Cell and Gene Therapies for Cancer: Future Promises and Challenges

Featured speakers:

J. Joseph Melenhorst, Ph.D., University of Pennsylvania

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

Moderated by:

Sean Sanders, Ph.D., Science/AAAS

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 takes 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.

Topics discussed during the webinar:

• Outline the current state of cell and gene therapies
• Speakers share their experience with the challenges of applying these therapies in a practical way
• 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.

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Recorded January 23, 2019