Final Program - American Society of Gene & Cell Therapy

Program Schedule, Wednesday, May 15, 2013
Marcelo N. Rivolta, MD, PhD
Restoration of Auditory Evoked Responses by hESC-derived Otic Progenitors
The presentation will discuss recent advances using stem cells in the search of a treatment for hearing loss. A method has been
developed to generate ear sensory cells from human embryonic stem cells (hESCs). By activating FGF signalling on hESCs using
ligands that induce the formation of the ear in vivo, we have generated otic progenitors that can produce sensory hair cell-like cells
and auditory neurons. We have then taken the hESC-derived otic progenitor cells and explore if they could repair a deaf ear in a
gerbil model of auditory neuropathy. When hESC-derived ear cells were transplanted into cochleae that have lost their auditory
neurons the cells survived, engrafted and differentiated. Moreover, they sent projections making connections with the hair cells and
with the brain and, more remarkably, they elicited a functional recovery represented by improved ABR thresholds. Related work,
developing them as a platform for gene and drug discovery will also be reviewed.
Scientific Symposium 103
8:00 am - 10:00 am
ROOM: 150 DEFG
Novel Approaches to Cancer Immunotherapy
CO-CHAIRS: Jennifer E. Adair, PhD and Elizabeth A. Mittendorf, MD, PhD
SPEAKERS
Wednesday, May 15, 2013
Elizabeth A. Mittendorf, MD, PhD
AE37: A Novel Hybrid Vaccine for Stimulating a Helper T Cell Response
Our group has been leading the clinical trial efforts evaluating AE37, a novel vaccine that combines AE36, a native HER2-derived
MHC class II epitope to the Ii-Key moiety. Linkage of the Ii-key moiety increases antigen presentation in vivo leading to increased
potency of the vaccine. Phase I trials have demonstrated AE37 to be safe and effective in eliciting a HER2-speciic immune response.
An ongoing phase II trial has shown evidence of clinical eficacy; at the time of a planned analysis at 24 month median follow-up,
the disease-free survival rate was 90% in vaccinated patients versus 83% in non-vaccinated controls, a 41% relative risk reduction.
This talk will focus on the preclinical and early clinical trial data available for the AE37 vaccine.
John J. Nemunaitis, MD
Clinical Update of bi-shRNA furin/GMCSF DNA Transfected Tumor Vaccine (FANG) in Cancer Patients
Exciting the immune system to attack cancer involves 3 critical components: education of tumor antigens, enhancement of cancer
targeting immune function, and blockade of pro-cancer immune inhibitors. FANG is a novel “triad” vaccine which utilizes both
cellular and DNA technology to accomplish all 3 components. Speciically, autologous harvested tumor cells are transfected with a
dual plasmid involving a GMCSF DNA cassette and a bifunctional RNA interference DNA cassette targeting furin, the proprotease
converting enzyme of TGFß1 and TGFß2. Phase I trial involving 42 advanced cancer patients (Senzer et. al. Mol Ther 2012: 679-
686) demonstrated patient safety, veriication of product mechanism and correlation of signiicant survival improvement with
induction of activated T-cells as measured by ELISPOT response. Follow up results of the Phase I trial with demonstration of median
survival advantage in comparison of FANG treated patients to FANG not treated patients of 562 days vs. 181 days (p