Cell Therapy News Volume 14.23 | Jun 17 2013

Wnt Activation in Nail Epithelium Couples Nail Growth to Digit Regeneration
Researchers showed that nail stem cells (NSCs) reside in the proximal nail matrix and that the mechanisms governing NSC differentiation are coupled directly with their ability to orchestrate digit regeneration. Early nail progenitors undergo Wnt-dependent differentiation into the nail. After amputation, this Wnt activation is required for nail regeneration and also for attracting nerves that promote mesenchymal blastema growth, leading to the regeneration of the digit. [Nature] Abstract | Press Release

Hydrogels that Mimic Developmentally Relevant Matrix and N-Cadherin Interactions Enhance MSC Chondrogenesis
Investigators functionalized hyaluronic acid hydrogels with N-cadherin mimetic peptides and evaluated their role in regulating chondrogenesis and cartilage matrix deposition by encapsulated mesenchymal stem cells (MSCs). Subcutaneous implantation of MSC-seeded constructs also showed superior neocartilage formation in implants functionalized with N-cadherin mimetic peptides compared with controls. [Proc Natl Acad Sci USA]
Abstract | Press Release

In Vivo-Directed Evolution of a New Adeno-Associated Virus for Therapeutic Outer Retinal Gene Delivery from the Vitreous
To address the need for broadly applicable gene delivery approaches, researchers implemented in vivo-directed evolution to engineer adeno-associated virus variants that deliver the gene cargo to the outer retina after injection into the eye’s easily accessible vitreous humor. [Sci Transl Med] Abstract | Press Release

Osteoblast-Targeted Suppression of PPARγ Increases Osteogenesis through Activation of mTOR Signaling
The authors describe an adenoviral-mediated peroxisome proliferator-activated receptor gamma (PPARγ) knock-out system in which suppression of PPARγ in mesenchymal stem cells enhanced osteoblast differentiation and inhibited adipogenesis in vitro. Data from both in vitro cultures of mesenchymal stem cells and in vivo μCT analysis of bones suggests that suppression of PPARγ activity in osteoblasts significantly increased osteoblast differentiation and trabecular number. [Stem Cells] Abstract

Tumor Imaging and Interferon-γ Inducible Protein-10 Gene Transfer Using a Highly Efficient Transferrin-Conjugated Liposome System in Mice
Researchers developed a PEGylated transferrin-conjugated liposomes system for the combined tumor imaging and targeted delivery of the interferon-γ inducible protein-10 gene in a single macromolecular construct. Here, they characterized and analyzed the utility of this system in a mouse model of breast cancer. [Clin Cancer Res] Abstract

Third-Party Mesenchymal Stem Cells Improved Human Islet Transplantation in a Humanized Diabetic Mouse Model
Investigators determined whether co-transplantation of human bone marrow derived mesenchymal stem cells could prevent immune rejection and improve human islet transplantation in a humanized NSG mouse model. [Mol Ther] Abstract

Toward RNAi Therapy for the Polyglutamine Disease Machado-Joseph Disease
Machado-Joseph disease (MJD) is a dominantly inherited ataxia caused by a polyglutamine-coding expansion in the ATXN3 gene. Suppressing expression of the toxic gene product represents a promising approach to therapy for MJD and other polyglutamine diseases. Researchers performed an extended therapeutic trial of RNA interference (RNAi) targeting ATXN3 in a mouse model expressing the full human disease gene and recapitulating key disease features. [Mol Ther] Abstract

Effects of Mesenchymal Stem Cell Therapy on the Time Course of Pulmonary Remodeling Depend on the Etiology of Lung Injury in Mice
Researchers found that mesenchymal stem cell therapy improves lung function through modulation of the inflammatory and remodeling processes. In pulmonary acute lung injury, a reduction in collagen fiber content was observed associated with a balance between metalloproteinase-8 and tissue inhibitor of metalloproteinase-1 expressions. [Crit Care Med] Abstract

AAV2-Mediated Striatum Delivery of Human CDNF Prevents the Deterioration of Midbrain Dopamine Neurons in a 6-Hydroxydopamine Induced Parkinsonian Rat Model
Scientists examined the neuroprotective and functional restorative effects of cerebral dopamine neurotrophic factor (CDNF) overexpression in the striatum via recombinant adeno-associated virus type 2 (AAV2.CDNF) in 6-hydroxydopamine injected rats. [Exp Neurol] Abstract

Beneficial Effects of Selective HDL-Raising Gene Transfer on Survival, Cardiac Remodeling and Cardiac Function after Myocardial Infarction in Mice
Post-myocardial infarction (MI) ejection fraction is decreased in patients with low high-density lipoprotein (HDL) cholesterol levels, independent of the degree of coronary atherosclerosis. Researchers evaluated whether selective HDL-raising gene transfer exerts cardioprotective effects post MI. [Gene Ther] Abstract

Mesenchymal Stem Cell Treatment for Ischemic Kidney Disease
This review focuses on recent progress on the use of mesenchymal stem cells to prevent kidney injury in ischemic kidney injury, with a focus on the chronic form. [Stem Cells] Abstract | Full Article

Evidence for High Translational Potential of Mesenchymal Stromal Cell Therapy to Improve Recovery from Ischemic Stroke
The authors discuss the clinical rationale of mesenchymal stromal cells for stroke therapy in the context of their emerging utility in other diseases, and their recent clinical approval for treatment of graft-versus-host disease. [J Cereb Blood Flow Metab] Abstract

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

Mayo Clinic First in US to Test Stem Cells in Pediatric Congenital Heart Disease Patients
Mayo Clinic has announced the first U.S. stem cell clinical trial for pediatric congenital heart disease. The trial aims to determine how stem cells from autologous umbilical cord blood can help children with hypoplastic left heart syndrome, a rare defect in which the left side of the heart is critically underdeveloped. [Mayo Clinic]
Press Release

NW Bio Has Initiated Its DCVax^®-Direct Phase I/II Trial for All Solid Tumor Cancers
Northwest Biotherapeutics (NW Bio) announced that its 60-patient Phase I/II clinical trial of DCVax-Direct for all inoperable solid tumor cancers has been initiated at The University of Texas MD Anderson Cancer Center in Houston, Texas. [Northwest Biotherapeutics, Inc.] Press Release

US Supreme Court Rules Patents on ‘Natural’ Human Genes Invalid
In one of the most anxiously awaited court decisions of the year, the US Supreme Court unanimously struck down patents on isolated, natural human genes. Patents on synthesized DNA, however, are still valid, the justices said. Included in that category are commercially important patents on cDNA – segments of DNA synthesized using an RNA template. [Supreme Court, United States] Press Release | Syllabus

UK Scientists Fear Further Cuts
Chancellor of the exchequer George Osborne is set to unveil the next comprehensive spending review, which sets spending for government departments. He said that he hoped to make clear the government’s “long-term commitment” to research in the new review, but scientists fear another budget freeze. [Chancellor of the Exchequer, United Kingdom] Press Release

Japan Eyes U.S. National Institutes of Health as Model for a New Agency
Japan’s government may create its own version of the U.S. National Institutes of Health to foster medical innovation and bridge the gap between the laboratory and the clinic. [Japanese Government, Japan]
Press Release

Protestors to Government: ‘Save Spanish Science’
Spanish researchers arrived at a closed gate at the Ministry of Economy and Competitiveness in Madrid, where they had wanted to deliver a clear message to their government: Save Spanish science. Protestors carried a 10-point wish list, signed by 45,000 supporters, that includes increasing public spending for research to 2% of the gross domestic product by 2016, improving opportunities for young researchers, and creating an independent funding agency. [Ministry of Economy and Competitiveness, Spain] Press Release

Scotland Continues to Forge Ahead in Stem Cell Research
Scotland’s standing as Europe’s largest and most highly regarded stem cell research community has been reaffirmed recently by announcements of funding from the UK Medical Research Council and the British Heart Foundation to the MRC Centre for Regenerative Medicine based at the Edinburgh BioQuarter. As part of a major $25 million boost for UK regenerative medicine, the Edinburgh center will play a key role in the development of new therapies. [PR Newswire Association LLC] Press Release

Immune Design Partners with Leading Cancer Organizations to Advance Novel Immunotherapy Research
The Cancer Research Institute, the Ludwig Institute for Cancer Research and Immune Design announced that they have signed a collaboration agreement to advance cancer immunotherapy research. Specifically, the partnership will focus on clinical trials to test novel combinations of immunotherapies, including two investigational drugs from Immune Design’s pipeline. [Ludwig Institute For Cancer Research Ltd] Press Release

Phase I Trial Uses Enhanced T Cells to Treat Neuroblastoma
A phase I clinical trial at Baylor College of Medicine and Texas Children’s Cancer Center using an immunotherapy approach to treating neuroblastoma has received support from the nonprofit organization Solving Kids’ Cancer. The organization provided $153,000 for the trial, which will start this summer. [Baylor College of Medicine]
Press Release

Argos Therapeutics Announces US and Global Expansion for Ongoing Pivotal Phase III ADAPT Study for Personalized Immunotherapy
Argos Therapeutics Inc. announced it has expanded its ADAPT Phase III clinical study for AGS-003 to additional top cancer centers in the United States, Canada and will soon be expanding into Europe and Israel. [Argos Therapeutics Inc.] Press Release

Stemline Therapeutics’ SL-401 Receives Orphan Drug Designation for the Treatment of Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
Stemline Therapeutics, Inc. announced that SL-401 has received Orphan Drug designation from the U.S. Food and Drug Administration for the treatment of BPDCN, a rare and aggressive hematologic malignancy for which there is no effective treatment. [Stemline Therapeutics, Inc.] Press Release

IntelliCell BioSciences Receives US Patent for Its Stem Cell Extraction Technology
IntelliCell BioSciences, Inc. announced that it has been notified by the US Patent office that its patent for stem cell extraction technology has been published. IntelliCell’s proprietary patented method for deriving blood vessel originated vascular cells from adipose tissue by use of ultrasonic cavitation has been made official. [PR Newswire Association LLC] Press Release

LifeSouth Reaches a Milestone with FDA License
A journey that began in Gainesville, Florida more than 15 years ago, that’s allowed newborn babies to become lifesavers, reached a milestone, when the Food and Drug Administration (FDA) notified LifeSouth that its license was approved for cord blood manufacturing. [LifeSouth Community Blood Centers] Press Release

Federal and State Governments Lend Support to Cord Blood Banking
As researchers continue to discover groundbreaking medical therapies using stem cells from umbilical cord blood, Federal and State governments in the United States are ramping up their efforts to support the cord blood industry and educate parents-to-be about banking their babies’ stem cells. The most recent development happened when the New York State Senate introduced a bill to create a public umbilical cord blood banking program within the New York State Department of Health to promote public awareness of the potential benefits of public cord blood banking. [PR Newswire Association LLC] Press Release

Global Expert in Lung Disease Joins Cedars-Sinai to Lead New Stem Cell Research Program
Barry R. Stripp, PhD, a recognized expert in lung disease and stem cell research, has been named director of the new Lung Stem Cell Research Program that spans the Women’s Guild Lung Institute and the Regenerative Medicine Institute at Cedars-Sinai. [Cedars-Sinai] Press Release

First Science Star for Wales Announced
A world-leading neurobiologist who will take up a post at the University as the first appointment under the Welsh Government’s £50 million flagship science initiative has been welcomed to Wales and Cardiff by First Minister for Wales and the Minister for Economy & Science. [Cardiff University] Press Release

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

Government-Owned Inventions; Availability for Licensing (FR Doc No: 2013-13895)

Center for Scientific Review; Notice of Closed Meeting (FR Doc No: 2013-14128)

National Institute of Biomedical Imaging and Bioengineering; Notice of Closed Meeting (FR Doc No: 2013-13743)

June 13, 2013 Approval Letter – HPC Cord Blood BLA 125432

Draft Guidance for Industry and FDA Staff: Investigational New Drug Applications for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic and Immunologic Reconstitution in Patients with Disorders Affecting the Hematopoietic System

Draft Guidance for Industry: Biologics License Applications for Minimally Manipulated, Unrelated Allogeneic Placental/Umbilical Cord Blood Intended for Hematopoietic and Immunologic Reconstitution in Patients with Disorders Affecting the Hematopoietic System

Food and Drug Administration (United States)

Agency Information Collection Activities; Proposed Collection; Comment Request: Investigational New Drug Safety Reporting Requirements for Human Drug and Biological Products and Safety Reporting Requirements for Bioavailability and Bioequivalence Studies in Humans (FR Doc No: 2013-13904)

Agency Information Collection Activities; Proposed Collection; Comment Request; General Licensing Provisions: Biologics License Application, Changes to an Approved Application, Labeling, Revocation and Suspension, Postmarketing Studies Status Reports, and Forms FDA 356h and 2567 (FR Doc No: 2013-13896)

NEW World Cord Blood Congress 2013
September 30-October 1, 2013
Cambridge, United States

NEW Cell Culture World Congress USA 2013
September 30-October 1, 2013
Cambridge, United States

NEW Stem Cells & Regenerative Medicine Congress 2013
September 30-October 1, 2013
Cambridge, United States

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

Director of Cell Processing Facility (S L Collins Associates, Inc.)

Postdoctoral Position – Developmental Neurobiology (North Carolina College of Veterinary Medicine)

Senior Postdoctoral Research Position – Molecular Cardiovascular and Renal Research (University of Oklahoma Health Sciences Center)

Postdoctoral Researcher – Hematopoietic Stem Cells (Albert Einstein College of Medicine)

Postdoctoral Position – Bone and Cartilage Tissue Engineering (University of California, Los Angeles)

Postdoctoral Fellow – Stem Cell and Cancer Biology (Johns Hopkins University School of Medicine)

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


Recruit Top Talent: Reach more than 50,000 potential candidates by posting your organization’s career opportunities on the Connexon Creative Job Board at no cost.