Volume 14.13 | Apr 8

Issue 14.13 April 8, 2013
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TOP STORY
Phase I ALS Trial Is First to Test Antisense Treatment of Neurodegenerative Disease
The initial clinical trial of a novel approach to treating amyotrophic lateral sclerosis (ALS) – blocking production of a mutant protein that causes an inherited form of the progressive neurodegenerative disease – may be a first step towards a new era in the treatment of such disorders. Investigators reported that infusion of an antisense oligonucleotide against SOD1, the first gene to be associated with familial ALS, had no serious adverse effects and the drug was successfully distributed throughout the central nervous system. [Press release from Massachusetts General Hospital discussing online prepublication in The Lancet Neurology]
Press Release | Abstract

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PUBLICATIONS (Ranked by impact factor of the journal)

A Tissue-Like Printed Material
Synthetic mimics of cells, such as liposomes, are typically incapable of cooperation and therefore cannot readily display sophisticated collective behavior. Researchers printed tens of thousands of picoliter aqueous droplets that become joined by single lipid bilayers to form a cohesive material with cooperating compartments. Printed droplet networks might be interfaced with tissues, used as tissue engineering substrates, or developed as mimics of living tissue. [Science] Abstract | Press Release

T-Cell Receptor Affinity and Avidity Defines Antitumor Response and Autoimmunity in T-Cell Immunotherapy
To determine the T-cell receptor (TCR) affinity threshold defining the optimal balance between effective antitumor activity and autoimmunity in vivo, investigators used a unique self-antigen system comprising seven human melanoma gp100(209-217)-specific TCRs spanning physiological affinities (1-100 μM). [Proc Natl Acad Sci USA] Abstract

Long-Term Detection of Human Adipose Derived Mesenchymal Stem Cells after Intra-Articular Injection
Researchers aimed to assess the toxicity of clinical grade human adipose-derived stem cells as well as their biodistribution after intra-articular injection in SCID mice. [Arthritis Rheum] Abstract

Alterations in the Cellular Immune Compartment of Patients Treated with Third-Party Mesenchymal Stromal Cells following Allogeneic Hematopoietic Stem-Cell Transplantation
Scientists assessed immune alterations in the T-cell, B-cell, NK-cell, dendritic cell, and monocytic compartments of steroid-refractory GvHD patients 30, 90, and 180 days after mesenchymal stromal cell or placebo infusion respectively. [Stem Cells] Abstract

Klf5 Controls Bone Marrow Homing of Stem Cells and Progenitors through Rab5-Mediated ß1/ß2-Integrin Trafficking
Researchers showed that Krüppel-like factor 5 (Klf-5)-deficient hematopoietic stem cells and progenitors fail to engraft after transplantation. In vivo-inducible gain-of-function of Krüppel-like factor 5 in hematopoietic stem cells increases hematopoietic stem cell and progenitor adhesion. Altogether, the data indicate that Krüppel-like factor 5 is indispensable for adhesion, homing, lodging and retention of hematopoietic stem cells and progenitors in the bone marrow through Rab5-dependent post-translational regulation of ß1/ß2 integrins. [Nat Commun] Abstract | Press Release

Oncolytic Adenovirus with Temozolomide Induces Autophagy and Antitumor Immune Responses in Cancer Patients
The combination of oncolytic adenovirus with low-dose temozolomide and metronomic cyclophosphamide increased tumor cell autophagy, elicited antitumor immune responses, and showed promising safety and efficacy. [Mol Ther] Abstract

Clinical-Grade Multipotent Adult Progenitor Cells Durably Control Pathogenic T Cell Responses in Human Models of Transplantation and Autoimmunity
Investigators used allogeneic islet transplantation as a model indication to assess the ability of clinical-grade multipotent adult progenitor cells to control T cell responses that drive immunopathology in human autoimmune disease and allograft rejection. [J Immunol] Abstract

Mesenchymal Stem Cells Engineered to Inhibit Complement-Mediated Damage
Although mesenchymal stem cells (MSC) express molecules, such as membrane cofactor protein, complement decay-accelerating factor, and protectin, which confer protection from complement-mediated lysis, MSC are recruited and activated by anaphylatoxins after transplantation, potentially causing MSC death and limiting therapeutic benefit. Researchers investigated whether engineering MSC to express US2, US3, US6, or US11 HCMV proteins can alter complement recognition, thereby better protecting MSC from complement attack and lysis. [PLoS One] Full Article | Press Release

Improved Methods for Reprogramming Human Dermal Fibroblasts Using Fluorescence Activated Cell Sorting
Researchers described an improved method for the derivation of induced pluripotent stem cell (iPSC) lines using fluorescence activated cell sorting (FACS) to isolate single cells expressing the cell surface marker signature CD13NEGSSEA4POSTra-1-60POS on day 7-10 after infection. FACS derived iPSC lines express common markers of pluripotency, and possess spontaneous differentiation potential in vitro and in vivo. [PLoS One]
Full Article | Press Release

Chimeric Antigen Receptor T Cells with Dissociated Signaling Domains Exhibit Focused Antitumor Activity with Reduced Potential for Toxicity In Vivo
To specify T cells for robust effector function that is selective for tumor but not normal tissue, researchers developed a trans-signaling chimeric antigen receptor (CAR) strategy, whereby T-cell activation signal 1 is physically dissociated from costimulatory signal 2 in two CARs of differing antigen specificity: mesothelin and a-folate receptor. [Cancer Immunol Res] Abstract | Full Article | Press Release

[New Webinar] Discover How to Reduce Variability in Primary Neuron Cultures with NeuroCult SM



REVIEWS
Cell-Based Therapeutics: The Next Pillar of Medicine
Two decades ago, the pharmaceutical industry – long dominated by small-molecule drugs – was revolutionized by the the advent of biologics. Today, biomedicine sits on the cusp of a new revolution: the use of microbial and human cells as versatile therapeutic engines. Here, researchers discuss the promise of this “third pillar” of therapeutics in the context of current scientific, regulatory, economic, and perceptual challenges. [Sci Transl Med] Abstract | Press Release

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.


SCIENCE NEWS

Immunotherapy Showed Promising Antileukemia Activity in Pediatric Patients
Researchers using patients’ own immune cells in an immunotherapy approach called “anti-CD19 chimeric antigen receptor T-cell therapy,” achieved responses in children whose acute lymphocytic leukemia had returned after a bone marrow transplant, according to preliminary results. [Press release from the American Association for Cancer Research (AACR) discussing research presented at the AACR Annual Meeting 2013, Washington]
Press Release

Two-Step Ovarian Cancer Immunotherapy Made from Patients’ Own Tumor Benefits Three Quarters of Trial Patients
As many as three quarters of advanced ovarian cancer patients appeared to respond to a new two-step immunotherapy approach, including one patient who achieved complete remission. The immunotherapy has two steps – a personalized dendritic cell vaccination and adoptive T-cell therapy. The team reports that in the study of 31 patients, vaccination therapy alone showed about a 61 percent clinical benefit, and the combination of both therapies showed about a 75 percent benefit. [Press release from the Perelman School of Medicine at the University of Pennsylvania discussing research presented at the AACR Annual Meeting 2013, Washington]
Press Release

Stem Cells Fill Gaps in Bones
For many patients the removal of several centimeters of bone from the lower leg following a serious injury or a tumor extraction is only the beginning of a long-lasting ordeal. Numerous surgeries are necessary to restore the leg to health, and bone regeneration takes a long time – provided it succeeds at all. Striving to accelerate and to boost the healing process, scientists have now begun deploying autologous stem cells. [Ruhr-Universitaet-Bochum] Press Release | Full Article

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POLICY
Canadian Official to Investigate Allegations That Government Scientists Are Being Muzzled
A simmering dispute over Canadian government rules on how federal researchers communicate with the public and the press has taken an unexpected turn. The country’s information commissioner, Suzanne Legault, confirmed that she has opened an investigation into whether scientists in seven government departments are being muzzled by senior politicians. [Canadian Information Commissioner, Canada] Press Release

As White House Embraces BRAIN Initiative, Questions Linger
For neuroscientist Rafael Yuste, sitting in an ornate White House chamber listening to President Barack Obama heap praise – and some $100 million – on a brain-mapping initiative that he helped hatch was a “luminous” experience. For all the lofty rhetoric, however, the White House didn’t provide many details about how the BRAIN (Brain Research through Advancing Innovative Neurotechnologies) Initiative will accomplish its mission. [White House, United States] Press Release

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BUSINESS

Researchers to Develop Next Generation Immunotherapy for Children with Deadly Solid Tumors
Researchers at the Children’s Hospital of Philadelphia, the University of Pennsylvania, and the Primary Children’s Medical Center of Salt Lake City received a $550,000 collaborative grant to test next generation T-cell immunotherapy strategies in children with neuroblastoma. The awardees were selected within a rigorous peer-review process through a new initiative called ACT FAST (Adoptive Cell Therapy For Adolescent/pediatric Solid Tumor), which is spearheaded by Solving Kids’ Cancer. [Solving Kids’ Cancer] Press Release

GenSight Biologics Raises €32 Million in a Series A Financing for Ophthalmic Gene Therapy
GenSight Biologics announced the closing of a €32 million Series A financing. GenSight will use the funds to develop a gene replacement therapy for Leber’s hereditary optic neuropathy (LHON) and an optogenetic therapy for retinitis pigmentosa. The lead product is expected to enter the clinic in 2013 in LHON patients. [PR Newswire Association LLC] Press Release

ACT Initiates Treatment of Higher-Dosage Cohort in Clinical Trials for Dry Age-Related Macular Degeneration and Stargardt’s Macular Dystrophy
Advanced Cell Technology, Inc. (ACT) announced treatment of patients in the third patient cohort, in each of its two U.S. clinical trials for forms of macular degeneration. The patients were injected with 150,000 human embryonic stem cell-derived retinal pigment epithelial cells, as compared with the 100,000-cell dose used in the previous three patients of the second cohort. [Advanced Cell Technology, Inc.] Press Release

Neuralstem Receives Notice of Allowance of Patent Covering Use of Stem Cells to Treat Amyotrophic Lateral Sclerosis
Neuralstem, Inc. announced that it received a notice of allowance for patent application 12/404,841. This patent covers methods for treating amyotrophic lateral sclerosis (ALS) with expanded spinal cord stem cells, including NSI-566. Neuralstem completed a Phase I safety trial of its NSI-566 stem cells in ALS earlier this year, for which it has also been granted an Orphan Drug Designation. The company plans to initiate Phase II of the trial, pending approval from the Food and Drug Administration. [Neuralstem, Inc.] Press Release

Cellular Biomedicine Group Marks the Launch of Clinical Trial for Knee Osteoarthritis (KOA)
Cellular Biomedicine Group held a ceremony to mark the launch of their clinical trial for haMPC (Human Adipose-derived Mesenchymal Progenitor Cells) therapy for KOA. The Phase I open label clinical research trial will test the safety and efficacy of intra-articular injections of autologous haMPC in order to reduce inflammation and regenerate damaged joint tissues. [PR Newswire Association LLC] Press Release

Dicerna Announces First Patent Grant in Japan to Dicer Substrate siRNA Molecules
Dicerna Pharmaceuticals, Inc. announced that the Japan Patent Office (JPO) has notified City of Hope of its intent to grant a patent from the Rossi patent family (JP Application Number 2007-504009). This patent family pertains to Dicer Substrate siRNA (DsiRNA) containing therapeutics, and the new allowance applies to 40 claims that confer exclusivity around DsiRNA compositions for reducing mammalian target gene expression, regardless of their specific sequence. [Dicerna Pharmaceuticals, Inc.] Press Release

Promising Stem Cell Therapy for Leukemia Patients
Leukemia patients receive a bone marrow transplant, which allows them to build a “new” immune system. However, this immune system not only attacks cancer cells but healthy tissue too. Special antibodies will be used to protect healthy tissue in future. [Fraunhofer-Gesellschaft] Press Release

Cord Blood Bank in Hong Kong Receives FACT Accreditation, Reflects High Quality Standards for Cord Blood Banking and Unit Administration
The Foundation for the Accreditation of Cellular Therapy (FACT) has accredited its first cord blood bank in Hong Kong. FACT accreditation demonstrates commitment to promote quality medical practices, laboratory processes, and banking to achieve consistent production of high quality placental and umbilical cord blood units for administration. The Hong Kong Red Cross Catherine Chow Cord Blood Bank, directed by Dr. Che Kit Lin, received accreditation and is accredited for unrelated donations. [Foundation for the Accreditation of Cellular Therapy] Press Release

China Cord Blood Corporation Received AABB Accreditation
China Cord Blood Corporation announced that the cord blood bank operated by its Beijing subsidiary has been granted the AABB Accreditation, as a certification of its quality excellence of providing cord blood processing and storage services. [China Cord Blood Corporation] Press Release

UW Stem Cell Pioneer Thomson Earns Innovation Award
Thomson, who first derived human embryonic stem cells in 1998 and reprogrammed human skin cells into induced pluripotent stem cells in 2007, has earned the McEwen Award for Innovation from the International Society for Stem Cell Research. He is being honored for his work in isolating human pluripotent stem cells, which revealed new possibilities for basic biology, drug discovery and cell-based therapies. [University of Wisconsin-Madison] Press Release



REGULATORY

Food and Drug Administration (United States)

Center for Biologics Evaluation and Research eSubmitter Pilot Evaluation Program for Investigational New Drug Applications (FR Doc No: 2013-08012)

User Fees and Refunds for Premarket Approval Applications and Device Biologics License Applications; Guidance for Industry and Food and Drug Administration Staff; Availability (FR Doc No: 2013-07577)

Draft Guidance for Industry and Food and Drug Administration Staff; Glass Syringes for Delivering Drug and Biological Products: Technical Information to Supplement International Organization for Standardization Standard 11040-4; Availability (FR Doc No: 2013-07685)

Vaccines and Related Biological Products Advisory Committee; Notice of Meeting (FR Doc No: 2013-07961)

Society of Clinical Research Associates-Food and Drug Administration: Food and Drug Administration Clinical Trial Requirements, Regulations, Compliance, and Good Clinical Practice (FR Doc No: 2013-07894)


Therapeutic Goods Administration (Australia)

Documents Released Under Section 11C of the Freedom of Information Act 1982



EVENTS

NEW The Jackson Laboratory: Modeling Human Malignancy with Patient-Derived Xenograft Cancer Models
April 18, 1:00PM (ET in US)
Webinar

NEW Select Biosciences: Translational Medicine Business Summit
June 6-7, 2013
Hamburg, Germany

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



JOB OPPORTUNITIES

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)


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