Cell Therapy News Volume 19.19 | Jun 4 2018

TOP STORY

CLL Patient Treated at Penn Goes into Remission Thanks to Single CAR T Cell

Researchers say a patient treated for chronic lymphocytic leukemia (CLL) in 2013 went into remission because of a single chimeric antigen receptor (CAR) T cell and the cells it produced as it multiplied, and has stayed cancer free in the five years since, with CAR T cells still present in his immune system. [Press release from Penn Medicine discussing online prepublication in Nature] Press Release | Abstract

(Ranked by impact factor of the journal)

Genetic Inactivation of CD33 in Hematopoietic Stem Cells to Enable CAR T Cell Immunotherapy for Acute Myeloid Leukemia

The authors generated CD33-deficient human hematopoietic stem and progenitor cells (HSPCs) and demonstrated normal engraftment and differentiation in immunodeficient mice. Autologous CD33 KO HSPC transplantation in rhesus macaques demonstrated long-term multilineage engraftment of gene-edited cells with normal myeloid function. [Cell] Abstract | Press Release | Graphical Abstract

A First-in-Human, Phase I Study of Neural Stem Cell Transplantation for Chronic Spinal Cord Injury

Scientists tested the feasibility and safety of human-spinal-cord-derived neural stem cell (NSI-566) transplantation for the treatment of chronic spinal cord injury (SCI). In this clinical trial, four subjects with T2–T12 SCI received treatment consisting of removal of spinal instrumentation, laminectomy, and durotomy, followed by six midline bilateral stereotactic injections of NSI-566 cells. [Cell Stem Cell] Abstract | Press Release 1 | Press Release 2 | Graphical Abstract

Enhancing the Therapeutic Effect via Elimination of Hepatocellular Carcinoma Stem Cells Using Bmi1 siRNA Delivered by Cationic Cisplatin Nanocapsules

Scientists report that Bmi1 siRNA (Bmi1siR) loaded in cationic nanocapsules of cisplatin (NPC) eliminated stem cells in situ hepatocellular carcinoma in mice. NPC/Bmi1siR was fabricated via electrostatic complexation of Bmi1 siRNA to NPCs, which had cores composed of cisplatin and were coated with cationic lipids. [Nanomedicine] Abstract

Endoscopic Administration of Mesenchymal Stromal Cells Reduces Inflammation in Experimental Colitis

The authors assessed whether endoscopic injections of mesenchymal stromal cells into the intestinal wall of the inflamed colon affect the course of experimental colitis. [Inflamm Bowel Dis] Abstract

Decellularized Skeletal Muscles Allow Functional Muscle Regeneration by Promoting Host Cell Migration

Researchers investigated the ability of three different decellularized skeletal muscle scaffolds to support muscle regeneration in a xenogeneic immune-competent model of volumetric muscle loss, in which the extensor digitorum longus muscle was surgically resected. [Sci Rep] Full Article

Sequential Intravenous Allogeneic Mesenchymal Stromal Cells as a Potential Treatment for Thromboangiitis Obliterans (Buerger’s Disease)

Scientists report the first case of a thromboangiitis obliterans patient at amputation risk treated with four sequential intravenous infusions of bone marrow-derived allogeneic mesenchymal stromal cells from a healthy donor. [Stem Cell Res Ther] Full Article

Rat Cranial Bone-Derived Mesenchymal Stem Cell Transplantation Promotes Functional Recovery in Ischemic Stroke Model Rats

Researchers aimed to demonstrate the therapeutic effects of transplantation with rat cranial bone-derived mesenchymal stem cells (rcMSCs) in ischemic stroke model rats. The mRNA expression of brain-derived neurotrophic factor and nerve growth factor was significantly stronger in rcMSCs than in rat bone marrow-derived MSCs. [Stem Cells Dev] Abstract

Mesenchymal Stromal Cells from Human Umbilical Cord Prevent the Development of Lung Fibrosis in Immunocompetent Mice

The authors investigated the anti-fibrotic activity in vivo of human mesenchymal stromal cells obtained from whole umbilical cord (hUC-MSCs). Adult immunocompetent C57BL/6 mice were injected intravenously with hUC-MSCs twice, 24 hours and seven days after endotracheal injection of bleomycin. [PLoS One] Full Article

Stem Cell Therapies for Treating Diabetes: Progress and Remaining Challenges

The authors describe current efforts aimed at generating a sustainable source of functional human stem cell-derived insulin-producing islet cells for cell transplantation and present state-of-the-art efforts to protect such cells via immune modulation and encapsulation strategies. [Cell Stem Cell] Full Article

Neural Stem Cell Therapies and Hypoxic-Ischemic Brain Injury

Scientists present an overview of neural stem cells during normal brain development and the effect of hypoxic-ischemic injury on the activation and function of endogenous neural stem cells in the brain. [Prog Neurobiol] Abstract

Gene Therapy and Genome Surgery in the Retina

Researchers highlight the translational progress of gene therapy and genome editing of several retinal disorders, including RPE65^–, CEP290^–, and GUY2D-associated Leber congenital amaurosis, as well as choroideremia, achromatopsia, Mer tyrosine kinase- and RPGR X-linked retinitis pigmentosa, Usher syndrome, neovascular age-related macular degeneration, X-linked retinoschisis, Stargardt disease, and Leber hereditary optic neuropathy. [J Clin Invest] Abstract

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

Kite Announces Initial Results from a Phase I Study of T Cell Receptor (TCR) Cell Therapy in HPV-16-Positive Solid Tumors

Kite announced results from an ongoing Phase I study conducted by the National Cancer Institute showing that clinical responses were observed with investigational TCR cell therapy targeting human papillomavirus (HPV) type 16 E7 in solid tumor cancers caused by HPV. [Press release from Kite (Business Wire, Inc.) discussing research presented at the 2018 American Society of Clinical Oncology (ASCO) Annual Meeting, Chicago] Press Release

MiNA Therapeutics Presents Initial Results from First-in-Human MTL-CEBPA Study in Advanced Liver Cancer Patients

MiNA Therapeutics announced preliminary results from its ongoing Phase I study of small activating RNA candidate MTL-CEBPA in advanced liver cancer. [Press release from MiNA Therapeutics Limited discussing research presented at the 2018 American Society of Clinical Oncology (ASCO) Annual Meeting, Chicago] Press Release

Updated Results of Ongoing Multicenter Phase I Study of bb2121 Anti-BCMA CAR T Cell Therapy Continue to Demonstrate Deep and Durable Responses in Patients with Late-Stage Relapsed/Refractory Multiple Myeloma

Celgene Corporation and bluebird bio, Inc. announced updated results from the ongoing CRB-401 Phase I clinical study of bb2121, an investigational anti-B-cell maturation antigen (BCMA) CAR T cell therapy, in 43 patients with late-stage relapsed/refractory multiple myeloma. [Press release from Celgene Corporation discussing research presented at the 2018 American Society of Clinical Oncology (ASCO) Annual Meeting, Chicago] Press Release

Mesoblast Partners with Cartherics to Develop ’Off-the-Shelf’ Cell-Based Immunotherapies for Solid Cancers

Mesoblast Limited announced that it has entered into a partnership with Cartherics Pty Ltd to develop allogeneic ‘off-the-shelf’ CAR-T cells armed with multiple targeting receptors for use in solid cancers. [Mesoblast Limited (GlobeNewswire, Inc.)] Press Release

BioTime Announces $1.9 Million Grant for Continued Development of OpRegen^® for Dry-AMD

ioTime, Inc. has been awarded a new grant for 2018 of up to 6.9 million Israeli New Shekels from the Israel Innovation Authority. [BioTime, Inc.] Press Release

Million-Dollar Kavli Prize Recognizes Scientist Scooped on CRISPR

Two biochemists widely credited with co-inventing the gene-editing technology, Emmanuelle Charpentier and Jennifer Doudna, were named on 31 May as the winners of this year’s Kavli Prize in Nanoscience. So was Virginijus Siksnys, a Lithuanian biochemist whose independent work on CRISPR has thus far garnered much less mainstream attention — and Nobel-prize buzz — compared with that of Charpentier, Doudna and some other scientists. [Nature News] Editorial

Cognate BioServices Selected By Medeor Therapeutics to Manufacture Stem Cell Derived Product Candidate MDR-102

Cognate BioServices announced an agreement with Medeor Therapeutics to manufacture MDR-102, a proprietary, organ donor-derived cellular immunotherapy product candidate for certain kidney transplant recipients. [Cognate BioServices (PR Newswire Association, LLC.)] Press Release

New Cell Therapy Could Help Millions Suffering from Back Pain

DiscGenics has engaged with CCRM and GE Healthcare, leaders in developing and commercializing cell and gene therapy technologies, to support the company’s efforts to scale and optimize manufacturing of its homologous, allogeneic, injectable cell therapy, which is referred to as IDCT. [DiscGenics] Press Release

Arrowhead Completes Enrollment in Single Dose Portion of Phase I/II Study of ARO-HBV

Arrowhead Pharmaceuticals Inc. announced that it has completed enrollment and dosing of all five planned cohorts of healthy adult volunteers in the single-ascending dose portion of its ongoing Phase I/II study of ARO-HBV, the company’s third generation subcutaneously administered RNA interference therapeutic being developed as a potentially curative therapy for patients with chronic hepatitis B virus (HBV) infection. [Arrowhead Pharmaceuticals Inc.] Press Release

‘Reprogrammed’ Stem Cells Approved to Mend Human Hearts for the First Time

Scientists in Japan now have permission to treat people who have heart disease with cells produced by a revolutionary reprogramming technique. The study is only the second clinical application of induced pluripotent stem cells. [Nature News] Editorial | Press Release

Abeona Therapeutics Announces Opening of Commercial Gene & Cell Therapy Manufacturing Facility in Ohio

Abeona Therapeutics Inc. announced the opening of The Elisa Linton Center for Rare Disease Therapies, the commercial GMP manufacturing facility for gene and cell therapies in Cleveland, Ohio. [Abeona Therapeutics Inc.] Press Release

CRISPR Therapeutics and Vertex Provide Update on FDA Review of Investigational New Drug Application for CTX001 for the Treatment of Sickle Cell Disease

CRISPR Therapeutics and Vertex Pharmaceuticals Incorporated announced that the FDA has placed a clinical hold on the Investigational New Drug Application (IND) for CTX001 for the treatment of sickle cell disease pending the resolution of certain questions that will be provided by the FDA as part of its review of the IND. [CRISPR Therapeutics] Press Release

Legend Announces FDA Clearance of IND Application on CAR-T Immuno-Cell Therapy for the Treatment of Multiple Myeloma

Legend Biotech announced that the FDA has authorized its development partner, Janssen Biotech, Inc., to commence a Phase Ib/II clinical trial in patients with relapsed or refractory multiple myeloma to evaluate the safety and efficacy of LCARB38M, a chimeric antigen receptor T cell (CAR-T) therapy. [Legend Biotechnology] Press Release

TxCell Names Lonza as Its CAR-Treg Cellular Product Manufacturer

TxCell SA and Lonza Pharma & Biotech announced entering into a Master Service Agreement for the manufacture of TxCell’s HLA-A2 CAR-Treg cellular product, which is in development for the prevention of chronic rejection after organ transplantation. [Lonza] Press Release

uniQure Announces the Issuance of New Patents Providing Broad Protection of the Padua Variant of Factor IX in Gene Therapy

uniQure N.V. announced that the U.S. Patent and Trademark Office has granted Patent Number 9,982,248 which broadly covers methods of treating coagulopathies, including hemophilia B, using AAV gene therapy with nucleic acid encoding the hyperactive Factor IX Padua variant. [uniQure N.V.] Press Release

Adaptimmune Announces First Patient to Receive One Billion Target Cell Dose after Positive Safety Data from Pilot Study with MAGE-A4 SPEAR T-Cells

Adaptimmune Therapeutics plc announced that the independent safety review committee has recommended dose escalation in the MAGE-A4 basket study, based on an acceptable safety profile in three patients dosed with 100 million cells. [Adaptimmune Therapeutics plc] Press Release

Europe’s Top Science Funder Shows High-Risk Research Pays Off

A popular and unusual self-review carried out by Europe’s most prestigious science funder is back. The annual assessment, now in its third year, found that nearly one in five projects by the European Research Council led to a scientific breakthrough. [Nature News] Editorial

‘Right-to-Try’ Law Intended to Weaken the FDA, Measure’s Sponsor Says in Blunt Remarks

Sen. Ron Johnson, the author of the federal “right-to-try” law signed by President Trump this week, wants to make one thing clear: His new law is meant to weaken the FDA. “This law intends to diminish the FDA’s power over people’s lives, not increase it,” he wrote in a letter to Commissioner Scott Gottlieb. [STAT News] Editorial

China to Crack Down on Fraud in Scandal-Hit Scientific Research amid ZTE Wrangle

China has issued the first national guidelines to enforce academic integrity in scientific research and vowed to punish academics and institutes for misconduct such as plagiarism and fabrication of data. [South China Morning Post Publishers Ltd] Editorial

Sweden Commits to Open Science with New Open-Access Publishing Deal

Swedish researchers can now publish their articles in Frontiers’ Open Access journals through a simplified process that covers publishing fees, thanks to a national agreement announced between Frontiers and the National Library of Sweden, on behalf of the organizations participating in the Bibsam Consortium. [EurekAlert] Editorial

FDA

Considerations for the Inclusion of Adolescent Patients in Adult Oncology Clinical Trials Guidance for Industry (FR Doc. No:Guidances) Notice

NEW American Society of Hematology (ASH) Meeting on Lymphoma Biology
August 2-5, 2018
Chicago, Illinois, United States

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

NEW Postdoctoral Fellow – Cell and Gene Therapies for Cardiac Arrhythmias and Heart Failure (Duke University)

NEW Postdoctoral Position – Human Pluripotent Stem Cell-Based Cell Therapy in Type 1 Diabetes (University of Copenhagen)

Research Officer – Immunology (The Walter and Eliza Hall Institute of Medical Research)

Postdoctoral Fellow – Gene Editing and Cellular Reprogramming (Houston Methodist Research Institute)

Faculty Position – Adoptive T Cell Therapy (Dana-Farber Cancer Institute)

Postdoctoral Position – Technology-Driven Research in Biomedicine (CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences)

Group Manager – AAV Vector Expression Systems (Genethon)

Postdoctoral Fellow – Gene Therapy in Cardiovascular Disease (Emory University)

Scientist/Senior Scientist – Lentivirus Production (Obsidian Therapeutics)

Scientist/Senior Scientist – Bioinformatics and Data Sciences (Obsidian Therapeutics)

Postdoctoral Fellow – Immunology, Immunotherapy, Gene Therapy (City of Hope)

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