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Pulmonary Cell News| Cell Therapy News 18.12 April 10, 2017 | |
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TOP STORYOptimizing Cardiac Delivery of Modified mRNA Investigators elucidated parameters by testing different nucleotide modifications, modified mRNA (modRNA) doses, and transfection reagents both in vitro and in vivo in cardiac cells and tissue. Their results indicated that optimal cardiac delivery of modRNA is with N1-Methylpseudouridine-5′-Triphosphate nucleotide modification and achieved using 0.013 μg modRNA/mm2/500 cardiomyocytes transfected with positively charged transfection reagent in vitro and 100 μg/mouse heart sucrose-citrate buffer in vivo. [Mol Ther] Abstract | |
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PUBLICATIONS(Ranked by impact factor of the journal)CXCR5-overexpressing mesenchymal stromal cells (MSCs) (MSCCXCR5) were generated, which retained their abilities of proliferation, differentiation, and immunomodulation. Furthermore, MSCCXCR5 showed significantly increased migrating ability toward CXCL13. Importantly, systemic infusion of MSCCXCR5 dramatically suppressed contact hypersensitivity (CHS) in mice, as evidenced by decreased levels of inflammatory cell infiltration and pro-inflammatory cytokines production. [Mol Ther] Abstract Scientists investigated the protective effect of pretreatment with SIRT1 activator SRT1720 on aged human mesenchymal stem cells (hMSCs). Transplantation with SRT1720 pretreated aged hMSCs achieved increased left ventricular ejection fraction and angiogenesis with reduced fibrosis of rat hearts as compared to DMSO pretreated group 28 days following myocardial infarction. [Cell Death and Dis] Full Article Researchers explored the role of systemically delivered induced neural stem cells (iNSCs) in complement activation following central nervous system injury. Their data showed that iNSC grafts decreased the levels of sera C3a and C5a and down-regulated the expression of C3d, C9, active Caspase-3 and Bax in the brain, kidney and lung tissues of closed head injury mice. [Sci Rep] Full Article Survival of Human Embryonic Stem Cells Implanted in the Guinea Pig Auditory Epithelium The authors evaluated the survival of human embryonic stem cells constitutively expressing GFP in deaf guinea pig cochleae that were pre-conditioned to reduce potassium levels. GFP-positive cells could be detected in the cochlea for at least seven days after the injection. The cells appeared spherical or irregularly shaped, and some were aggregated. [Sci Rep] Full Article This Phase I clinical trial tested whether transplanting autologous cell–sheets derived from skeletal muscle is feasible, safe, and effective for treating severe congestive heart failure. Scaffold‐free cell sheets of 3 to 9×108 cells derived from autologous muscle were transplanted over the LV free wall via left thoracotomy, without additional interventional treatments. [J Am Heart Assoc] Full Article Human Kidney-Derived Cells Ameliorate Acute Kidney Injury without Engrafting into Renal Tissue Researchers evaluated the impact of the CD133+ cells on renal function by undertaking longitudinal measurements of the glomerular filtration rate using a novel transcutaneous device. Using histological assays, they assessed whether the human kidney cells could promote renal regeneration, and if this was related to their ability to integrate into the damaged kidneys. Their results showed that both CD133+ and CD133− cells improve renal function and promote renal regeneration to a similar degree. [Stem Cells Transl Med] Full Article Fabrication and Characterization of a Decellularized Bovine Tendon Sheet for Tendon Reconstruction Scientists developed a new decellularization protocol, including physical methods and enzymatic solutions for processing bovine Achilles tendons, and produced a decellularized bovine tendon sheet (DBTS) scaffold for tendon reconstruction. The decellularization effectiveness was demonstrated by DNA quantification and histological qualification. When implanted into rat subcutaneous tissue, the DBTS scaffold displayed excellent histocompatibility in vivo. [J Biomed Mater Res A] Abstract Engineered Stem Cell Niche Matrices for Rotator Cuff Tendon Regenerative Engineering Scientists investigated structured matrices mimicking the tendon microenvironment as cell delivery vehicles in a rat rotator cuff (RC) tear model. RC injuries augmented with a matrix delivering rat mesenchymal stem cells (rMSCs) showed enhanced regeneration over suture repair alone or repair with augmentation, at 6 and 12-weeks post-surgery. The local delivery of rMSCs led to increased mechanical properties and improved tissue morphology. [PLoS One] Full Article Dual-Phase Iontophoresis for the Delivery of Antisense Oligonucleotides Investigators improved previous results of iontophoresis to deliver RNA interference-based oligonucleotides. By using a electromechanics-based approach, they were able to devise a two-phase solution that separated the buffering solution from the antisense oligonucleotide solution. In vivo studies demonstrated short-term bioavailability of at least 24 hours, a lack of chemical or thermal injury, a lack of interference in the healing of a corneal injury, and an antisense effect till at least day 7, but not day 14. [Nucleic Acid Ther] Abstract | |
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REVIEWSTherapeutic Gene Editing: Delivery and Regulatory Perspectives The authors address current research trends and delivery strategies for gene editing-based therapeutics in non-clinical and clinical settings and considers the associated regulatory issues. [Acta Pharmacol Sinica] Abstract Restoring Heart Function and Electrical Integrity: Closing the Circuit Scientists review the state-of-art in the area of myocardial electrical integrity, while briefly describing the biological principles underlying the heart electrical/conduction system and how this system can be disrupted in heart disease. Suggestions regarding targets for future studies are also presented. [npj Regenerative Medicine] Full Article Visit our reviews page to see a complete list of reviews in the cell therapy research field. | |
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SCIENCE NEWSKite Pharma, Inc. announced new data presentations from preclinical studies related to KITE-585, a fully human anti-B cell maturation antigen chimeric antigen receptor (CAR) T-cell product candidate for the treatment of multiple myeloma. [Press release from Kite Pharma, Inc. discussing research presented at the 2017 American Association of Cancer Research (AACR) Annual Meeting, Washington D.C.] Press Release Immunotherapy for Ovarian Cancer Shows Promise in Mouse Model, but Hurdles Remain Fred Hutchinson Cancer Research Center immunotherapy researchers Drs. Kristin Anderson and Phil Greenberg and their colleagues are working on ways to tweak their team’s early successes with T-cell therapy for leukemia to apply to solid tumors. In a presentation, Anderson described her preclinical results working toward T-cell therapy for ovarian tumors — and the hurdles any clinical version of this therapy will need to overcome. [Press release from Fred Hutchinson Cancer Research Center discussing research presented at the 2017 American Association of Cancer Research (AACR) Annual Meeting, Washington D.C.] Press Release | Abstract Spark Therapeutics announced updated preliminary data from ten infused participants in the ongoing Phase I/II clinical trial of investigational SPK-9001 for hemophilia B. All participants have experienced consistent and sustained increases in factor IX activity following administration of the investigational therapy. [Press release from Spark Therapeutics discussing research presented at the Hemostasis and Thrombosis Research Society (HTRS) 2017 Scientific Symposium, Arizona] Press Release | |
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INDUSTRY NEWSSylvester Comprehensive Cancer Center and Syapse announced that they will team up to launch a new precision medicine initiative at Sylvester to help make personalized cancer care in South Florida and throughout the Southeastern U.S. more effective. [Syapse] Press Release Evotec Achieves First Milestone in Diabetes Alliance with Sanofi Evotec AG announced that its strategic alliance with Sanofi in the field of diabetes has reached an important milestone. The goal of this collaboration is to develop a beta cell replacement therapy based on functional beta cells derived from human stem cells. [Evotec AG] Press Release Mesoblast Limited announced that the Phase III trial of its allogeneic mesenchymal precursor cell (MPC) product candidate MPC-150-IM in patients with moderate to advanced chronic heart failure was successful in the pre-specified interim futility analysis of the efficacy endpoint in the trial’s first 270 patients. It is expected that the trial will enroll in total approximately 600 patients. [Mesoblast Limited] Press Release (Download) U.S. Stem Cell, Inc.’s MARVEL Phase II/III Trial Receives Reactivation Status with the FDA U.S. Stem Cell, Inc. has received reactivation status of the MARVEL Phase II/III trial with the Food and Drug Administration (FDA). [U.S. Stem Cell, Inc.] Press Release Positive Results from RepliCel’s RCS-01 Phase I Skin Trial Are the Company’s Most Compelling to Date RepliCel Life Sciences Inc. reported statistically and clinically significant positive data from the interim analysis of its Phase I study evaluating RCS-01 for the treatment of aging and sun-damaged skin. [RepliCel Life Sciences Inc.] Press Release Cytori Receives U.S. FDA Approval for Burn Clinical Trial Related to BARDA Contract Cytori Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved an Investigational Device Exemption for a pilot clinical trial to evaluate Cytori Cell Therapy™ in patients with thermal burn injury. This trial, named the RELIEF trial, is a continuation of Cytori’s ongoing research and development efforts under its contract with the Biomedical Advanced Research and Development Authority (BARDA), a division of the U.S. Department of Health and Human Services. [Cytori Therapeutics, Inc.] Press Release Indian FDA Approves the First Autologous Dendritic Cell-Based Immuno-Oncology Product, APCEDEN® APAC Biotech was granted with a commercial license by Indian FDA (CDSCO – Central Drugs Standard Control Organization) to market product, APCEDEN®, a Dendritic cell-based autologous immuno-oncology product for four cancer indication namely prostate, ovarian, colorectal and non small cell lung carcinoma. [APAC Biotech (PR Newswire Association, LLC.)] Press Release Kite Pharma, Inc. announced that it won the Clinical Trial Result of the Year award for ZUMA-1, its pivotal CAR-T trial of axicabtagene ciloleucel, in patients with refractory aggressive B-cell non-Hodgkin lymphoma at the Clinical and Research Excellence Awards. The award recognizes clinical achievements in the pharmaceutical industry and contribution to the advancement of therapies for unmet medical needs. [Kite Pharma, Inc.] Press Release | |
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POLICY NEWSCongress and FDA Nominee Heap Love on ‘Adaptive Trials’ As President Donald Trump’s nominee to head FDA, Gottlieb sat before Republican lawmakers hungry for promises of “shorter time frames” for drug and device approvals, and expressed his zeal—repeatedly—for adaptive trial designs. If confirmed to be FDA’s head, as expected, Gottlieb suggested he’d promote wider use of the approach. [ScienceInsider] Editorial Geophysics Society Hopes to Define Sexual Harassment as Scientific Misconduct A major U.S.-based scientific society is on the verge of expanding its definition of research misconduct to include sexual harassment. The American Geophysical Union is making the change to emphasize the serious threat that harassment and other forms of discrimination pose to the scientific enterprise, say society officials. [ScienceInsider] Editorial
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REGULATORYFDAAntibody Mediated Rejection in Kidney Transplantation; Public Workshop; Request for Comments (FR Doc. No:2017-06700) Notice Food and Drug Administration/Xavier University Medical Device Conference (MedCon) (FR Doc. No:2017-06699) Notice
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EVENTSNEW Gordon Research Conferences: Tissue Repair & Regeneration Visit our events page to see a complete list of events in the community.
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JOB OPPORTUNITIESNEW Research Fellow – Functional Roles of Stem Cell Derived Extracellular Vesicles (Mayo Clinic) Executive Director – Global CAR-T Supply (Celgene Corporation) Postdoctoral Associate – Gene Therapy (University of Massachusetts Medical School) Postdoctoral Fellow – Gene and Stem Cell Therapy Program (Centenary Institute) Postdoctoral Research Fellow – Preclinical Models of Cancer Immunotherapy (Providence Cancer Center) 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 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 18.12 | Apr 10 2017
