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Cell and gene therapies for cancer: Future promises and challenges

This webinar is brought to you by the Science/AAAS Custom Publishing Office

Cell and gene therapies for cancer: Future promises and challenges

Recorded 23 January 2019


The field of immuno-oncology has exploded in the clinic, in the press, and on Wall Street, particularly regarding the use of genetically modified immune cells to fight cancer. This “explosion” is largely due to the success of chimeric antigen receptor (CAR) T-cell therapy. The fields of immunology and gene therapy have converged to harness recombinant viruses to deliver “living drugs” with remarkable clinical efficacy, especially for patients with blood cancers. Multiple approaches to engineer immune cells—mostly T cells but also other immune cell types—make use of CARs, while tumor-specific T-cell receptors continue to gain a foothold in the field, mostly for the treatment of solid tumors. Several genome-editing approaches are now available, including established technologies such as zinc-finger nucleases, and newer modalities like the CRISPR/Cas9 system. These methodologies have recently been applied to augment the antitumor efficacy of adoptively transferred cells in the clinic by knocking out negative regulatory molecules such as PD1. Currently, lentiviruses provide the vehicle of choice to stably express immune receptors in T cells, while transposon/transposase systems are an up-and-coming new technology. Additionally, researchers are testing in vivo engineering of tumors using oncolytic viruses. These treatment modalities and technologies have fundamentally changed the way we think about remedying disease, creating the possibility for curing or managing previously untreatable disorders and broadening the clinical appeal of genetically modified T cells. These and other advances are also addressing issues associated with the cost and complexity of bioengineering cells for cancer treatment. This webinar will take an honest look at cell and gene therapies, exploring the potential of these techniques as well as exposing the challenges they present and suggesting how to navigate them.

During the webinar, the speakers will:

  • Outline the current state of cell and gene therapies
  • Share their experience with the challenges of applying these therapies in a practical way
  • Speculate on possible future directions and applications for cell and gene therapy, including adoptive immunotherapy safety and efficacy, and advances in T-cell bioengineering methodologies
  • Answer questions from the online audience during the live broadcast.

This webinar will last for approximately 60 minutes.

Speaker bios

J. Joseph Melenhorst, Ph.D.

University of Pennsylvania
Philadelphia, PA

Dr. Melenhorst is a translational immunologist who has made his mark in fields as diverse as autoimmune disorders, the biology of graft-versus-host disease, and antitumor responses in the allogeneic transplant setting. He was recruited to the University of Pennsylvania in 2012 by Dr. Carl June, first as deputy director of the Clinical Cell and Vaccine Production Facility [where the first FDA-approved chimeric antigen receptor (CAR) product, Kymriah (tisagenlecleucel), was developed] and later as director of Product Development & Correlative Sciences. In this role, he contributed significantly to the evolution of the CAR manufacturing process and to in-depth mechanistic studies into the immunobiology of CAR T-cell therapies. Dr. Melenhorst’s research aims to understand and improve the efficacy and safety of adoptive cell immunotherapy through biomarker, mechanistic, and product development studies. His laboratory has uncovered key biomarkers of cytokine release syndrome, a CAR T-cell therapy–associated toxicity, and elucidated the basic principles governing CAR T-cell therapy potency in leukemia as well as the role of epigenetic mechanisms in CAR T-cell–mediated cancer therapy.

Laurence J. N. Cooper, M.D., Ph.D.

Ziopharm Oncology
Boston, MA

Dr. Cooper joined Ziopharm Oncology as chief executive officer in 2015 after Ziopharm and Intrexon Corporation together licensed technology for a nonviral approach for genetically modifying T cells from the University of Texas MD Anderson Cancer Center and the University of Minnesota. This technology is designed to reduce the cost and complexity of generating genetically modified T cells. Prior to joining Ziopharm, he was a tenured professor at MD Anderson with joint appointments in the Division of Pediatrics and Department of Immunology. There, he served as section chief of cell therapy at the Children’s Cancer Hospital and helped lead scientific efforts to develop new treatment approaches that pair genetic engineering with immunotherapies. He remains a visiting scientist at MD Anderson. Dr. Cooper has coauthored dozens of peer-reviewed journal articles, abstracts, and book chapters. He has initiated multiple gene therapy trials testing infused T cells. He obtained his M.D. and Ph.D. degrees at Case Western Reserve University in Cleveland, followed by training in pediatric oncology and bone marrow transplantation at the Fred Hutchinson Cancer Research Center in Seattle, Washington.

Sean Sanders, Ph.D.

Washington, DC

Dr. Sanders did his undergraduate training at the University of Cape Town, South Africa, and his Ph.D. at the University of Cambridge, UK, supported by the Wellcome Trust. Following postdoctoral training at the National Institutes of Health and Georgetown University, Dr. Sanders joined TranXenoGen, a startup biotechnology company in Massachusetts working on avian transgenics. Pursuing his parallel passion for writing and editing, Dr. Sanders joined BioTechniques as an editor, before joining Science/AAAS in 2006. Currently, Dr. Sanders is the Director and Senior Editor for Custom Publishing for the journal Science and Program Director for Outreach.

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