Cell Therapy News Volume 19.15 | Apr 30 2018

TOP STORY

T Cell Biomarker Predicts Which CLL Patients Will Respond to CAR T Cell Therapy

Researchers may have found the reason why some patients with advanced chronic lymphocytic leukemia (CLL) don’t respond to chimeric antigen receptor (CAR) T cell therapy, and the answer is tied to how primed patients’ immune systems are before the therapy is administered. [Press release from Penn Medicine discussing online prepublication in Nature Medicine] Press Release | Abstract

(Ranked by impact factor of the journal)

Inducible Expression of GDNF in Transplanted iPSC-Derived Neural Progenitor Cells

Scientists engineered a doxycycline-regulated vector, allowing inducible and reversible expression of a therapeutic molecule. Human induced pluripotent stem cell (iPSC)-derived neural progenitors were stably transfected with the vector and transplanted into the adult mouse brain. [Stem Cell Reports] Full Article | Graphical Abstract

Ultrasound-Mediated Gene Delivery Enhances Tendon Allograft Integration in Mini-Pig Ligament Reconstruction

A collagen scaffold was implanted in the reconstruction sites to facilitate recruitment of endogenous mesenchymal stem cells. Ultrasound-mediated reporter gene delivery successfully transfected 40% of cells recruited to the reconstruction sites. [Mol Ther] Abstract

Treatment of Bone Defects by Transplantation of Genetically Modified Mesenchymal Stem Cell Spheroids

Transplanting runt-related transcription factor 2-transfected mesenchymal stem cell (MSC) spheroids into bone defects on rat femurs induced significantly faster bone regeneration compared with non-transfected MSC spheroids or genetically modified MSCs from conventional monolayer culture. [Mol Ther Methods Clin Dev] Full Article | Graphical Abstract

Using Tyrosinase as a Tri-Modality Reporter Gene to Monitor Transplanted Stem Cells in Acute Myocardial Infarction

Researchers investigated the feasibility of non-invasive monitoring of bone marrow mesenchymal stem cells transduced with the tyrosinase reporter gene for acute myocardial infarction with photoacoustic imaging, magnetic resonance imaging, and positron emission tomography in vitro and in vivo. [Exp Mol Med] Full Article

Long-Term Viability and Function of Transplanted Islets Macroencapsulated at High Density Are Achieved by Enhanced Oxygen Supply

Encapsulated islets implanted with surface density as high as 4,800 islet equivalents/cm³ in diabetic rats maintained normoglycemia for more than seven months and provided near-normal intravenous glucose tolerance tests. [Sci Rep] Full Article | Press Release

Transplantation of Adipose-Derived Mesenchymal Stem Cells Attenuates Pulmonary Fibrosis of Silicosis via Anti-Inflammatory and Anti-Apoptosis Effects in Rats

Scientists investigated the anti-pulmonary fibrosis effects of the transplantation of adipose-derived mesenchymal stem cells in rats in which lung fibrosis was induced by oral tracheal intubation with silica suspension. [Stem Cell Res Ther] Full Article

Human Carbonic Anhydrase-8 AAV8 Gene Therapy Inhibits Nerve Growth Factor Signaling Producing Prolonged Analgesia and Anti-Hyperalgesia in Mice

Investigators showed that gene transfer using AAV8-V5-CA8^WT viral particles via sciatic nerve injection demonstrates retrograde transport to dorsal root ganglia producing prolonged V5-CA8^WT expression, pITPR1 and pTrkA inhibition, and profound analgesia and anti-hyperalgesia in male C57BL/6J mice. [Gene Ther] Abstract

Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for Ex Vivo Gene Therapy of X-Linked Chronic Granulomatous Disease

Transduced CD34⁺ cells derived from nicotinamide dinucleotide phosphate (NADPH) oxidase complex patients engrafted and differentiated similarly to their non-transduced counterparts in xenograft mouse models and generated therapeutically relevant levels of NADPH activity in myeloid cells expressing gp91^phox. [Hum Gene Ther Clin Dev] Abstract

Hepatocyte Transplantation and Advancements in Alternative Cell Sources for Liver-Based Regenerative Medicine

The authors review the current status of hepatocyte transplantation, alternative cell sources to primary human hepatocytes and their potential in liver regeneration. They also describe recent clinical trials using hepatocytes derived from stem cells and their role in improving the phenotype of several liver diseases. [J Mol Med] Full Article

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

AveXis Presents Initial Data from Pivotal U.S. Trial for SMA Type 1 and 24-Month Follow-Up Data from Phase I Trial of AVXS-101 in SMA Type 1

AveXis, Inc. will present initial results from the Type 1 U.S. Pivotal trial and 24-month follow-up data from the Phase I trial of AVXS-101 for the treatment of spinal muscular atrophy (SMA) Type 1. [Press release from AveXis, Inc. discussing research to be presented at the 2018 Annual Meeting of the American Academy of Neurology (AAN) Annual Meeting, Los Angeles] Press Release

STEMCELL Technologies Announces $45 Million Government Funding for Advanced Manufacturing Facility

STEMCELL Technologies announced a $45 million joint funding agreement between the Governments of Canada and British Columbia to help the company build a state-of-the-art advanced manufacturing facility. [STEMCELL Technologies Inc.] Press Release

Therapies Targeting Cancer, Deadly Immune Disorder and Life-Threatening Blood Condition Get Almost $32 Million Boost from CIRM Board

Researchers at the Stanford University School of Medicine were awarded $11.9 million to test their Chimeric Antigen Receptor (CAR) T Cell Therapy in patients with B cell leukemias who have relapsed or are not responding after standard treatments, such as chemotherapy. [California Institute for Regenerative Medicine] Press Release

Doudna’s Latest CRISPR Company Debuts, Joining the Crowds in Diagnostics and Genome-Editing

After a year spent wooing investors, hiring, and refining its plans, Mammoth Biosciences, co-founded by CRISPR developer Jennifer Doudna, is announcing its entry into the ever-more-crowded fields of diagnostics and genome-editing, and hoping to find a sweet spot at their intersection. [STAT News] Press Release

AveXis Announces First Patient Dosed in Phase III Trial of AVXS-101 in Pre-Symptomatic SMA Types 1, 2 and 3

AveXis, Inc. announced that the first patient has been dosed in a Phase III trial evaluating AVXS-101 in pre-symptomatic patients with spinal muscular atrophy (SMA) Types 1, 2 and 3. [AveXis, Inc.] Press Release

U.S. FDA Clears Pluristem’s Phase III Study in Treatment of Muscle Injury Following Hip Fracture Surgery

Pluristem Therapeutics Inc. announced that the FDA has cleared Pluristem’s Investigational Drug Application for a Phase III study of its PLX-PAD cell therapy in the treatment of muscle injury following surgical repair of the hip joint due to fracture. [Pluristem Therapeutics Inc.] Press Release

Capricor Announces Initiation of HOPE-2 Clinical Trial of CAP-1002 for Duchenne Muscular Dystrophy

Capricor Therapeutics, Inc. announced it has initiated the HOPE-2 clinical trial at UC Davis Medical Center. The trial will test the safety and efficacy of Capricor’s novel cellular therapy, CAP-1002, in boys and young men with Duchenne muscular dystrophy, a devastating and fatal genetic disorder with limited treatment options and no cure. [Capricor Therapeutics, Inc.] Press Release

Sending Nanomedicine to the Back of Your Eye to Cure Glaucoma

Marianna Foldvari is one of a handful of researchers worldwide focusing on non-invasive gene therapy and drug delivery, using nanotechnology to treat and cure neurodegenerative diseases such as glaucoma. [University of Waterloo] Editorial

DiscGenics Announces First Patient Treated in U.S. Clinical Trial of IDCT for Degenerative Disc Disease

DiscGenics, Inc. announced the first patient has been treated in its Phase I/II U.S. clinical trial of IDCT for mild-to-moderate degenerative disc disease. [DiscGenics, Inc.] Press Release

Kadimastem Announces the Enrollment of the First Patient for Its Clinical Trial in ALS Patients

Kadimastem announced the enrollment of the first patient for its clinical trial in ALS patients, using the cell therapy product developed by the company, AstroRx^®. [Kadimastem] Press Release

Asklepios BioPharmaceutical, Inc. Announces Former Portfolio Company’s Preclinical Asset for Duchenne Muscular Dystrophy Has Advanced into the Clinic

Asklepios BioPharmaceutical, Inc. announced that a pre-clinical GT asset for Duchenne Muscular Dystrophy has advanced into the clinic. [Asklepios BioPharmaceutical, Inc] Press Release

BioCardia Receives New Potency Assay Patent, Providing Further Protection to Autologous Cell Therapy Programs in Heart Failure and Refractory Angina

BioCardia^®, Inc. announced the issuance of United States Patent No. 9,945,854 relating to methods of measuring therapeutic potency potential and defining dosages for autologous cell therapy. [BioCardia^®, Inc.] Press Release

A House Too Far: Two Scientists Abandon Their Bids for Congress

When Phil Janowicz and Kristopher Larsen began their campaigns for a seat in the U.S. House of Representatives, they joined what appears to be a record number of Ph.D. scientists running for national office. But after spending months on the campaign trail, the two Democrats—an organic chemistry professor from southern California and a space physics researcher from Colorado, respectively—decided to drop out before a single ballot had been cast in their states. [ScienceInsider] Editorial

Two Key FDA Officials to Retire, Leaving Three Divisions without Permanent Leaders

Two Food and Drug Administration directors will retire this week, according to the agency. The departures leave three of the FDA’s 19 drug review divisions without permanent leaders. [STAT News] Editorial

Disgraced Surgeon Is Still Publishing on Stem Cell Therapies

Paolo Macchiarini, an Italian surgeon, has been fired from two institutions and faces the retraction of many of his papers after findings of scientific misconduct and ethical lapses in his research—yet this hasn’t prevented him from publishing again in a peer-reviewed journal. [ScienceInsider] Editorial

Famed Cancer Biologist Allegedly Sexually Harassed Women for Decades

Inder Verma, the prominent geneticist and cancer scientist who has made his mark on U.S. research for decades, has sexually harassed women for just as long, according to allegations from eight women. [ScienceInsider] Editorial

Columbia University Graduate Students Go on Strike

Administration officials contend that research and teaching assistants are students, not workers, and want to take the matter to federal court. [The Scientist] Editorial

FDA

Vaccines and Related Biological Products Advisory Committee; Notice of Meeting (FR Doc. No:2018-08711) Notice

NEW 23^rd Congress of European Hematology Association (EHA)
June 14-17, 2018
Stockholm, Sweden

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

NEW Gene & Cell Therapy Review Scientists (Center for Biologics Evaluation and Research)

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

Research Associate – Gene and Cell Therapy (University College London)

PhD Student – Immunology of Cell Death & Cancer Immunotherapy (KU Leuven)

Scientist/Senior Scientist – Lentivirus Production (Obsidian Therapeutics)

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

Postdoctoral Associate – Cancer Therapy (St. Jude Children’s Research Hospital)

Scientist – Discovery Biology (Prevail Therapeutics)

Postdoctoral Research Fellow – Cell/Gene Therapy (Stanford University)

Postdoctoral Fellow – Precision Therapy of Aggressive Lymphoma (AstraZeneca)

Postdoctoral Fellow – Leukemia (Necker Institut Enfants Malades)

Postdoctoral Research Fellow – Hematopoiesis (Fred Hutchinson Cancer Research Center)

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

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