Cell Therapy News Volume 12.08 | Feb 28 2011

May 12, 2022

TOP STORY

Newborn Heart Muscle Can Grow Back By Itself
In a promising science-fiction-meets-real-world juxtaposition, researchers have discovered that the mammalian newborn heart can heal itself completely. [Press release from the University of Texas Southwestern Medical Center discussing online prepublication in Science]

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SCIENCE NEWS

Scientists Discover Cause of Rare Skin Cancer That Heals Itself
Scientists have discovered the gene behind a rare skin cancer which grows rapidly for a few weeks before healing spontaneously. [Press release from the University of Dundee discussing online prepublication in Nature Genetics]

New Nanoparticles Could Lead to Powerful Vaccines for HIV and Other Diseases
Engineers have designed a new type of nanoparticle that could safely and effectively deliver vaccines for diseases such as HIV and malaria. [Press release from Massachusetts Institute of Technology discussing online prepublication in Nature Materials]

Hot Off the Presses, New Skin for Burn Victims
Designing organs on a computer and printing them out in a matter of minutes definitely beats growing them in vats. But is it possible? Scientists say the technology is closer then you might think, and could revolutionize transplantation and wound repair. [Press release from ScienceNOW discussing research presented at the Annual Meeting of the American Association for the Advancement of Science]

Scientists Tie Cell Cycle “Clock” to Childhood Cancers
Abnormal chromosomes have long been detected in children with leukemias and lymphomas, and now, research has linked such abnormalities with a molecular clock that controls the timing of a high-stakes genetic exchange inside dividing immune system cells. [Press release from Johns Hopkins University discussing online prepublication in Immunity]

Cancer-Related Pathways Reveal Potential Treatment Target for Congenital Heart Disease
Cross-disciplinary teams of scientists studying genetic pathways that are mutated in many forms of cancer, but which also cause certain forms of congenital heart disease — including hypertrophic cardiomyopathy (HCM) — have introduced these mutations into mice and successfully treated HCM in the lab. [Press release from EurekAlert! discussing online prepublication in the Journal of Clinical Investigation]

Study Identifies Genes Associated with Binge Drinking
Scientists found that manipulating two receptors in the brain, GABA receptors and toll-like receptor 4, “caused profound reduction” of binge drinking for two weeks in rodents that had been bred and trained to drink excessively. [Press release from the University of Maryland School of Medicine discussing online prepublication in the Proceedings of the National Academy of Sciences USA]

Personalized Medicine Comes Within Reach
A team of scientists has established a strategy for identifying biomarkers. If a particular pattern of these biomarkers can be detected in the blood, this indicates a cancerous disease. [Press release from Swiss Federal Institute of Technology Zurich discussing online prepublication in the Proceedings of the National Academy of Sciences USA]

Scientists Devise Artificial Intestine to Help Engineer Disease-Fighting Gut Bacteria
3D hydrogel scaffolds developed by researchers will allow scientists to grow cells under realistic physiological conditions. [Press release from Cornell University discussing online prepublication in Lab on a Chip]

Vaccine Made with Synthetic Gene Protects Against Deadly Pneumonia
Researchers have developed an experimental vaccine that appears to protect against an increasingly common and particularly deadly form of pneumococcal pneumonia. [Press release from Albert Einstein College of Medicine of Yeshiva University discussing online prepublication in the Journal of Infectious Diseases]

Human Umbilical Cord Blood Cells Aid Diabetic Wound Healing
Transplanting human umbilical cord blood-derived endothelial progenitor cells has been found to “significantly accelerate” wound closure in diabetic mouse models, said a team of Korean researchers. [Press release from EurekAlert! discussing online prepublication in Cell Transplantation]

Gene Fuelled Transporter Causes Breast Cancer Cells to Self-Destruct
Scientists have shown that they can deliver a gene directly into breast cancer cells causing them to self-destruct, using an innovative, miniscule gene transport system. [Press release from Queen’s University Belfast discussing online prepublication in the International Journal of Pharmaceutics]

Designing New Molecular Tools to Study the Life and Death of a Cancer Cell
Researchers have developed new synthetic molecules as models to study the structural and functional role of the proline residue and tetrapeptide sequence important for the regulation of cancer cell apoptosis by the XIAP protein. [Press release from ScienceDaily discussing online prepublication in Experimental Biology and Medicine]

Researchers First Worldwide to Generate Pluripotent Stem Cells from Horses
In a world first, pluripotent stem cells have been generated from horses. The findings will help enable new stem cell based regenerative therapies in veterinary medicine, and because horses’ muscle and tendon systems are similar to our own, aid the development of preclinical models leading to human applications. [Press release from the University of Montreal discussing online prepublication in Stem Cell Reviews and Reports]

BrainStorm’s NurOwn™ Stem Cell Technology Shows Promise for Treating Sciatic Nerve Injury
BrainStorm Cell Therapeutics Inc. announced that intramuscular transplantation of autologous, astrocyte-like cells that produce and secrete neurotrophic factors, representing the company’s NurOwn™ technology platform, preserved motor function, significantly inhibited the degeneration of the neuromuscular junctions, and preserved the myelinated motor axons in an animal sciatic nerve injury model. [Press release from BrainStorm Cell Therapeutics Inc. discussing online prepublication in Stem Cell Reviews and Reports]

Clinical Responses Demonstrated in Metastatic Castrate Resistant Prostate Cancer Patients Treated with BPX-101 Cancer Vaccine
Bellicum Pharmaceuticals, Inc. announced the presentation of Phase I/II safety and efficacy data for BPX-101, a novel drug-activated dendritic cell vaccine for metastatic castrate resistant prostate cancer. [Press release from Bellicum Pharmaceuticals, Inc. discussing research presented at the 2011 Genitourinary Cancers Symposium]

Sangamo BioSciences Announces Presentation of Positive Clinical Data from Novel ZFN Therapeutic Approach for HIV/AIDS Treatment
Sangamo BioSciences, Inc. announced the presentation of positive preliminary clinical data from its Phase I trial (SB-728-902). The study is designed to evaluate safety and clinical outcomes of Sangamo’s zinc finger nuclease (ZFN)-generated CCR5-modified, autologous T-cell product (SB-728-T) for the treatment of HIV/AIDS. [Press release from Sangamo BioSciences, Inc. discussing research presented at the Conference on Retroviruses and Opportunistic Infections]

UCLA and Sound Pharmaceuticals Identify a Key Hearing Regeneration Protein in the Human Inner Ear
In collaboration with scientists and clinicians from the University of California Los Angeles, scientists from Sound Pharmaceuticals have found p27Kip1 to be expressed in the adult and aged human inner ear including the auditory and vestibular sensory organs. These findings are identical to what has been observed and reported in neonatal and adult rodents, further validating p27Kip1 as a key regeneration target in the deafened mammalian inner ear. [Press release from PR Newswire discussing research presented at the 34th Annual Midwinter Meeting of the Association for Research in Otolaryngology]

CURRENT PUBLICATIONS (Ranked by Impact Factor of the Journal)

Multiple Self-Healing Squamous Epithelioma Is Caused by a Disease-Specific Spectrum of Mutations in TGFBR1
High-throughput genomic sequencing of a conservative estimate (24.2 Mb) of the disease locus on chromosome 9 using exon array capture identified independent mutations in TGFBR1 in three unrelated families. [Nat Genet]

Transient Regenerative Potential of the Neonatal Mouse Heart
Researchers found that the hearts of 1-day-old neonatal mice can regenerate after partial surgical resection, but this capacity is lost by 7 days of age. [Science]

Interbilayer-Crosslinked Multilamellar Vesicles as Synthetic Vaccines for Potent Humoral and Cellular Immune Responses
Researchers found that their antigen/adjuvant-carrying vesicles form an extremely potent whole-protein vaccine, eliciting endogenous T-cell and antibody responses comparable to those for the strongest vaccine vectors. [Nat Mater]

Coupling of V(D)J Recombination to the Cell Cycle Suppresses Genomic Instability and Lymphoid Tumorigenesis
Scientists infer that cell cycle control of the V(D)J recombinase limits the potential genomic damage that could otherwise result from RAG-mediated DNA cleavage. [Immunity]

MEK-ERK Pathway Modulation Ameliorates Disease Phenotypes in a Mouse Model of Noonan Syndrome Associated with the Raf1^L613V Mutation
Data show that different Noonan syndrome (NS) genes have intrinsically distinct pathological effects, demonstrate that enhanced MEK-ERK activity is critical for causing hypertrophic cardiomyopathy and other RAF1-mutant NS phenotypes, and suggest a mutation-specific approach to the treatment of RASopathies. [J Clin Invest]

Rapamycin Reverses Hypertrophic Cardiomyopathy in a Mouse Model of LEOPARD Syndrome-Associated PTPN11 Mutation
Results demonstrate that LEOPARD syndrome (LS) mutations have dominant-negative effects in vivo, identify enhanced mTOR activity as critical for causing LS-associated hypertrophic cardiomyopathy (HCM), and suggest that TOR inhibitors be considered for treatment of HCM in LS patients. [J Clin Invest]

Binge Alcohol Drinking Is Associated with GABAA Alpha2-Regulated Toll-like Receptor 4 (TLR4) Expression in the Central Amygdala
Researchers report that a GABAA alpha2 siRNA vector (pHSVsiLA2) infused into the central nucleus of the amygdala of alcohol-preferring rats caused profound and selective reduction of binge drinking associated with inhibition of alpha2 expression, decreased GABAA receptor density, and inhibition of Toll-like receptor 4. [Proc Natl Acad Sci U S A]

Cancer Genetics-Guided Discovery of Serum Biomarker Signatures for Diagnosis and Prognosis of Prostate Cancer
Researchers’ approach suggests a general path to rational cancer biomarker discovery and initial validation guided by cancer genetics and based on the integration of experimental mouse models, proteomics-based technologies, and computational modeling. [Proc Natl Acad Sci U S A]

Microscale 3D Hydrogel Scaffold for Biomimetic Gastrointestinal (GI) Tract Model
Researchers describe a simple and efficient method for fabricating natural and synthetic hydrogels into 3D geometries with high aspect ratio and curvature. [Lab Chip]

Human Cord Blood-Derived Endothelial Progenitor Cells and their Conditioned Media Exhibit Therapeutic Equivalence for Diabetic Wound Healing
In this study, human cord blood-derived endothelial progenitor cell transplantation stimulated keratinocyte and fibroblast proliferation substantially as early as 3 days after injury, leading to significantly accelerated wound closure in streptozotocin-induced diabetic nude mice, compared to PBS control. [Cell Transplant]

POLICY

Government of Canada Boosts Efforts to Find Treatments for Pediatric Cancers and Rare Genetic Diseases
Two new research teams of top doctors and scientists from across Canada will embark on an ambitious goal to identify the genes that cause the most challenging types of cancer and rare diseases in children, and find new treatments. [Canadian Institutes of Health Research, Canada]

Singapore Infuses Another $1 Billion into R&D Fund
Singapore’s National Research Fund will get an extra $1 billion in its budget for the coming year. [National Research Fund, Singapore]

Aussie Science Chief Resigns
Australia’s chief scientist, astronomer Penny Sackett, surprised the scientific community by announcing her resignation effective 4 March — just halfway through her 5-year tenure. [Department of Innovation, Industry, Science and Research, Australia]

Big Increase for Indian Research Is Not Good Enough, Say Scientists
The Indian government plans to spend 21% more this year on science and technology, Finance Minister Pranab Mukherjee announced. But some scientists think the government, which finances about 80% of the country’s investment in research and development, should take advantage of a booming economy to enact even larger increases. [Indian Ministry of Science and Technology, India]

BUSINESS

TiGenix Announces Proposed Combination with Cellerix and Capital Increase Through a Public Rights Offering
TiGenix NV and Cellerix SA, a privately held company based in Spain, announce that the two cell therapy-focused biotechnology companies and Cellerix’ shareholders have entered into a contribution agreement to combine the operations of both companies by means of a share for share exchange. [Cellerix SA Press Release]

Horizon Secures Exclusive License from University of Pittsburgh to Panel of New Human Isogenic Cell Lines
Horizon Discovery announced that it has secured worldwide exclusive rights to a panel of new human isogenic cell models developed by Dr.Jian Yu and Dr.Lin Zhang of the University of Pittsburgh using techniques that are the basis of the Company’s proprietary rAAV GENESIS gene-editing platform. [Horizon Discovery Limited Press Release]

Acquisition of iPS Cell Patent License from iPS Academia Japan
Univercell-Biosolutions S.A.S., a leading provider of human cardiac cells for drug discovery, and iPS Academia Japan, Inc. (Kyoto, Japan), announced a worldwide non-exclusive licensing agreement for iPS Academia Japan’s iPSC (induced pluripotent stem cell) patent portfolio. [Univercell-Biosolutions S.A.S. Press Release]

CellSeed and LA BioMed to Launch Joint Research Project on Regenerative Medicine Technology
CellSeed Inc. and Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center (LA BioMed) have entered into a two-year research agreement to study CellSeed’s proprietary core technology in regenerative medicine, “Cell Sheet Engineering,” CellSeed President and CEO Yukio Hasegawa, PhD, announced. [CellSeed Inc. Press Release]

Quintiles Supports Samsung’s Entry Into Biopharma Market Through Joint Venture
Samsung Electronics Co., Ltd., the world’s largest electronics company, and its affiliated companies have entered into a strategic partnership with Quintiles, the world’s leading pharmaceutical services company, to support Samsung’s entry into the biopharmaceuticals market. [Quintiles Transnational Corporation Press Release]

Elan and Celgene Enter into Settlement and License Agreement Related to ABRAXANE®
Elan Corporation plc and Celgene Corporation, announced that Elan subsidiary Elan PharmaInternational Limited has entered into a settlement and license agreement with Celgene Corporation resolving the patent infringement litigation involving ABRAXANE®. [Elan Corporation plc Press Release]

ACT Secures Patent to Generate Embryonic Stem Cells Without Embryo Destruction
Advanced Cell Technology, Inc. (ACT), a leader in the field of regenerative medicine, announced that it has been issued a patent on its “single-blastomere” technique. [Advanced Cell Technology, Inc. Press Release]

Silence Issued New U.S. Patent Covering Fundamental RNA Interference Technology Currently in Several Clinical Trials
Silence Therapeutics plc announced the issuance of United States patent 7,893,245, titled “Interfering RNA Molecules,” by the United States Patent and Trademark Office. [Silence Therapeutics plc Press Release]

Cell-Based Gene Therapy Against Cancer (MGN1601) Granted Patent Protection in Europe
The German biotechnology company MOLOGEN AG revealed in Berlin that the European Patent Office has announced that it will be granting a European patent for the company’s allogenic tumor therapy. [MOLOGEN AG Press Release]

Santarus and Pharming Announce Receipt of FDA Refusal to File Letter for Rhucin Biologics License Application
Specialty biopharmaceutical company Santarus, Inc. and biotech company Pharming Group NV announced the receipt of a “refusal to file” letter from the U.S. Food and Drug Administration (FDA) for the RHUCIN® (recombinant human C1 inhibitor) Biologics License Application submitted by Pharming. [Pharming Group NV Press Release]

ARCALYST® (rilonacept) Meets Primary and All Secondary Endpoints in Second Phase III Trial for Prevention of Gout Flares in Patients Initiating Uric Acid-Lowering Therapy
Regeneron Pharmaceuticals, Inc. announced that in a second Phase III study (PRE-SURGE 2) in gout patients initiating allopurinol therapy, ARCALYST (rilonacept) met the primary and all secondary study endpoints. [Regeneron Pharmaceuticals, Inc. Press Release]

Regulatory Scientific Advice on CVac Phase III Trial
Australian health care company Prima BioMed Limited announced that there has been an agreement for strategy and design of the Phase III clinical registration trial for the CVac™ immunotherapy therapeutic ovarian cancer vaccine. [Prima BioMed Limited Press Release]

Fate Therapeutics Announces Emerging Data from Proof-of-Concept FT1050 Clinical Trial and Receives Orphan Drug Designation
Fate Therapeutics, Inc. presented encouraging preliminary data from an ongoing Phase Ib clinical trial of FT1050 at the 2011 BMT Tandem Meetings in Honolulu, Hawaii (Abstract Number: 198; entitled, “Ex Vivo Treatment of Hematopoietic Stem Cells with 16, 16-dimethyl Prostaglandin E2 (FT1050) Improves Engraftment and Hematopoietic Reconstitution”). [Fate Therapeutics, Inc. Press Release]

Stem Cell Researchers at UM Embark on New Trial: First Trial to Compare Autologous Cells with Donor Cells for Dilated Cardiomyopathy
Joshua M. Hare, M.D., professor of medicine and director of the University of Miami Miller School of Medicine’s Interdisciplinary Stem Cell Institute, has received approval from the U.S. Food and Drug Administration to begin the first clinical trial in the nation comparing autologous stem cells to donor stem cells for patients with dilated cardiomyopathy. [University of Miami Miller School of Medicine Press Release]

Scientists Contributing to Regenerative Medicine Are Recognized by the CSC
The Carnegie Science Center (CSC) announced the 2011 award recipients and will honor them during the Fifteenth Annual Carnegie Science Awards celebration, to be held on May 6, 2011, at Carnegie Museums of Pittsburgh in Oakland. [McGowan Institute for Regenerative Medicine Press Release]

NIH

Research Center’s Death Warrant Signed by NIH Advisers
The Scientific Management Review Board (SMRB), which was created by Congress to offer advice on National Institutes of Health’s (NIH) structure, met for one and a half hours by teleconference to hear updates on two changes — the creation of the new center, which SMRB recommended, and NIH’s plan to simultaneously dismantle National Center for Research Resources.

CBER

Order to Cease Manufacturing of HCT/Ps – Sunrise Medical Laboratories, Inc.

February 17, 2011 Approval Letter – Fluzone/Fluzone High-Dose

Corifact – Approval Letter and Package Insert

REGULATORY

FOOD AND DRUG ADMINISTRATION (United States)

FDA and Georgetown University Medical Center Announce Partnership
The U.S. Food and Drug Administration (FDA) and Georgetown University Medical Center announced a new partnership to stimulate innovation in regulatory science, ethics, education, and training. The partnership enhances the capabilities of both institutions to meet their common goal of improving public health.

Agency Information Collection Activities; Announcement of Office of Management and Budget Approval; Charging for Investigational Drugs [Docket No. FDA-2006-N-0237]

Draft Guidance for Industry on Medication Guides — Distribution Requirements and Inclusion of Medication Guides in Risk Evaluation and Mitigation Strategies; Availability [Docket No. FDA-2011-D-0074]

Clinical Pharmacogenomics: Premarketing Evaluation in Early Phase Clinical Studies

EUROPEAN MEDICINES AGENCY (European Union)

Adjusted Fees for Applications to European Medicines Agency on 1 April 2011
The European Medicines Agency reminds applicants and marketing-authorization holders that adjusted fees will be coming into effect on Friday 1 April.

EVENTS

NEW RNAi 2011
March 29-31, 2011
Oxford, United Kingdom

NEW RNAi and High Content Imaging 2011
April 1, 2011
Oxford, United Kingdom

NEW International Association of Neurorestoratology (IANR) IV & 8th Global College of Neuroprotection and Neuroregeneration (GCNN) Conference
April 27-30, 2011
Amman, Jordan

NEW Stem Cell Programming and Reprogramming
December 8-11, 2011
Lisbon, Portugal

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

JOB OPPORTUNITIES

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

Manager Clinical Affairs (Bioheart, Inc.)

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

Postdoctoral Position – Germline Stem Cells (R. L. Brinster, School of Veterinary Medicine, University of Pennsylvania)

Scientific Director (Adipose Derived Stem Cell Bank)

Process Technical Development Scientist (StemCells, Inc.)

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)

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

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

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