Volume 14.14 | Apr 15

Issue 14.14 April 15, 2013
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Team Develops Implantable, Bioengineered Rat Kidney
Bioengineered rat kidneys successfully produced urine both in a laboratory apparatus and after being transplanted into living animals. The research team describes building functional replacement kidneys on the structure of donor organs from which living cells had been stripped, an approach previously used to create bioartificial hearts, lungs and livers. [Press release from Massachusetts General Hospital discussing online prepublication in Nature Medicine] Press Release | Abstract

Achieve Superior Differentiation of Mouse MSC & MEF into Osteoblasts with the New MesenCult™ Osteogenic Stimulatory Kit (Mouse)

PUBLICATIONS (Ranked by impact factor of the journal)

Transcription Factor-Mediated Reprogramming of Fibroblasts to Expandable, Myelinogenic Oligodendrocyte Progenitor Cells
Investigators described direct conversion of mouse embryonic and lung fibroblasts to induced oligodendrocyte progenitor cells (iOPCs) using sets of either eight or three defined transcription factors. When transplanted to hypomyelinated mice, iOPCs are capable of ensheathing host axons and generating compact myelin. [Nat Biotechnol] Abstract | Press Release

Reestablishment of the Endothelial Lining by Endothelial Cell Therapy Stabilizes Experimental Abdominal Aortic Aneurysms
Researchers hypothesized that reendothelialization by cell therapy would modulate aortic wall destruction and ultimately stabilize abdominal aortic aneurysms (AAAs). They evaluated the impact of local seeding of rat aortic endothelial cells (ECs) or peripheral blood-derived outgrowth ECs on AAA evolution. [Circulation] Abstract

LDL Receptor and Its Family Members Serve as the Cellular Receptors for Vesicular Stomatitis Virus
Recombinant forms of vesicular stomatitis virus (VSV) and VSV-G-pseudotyped viral vectors are being developed for gene therapy, vaccination, and viral oncolysis and are extensively used for gene transduction in vivo and in vitro. Researchers showed that the LDL receptor (LDLR) serves as the major entry port of VSV and of VSV-G-pseudotyped lentiviral vectors in human and mouse cells, whereas other LDLR family members serve as alternative receptors. [Proc Natl Acad Sci USA] Abstract

Cardiopoietic Stem Cell Therapy in Heart Failure: The C-CURE Multicenter Randomized Trial with Lineage-Specified Biologics
The authors evaluated the feasibility and safety of autologous bone marrow-derived and cardiogenically-oriented mesenchymal stem cell therapy, and probed for efficacy signals in patients with chronic heart failure. [J Am Coll Cardiol] Abstract | Press Release

Gene Silencing via RNAi and siRNA Quantification in Tumor Tissue Using MEND, a Liposomal siRNA Delivery System
Scientists developed a liposomal small interfering RNA (siRNA) carrier, a multifunctional envelope-type nanodevice (MEND). They report on the development of an in vivo siRNA delivery system that is delivered by systemic injection and an analysis of the pharmacokinetics of an intravenously administered siRNA molecule in tumor tissue. [Mol Ther] Abstract

Transient Photoreceptor Deconstruction by CNTF Enhances rAAV-Mediated Cone Functional Rescue in Late Stage CNGB3-Achromatopsia
Recombinant adeno-associated virus of serotype 5 (rAAV5)-mediated gene transfer of the human β-subunit, CNGB3, of the cone cyclic nucleotide-gated (CNG) channel cDNA to mutant dog cones results in functional and structural rescue in dogs less than 0.5 years of age, but treatment is minimally effective in dogs greater than 1 year. Researchers tested a new therapeutic concept by combining gene therapy with the administration of ciliary neurotrophic factor (CNTF). [Mol Ther] Abstract

Circulating Mononuclear Progenitor Cells: Differential Roles for Sub-Populations in Repair of Retinal Vascular Injury
Researchers examined the effect on retinal vascular homing of exogenous CD34+ and CD14+ progenitor cells using mouse models of chronic (streptozotocin-induced diabetes) and acute (ischemia/reperfusion) ocular vascular injury. [Invest Ophthalmol Vis Sci] Abstract

Lower Baseline Prostate-Specific Antigen Is Associated with a Greater Overall Survival Benefit from Sipuleucel-T in the Immunotherapy for Prostate Adenocarcinoma Treatment (IMPACT) Trial
The authors explored the prognostic and predictive value of baseline variables in 512 patients with metastatic castration-resistant prostate cancer from the phase III IMPACT trial who were randomized to receive sipuleucel-T or control. [Urology] Abstract | Press Release

Air-Liquid Interface Culture for Respiratory Research: Watch Q&A Video


Molecular Imaging: The Key to Advancing Cardiac Stem Cell Therapy
The authors discus the various molecular imaging techniques validated to date for studying stem cells in living subjects, with a particular emphasis on their utilities in assessing the acute retention and the long-term survival of transplanted stem cells. These imaging techniques will be essential for advancing cardiac stem cell therapy by providing the means to both guide ongoing optimization and predict treatment response in humans. [Trends Cardiovasc Med] Abstract

Therapeutic Angiogenesis for Revascularization in Peripheral Artery Disease
Researchers focused on previous and recent gene and cell therapy studies for limb ischemia, including both experimental and clinical research, and summarized some important papers published in this field. [Gene] Abstract

Visit our reviews page to see a complete list of reviews in the cell, gene and immunotherapy research field.

Complimentary Wallchart: Human Hematopoietic Progenitors. Request Your Copy Today.


Experimental Study Suggests Bone-Marrow Grafts Show Promise for Some Sufferers of Low-Back Pain
A new study suggests that the type of bio-cellular grafts increasingly used by surgeons to repair damaged tissue may be useful for treating low-back pain. However, not all sufferers responded equally to the novel therapy. [Press release from the American Academy of Pain Medicine (AAPM) discussing research presented at the 29th Annual Meeting of the AAPM, Fort Lauderdale] Press Release

Tekmira Presents Data from Its TKM-PLK1 Phase I Clinical Trial
Tekmira Pharmaceuticals Corporation announced the results of its Phase I clinical trial with TKM-PLK1, an RNAi therapeutic for the treatment of solid tumors. [Press release from Tekmira Pharmaceuticals Corporation discussing research presented at the American Association for Cancer Research (AACR) Annual Meeting 2013, Washington] Press Release

Tekmira’s Collaborators at the National Cancer Institute Present Preclinical Data
Preclinical results indicate that a statistically significant decrease in tumor size was observed in the animals treated with either CSN5 siRNA or a combination of CSN5 and WEE1 siRNA. [Press release from Tekmira Pharmaceuticals Corporation discussing research presented at the AACR Annual Meeting 2013, Washington] Press Release

Meta-Analysis of VELCADE® (bortezomib)-Based Induction Therapy followed by Stem-Cell Transplantation Showed Significantly Greater Response and Longer Progression-Free and Overall Survival in Patients with Multiple Myeloma
Millennium: The Takeda Oncology Company with its parent company Takeda Pharmaceutical Company Limited announced data from a meta-analysis of VELCADE-based regimens compared to non-VELCADE-based regimens as induction therapy prior to autologous stem cell transplant in patients with previously untreated multiple myeloma. [Press release from Millennium Pharmaceuticals, Inc. discussing research presented at the 14th International Myeloma Workshop, Kyoto] Press Release

11th Annual International Cord Blood Symposium | San Francisco


Researchers and Advocates Gather in Washington, D.C., to Protest Cuts to Biomedical Research
Thousands of scientists and patient advocates poured into a square in downtown Washington, D.C., to hold what organizers billed as the largest-ever rally to call for more funding for biomedical research. The event held in conjunction with the annual meeting of the American Association for Cancer Research with support from over 200 organizations, aimed to draw attention to the 5% cut to the National Institutes of Health’s (NIH’s) $31 billion budget imposed by Congress through sequestration as well as the flat growth of NIH’s budget over the past decade. [National Institutes of Health, United States] Press Release

Obama’s 2014 Science Budget: Research Gets Some Help, and Hurt
President Barack Obama submitted to Congress his spending plan for the 2014 fiscal year that begins on 1 October. This story detail requests for specific agencies, programs, or initiatives and offer some insight into the Obama administration’s vision for science. Plus, reactions from the research community and around the web. [US President, United States] Press Release

Obama Plays Scientific Favorites
US President Barack Obama released a budget plan for fiscal year 2014 that proposes US$143 billion for research and development across the federal government, an increase of about 1% above 2012 levels. But even this modest rise may be wishful thinking. White House budget requests often bear little resemblance to the spending choices made later by Congress, although they can reveal an administration’s policy priorities. [US President, United States] Press Release

Stem Cells Boom in Vet Clinics
New guidance from the US Food and Drug Administration (FDA) could soon rein in veterinary uses of stem cells, a practice that has exploded in the United States over the past decade, even though most therapies are unproven. Many researchers and veterinarians say that the guidance, a draft of which the agency plans to issue by the end of the year, is overdue. But others worry that FDA interference could hamper research that could benefit animals – and their human companions. [Food and Drug Administration, United States] Press Release

From our sponsor: Free Nature Reviews Immunology poster – Immune Response to HIV. Request a copy.


BrainStorm Partners with Dana-Farber for NurOwn™ Production in Phase II ALS Clinical Trial
BrainStorm Cell Therapeutics announced that Dana-Farber Cancer Institute has entered into an agreement to provide cGMP-compliant clean room facilities for production of BrainStorm’s NurOwn™ stem cell candidate during its upcoming Phase II ALS trial in the USA. [BrainStorm Cell Therapeutics] Press Release

Cell Therapy Catapult & the UK Stem Cell Foundation Agree to Collaborate: Working Together on Next-Generation Medicines for Patient Benefit
The Cell Therapy Catapult and the UK Stem Cell Foundation announced that they will be working together to progress promising stem cell-based therapies in the UK. The two organizations, which have entered into a memorandum of understanding to cover their co-operation, are both focused on translational activities, moving potential therapeutic approaches on from early-stage research to the later stages of development. [Cell Therapy Catapult] Press Release

Immunotherapies against Cancer: German Cancer Research Center to Broaden Strategic Alliance with Bayer HealthCare
The German Cancer Research Center and Bayer HealthCare will extend their successful strategic research alliance in search of novel cancer therapeutics by focusing their activities also on the field of immunotherapy. [German Cancer Research Center] Press Release

Putin Watches as UMCG Signs Cooperation Agreement to Set Up Stem Cell Institute in Russia
President Vladimir Putin of the Russian Federation and Dutch Prime Minister Mark Rutte watched as Folkert Kuipers, Dean of the Faculty of Medical Sciences and the Supervisory Board of the University Medical Center Groningen (UMCG), signed a cooperation agreement aimed at establishing a stem cell institute in Skolkovo, Russia. President Edward Crawley and director Nikolai Yankovsky signed the agreement on behalf of the Russian partners, the Skolkovo Institute of Science and Technology and the Vavilov Institute of General Genetics, respectively. [University Medical Center Groningen] Press Release

StemCells, Inc. Enters Agreement to Receive $19.3 Million from California Institute for Regenerative Medicine to Help Fund IND for Alzheimer’s Disease
StemCells, Inc. announced that it has entered into an agreement with the California Institute for Regenerative Medicine (CIRM) under which CIRM will provide approximately $19.3 million to help fund preclinical development and IND-enabling activities of the Company’s proprietary HuCNS-SC® product candidate (purified human neural stem cells) for Alzheimer’s disease. [StemCells, Inc.] Press Release

BCM Major Part of New Pediatric Cancer Dream Team Initiative
Baylor College of Medicine’s (BCM’s) Texas Children’s Cancer Center is part of a Dream Team of childhood cancer researchers that will focus on melding the fields of genomics and immunotherapy to advance treatment for pediatric cancer. [Baylor College of Medicine] Press Release

Gamida Cell Reports StemEx® Phase II/III Study Safety and Efficacy Data
Gamida Cell announced today additional analyses for the Phase II/III, multi-center, multi-national, historical cohort-controlled study to evaluate efficacy and safety of StemEx® as an alternative transplantation treatment for patients with high risk leukemia and lymphoma. [Gamida Cell] Press Release

Ireland’s Genable Technologies Ltd Secures Orphan Drug Designation by FDA for Gene Therapy Product GT308
Genable Technologies Ltd gene therapy product, GT038, has been granted Orphan Drug Designation by the Federal Drug Administration (FDA) in the USA for the treatment of Retinitis Pigmentosa. [Genable Technologies Ltd] Press Release

Immune Therapy from Austria Receives Orphan Drug Designation from European Medicines Agency
The European Medicines Agency recently awarded the Austrian biotech company Activartis an Orphan Drug Designation for its innovative Cancer Immune Therapy AV0113. The Orphan Drug Designation applies specifically to the use of AV0113 for the treatment of glioma, a type of brain tumor, which afflicts around one in 10,000 people in the EU. [Activartis Biotech GmbH] Press Release


Submission for OMB Review; 30-Day Comment Request: The Clinical Trials Reporting Program (CTRP) Database (NCI)(FR Doc No: 2013-08270)

Government-Owned Inventions; Availability for Licensing (FR Doc No: 2013-08414)

Notice to Clarify and Update Research Objectives of PAS-13-031 “Stimulating Hematology Investigation:  New Endeavors (SHINE) (R01)”(NOT-DK-13-011)

Broad Agency Announcement (BAA): Development of Medical Countermeasures to Enhance Platelet Regeneration and Survival following Radiation Exposure from a Radiological/Nuclear Incident, BAA-NIAID-DAIT-NIH-AI-2013-166 (NOT-AI-13-039)

Office of Compliance and Biologics Quality (OCBQ)


Food and Drug Administration (United States)

Medical Device Classification Product Codes; Guidance for Industry and Food and Drug Administration Staff; Availability (FR Doc No: 2013-08442)


NEW The Jackson Laboratory: Immune Cells Gone Rogue – Mouse Models of Inflammatory Bowel Disease and Psoriasis
April 25, 1:00PM (ET in US)

NEW Gordon-Merck Research Seminar: Cell Contact & Adhesion
June 1-2, 2013
Lucca (Barga), Italy

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


Faculty Position – Stem Cell, Vascular and Developmental Biology in Ophthalmology (Zhongshan Ophthalmic Center, Sun Yat-Sen University)

Postdoctoral Position – Adult Neurogenesis and Neural Survival Signaling (University of Kentucky)

Cord Blood Laboratory Manager (Puget Sound Blood Center)

Postdoctoral Fellow – Regenerative Medicine (The University of Texas Health Science Center at Houston)

Senior Scientist – Stem Cell Therapy: Metabolic Diseases (Paramount Recruitment)

Postdoctoral Research Fellow – Immunology (Beth Israel Deaconess Medical Center, Harvard Medical School)

Postdoctoral Research Fellow – Regenerative Medicine (Loma Linda University)

Postdoctoral Research Associate (Washington State University)

Postdoctoral Position – Synthetic Biology, RNAi & Gene Therapy (University of Heidelberg Medical School)

Postdoctoral Fellowships – Muscular Dystrophy, Cardiomyopathy, Respiratory Failure, Gene Therapy (University of Pennsylvania)

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