Cell Therapy News 18.09 March 20, 2017 | |
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TOP STORYThe authors designed a tumor extracellular acidity-responsive chimeric peptide with geometrical shape switch for enhanced tumor internalization and photodynamic therapy. Both in vitro and in vivo studies demonstrated that this acidity-triggered geometrical shape switch endowed chimeric peptide with accelerated internalization in tumor cells, prolonged accumulation in tumor tissue, enhanced photodynamic therapy, and minimal side effects. [ACS Nano] Abstract | Graphical Abstract | |
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PUBLICATIONS(Ranked by impact factor of the journal)Angiogenic Mechanisms of Human CD34+ Stem Cell Exosomes in the Repair of Ischemic Hindlimb Scientists investigated the mechanisms of membrane-bound nano-vesicles called exosomes (CD34Exo)-mediated ischemic tissue repair and therapeutic angiogenesis by studying their miRNA content and uptake. When injected into mouse ischemic hindlimb tissue, CD34Exo, but not the CD34exo-depleted conditioned media, mimicked the beneficial activity of their parent cells by improving ischemic limb perfusion, capillary density, motor function and their amputation. [Circ Res] Abstract Researchers packaged secreted factors from human bone marrow-derived mesenchymal stem cells (MSC) into Poly(lactic-co-glycolic acid) PLGA microparticles and then coated them with MSC membranes. Synthetic MSC (synMSC) promoted cardiomyocyte functions and displayed cryopreservation and lyophilization stability in vitro and in vivo. In a mouse model of acute myocardial infarction, direct injection of synMSC promoted angiogenesis and mitigated left ventricle remodeling. [Circ Res] Abstract Scientists showed that nanoparticle geometry can be harnessed to mediate bone regeneration in a rat cranial defect model. They successfully synthesized hydroxyapatite nanoparticles (NPs) with well-defined morphologies using a modified liquid–solution–solid method. The NPs showed differential effects on stem cell behaviors such as particle uptake, autophagy activation and osteogenic differentiation. [Nanoscale] Abstract | Graphical Abstract Phase I Escalating-Dose Trial of CAR-T Therapy Targeting CEA+ Metastatic Colorectal Cancers The authors investigated chimeric antigen receptor T (CAR-T) therapy targeting carcino-embryonic antigen (CEA) positive colorectal cancer (CRC) patients with metastases to evaluate its safety and efficacy. Five escalating dose levels of CAR-T were applied in ten CRC patients. Their data showed that severe adverse events related to CAR-T therapy were not observed. [Mol Ther] Abstract Researchers generated and evaluated two novel and distinct ssRNA molecules (Immunogenic Cell- killing RNA (ICR)2 and ICR4). ICR2 and ICR4 differentially stimulated cell death and pattern recognition receptor signaling pathways and induced different patterns of cytokine expression in cancer and innate immune cells. ICR2 and ICR4 inhibited in vivo tumor growth as effectively as polyI:C. [Mol Ther] Abstract Researchers found that a single administration of EVs improved functional recovery, fiber tract integrity, axonal sprouting and white matter repair markers in an experimental animal model of subcortical stroke. [Sci Rep] Full Article Lentiviral Vectors Can Be Used for Full-Length Dystrophin Gene Therapy Investigators report a novel approach for delivering large transgenes in lentiviruses, in which they demonstrated proof-of-concept for a ‘template-switching’ lentiviral vector that harnesses recombination events during reverse-transcription. During this work, they discovered that a standard, unmodified lentiviral vector was efficient in delivering full-length dystrophin to target cells, within a total genomic load of more than 15,000 base pairs. [Sci Rep] Full Article The authors sought to determine the safety and efficacy of allogeneic cardiosphere-derived cells (CDCs) in a large animal (canine) model of spontaneous dilated cardiomyopathy (DCM). Canine CDCs (cCDCs) were grown from a donor dog heart. Similar to human CDCs, cCDCs express CD105 and are slightly positive for c-kit and CD90. Thirty million of allogeneic cCDCs were infused into the coronary vessels of Doberman pinscher dogs with spontaneous DCM. [J Cell Mol Med] Full Article The authors showed the long-term updated outcomes of a multicenter retrospective study which analyzed 843 patients with myelodysplastic syndrome who underwent transplantation with an HLA-identical sibling donor with either reduced-intensity conditioning in 213 patients, or standard myeloablative conditioning in 630 patients. [Bone Marrow Transplant] Abstract | |
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REVIEWSCharged-Particle Therapy in Cancer: Clinical Uses and Future Perspectives Researchers review several randomized trials of charged-particle therapies that are ongoing, with results that will enable selective delivery to patients who are most likely to benefit from them. They also discuss aspects related to radiobiology, including the immune response and hypoxia, which will need to be taken into consideration in future randomized trials to fully exploit the potential of charged particles. [Nat Rev Clin Oncol] Abstract Ex Vivo T-Cell Depletion in Allogeneic Hematopoietic Stem Cell Transplant: Past, Present and Future The authors discuss the clinical outcome of current and emerging strategies of T-cell depletion in allogeneic hematopoietic stem cell transplant that have developed during the modern transplantation era, focusing specifically on ex vivo strategies that target selected T-cell subsets. [Bone Marrow Transplant] Full Article Visit our reviews page to see a complete list of reviews in the cell therapy research field. | |
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SCIENCE NEWSAudentes Therapeutics, Inc. announced that data from RECENSUS, a medical chart review of patients with XLMTM, will be presented. These data provide new insights into the significant medical burden for children with XLMTM, their families and caregivers. [Press release from Audentes Therapeutics, Inc. discussing research presented at the 2017 Muscular Dystrophy Association (MDA) Scientific Conference, Arlington] Press Release | |
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INDUSTRY NEWSMedicenna BioPharma Inc. announced that it has entered into a sponsored research agreement with The University of Texas MD Anderson Cancer Center to develop next generation fusion protein therapeutics targeting the IL-4 Receptor. [Medicenna BioPharma Inc.] Press Release Ferring Pharmaceuticals and Brazil-based Instituto de Ciências Farmacêuticas (ICF) announced a research collaboration aimed at developing a mucoadhesive rectal delivery system to treat anal fissures and to improve quality of care for patients with inflammatory bowel disease (IBD). [Ferring Pharmaceuticals] Press Release AMO Pharma Limited announced it has entered a collaboration agreement with Ranedis Pharmaceuticals to progress the development of RND-001 for rare genetic diseases, including certain lysosomal storage disorders and other diseases affecting the central nervous system. [AMO Pharma Limited (PR Newswire Association LLC.)] Press Release Allergan plc and Editas Medicine, Inc. announced that Allergan’s wholly-owned subsidiary, Allergan Pharmaceuticals International Limited, and Editas Medicine have entered into a strategic research and development alliance under which Allergan will receive exclusive access and the option to license up to five of Editas Medicine’s genome-editing ocular programs, including its lead program for Leber Congenital Amaurosis, which is currently in pre-clinical development. [Allergan plc] Press Release Pharnext SA announced that the first two patients have entered the international Phase III Extension Study PLEO-CMT-FU of PXT3003 at La Timone University Hospital. PXT3003 is Pharnext’s lead PLEODRUGâ„¢ for the treatment of patients with mild-to-moderate Charcot-Marie-Tooth Disease Type 1A, a rare and debilitating inherited peripheral neuropathy for which there are no satisfactory approved treatments available. [Pharnext SA] Press Release BioLineRx Ltd. reported partial results data from its open-label Phase II trial for BL-8040 as a novel monotherapy approach for the mobilization and collection of blood forming stem and progenitor cells from the peripheral blood. [BioLineRx Ltd.] Press Release Mesoblast Limited announced 36-month results from the randomized, placebo-controlled 100-patient Phase II trial of its proprietary allogeneic Mesenchymal Precursor Cells (MPCs) in patients with chronic low back pain due to intervertebral disc degeneration. A single intra-discal injection of 6 million MPCs resulted in meaningful improvements in both pain and function that were durable for at least 36 months. [Mesoblast Limited] Press Release (Download) NightstaRx Ltd. announces that the company has commenced enrolling and dosing subjects in a Phase I/II clinical trial to treat patients with X-Linked Retinitis Pigmentosa (XLRP). This is the first clinical trial of its type in XLRP worldwide, and has been progressed from licensing to clinical trial in a period of just 18 months. [NightstaRx Ltd.] Press Release RepliCel’s Phase I Clinical Trial for Hair Loss Succeeds in Meeting Primary Endpoints RepliCel Life Sciences Inc. announced the successful completion of its first-in-human clinical study of the company’s autologous cell therapy for the treatment of androgenetic alopecia (pattern baldness). [RepliCel Life Sciences Inc.] Press Release Longeveron Achieves Milestone in Groundbreaking Stem Cell Trial for Alzheimer’s Disease Longeveron announced the successful completion of enrollment in the first phase of its clinical research trial of Longeveron allogeneic human mesenchymal stem cells to treat Alzheimer’s disease. [Longeveron (PR Newswire Association LLC.)] Press Release Fate Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has cleared an investigational new drug application for FATE-NK100, the company’s first-in-class adaptive memory natural killer cell product candidate. The company expects a first-in-human clinical trial of FATE-NK100 for advanced acute myeloid leukemia to open enrollment at the Masonic Cancer Center, University of Minnesota following approval of the center’s institutional review board. [Fate Therapeutics, Inc.] Press Release Kadimastem Granted a Patent in the US for Treatment of Neurodegenerative Diseases Kadimastem announced that it has been granted a patent from the United States Patent and Trademark Office for its technology in the field of cell based treatment for diseases of the nervous system. The United States patent joins additional patents granted in Israel and Europe. [Kadimastem] Press Release Arbutus Biopharma Corporation announced it has licensed to Alexion Pharmaceuticals, Inc. its proprietary lipid nanoparticle (LNP) technology for exclusive use in one of Alexion’s rare disease programs. This transaction enables Alexion to address delivery for therapeutic application of messenger RNA (mRNA) and rapidly enter clinical development with its mRNA product candidate. [Arbutus Biopharma Corporation] Press Release Stem Cell, Regenerative Medicine Leaders Join Forces to Advance Canada’s Position in the Field Stem cell science belongs to Canada and it is powering regenerative medicine. Leaders from across this emerging sector have joined forces to advance the field, through the newly created Regenerative Medicine Alliance of Canada. [University of Toronto] Press Release | |
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POLICY NEWSJailed Iranian Researcher’s Health Worsening Rapidly An Iranian researcher jailed in Tehran for the last 11 months is in declining health after spending more than two months on hunger strike. This month, researchers around the world made urgent appeals for his release. [Nature News] Editorial Trump’s NIH Budget May Include Reducing Overhead Payments to Universities The Trump administration may be planning to help pay for a massive 18% cut to the National Institutes of Health (NIH) by slashing payments to universities and research institutes for overhead costs, ScienceInsider has learned. [ScienceInsider] Editorial
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REGULATORYFDAIdentification and Characterization of the Infectious Disease Risks of Human Cells, Tissues, and Cellular and Tissue-Based Products; Public Workshop; Correction (FR Doc. No:2017-05417) Notice Agency Information Collection Activities; Proposed Collection; Comment Request; Medical Devices; Reports of Corrections and Removals (FR Doc. No:2017-05415) Notice Agency Information Collection Activities; Proposed Collection; Comment Request; Guidance for Industry on Pharmacogenomic Data Submissions (FR Doc. No:2017-05293) Notice
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EVENTSNEW NYSTEM 2017 NEW Biomed 2017: 22nd International Biomedical Science & Technology Symposium Visit our events page to see a complete list of events in the community.
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JOB OPPORTUNITIESPostdoctoral Fellow – Gene and Stem Cell Therapy Program (Centenary Institute) Research Scientist – Immunotherapies for Cancer (GammaDelta Therapeutics Limited) Postdoctoral Research Fellow – Preclinical Models of Cancer Immunotherapy (Providence Cancer Center) Associate/Full Professor – Loretta Rogers Chair in Immunobioengineering (University of Toronto) Medical Director – Clinical Research Physician (Celgene Corporation) Postdoctoral Fellow – Various Projects (University of Oklahoma) GMP Manufacturing Director – Cell and Gene Therapy (Fred Hutchinson Cancer Research Center) Assistant Professor – Molecular Therapeutics of Cancer (Dartmouth College) Assistant or Associate Member – Stem CellGene Therapy (Fred Hutchinson Cancer Research Center) Recruit Top Talent: Reach potential candidates by posting your organization’s career opportunities on the Connexon Creative Job Board at no cost.
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Home Cell Therapy News Volume 18.09 | Mar 20 2017