Volume 12.07 | Feb 21

     In this issue: Science | Policy | Business | NIH | CBER | Regulatory | Events
Researcher Discovers Potential Cancer Therapy Target
A researcher has discovered the mechanism by which p53 is inactivated in cancerous cells, allowing tumors to grow. [Press release from the University of Alberta discussing online prepublication in Nature Medicine]


Feature: Inside DNA Vaccines
A radical new type of vaccine is on the horizon, one that uses the body’s own cellular machinery to provoke a comprehensive immune response. DNA vaccines offer many advantages over conventional vaccines. [Australian Life Scientist]

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Human and Mouse Studies Sharpen Focus on Cause of Celiac Disease
Blocking a factor that can activate the human immune response against intestinal bacteria or certain foods could prevent the development of celiac disease in those most at risk. The study points to two chemical signals–interleukin 15 and retinoic acid–as triggers for the inflammatory response to gluten, a protein found in many grains that causes celiac disease. [Press release from the University of Chicago Medical Center discussing online prepublication in Nature]

Phase III Data Show Baxter’s PREFLUCEL Protective Against Seasonal Influenza
Baxter International Inc. announced results of a study that demonstrate effectiveness and tolerability of Baxter’s PREFLUCEL in protecting against seasonal influenza. The study data show nearly 80 percent protective efficacy against the influenza strains contained in the vaccine and a low adverse event profile. [Press release from Baxter International Inc. discussing online prepublication in The Lancet]
Compound Shows Promise Against Treatment-Resistant Cancer Cells in Lab Tests
A new compound identified by UK scientists may help to destroy stubborn cancer cells that are resistant to chemotherapy drugs. Early laboratory tests suggest the compound, ABT-737, could be used to trigger the death of cancer cells in hypoxic regions of solid tumors. [Press release from Cancer Research UK discussing online prepublication in the Journal of Clinical Investigation]
Study Sheds Light on RNA “On/Off Switches”
Scientists have shed new light on a molecular switch that turns genes on or off in response to a cell’s energy needs. The study shows these recently discovered RNA “riboswitches” are capable of more complex functions than originally thought. In addition, because riboswitches so far have been found primarily in bacteria, the study may have implications for designing new antibiotics against harmful bacteria. [Press release from The Scripps Research Institute discussing online prepublication in Nature Structural and Molecular Biology]

Erg Gene Key to Blood Stem Cell ‘Self-Renewal’
Scientists have begun to unravel how blood stem cells regenerate themselves, identifying a key gene required for the process. The discovery that the Erg gene is vitally important to blood stem cells’ unique ability to self-renew could give scientists new opportunities to use blood stem cells for tissue repair, transplantation and other therapeutic applications. [Press release from the Walter and Eliza Hall Institute discussing online prepublication in Genes and Development]

Identification of Glaucoma Gene Brightens View for Future Therapies
A team of investigators from Vanderbilt University and the University of Florida has identified a new candidate gene for the most common form of glaucoma, primary open angle glaucoma. [Press release from EurekAlert! discussing online prepublication in PLoS Genetics]

Reprogrammed Stem Cells Hit a Roadblock: Reprogramming Cells Leads to Genomic Aberrations
A study conducted by the Department of Biochemistry at the University of Geneva and the European Institute of Oncology in Milan concludes that reprogrammed cells exhibit a “genomic instability” that appears to be caused by the process used to return the cells to their embryonic state. Even more serious, the genetic mutations observed resemble mutations that are found in cancer cells. [Press release from ScienceDaily discussing online prepublication in Cell Death and Differentiation]

Scientists Bioengineer a Protein to Fight Leukemia
Scientists have identified a protein called CD19-ligand (CD19-L) located on the surface of certain white blood cells that facilitates the recognition and destruction of leukemia cells by the immune system. This work represents the first report of a bioengineered version of CD19-L, a recombinant human biotherapeutic agent, targeting CD19-positive leukemic stem cells. [Press release from Children’s Hospital Los Angeles discussing online prepublication in the British Journal of Haematology]

Ceregene Reports New Findings Regarding How Parkinson’s Brains Respond to Neurotrophic Factors
Ceregene, Inc. reported that its scientists and collaborators have published new experimental findings in support of its Parkinson’s disease program. The publication reports the first evidence that gene transfer can provide targeted expression of a neurotrophic factor, i.e., neurturin or NRTN, intended to restore and preserve dying neurons. [Press release from Ceregene, Inc. discussing online prepublication in Movement Disorders]

World’s First Chemical Guided Missile Could Be the Answer to Wiping Out Cancer
Scientists have created the world’s first cancer stem cell-targeting chemical missile, placing them a step closer to creating a medical ‘smart bomb’ that would seek out and eradicate the root of cancer cells. [Press release from Deakin University discussing online prepublication in Cancer Science]

Tumor Microvesicles Reveal Detailed Genetic Information
The research team that first discovered tumor-associated RNA in tiny membrane-enclosed sacs released into the bloodstream by cancer cells has now found that these microvesicles also contain segments of tumor DNA, including retrotransposons — also called “jumping genes”– that copy and insert themselves into other areas of the genome. The investigators’ report is the first to show that microvesicles are involved in transferring retrotransposons between cells.  [Press release from Massachusetts General Hospital discussing online prepublication in Nature Communications]

Stem Cell Transplants Help Kidney Damage
Transplanting autologous renal progenitor cells into rat models with kidney damage from pyelonephritis has been found to improve kidney structure and function. [Press release from EurekAlert! discussing online prepublication in Cell Medicine]

Scientists Discover First Breast Cancer ‘Oncogene’ for Five Years
Scientists have pinpointed a key cancer-causing gene that, when overactive, triggers a particularly aggressive type of breast cancer to develop. [Press release from the British Columbia Cancer Agency discussing online prepublication in EMBO Molecular Medicine]

Fibrocell Science, Inc. Announces Positive Results of Its Pilot Study for Treatment of Vocal Fold Scarring with Fibroblasts
Fibrocell Science, Inc. announced publication of results of a five-patient pilot study evaluating the safety and effectiveness of autologous fibroblasts to treat vocal fold scarring. [Fibrocell Science, Inc. Press Release]
CURRENT PUBLICATIONS (Ranked by Impact Factor of the Journal)

Co-Adjuvant Effects of Retinoic Acid and IL-15 Induce Inflammatory Immunity to Dietary Antigens
Researchers find in mice that in conjunction with IL-15, a cytokine greatly upregulated in the gut of celiac disease patients, retinoic acid rapidly activates dendritic cells to induce JNK (also known as MAPK8) phosphorylation and release the proinflammatory cytokines IL-12p70 and IL-23. [Nature]
Efficacy, Safety, and Immunogenicity of a Vero-Cell-Culture-Derived Trivalent Influenza Vaccine: A Multicentre, Double-Blind, Randomized, Placebo-Controlled Trial
The use of cell-culture technologies for the manufacture of influenza vaccines might contribute to improved strain selection and robust vaccine supplies. Researchers investigated the safety, immunogenicity, and protective efficacy of a Vero-cell-culture-derived influenza vaccine, and assessed the correlation between vaccine efficacy and hemagglutination inhibition antibody titer. [Lancet]
UBE4B Promotes Hdm2-Mediated Degradation of the Tumor Suppressor p53
Data indicate that amplification and overexpression of UBE4B represent previously undescribed molecular mechanisms of inactivation of p53 in brain tumors. [Nat Med]
Hypoxic Human Cancer Cells Are Sensitized to BH-3 Mimetic-Induced Apoptosis via Downregulation of the Bcl-2 Protein Mcl-1
Findings suggest that myeloid cell leukemia sequence 1 (Mcl-1)-sparing Bcl-2 homology domain 3 (BH-3) mimetics may induce apoptosis in hypoxic tumor cells that are resistant to other chemotherapeutic agents and may have a role in combinatorial chemotherapeutic regimens for treatment of solid tumors. [J Clin Invest]
The glmS Riboswitch Integrates Signals from Activating and Inhibitory Metabolites In Vivo
Contrary to the conventional view that a riboswitch responds to just a single cognate metabolite, this new model proposes that a single riboswitch integrates information from an array of chemical signals to modulate gene expression based on the overall metabolic state of the cell. [Nat Struct Mol Biol]
ERG Dependence Distinguishes Developmental Control of Hematopoietic Stem Cell Maintenance from Hematopoietic Specification
Using in vitro and in vivo models of hematopoiesis and hematopoietic stem cell (HSC) development, researchers provide strong evidence that ERG is at the center of a distinct regulatory program that is not required for hematopoietic specification or differentiation but is critical for HSC maintenance during embryonic development. [Genes Dev]
Mapping of the Disease Locus and Identification of ADAMTS10 as a Candidate Gene in a Canine Model of Primary Open Angle Glaucoma
The Gly661Arg variant in ADAMTS10 found in the POAG Beagles suggests that altered processing of extracellular matrix and/or defects in microfibril structure or function may be involved in raising intraocular pressure, offering specific biochemical targets for future research and treatment strategies. [PLoS Genet]
Genomic Instability in Induced Stem Cells
Comparative genomic hybridization analysis of stem cells derived by the present protocols revealed the presence of genomic deletions and amplifications, whose signature was suggestive of oncogene-induced DNA replication stress. The genomic aberrations were to a significant degree dependent on c-Myc expression and their presence could explain why p53 inactivation facilitates stem cell reprogramming. [Cell Death Differ]

Tumor Microvesicles Contain Retrotransposon Elements and Amplified Oncogene Sequences
Findings expand the nucleic acid content of tumor microvesicles to include: elevated levels of specific coding and non-coding RNA and DNA, mutated and amplified oncogene sequences and transposable elements. Thus, tumor microvesicles contain a repertoire of genetic information available for horizontal gene transfer and potential use as blood biomarkers for cancer. [Nat Commun]
Autografting of Renal Progenitor Cells Ameliorates Kidney Damage in Experimental Model of Pyelonephritis
Transplanting renal progenitor cells showed the potential for partial augmentation of kidney structure and function in pyelonephritis. Cellular repair was seen predominantly in the proximal tubule, the major site of injury in pyelonephritis. [Cell Med]



Obama Budget Targets Brand Name Medicines
Big pharmaceutical companies could face increased competition from generic drugmakers under two proposals put forth by the Obama administration despite earlier savings extracted from drugmakers as part of last year’s healthcare law. [Obama Administration, United States]

CDC’s Pinched Budget Focuses on Chronic Disease Prevention
The purse strings are getting tighter at the U.S. Centers for Disease Control and Prevention (CDC), which has already seen budget drops in recent years. The agency’s discretionary budget request in 2012 will dip to $5.89 billion, down from $6.47 billion in 2010. [Centers for Disease Control and Prevention, United States]



Sanofi-aventis to Acquire Genzyme for $74.00 in Cash per Share Plus Contingent Value Right
Sanofi-aventis and Genzyme Corporation announced that they have entered into a definitive agreement under which sanofi-aventis is to acquire Genzyme for $74.00 per share in cash, or approximately $20.1 billion. [Sanofi-aventis Press Release]

Exclusive License with Transgene for Novel Skin Cancer Immunotherapeutic Treatment
Australian bio-pharmaceutical company Virax Holdings Limited announced that it has signed a worldwide exclusive License Agreement with leading French biopharmaceutical company Transgene for Virax to develop Transgene’s TG1042 a clinical-stage skin cancer immunotherapy product. [Virax Holdings Limited Press Release]

Tengion Addresses Recent Trading Activity in its Common Stock
Tengion, Inc. announced that it has been engaged in negotiations with a publicly traded company relating to the acquisition of Tengion through a stock-for-stock merger. As a result of the recent increases in the trading price and volume of Tengion’s common stock, Tengion’s potential acquirer has terminated merger negotiations. [Tengion, Inc. Press Release]

Tissue Engineering Methods Earn Funding to Heal Little Hearts
Texas Children’s Hospital in Houston announced that a researcher in its Pediatric Cardiac Bioengineering Laboratory has earned a National Science Foundation Early Career Development (CAREER) Award for his work in studying the causes of congenital heart disease, heart defects, and the development of tissue engineering therapies using stem cells derived from human amniotic fluid. [EurekAlert!]

Stemedica Receives Approval from UCSD to Initiate Ischemic Stroke Study with Adult Allogeneic Stem Cells
Stemedica Cell Technologies, Inc., a leader in adult allogeneic stem cell manufacturing, research and development announced that it has received IRB approval from the University of California, San Diego (UCSD) to initiate a Stemedica clinical study entitled, “A Phase I/II, Multi-Center, Open-Label Study to Assess the Safety, Tolerability and Preliminary Efficacy of a Single Intravenous Dose of Allogeneic Mesenchymal Bone Marrow Cells to Subjects with Ischemic Stroke”.  [Stemedica Cell Technologies, Inc. Press Release]

UMASS Medical School’s Human Stem Cell Bank Makes Available First Seven Stem Cell Lines
The first seven stem cell lines grown and banked at the University of Massachusetts (UMASS) Medical School’s Human Stem Cell Bank are ready for worldwide distribution to researchers working on discovering new therapeutic treatments for diseases such as cancer, juvenile diabetes, Alzheimer’s and Parkinson’s, among others. [EurekAlert!]

Halozyme Announces Roche Doses First Patient in Phase III Clinical Trial with Subcutaneous MabThera® (rituximab)
Halozyme Therapeutics, Inc. and Roche announced the first patient received subcutaneous MabThera (rituximab), an anticancer biologic, in a Phase III registration trial using Enhanze™ technology (rHuPH20, recombinant human hyaluronidase). [Halozyme Therapeutics, Inc. Press Release]

Patent Update: Strengthening and Expansion of Benitec’s Dominant Patent Position in RNAi
Benitec Limited announced that the United States Patent and Trademark Office (USPTO) has indicated that it is issuing the Re-examination Certificate, the formal final step in the USPTO’s confirmation of patentability of claims in the key ‘099 Graham Patent [US Patent No. 6,573,099] “Genetic Constructs for Delaying or Repressing the Expression of a Target Gene”, the wide-ranging platform technology patent covering the use of expressed RNA Interference (RNAi) in human therapeutic applications. [Benitec Limited Press Release]

BrainStorm Receives FDA Orphan Drug Designation for NurOwn™ in the Treatment of Amyotrophic Lateral Sclerosis
BrainStorm Cell Therapeutics Inc. announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the Company’s NurOwn™ autologous adult stem cell product candidate for the treatment of amyotrophic lateral sclerosis, often referred to as Lou Gehrig’s Disease. [BrainStorm Cell Therapeutics Inc. Press Release]

Whitehead Member Rudolf Jaenisch Honored for Groundbreaking Stem Cell Research
Israel’s Wolf Foundation, whose stated mission is “to promote science and art for the benefit of mankind,” has named Whitehead Institute Founding Member Rudolf Jaenisch a recipient of the prestigious 2011 Wolf Prize in Medicine. [Whitehead Institute for Biomedical Research Press Release]

Oxford BioMedica Appoints James Christie as New Head of Manufacturing
Oxford BioMedica plc, a leading gene therapy company, announced the appointment of James Christie, BSc, MBA to the newly created role of Head of Manufacturing with immediate effect. [Oxford BioMedica plc Press Release]

Researching Regenerative Medicine
A new facility at the University of Manitoba will allow researchers to pursue cutting-edge work in the field of regenerative medicine, which could lead to important new treatments for patients. The university’s Regenerative Medicine Program is the first of its kind in Western Canada. [University of Manitoba Press Release]

Promise of Genomics Research Needs a Realistic View
A new commentary by four internationally prominent genetic medicine and bioethics experts cautions against the dangers of inflated expectations of genomics research — an unsustainable genomic bubble — and it offers ways to avoid it while still realizing “the true — and considerable — promise of the genomic revolution.” [University of North Carolina at Chapel Hill School of Medicine Press Release]

Success Rates for Experimental Drugs Falls: Study
The success rate in bringing new medicines to market in recent years is only about half of what it had been previously, but biotech drugs are twice as likely to gain U.S. approval than more traditional chemical drugs, according to a new study. [Reuters]

Drug Firms Attack U.S. Patent Plans
A U.S. government proposal to bring cheaper generic drugs to the market more quickly and help curb health-care costs has triggered criticism from the European pharmaceutical sector. [Wall Street Journal]


President Would Give NIH Slight 2.4% Boost in 2012
The president’s budget would give the National Institutes of Health (NIH) a modest 2.4% raise in 2012 of $745 million over the 2010 level, bringing the total to $31.8 billion. The request includes one major new program: the proposed National Center for Advancing Translational Sciences, which would “re-engineer the pipeline for diagnostics and therapeutics discovery and development,” a budget document explains.

Frustrated Researchers to Petition NIH to Change Rule on Resubmitting Grants
A draft petition urging National Institutes of Health (NIH) leaders to rescind a recent rule limiting the number of times a grant application can be submitted is generating a buzz — not all of it positive — in the scientific community.

Notice of Intent to Publish a Request for Applications on DNA Repository for the NIAAA National Epidemiologic Survey on Alcohol and Related Condition-III (NESARC-III) (U24) (NOT-AA-11-003)

National Cancer Institute Participation in the NIH Parent Program Announcement for Ruth L. Kirschstein National Research Service Awards for Individual Postdoctoral Fellows (PA-11-113) (NOT-CA-11-008)

Notice of Intent to Publish a Funding Opportunity Announcement for Induced Pluripotent Stem Cell (iPSC) Resources promoting Translational Research Advancements in Neurodegenerative Diseases (U24) (NOT-NS-11-011)

NHLBI Ruth L. Kirschstein National Research Service Awards for Individual Predoctoral MD/PhD Fellows (F30) (PA-11-125)
National Human Genome Research Institute; Notice of Closed Meeting (FR Doc. 2011-3482)
National Cancer Institute; Notice of Closed Meetings (FR Doc. 2011-3631)
Center for Scientific Review; Notice of Closed Meetings (FR Doc. 2011-3633)

Terrapinn Stem Cells 645x110


Final Guidance for Industry: Potency Tests for Cellular and Gene Therapy Products 
Roster of the Vaccines and Related Biological Products Advisory Committee

Roster of the Blood Products Advisory Committee 

February 11, 2011 Approval Letter — Adacel

February 25, 2011: Vaccines and Related Biological Products Advisory Committee Meeting Members, Speakers and Participant Roster

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



FDA’s Expanded Mandate in Limbo
The U.S. Food and Drug Administration (FDA) could get a big budget boost this year, with the president requesting $4.3 billion, more than a third of it from regulatory fees paid by companies. But despite an outwardly rosy picture, the agency still doesn’t have the cash in hand it needs to fulfill an expanded mandate. [Food and Drug Administration, United States]

FDA Chief Backs Fees for Generic Drug Reviews
Dr. Margaret Hamburg, commissioner of the Food and Drug Administration, called for a new process to allow makers of generic drugs to pay user fees to the FDA to speed up approval of the low-cost drugs.

FDA’s Rules for Copying Amgen, Roche Drugs Coming ‘Very Soon’
U.S. regulators plan to unveil rules for reviewing the first copies of biologic medicines “very soon,” according to Food and Drug Administration Commissioner Margaret Hamburg.

Draft Guidance for Industry on Clinical Pharmacogenomics: Premarketing Evaluation in Early Phase Clinical Studies; Availability [Docket No. FDA-2011-D-0082]


First Clinical Biomarker Qualification Released for Public Consultation
The European Medicines Agency has released the first qualification opinion for a clinical biomarker for public consultation. The biomarker is intended to identify patients who can be recruited for clinical trials of treatments for pre-dementia Alzheimer’s disease.

Meeting Highlights from the Committee for Medicinal Products for Human Use (CHMP) 14-17 February 2011


Therapeutic Goods Committee 36th Meeting, Summary of Key Resolutions

Compliance with Ministerial and Default Standards


NEW Nanomedicine 2011
June 19-24, 2011
Wittenberg, Germany

NEW BioBanking
July 11-12, 2011
London, United Kingdom

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)

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)
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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