Cell Therapy News Volume 19.40 | Dec 3 2018

TOP STORY

Risk Factors Affecting Outcome of Unrelated Cord Blood Transplantation for Children with Familial Hemophagocytic Lymphohistiocytosis

Researchers analyzed the outcomes of 118 children with familial hemophagocytic lymphohistiocytosis undergoing single‐unit umbilical cord blood transplantation performed from 1996 to 2014. [Br J Haematol] Abstract

(Ranked by impact factor of the journal)

Mesenchymal Stem Cell Administration Attenuates Colon Cancer Progression by Modulating the Immune Component within the Colorectal Tumor Microenvironment

Treatment with bone marrow-derived mesenchymal stem cells significantly reduced both cancer initiation and cancer progression by increasing the number of tumor-free animals as well as decreasing the number and the size of the tumors by half, thereby extending their lifespan. [Stem Cells Transl Med] Full Article

Long-Term Therapeutic Effect of Cell Therapy on Improvement in Erectile Function in a Rat Model with Pelvic Neurovascular Injury

A dual neurovascular injured erectile dysfunction model was established in athymic rats by crushing the bilateral cavernous nerves and ligations of the bilateral internal pudendal nerve-vessel bundles. At the time of defect creation, three different types of human cell populations, umbilical vein endothelial cells, adipose derived stem cells and amniotic fluid derived stem cells, were injected intracavernously into the penile tissue. [BJU Int] Abstract

Understanding the Role of Mesenchymal Stem Cells in Urinary Bladder Regeneration — A Preclinical Study on a Porcine Model

Scientists evaluated the role of adipose-derived stem cells in regeneration of clinically significant urinary bladder wall defect in a large animal model. [Stem Cell Res Ther] Full Article

Characterization, Recellularization, and Transplantation of Rat Decellularized Testis Scaffold with Bone Marrow-Derived Mesenchymal Stem Cells

In vivo implant of the scaffolds with or without mesenchymal stem cells (MSCs) showed that appropriate positions for transplantation were the mesentery and liver and the scaffolds could induce donor-loaded MSCs or host migrating cells to differentiate to the cells with phenotype of the sertoli- and leydig-like cells. [Stem Cell Res Ther] Full Article

Effect of Pretreatment with the NADPH Oxidase Inhibitor Apocynin on the Therapeutic Efficacy of Human Placenta-Derived Mesenchymal Stem Cells in Intracerebral Hemorrhage

Researchers suggested apocynin, a well-known nicotinamide adenine dinucleotide phosphate (NADPH) oxidase inhibitor, as a novel preconditioning regimen to enhance the therapeutic efficacy of mesenchymal stem cells in intracerebral hemorrhage (ICH). Rat ICH models were made using bacterial collagenase. 24 hours after ICH induction, the rats were randomly divided into apocynin-preconditioned MSC-treated, naïve MSC-treated and control groups. [Int J Mol Sci] Full Article | Graphical Abstract

The Effect of Herpes Simplex Virus Vector-Mediated Gene Therapy of Protein Phosphatase 1α on Bladder Overactivity and Nociception

Using adult female Sprague-Dawley rats, non‐replicating herpes simplex virus vectors carrying PP1α or green fluorescent protein were injected into the bladder wall. At one week after vector inoculation, cystometry and Western blot assay were performed, whereas the other experiments were performed at two weeks after vector inoculation. [Neurourol Urodyn] Abstract

Biomechanical Evaluation of Tendon Regeneration with Adipose-Derived Stem Cell

Flexor digitorum superficialis tendon lesion was performed on both legs of eleven New Zealand rabbits and treated as follows: suture alone, suture associated with adipose‐derived stem cells or without suture. At four weeks after the tendon surgery, the animals were euthanized, and the tendon evaluated. [J Orthop Res] Abstract

Transparenchymal Renal Pelvis Injection of Recombinant AAV9 Vectors Is a Practical Approach for Gene Delivery in the Kidney

To find an effective gene delivery approach in the kidney, investigators developed transparenchymal renal pelvis injection. Using AAV9 vectors, they evaluated the gene delivery efficiency and safety of this administration method. [Hum Gene Ther Methods] Abstract

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Renewed Assessment of the Risk of Emergent Advanced Cell Therapies to Transmit Neuroproteinopathies

The authors assess the prospective global dissemination of advanced cell therapies founded on transplantation or exposure to allogeneic human cells, recap lessons learned from the historical precedents of Creutzfeldt-Jakob disease transmission and review recent advances and current limits in understanding of prion and other neurodegenerative disease prion-like susceptibility and transmission. [Acta Neuropathol] Abstract

Entering the Modern Era of Gene Therapy

While the strategies being translated from the lab to the clinic are numerous, this review focuses on the clinical research that has forged the gene therapy field as it currently stands. [Annu Rev Med] Abstract

Gene Therapy for Lysosomal Storage Diseases and Peroxisomal Diseases

The author provides an overview of recently developed gene therapies for various lysosomal storage diseases and peroxisomal diseases, including the results of clinical trials, with an emphasis on the benefits of this approach for these diseases. [J Hum Genet] Abstract

Visit our reviews page to see a complete list of reviews in the cell therapy research field.

Kite Announces Two-Year Data for Yescarta^® (Axicabtagene Ciloleucel) in Patients with Refractory Large B-Cell Lymphoma

Kite announced two-year efficacy and safety data from the pivotal ZUMA-1 trial of Yescarta^® in patients with refractory large B-cell lymphoma. With a minimum follow-up of two years after a single infusion of Yescarta, 39 percent of patients were in an ongoing response. [Press release from Gilead discussing research presented at the Annual Meeting of the American Society of Hematology, San Diego] Press Release

bluebird bio and Celgene Corporation Present Initial Data from Ongoing Phase I Clinical Study of Next-Generation Anti-BCMA CAR T Cell Therapy bb21217 in Patients with Relapsed/Refractory Multiple Myeloma

bluebird bio, Inc. and Celgene Corporation announced initial data from the ongoing Phase I clinical study of bb21217, an investigational next-generation anti-BCMA CAR T cell therapy being studied in patients with relapsed/refractory multiple myeloma. [Press release from bluebird bio, Inc. discussing research presented at the Annual Meeting of the American Society of Hematology, San Diego] Press Release

uniQure Announces Long-Term Clinical Data from Ongoing Phase I/II Trial of AMT-060 and Confirms Dose for AMT-061 Pivotal Study in Hemophilia B

uniQure N.V. announced updated results from its ongoing Phase I/II trial of AMT-060, and provided an update on AMT-061, the company’s next-generation gene therapy candidate currently in late-stage clinical development for patients with hemophilia B. [Press release from uniQure N.V. discussing research presented at the Annual Meeting of the American Society of Hematology, San Diego] Press Release

Allogene Therapeutics Presents Data from Preclinical Study of ALLO-715 (Anti-BCMA) Demonstrating Allogeneic CAR T Potential in Multiple Myeloma

Allogene Therapeutics, Inc. announced results of a preclinical study of ALLO-715, an AlloCAR T therapy targeting B-cell maturation antigen (BCMA) in development by Allogene. [Press release from Allogene Therapeutics, Inc. discussing research presented at the Annual Meeting of the American Society of Hematology, San Diego] Press Release

Fate Therapeutics Secures Exclusive Option to Novel Humanized Anti-BCMA CAR Constructs for Development of iPSC-derived Cell Products

Fate Therapeutics, Inc. announced that it has secured an exclusive option to an intellectual property portfolio owned by the Max Delbrück Center for Molecular Medicine covering novel humanized chimeric antigen receptor (CAR) constructs that uniquely and specifically bind B-cell Maturation Antigen (BCMA). [Fate Therapeutics, Inc.] Press Release

Novartis Announces FDA Filing Acceptance and Priority Review of AVXS-101, a One-Time Treatment Designed to Address the Genetic Root Cause of SMA Type 1

Novartis announced that the FDA has accepted the company’s Biologics License Application for AVXS-101, now known as ZOLGENSMA^®, an investigational gene replacement therapy for the treatment of spinal muscular atrophy Type 1. [Novartis AG] Press Release

Fate Therapeutics Announces FDA Clearance of Landmark IND for FT500 iPSC-Derived, Off-the-Shelf NK Cell Cancer Immunotherapy

Fate Therapeutics, Inc. announced that the FDA has allowed its Investigational New Drug (IND) Application for FT500, the company’s universal, off-the-shelf natural killer (NK) cell product candidate derived from a clonal master induced pluripotent stem cell (iPSC) line. [Fate Therapeutics, Inc.] Press Release

Addressing the Sector Skills Gap with First Apprenticeship Program for Advanced Therapies

The Cell and Gene Therapy Catapult launched the first Advanced Therapy Medicinal Products (ATMP) focused apprenticeship program developed in partnership with The Medicine Manufacturing Industry Partnership and aimed at building the skills base needed to support the growing UK industry in the development of ATMPs. [The Cell and Gene Therapy Catapult] Press Release

Editas Medicine Announces FDA Acceptance of IND Application for EDIT-101

Editas Medicine, Inc. announced the FDA has accepted the company’s Investigational New Drug (IND) application for EDIT-101, an experimental CRISPR genome editing medicine being investigated for the treatment of Leber Congenital Amaurosis type 10. [Editas Medicine, Inc.] Press Release

Editas Medicine Announces Pre-Clinical Data Supporting Novel Approach for Treatment of Sickle Cell Disease and Beta-Thalassemia

Editas Medicine, Inc. announced results from experiments to demonstrate expanded CRISPR genome editing strategies in hematopoietic stem cells for the treatment of sickle cell disease and beta-thalassemia. [Editas Medicine, Inc.] Press Release

Celgene Corporation and bluebird bio Complete Enrollment of Pivotal KarMMa Study of Anti-BCMA CAR T Cell Therapy bb2121 in Patients with Relapsed and Refractory Multiple Myeloma

Celgene Corporation and bluebird bio, Inc. announced the completion of enrollment for the KarMMa pivotal study of bb2121, the companies’ lead investigational anti-BCMA CAR T cell therapy candidate for patients with relapsed and refractory multiple myeloma. [Celgene Corporation] Press Release

Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy (RMAT) and Fast Track Designations for RP-L102 Gene Therapy for Fanconi Anemia

Rocket Pharmaceuticals, Inc. announced that the FDA has granted RMAT and Fast Track designations to RP-L102, the company’s lentiviral vector-based gene therapy for the treatment of Fanconi anemia. [Rocket Pharmaceuticals, Inc.] Press Release

Vybion Receives Orphan Drug Designation for INT41 in Huntington’s Disease

Vybion Inc. announced that the FDA has granted orphan drug designation for INT41, the company’s Intrabody delivered with AAV for the treatment of Huntington’s disease. [Vybion Inc. (Business Wire, Inc.)] Press Release

Celyad Doses First mCRC Patient in the Phase I alloSHRINK Trial Evaluating Non-Gene Edited Allogeneic CAR-T Candidate, CYAD-101

Celyad announced the injection of the first patient in the Phase I alloSHRINK trial evaluating the company’s non-gene edited allogeneic CAR-T therapy, CYAD-101, administered concurrently with FOLFOX chemotherapy in the treatment of patients with unresectable metastatic colorectal cancer (mCRC). [Celyad] Press Release

Cytori Announces Japan Class III Medical Device Approval for Cell Therapy Product

Cytori Therapeutics announced that the company has received approval for the Celution Cell Therapy System consumable bundle in Japan as a Class III medical device. [Cytori Therapeutics Inc.] Press Release

First CRISPR Babies: Six Questions that Remain

The meeting at which He Jiankui explained his extraordinary claim to have helped produce the first babies — twin girls — born with edited genomes came to a close with a statement that came down hard on the scientist. “We heard an unexpected and deeply disturbing claim that human embryos had been edited and implanted, resulting in a pregnancy and the birth of twins,” reads the statement released by the organizing committee of the Second International Summit on Human Genome Editing in Hong Kong on 29 November. “Even if the modifications are verified, the procedure was irresponsible and failed to conform with international norms.” [Nature News] Editorial

NIH Director Says There’s Work to Do on Regulating Genome Editing Globally

The apparent birth this month of the first genetically modified babies is “a lesson in the potential for human hubris to overtake us,” Dr. Francis Collins, the director of the National Institutes of Health, told STAT, but he said there is little U.S. officials can do to influence how China sanctions the rogue scientist who claims to have led the ethically dubious scientific breakthrough. [STAT News] Editorial

NEW Keystone Symposia: Cellular Plasticity: Reprogramming, Regeneration and Metaplasia
January 27-31, 2019
Keystone, Colorado, United States

Visit our events page to see a complete list of events in the community.

NEW Faculty Position – Cancer Bioengineering (Ecole Polytechnique Fédérale de Lausanne)

NEW Postdoctoral Position – Regenerative Medicine, Stem Cells and Bioengineering (Igenomix Foundation)

Postdoctoral Associate – Gene Therapy (University of Massachusetts)

Research Scientist – Cellular Manufacturing (California Institute for Biomedical Research)

Assistant Project Scientist – Hematology and Oncology (University of California, Davis)

Executive Director – Cell and Gene Medicine (Stanford University)

Faculty Positions – Hematology and Oncology (University of California Davis)

Lab Technologist – Cell Therapy (University of Maryland Medical Center)

Open Rank Professor – Biopharmaceutical Discovery (University of Delaware)

Assistant Professor – Biology (University of Delaware)

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