children’s health. [University of California, San Diego Press Release]

Drug Developer Aldagen Withdraws IPO For Second Time
Aldagen Inc. has withdrawn its proposed initial public offering after 18 months in registration, a period in which the drug developer suffered a series of setbacks in the clinic and shuffled its senior management multiple times. [The Wall Street Journal]

Blueprint Medicines Secures $40 Million Series A Financing to Translate New Molecular Data into Personalized Cancer Treatments
Blueprint Medicines, a company harnessing the understanding of the molecular blueprint of cancer to develop personalized, highly-selective cancer therapies, announced the closing of a $40 million Series A financing led by Third Rock Ventures. [BusinessWire]

Cardio3 BioSciences Announces International C-Cure® Phase II Clinical Trial at the 60th Annual American College of Cardiology Meeting
The Belgian biotechnology company, Cardio3 BioSciences presented detailed data from the Phase II clinical trial of C3BS-CQR-1 (C-Cure®), its novel stem cell therapy for ischemic cardiomyopathy, at the 60th annual American College of Cardiology in New Orleans, USA. [Cardio3 BioSciences Press Release]

CEL-SCI Corporation Receives Government Approval in India to Commence Phase III Clinical Trial of Multikine in Head and Neck Cancer
CEL-SCI Corporation announced that it has received approval to begin enrollment of patients in its Phase III clinical trial of Multikine® in India from the Directorate General of Health Services Office of Drug Controller General (India). [CEL-SCI Corporation Press Release]

Proteonomix, Inc. Files New Patent Application for a New Technology to Focus on Mobilization of Bone Marrow Stem Cells and Begins to Prepare for a Clinical Trial Based on the Patent Application
Proteonomix, Inc. announced that it has filed a provisional patent application in anticipation of commencement of its initial clinical trial of a drug combination thought to extend life expectancy for a class of terminally ill patients awaiting liver transplants. [BusinessWire]

Parkinson’s Patients Needed for Trial of Possible Gene Therapy
Researchers at the Stanford University School of Medicine are recruiting people who have Parkinson’s disease to participate in a clinical trial of a potential gene therapy. [Stanford University School of Medicine Press Release]

HemaCare Names Dr. Scott Burger Scientific Advisory Board Chair
HemaCare Corporation named Scott R. Burger, MD as the Chairperson of its Scientific Advisory Board. HemaCare has also engaged Dr. Burger, a world renowned cell therapy expert, in a comprehensive consulting services agreement. [BusinessWire]

Walk Away With A RoboSep™ Facebook Photo Contest Winners
STEMCELL Technologies has announced the Grand Prize Winner and Finalists of the “Walk Away With A RoboSep™” Facebook photo contest. [STEMCELL Technologies, Inc. Press Release]

First Patient to Get Stem Cell Therapy Comes Forward
In the six months since scientists announced they had infused a drug made from human embryonic stem cells into a partially paralyzed patient’s spine, the identity of the recipient has been shrouded in secrecy. Now, a 21-year-old Alabama nursing student who was paralyzed from the chest down in a car crash in September has come forward to identify himself as the volunteer. [The Washington Post]

Announcing the First Stem Cell Translational Journal
AlphaMed Press announces the launch of a major peer-reviewed journal, STEM CELLS Translational Medicine, answering an acknowledged need within the research and clinical communities for comprehensive coverage of stem cell science, stem cell-based regenerative medicine and tissue engineering, stem cell-based predictive toxicology, and cancer stem cell investigation. [AlphaMed Press Press Release]


Budget Freeze Forces NIH to Slash Electronic Grants Staff
In a sign of grim budget times, the National Institutes of Health (NIH) is cutting 20% of the contractors who help run its electronic grants system.

NIH Launches Training Institute on Dissemination and Implementation Research
Although experts know the best evidence-based strategies to treat many conditions, health care providers do not always practice those strategies. How to conduct research to close this gap (called dissemination and implementation health research) will be the subject of a five-day training session.

Pilot Studies to Develop and Test Novel, Low- Cost Methods for the Conduct of Clinical Trials (R01) (RFA-HL-12-019)

National Human Genome Research Institute; Notice of Closed Meeting (FR Doc. 2011-8437)

National Cancer Institute; Notice of Closed Meetings (FR Doc. 2011-8165)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-8156)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-8162)

Center for Scientific Review; Notice of Meeting (FR Doc. 2011-8315)

Center for Scientific Review; Notice of Closed Meeting (FR Doc. 2011-8442)



May 31, 2011: Cellular, Tissue and Gene Therapies Advisory Committee Meeting Announcement

April 28-29, 2011: Blood Products Advisory Committee Meeting Waivers for Conflicts of Interest

April 1, 2011 Approval Letter-Menevo



Request for Notification From Industry Organizations Interested in Participating in the Selection Process for a Nonvoting Industry Representative on the Allergenic Products Advisory Committee and Request for Nominations for a Nonvoting Industry Representative on the Allergenic Products Advisory Committee [FR Doc. 2011-8125]


EMA/COMP/430447/2010: Recommendation for Maintenance of Orphan Designation at the Time of Marketing Authorisation: Tepadina (Thiotepa) for the Conditioning Treatment Prior to Haematopoietic Progenitor Cell Transplantation, Adopted

EU/3/10/774: Public Summary of Opinion on Orphan Designation: Allogeneic Human Dermal Fibroblasts for the Treatment of Epidermolysis Bullosa


NEW Genetic Stability, Effects of Genetic Background, and Nomenclature
April 21, 2011

NEW 2nd Else Kröner-Fresenius Symposium on Molecular Mechanisms of  Adult Stem Cell Aging
May 20-22, 2011
Günzburg, Germany

NEW 2011 American Society of Clinical Oncology Annual Meeting
June 3-7, 2011
Chicago, United States

NEW 2011 American Society for Histocompatibility & Immunogenetics (ASHI) – Regional Education Workshop 2
June 16-18, 2011
Seattle, United States

NEW National Cancer Institute – Cancer Research Imaging Camp
June 19-24, 2011
St. Louis, United States

NEW The American Society of Clinical Oncology and the American Association for Cancer Research: Methods in Clinical Cancer Research
July 30-August 4, 2011
Vail, United States

NEW Practical Course on Gene Expression Regulation and Data Integration 2011
August 27-September 4, 2011
Debrecen, Hungary

NEW Royan International Twin Congress on Reproductive Biomedicine and Stem Cells Biology & Technology
September 7-9, 2011
Tehran, Israel

NEW Stem Cells Update 2011 – From Basic Research to the Clinic
October 5-6, 2011
Uppsala, Sweden

NEW American Association for Cancer Research: Metabolism and Cancer
October 16-19, 2011
Baltimore, United States

NEW 10th Annual American Association for Cancer Research Conference on Frontiers in Cancer Prevention Research
October 22-25, 2011
Boston, United States

NEW BioProcess International Conference & Exhibition
October 31-November 4, 2011
Long Beach, United States

NEW AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics: Discovery, Biology, and Clinical Applications
November 12-16, 2011
San Francisco, United States

NEW Up Close and Personalized, International Congress on Personalized Medicine (UPCP)
February 2-5, 2012
Florence, Italy

NEW The 2012 London Regenerative Medicine Event
February 16, 2012
London, United Kingdom

NEW AAAS Annual Meeting
February 16-20, 2012
Vancouver, Canada

our events page to see a complete list of events in the cell, gene and immunotherapy community.


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

Implementation Coordinator – Maternity Leave Contract (STEMSOFT Software Inc.)

Tenure Track, Assistant/Associate Professor (University of Missouri-Kansas City School of Medicine)

Quality Assurance Manager – Program in Children’s Regenerative Medicine (The University of Texas Medical School at Houston)

Postdoc – Laboratory of Molecular Tumor Genetics (Duke-NUS Graduate Medical School)

Postdoctoral Research Position – Mucosal Immunology (Department of Microbiology and Immunology, Columbia University)

Postdoctoral Position – Vascular Smooth Muscle Biology (Boston University Medical School)

Supervisor Cell Therapy Laboratory – Cord Blood Bank (The University of Texas MD Anderson Cancer Center)

Supervisor Cell Therapy Lab (The University of Texas MD Anderson Cancer Center)

Postdoctoral Position (Johns Hopkins University School of Medicine)

Manager Clinical Affairs (Bioheart, Inc.)

Quality Assurance Specialist – Gene Transfer & Somatic Cell Engineering Facility (Memorial Sloan-Kettering Cancer Center)

Scientific Director (Adipose Derived Stem Cell Bank)

Process Technical Development Scientist (StemCells, Inc.) 

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

Postdoctoral Fellow in Gene Expression/Cancer and Stem Cell Biology (University of Cincinnati College of Medicine)

Postdoctoral Positions in Immune Cell Activation (Institut de recherches cliniques de Montréal)

Postdoctoral Position in Developmental Neurobiology (Institut de recherches cliniques de Montréal)

Postdoctoral Research Fellows – Transcriptional Control in Hematopoietic Stem Cells and Leukemia (Institut de recherches cliniques de Montréal)

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