Cell Therapy News Volume 18.25 | Jul 24 2017

TOP STORY

Intracerebral Gene Therapy in Children with Mucopolysaccharidosis Type IIIB Syndrome: An Uncontrolled Phase I/II Clinical Trial

Mucopolysaccharidosis type IIIB syndrome is a lysosomal storage disease resulting in progressive deterioration of cognitive acquisition after age two–four years. Scientists assessed the safety and efficacy of a novel intracerebral gene therapy. [Lancet Neurol] Abstract

(Ranked by impact factor of the journal)

A Single Dose of Peripherally Infused EGFRvIII-Directed CAR T Cells Mediates Antigen Loss and Induces Adaptive Resistance in Patients with Recurrent Glioblastoma

Scientists conducted a first-in-human study of intravenous delivery of a single dose of autologous T cells redirected to the epidermal growth factor receptor variant III (EGFRvIII) mutation by a CAR. [Sci Transl Med] Abstract | Press Release

Gene Therapy for Wiskott-Aldrich Syndrome in a Severely Affected Adult

Investigators describe a case of a pre-splenectomised 30 year old patient with severe WAS manifesting as cutaneous vasculitis, inflammatory arthropathy, intermittent polyclonal lymphoproliferation, significant chronic kidney disease and requiring long-term immunosuppressive treatment. Following reduced intensity conditioning there was rapid engraftment and expansion of a polyclonal pool of transgene-positive functional T cells, and sustained gene marking in myeloid and B cell lineages up to 20 months of observation. [Blood] Abstract

In Vivo Imaging to Monitor Differentiation and Therapeutic Effects of Transplanted Mesenchymal Stem Cells in Myocardial Infarction

Researchers used a noninvasive multimodality imaging approach to monitor differentiation of transplanted bone marrow mesenchymal stem cells and recovery of cardiac function in an in vivo model of myocardial infarction. [Sci Rep] Full Article

Aptamer-Mediated Survivin RNAi Enables 5-Fluorouracil to Eliminate Colorectal Cancer Stem Cells

Investigators showed that EpCAM-aptamer-guided survivin RNAi effectively downregulated survivin both in colorectal cancer cells in vitro and in a mouse xenograft model for colorectal cancer. [Sci Rep] Full Article

Optimal Timing and Frequency of Bone Marrow Soup Therapy for Functional Restoration of Salivary Gland Injured by Single Dose or Fractionated Irradiation

The authors tested the optimal starting time and frequency of bone marrow soup injections in mice radiated with either a single dose or fractionated dose. [J Tissue Eng Regen Med] Abstract

A Small-Molecule/Cytokine Combination Enhances Hematopoietic Stem Cell Proliferation via Inhibition of Cell Differentiation

Small-molecule candidates were individually screened and then combined at their optimal concentration with the presence of cytokines to achieve maximum capacity for stimulating the human CD34⁺ cell expansion ex vivo. Expanded cells were transplanted into sublethally irradiated NOD/SCID mice for the assessment of human cell viability and engraftment potential in vivo. [Stem Cell Res Ther] Full Article

Phase II Study of Sequential Infusion of DLI and Cytokine Induced Killer Cells for Patients Relapsed after AlloHSCT

Seventy-four patients relapsed after allogeneic stem cell transplantation were enrolled in a Phase IIA study and treated with the sequential infusion of donor lymphocytes (DLI) followed by cytokine induced killer cells. [Biol Blood Marrow Transplant] Abstract

Preclinical Safety Evaluation of Allogeneic iPS Cell-Based Therapy in a Swine Model of Myocardial Infarction

Researchers used porcine induced pluripotent stem cell (p-iPSC), in conjunction with bioengineered constructs that support recovery after acute myocardial infarction. Histopathology analyses found no evidence of p-iPSC or p-iPSC-derived cells within the host myocardium or biomatrices after 30-day and 90-day of follow-up. [Tissue Eng Part C Methods] Abstract

Allogeneic Cell Therapy Manufacturing: Process Development Technologies and Facility Design Options

The authors highlight the recent advances in the development of allogeneic products, the available options to develop robust manufacturing processes, and facility design considerations. [Expert Opin Biol Ther] Abstract

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

Longeveron to Sponsor Industry Symposium on Regenerative Medicine

Longeveron LLC is sponsoring a regenerative medicine symposium. The symposium, titled Regenerative Medicine Alternatives for Aging Conditions, will feature a global panel of physicians who are experts in stem cell therapy and geriatric conditions, such as Aging Frailty and Alzheimer’s disease. [Press release from Longeveron LLC discussing a symposium to be presented at the 2017 International Association of Gerontology and Geriatrics (IAGG) World Congress, San Francisco] Press Release

Breathing in a New Gene Therapy to Treat Pulmonary Hypertension

Mount Sinai has partnered with Theragene Pharmaceuticals, Inc. to advance a novel airway-delivered gene therapy for treating pulmonary hypertension, a form of high blood pressure in blood vessels in the lungs that is linked to heart failure. [Mount Sinai (Newswise, Inc)] Press Release

Abeona Therapeutics Receives Guidance from FDA to Commence Pivotal Phase III for EB-101 Gene Therapy for Patients with Epidermolysis Bullosa

Abeona Therapeutics Inc. announced guidance from a recent Type-C meeting with the FDA which has recommended accelerating the EB-101 program into a pivotal Phase III trial. [Abeona Therapeutics Inc.] Press Release

Brainstorm Awarded $16 Million Non-Dilutive Grant from CIRM in Support of Phase III Clinical Trial of NurOwn^® in ALS

Brainstorm Cell Therapeutics Inc. announced that the California Institute for Regenerative Medicine (CIRM) has awarded Brainstorm a grant of $16 million to support the pivotal Phase III study of NurOwn^®, for the treatment of amyotrophic lateral sclerosis (ALS). [Brainstorm Cell Therapeutics Inc.] Press Release

BrainStorm Announces Agreements with Mass. General Hospital and California Pacific Medical Center to Participate in Phase III Trial of NurOwn^® in ALS

BrainStorm Cell Therapeutics Inc. announced that it has signed definitive agreements with Massachusetts General Hospital and California Pacific Medical Center to enroll patients in the planned Phase III clinical trial of NurOwn^® in amyotrophic lateral sclerosis (ALS), pending FDA and Institutional Review Board approvals. [BrainStorm Cell Therapeutics Inc.] Press Release

Jeffrey and Marieke Rothschild Gift Establishes Stanford Center for Cancer Cell Therapy

The Stanford Cancer Institute has received a $10 million gift from Silicon Valley entrepreneur Jeffrey Rothschild and his wife, Marieke, to advance research in cancer cell therapy. The gift launches the Stanford Center for Cancer Cell Therapy, which will support research and clinical trials of treatments that use the power of the patients’ own immune systems to attack and kill tumor cells. [Stanford Medicine] Press Release

Celyad Announces Initiation of the SHRINK Trial

Celyad SA/NV announced the initiation of the SHRINK trial, a third clinical trial with their lead product candidate CYAD-01 targeting metastatic colorectal patients. [Celyad SA/NV] Press Release

4D Molecular Therapeutics and Foundation Fighting Blindness Partner to Develop Gene Therapies for Retinal Diseases

4D Molecular Therapeutics (4DMT) and the Foundation Fighting Blindness announced a partnership to develop intravitreal gene therapeutics for patients with these blinding conditions using 4DMT-proprietary adeno-associated virus vectors. [4D Molecular Therapeutics] Press Release

U.S. FDA Designates Mallinckrodt’s StrataGraft^® as Regenerative Medicine Advanced Therapy

Mallinckrodt plc announced that StrataGraft^® regenerative skin tissue is among the first products to be designated as a Regenerative Medicine Advanced Therapy by the FDA under the provisions of the 21^st Century Cures Act. [Mallinckrodt plc] Press Release

Capricor Receives Rare Pediatric Disease Designation from FDA for CAP-1002 for Patients with Duchenne Muscular Dystrophy

Capricor Therapeutics, Inc. announced that the FDA has granted Rare Pediatric Disease Designation to CAP-1002, Capricor’s development candidate for the treatment of Duchenne muscular dystrophy. [Capricor Therapeutics, Inc.] Press Release

ViraCyte Enters Exclusive License Agreement with Baylor College of Medicine for T Cell Immunotherapies Targeting Viruses

ViraCyte, LLC announced that it has exercised its exclusive option with Baylor College of Medicine to license Baylor’s virus-specific T cell therapy technology for the prevention and/or treatment of viral infections. [ViraCyte, LLC] Press Release

US Defence Agencies Grapple With Gene Drives

The JASONs, a group of elite scientists that advises the US government on national security, has weighed in on issues ranging from cyber security to renewing America’s nuclear arsenal. But at a meeting in June, the secretive group took stock of a new threat: gene drives, a genetic-engineering technology that can swiftly spread modifications through entire populations and could help vanquish malaria-spreading mosquitoes. [Nature News] Editorial

Salk Institute Hit with Discrimination Lawsuit by Third Female Scientist

Following two gender discrimination lawsuits filed last week, a third senior female professor at the Salk Institute for Biological Studies has similarly sued the storied independent institute in San Diego, California. [ScienceInsider] Editorial

Some Scientists Hate NIH’s New Definition of a Clinical Trial. Here’s Why

Scientists in behavioral research fields — from cognitive psychology to vision science — were dismayed to learn that the U.S. National Institutes of Health (NIH) in Bethesda, Maryland, could soon deem their studies to be clinical trials. [ScienceInsider] Editorial

FDA

Agency Information Collection Activities; Proposed Collection; Comment Request; Adverse Experience Reporting for Licensed Biological Products; and General Records (FR Doc. No:2017-15004) Notice

Agency Information Collection Activities; Submission for Office of Management and Budget Review; Comment Request; Current Good Manufacturing Practice Regulations for Type A Medicated Articles (FR Doc. No:2017-14996) Notice

NEW Mammalian Genetics and Genomics: From Molecular Mechanisms to Translational Applications
October 24-27, 2017
Heidelberg, Germany

NEW Singapore Stem Cell Society Symposium 2017
November 16-17, 2017
Singapore, Singapore

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

NEW Postdoctoral Research Associate – Molecular Mechanisms of Stem Cells in the Heart (University of Louisville)

Postdoctoral Research Associate – Gene Therapy (King’s College London)

Senior Research Associate – Cellular Therapy Core (University of Texas Health Science Center at Houston)

Postdoctoral Fellow – Single Cell Genomics (BC Cancer Agency Research Center)

Research Associate – Cell Engineering (Caribou Biosciences, Inc.)

Quality Improvement Coordinator – Cellular Therapy Core (University of Texas Health Science Center at Houston)

Scientist – Analytical Development (KBI Biopharma)

GMP Manufacturing Director – Cell and Gene Therapy (Fred Hutchinson Cancer Research Center)

Research Fellow – Functional Roles of Stem Cell Derived Extracellular Vesicles (Mayo Clinic)

Professor and Faculty Positions – Various Projects (Academy of Medical Sciences, Zhengzhou University)

Postdoctoral Fellow – Various Projects (University of Oklahoma)

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