Volume 12.15 | Apr 18

Cell Therapy News 12.15, April 18, 2011
     In this issue: Science | Policy | Business | NIH | CBER | Regulatory | Events
Cell Therapy News on Facebook  Cell Therapy News on Twitter

Scientists Recreate Brain Cells from Skin Cells to Study Schizophrenia
A team of scientists has developed a method for recreating a schizophrenic patient’s own brain cells, which then can be studied safely and effectively in a Petri dish. [Press release from The Pennsylvania State University discussing online prepublication in Nature]



Study Suggests Enzyme Crucial to DNA Replication May Provide Potent Anti-Cancer Drug Target
An enzyme essential for DNA replication and repair in humans works in a way that might be exploited as anti-cancer therapy. [Press release from Scripps Research Institute discussing online prepublication in Cell]

Successful Strategy Developed to Regenerate Blood Vessels
Researchers have discovered a strategy for stimulating the formation of highly functional new blood vessels in tissues that are starved of oxygen. [Press release from The University of Western Ontario discussing online prepublication in Nature Biotechnology]

New Therapy Improves Immune Response in Teen with Rare Disease
In a novel approach that works around the gene defect in Wiskott-Aldrich syndrome, an inherited immune deficiency disorder, researchers used an alternative cell signaling pathway to significantly improve immune function in a 13-year-old boy with the disease. [Press release from The Children’s Hospital of Philadelphia discussing online prepublication in the Journal of Clinical Investigation]

NIH Scientists Identify Gene That Could Hold the Key to Muscle Repair
Researchers have long questioned why patients with Duchenne muscular dystrophy tend to manage well through childhood and adolescence, yet succumb to their disease in early adulthood, or why elderly people who lose muscle strength following bed rest find it difficult or impossible to regain. Now, researchers are beginning to find answers in a specialized population of cells called satellite cells. [Press release from the National Institute of Arthritis and Musculoskeletal and Skin Diseases discussing online prepublication in Genes & Development]

Team Perfects Non-Lethal Way of Switching Off Essential Genes in Mice
Researchers have overcome the lethal problem of switching off essential genes by using RNA interference technology to temporarily turn off any essential gene in adult mice and then turn it back on before the change kills the animals. [Press release from Cold Spring Harbor Laboratory discussing online prepublication in the Proceedings of the National Academy of Sciences]

First Clinical Trial of Gene Therapy for Pain Reported by Neurologists
In the first clinical trial of gene therapy for treatment of intractable pain, researchers observed that the treatment appears to provide substantial pain relief. [Press release from the University of Michigan Health System discussing online prepublication in the Annals of Neurology]

Pig Stem Cell Transplants: The Key to Future Research into Retina Treatment
Scientists studying the role of stem cells in repairing damaged retina tissue have found that pigs represent an effective proxy species to research treatments for humans. [Press release from Wiley discussing online prepublication in Stem Cells]

Gene Find Could Aid Nerve Repair
Scientists have pinpointed a gene that controls how quickly a person’s nerves can regenerate after injury or disease. [Press release from The University of Edinburgh discussing online prepublication in Human Molecular Genetics]

Tiny Antibody Fragments Raised in Camels Find Drug Targets in Human Breast Cancer Cells
A new discovery promises to help physicians identify patients most likely to benefit from breast cancer drug therapies. [Press release from ScienceDaily discussing online prepublication in The FASEB Journal]

Adding Stem Cells to Common Bypass Surgery May Reduce Heart Failure
In a new research study under way, surgeons are adding a patient’s own stem cells to the heart during cardiac bypass surgery. The goal of this research study is to determine whether the stem cell infusion will generate new blood vessels and improve heart function more than what is seen through bypass surgery alone. [Methodist DeBakey Heart & Vascular Center Press Release]

(Ranked by Impact Factor of the Journal)

Modeling Schizophrenia Using Human Induced Pluripotent Stem Cells
To elucidate the cellular and molecular defects of schizophrenia (SCZD), researchers directly reprogrammed fibroblasts from SCZD patients into human induced pluripotent stem cells (hiPSCs) and subsequently differentiated these disorder-specific hiPSCs into neurons. [Nature]

Human Flap Endonuclease Structures, DNA Double-Base Flipping, and a Unified Understanding of the FEN1 Superfamily
Here, structural and functional analyses of human Flap endonuclease (FEN1):DNA complexes show structure-specific, sequence-independent recognition for nicked dsDNA bent 100° with unpaired 3′ and 5′ flaps. [Cell]

Fibroblast Growth Factor 9 Delivery During Angiogenesis Produces Durable, Vasoresponsive Microvessels Wrapped by Smooth Muscle Cells
Using microarray expression analysis, scientists discovered that fibroblast growth factor 9 was highly upregulated as human vascular smooth muscle cells assemble into layered cords. [Nat Biotechnol]

IL-2 Induces a WAVE2-Dependent Pathway for Actin Reorganization That Enables Wiskott-Aldrich Syndrom Protein-Independent Human NK Cell Function
Results demonstrate how overlapping cytoskeletal activities can utilize immunologically distinct pathways to achieve synonymous immune function. [J Clin Invest]

Polycomb EZH2 Controls Self-Renewal and Safeguards the Transcriptional Identity of Skeletal Muscle Stem Cells
Scientists report that EZH2, the enzymatic subunit of the polycomb-repressive complex 2, is expressed in both Pax7+/Myf5 stem cells and Pax7+/Myf5+ committed myogenic precursors and is required for homeostasis of the adult satellite cell pool. [Genes Dev]

Reversible Suppression of an Essential Gene in Adult Mice Using Transgenic RNA Interference 
Scientists demonstrate the feasibility of transgenic inducible RNAi for suppression of essential genes. [Proc Natl Acad Sci U S A]

Gene Therapy for Pain: Results of a Phase I Clinical Trial
Investigators conducted a multicenter, dose-escalation, Phase I clinical trial of NP2, a replication defective HSV-based vector expressing human preproenkephalin in subjects with intractable focal pain caused by cancer. [Ann Neurol]

Differentiation of Swine iPSC into Rod Photoreceptors and their Integration into the Retina
Scientists subjected swine induced pluripotent stem cell (iPSC) to a rod photoreceptor differentiation protocol consisting of floating culture as embryoid bodies followed by differentiation in adherent culture. [Stem Cells]

ApoE Isoform-Specific Regulation of Regeneration in the Peripheral Nervous System
Researchers have investigated isoform-specific effects of human Apolipoprotein E (apoE) on the mouse peripheral nervous system. [Human Mol Genet]

Preclinical Screening of Anti-HER2 Nanobodies for Molecular Imaging of Breast Cancer
Researchers describe the generation of a panel of 38 nanobodies, small human epidermal growth factor receptor 2 (HER2)-binding fragments that are derived from heavy-chain-only antibodies raised in an immunized dromedary. [FASEB J]



Bill Would Necessitate Stem Cell Reporting
Stem cell researchers at Michigan State University and other state universities might face new transparency requirements because of a provision attached to a higher education appropriations bill passed Wednesday. [State Congress, United States]

As Congress Slashes EPA Budget, Research Least Harmed
Science and technology at the Environmental Protection Agency (EPA) will be sliced 3.9%, to $815 million, under the new budget for FY 2011. [Congress, United States]

US Legislation Aims to Simplify Rules for Inventors
The US Congress is poised to enact the most sweeping overhaul of the country’s patent legislation for almost 30 years. [Congress, United States]

Global Treatment Evaluation for Parkinson’s Disease
The International Cellular Medicine Society, the global leader in providing best practice standards for cell based medicine, announced the initiation of its first step toward conducting worldwide evaluations of current stem cell treatment offerings. [International Cellular Medicine Society, United States]

Australian Medical Scientists Protest Hefty Budget Cuts
Chanting “cures not cuts,” some 7000 medical researchers took to the streets in five Australian cities this week to protest a steep cut—about 20% per year—to medical research spending anticipated in the federal government’s 2011 budget, set for release next month. [Federal Government, Australia]

NATIONAL SCIENCE FOUNDATION; Notice of Intent To Seek Approval To Continue an Information Collection (FR Doc. 2011-9032) [National Science Foundation, United States]



Cellular Dynamics Licenses Technology from ViaCyte
Cellular Dynamics International announced it has strengthened its intellectual property portfolio by licensing patented definitive endoderm differentiation technology from ViaCyte, Inc., a stem cell company focused on diabetes therapy. [Cellular Dynamics International Press Release]

King’s Set to Join Scientific Powerhouse
King’s College London and Imperial College London are set to join the partners behind the world-leading medical research institute: the UK Centre for Medical Research and Innovation. [King’s College London Press Release]

LCT and Otsuka of Japan Share Common DIABECELL® Vision
Living Cell Technologies (LCT) Limited announced that it has entered into an agreement to raise $3 million through a placement of shares to Otsuka Pharmaceutical Factory, Inc. [Living Cell Technologies Limited Press Release]

Takeda and Samyang Form Research Collaboration in Novel DDS Platform Technology for RNAi Therapeutics
Takeda Pharmaceutical Company Limited and Samyang Corporation announced that they have formed a research collaboration agreement for discovering a novel DDS platform technology for RNAi therapeutics. [Takeda Pharmaceutical Company Limited Press Release]

Access Pharmaceuticals Signs Agreement with Major Global Pharmaceutical Company for CobaCyte® and CobOral® RNAi Therapeutic Delivery Technologies
Access Pharmaceuticals, Inc. announced it has entered into an agreement with a major pharmaceutical company to exploit its CobaCyte and CobOral technology for the targeted delivery of RNAi therapeutics. [Access Pharmaceuticals, Inc. Press Release]

Quest PharmaTech Announces Clinical Development Agreement with Hemispherx Biopharma Inc. to Evaluate a Combinatorial Immunotherapy Technology
Quest PharmaTech Inc. announced a clinical development arrangement with U.S. based Hemispherx Biopharma Inc. to evaluate the clinical utility of combining Quest’s antibody immunotherapy technology with Hemispherx’s immune activator in a thirty patient clinical trial. [Quest PharmaTech Inc. Press Release]

NCKU and NRC Made Innovative Cancer Diagnostic and Treatment Discoveries
A Taiwanese research team led by Prof. Dar-Bin Shieh of Institute of Oral Medicine in Medical College at National Cheng Kung University (NCKU) has partnered with a research group led by Dr. Dennis Whitefield from National Research Council (NRC) of Canada to develop innovative cancer diagnostic and treatment technologies. [BusinessWire]

Regulus Therapeutics and UC San Diego to Collaborate on Angiogenic Disease Research Utilizing microRNA Technology
Regulus Therapeutics Inc. announced it is collaborating with researchers at the University of California (UC), San Diego School of Medicine seeking novel treatments for angiogenic diseases using microRNA therapeutics. [Regulus Therapeutics Inc. Press Release]

Sanofi-aventis Enters into Collaboration Agreement with Stanford University Bio-X to Focus on Early-Stage Scientific Research
Sanofi-aventis announced that it has entered into a multi-year research collaboration agreement through the Stanford University Bio-X program that supports, organizes, and facilitates interdisciplinary, collaborative and innovative research projects in the early phases of development. [PR Newswire]

RXi Pharmaceuticals Completes Apthera Acquisition
RXi Pharmaceuticals Corporation announced the completion of its previously announced acquisition of Apthera, Inc. [RXi Pharmaceuticals Corporation Press Release]

New Crop of Taubman Scholars Named; U-M Clinician-Scientists Receive Unrestricted Funding to Work Toward Cures
The second round of Taubman Scholars has been announced at the University of Michigan’s (U-M) A. Alfred Taubman Medical Research Institute. [University of Michigan Health Systems Press Release]

Nature Technology Corporation / Johns Hopkins University Receive $920,000 National Institutes of Health Grant for Cutaneous Gene Therapy Development
Nature Technology Corporation announced the award of a Small Business Innovation Research Phase II grant award from the National Institute of General Medical Sciences. [PRWeb]

Merrimack Pharmaceuticals Raises $77 Million in Private Financing
Merrimack Pharmaceuticals, Inc. announced that it has raised $77 million in a Series G private financing with participation by new and existing investors. [Merrimack Pharmaceuticals, Inc. Press Release]

Tekmira Announces USPTO Issuance of Key Patents Covering LNP Manufacturing Process and Mitigation of siRNA Immune Stimulation
Tekmira Pharmaceuticals Corporation announced that the United States Patent & Trademark Office (USPTO) has recently issued two key patents: one covering its lipid nanoparticle (LNP) manufacturing process and the other covering the chemical modification of siRNA to mitigate immune stimulation. [Tekmira Pharmaceuticals Corporation Press Release]

Stem Cell Assurance Announces Hiring of Vice President of Research and Development
Stem Cell Assurance, Inc. has announced that Francisco J. Silva has been hired as its Vice President of Research and Development. [PR Newswire]

Organogenesis, Life Sciences Center Break Ground on World’s Largest Automated Living Cell Manufacturing Plant
Massachusetts Life Sciences Center President & CEO Susan Windham-Bannister joined state and local officials, Organogenesis, Inc. President & CEO Geoff MacKay and employees to celebrate the start of construction of 275 Dan Road. Upon completion in 2013, the company expects the facility to be the world’s largest automated living cell manufacturing plant. [Organogenesis, Inc. Press Release]

UC San Diego Selected as Study Site for National Research Network
The University of California (UC), San Diego School of Medicine is among 27 research institutions selected across North America to be a part of the Cancer Immunotherapy Trials Network, funded by the National Cancer Institute. [University of California, San Diego Press Release]

Novartis, Pfizer Cancer Drugs Win U.S. Panel Vote
A U.S. advisory panel backed cancer drugs by Pfizer Inc and Novartis AG for treating a rare kind of pancreatic cancer that has few treatment options. [Reuters]


2011 Spending Deal Spares NIH Major Cuts
Just as White House officials promised, the 2011 funding bill agreed to by Congress and the White House last Friday spares biomedical research from major cuts.

Limited Competition for Continuation of Induced Pluripotent Stem Cell (iPSC) Resources Promoting Translational Research Advancements in Neurodegenerative Diseases (U24) (RFA-NS-11-011)

National Cancer Institute; Notice of Closed Meeting (FR Doc. 2011-8713)

National Cancer Institute; Notice of Closed Meeting (FR Doc. 2011-8928)

National Cancer Institute; Notice of Meeting (FR Doc. 2011-8929)
National Cancer Institute; Notice of Closed Meeting (FR Doc. 2011-9134)

Center for Scientific Review; Notice of Closed Meeting (FR Doc. 2011-8927)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-8605)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-8933)

Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-9132)

Center for Scientific Review; Notice of Closed Meeting (FR Doc. 2011-9133)

National Institute of Diabetes and Digestive and Kidney Diseases; Notice of Closed Meetings (FR Doc. 2011-8717)

National Institute of Neurological Disorders and Stroke; Notice of Closed Meeting (FR Doc. 2011-8988)

Public Meeting of the Presidential Commission for the Study of Bioethical Issues (FR Doc. 2011-9123)

Office of the Director, National Institutes of Health; Notice of Meeting (FR Doc. 2011-9131)

Office of the Director, National Institutes of Health; Notice of Meeting (FR Doc. 2011-9320)

Appeals of NIH Initial Peer Review (NOT-OD-11-064)

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



Translational Regenerative Medicine Forum Presentation

April 28-29, 2011: Blood Products Advisory Committee Meeting Document: Topic I: Testing Source Plasma for Hepatitis B Virus by Nucleic Acid Testing

April 28-29, 2011: Blood Products Advisory Committee Meeting Draft Agenda

April 28-29, 2011: Blood Products Advisory Committee Meeting Public Roster 

April 28-29, 2011: Blood Products Advisory Committee Meeting Document: Topic II: Current Considerations on Plasma for Further Manufacturing Obtained from Whole Blood Donors

April 28-29, 2011: Blood Products Advisory Committee Meeting Document: Topic III: Blood Donor Written Statement of Understanding 

May 12, 2011: Allergenic Products Advisory Committee Waivers for Conflicts of Interest

December 14, 2010: Cellular, Tissue and Gene Therapies Advisory Committee Meeting Minutes  




Agency Information Collection Activities; Proposed Collection; Comment Request; Guidance for Industry: Fast Track Drug Development Programs: Designation, Development, and Application Review [Docket No. FDA-2011-N-0157]

Agency Information Collection Activities; Proposals, Submissions, and Approvals: Application for Food and Drug Administration Approval to Market a New Drug [Docket No. FDA-2010-N-0627]

Agency Information Collection Activities; Proposed Collection; Comment Request; Guidance for Industry: Fast Track Drug Development Programs: Designation, Development, and Application Review [Docket No. FDA-2011-N-0157]

Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; Presubmission Conferences, New Animal Drug Applications and Supporting Regulations, and Food and Drug Administration Form 356V [Docket No. FDA-2011-N-0049]

Determination of Regulatory Review Period for Purposes of Patent Extension; ATRYN; Correction [Docket No. FDA-2009-E-0241]

Pediatric Ethics Subcommittee; Notice of Meeting [Docket No. FDA-2011-N-0184]


European Medicines Agency Issues Reminder on PIP Deferral Obligations

European Medicines Agency Launches Consultation on its Position on Replacement of Animal Studies


Vigilance and Risk Management of Medicines (VRMM) Division Realignment


NEW The World Congress on Industrial Biotechnology and Bioprocessing
May 8-11, 2011
Toronto, Canada

NEW ImVacS: The Immunotherapeutics & Vaccine Summit
August 16-18, 2011
Cambridge, United States

 The EMBO Meeting 2011
September 10-13, 2011
Vienna, Austria

 Cell & Gene Therapy Forum Asia 2011
September 19-21, 2011

 Vaccine Forum Singapore 2011
September 19-21, 2011

 Cell Symposia: Regulatory RNAs
October 10-12, 2011
Chicago, United States

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)

Production Manager – Cord Blood Bank (Murdoch Childrens Research Institute – BMDI Cord Blood Bank) 
Transplant Coordinator – Cord Blood Bank (Murdoch Childrens Research Institute – BMDI Cord Blood Bank)

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

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

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)

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)

Recruit Top Talent

Reach more than 11,000 potential candidates by posting your organization’s career opportunities on the Connexon Creative Job Board at no cost.

Jobs are categorized regionally:
United States, Canada, Europe, Asia Pacific

here to post your career opportunities.

Have we missed an important article or publication in Cell Therapy News? Click here to submit!

Comments or Suggestions? Email [email protected] with your feedback.

Learn more about Cell Therapy News: Archives | Events | Contact Us