Final Program - American Society of Gene & Cell Therapy

Program Schedule, Thursday, May 16, 2013
Christian Mueller, PhD
MicroRNA’s and Alpha-1 Antitrypsin Deiciency
Terence R. Flotte, MD
Advances in Clinical Gene Therapy Trials for Alpha-1 Anti-trypsin Deiciency
Scientific Symposium 211
10:30 am - 12:30 pm
ROOM: BALLROOM C
Clinical Updates of Stem Cell Therapies
CO-CHAIRS: Jane S. Lebkowski, PhD and Eirini P. Papapetrou, MD, PhD
SPEAKERS
Michele De Luca, MD
Corneal Regeneration By Cultured Limbal Stem Cells
Adult stem cells are cells with a high capacity for self-renewal that can produce terminally differentiated progeny. Stem cells
generate an intermediate population of committed progenitors, often referred to as transit amplifying (TA) cells, that terminally
differentiate after a limited number of cell divisions. Human keratinocyte stem cells are clonogenic and are known as holoclones.
Human corneal stem cells are segregated in the limbus while limbal-derived TA cells form the corneal epithelium. Cultivated limbal
stem cells generate sheets of corneal epithelium suitable for clinical application. We report long-term (up to 15 years) clinical
results obtained in an homogeneous group of patients presenting with corneal opaciication and visual loss due to chemical burndependent
limbal stem cell deiciency. The corneal epithelium and the visual acuity of these patients have been restored by grafts of
autologous cultured limbal keratinocytes. In post hoc analyses, success was associated with the percentage of p63-bright holocloneforming
stem cells in culture, but not with total number of clonogenic cells, colony size or growth rate of the cultures. Graft failure
was also associated with the type of initial ocular damage and postoperative complications.
Joshua Hare, MD
Cell Therapy for Chronic Ischemic Heart Disease from Concept to Clinic
Thursday, May 16, 2013
Brian W. Bigger, PhD
Hematopoietic Stem Cell Gene Therapy for Neurological Diseases
Haematopoietic stem cell transplantation (HSCT) has traditionally been used to treat a select group of single gene neurological
diseases, with particular success in the neurodegenerative lysosomal disease Hurler syndrome. In many lysosomal diseases
exogenous enzyme can be taken up by, and cross-correct affected cells, but the blood brain barrier excludes enzyme from the
brain. Neurological correction after HSCT relies on monocyte/microglial donor cell traficking into the brain to deliver enzyme, and
enzyme dose is thought to be one of the factors limiting HSCT to a relatively narrow range of neurological indications. Autologous
haematopoietic stem cell gene therapy, using retroviral or lentiviral vectors to overexpress enzyme in ex vivo transduced and
transplanted cells has been used to great effect in X-SCID and other immunodeiciencies and more recently has been adopted as a
potential treatment for neurological lysosomal and related diseases. In this talk the initial clinical trial results in this ield will be
discussed, together with the current challenges faced by this therapeutic approach, what is being done to address them and where
the future for this exciting therapy lies.
Gary K. Steinberg, MD, PhD
Stem Cell Therapy for Stroke
No treatment currently exists to restore lost brain function after stroke. Animal studies have demonstrated that post-ischemic
intraparenchymal, intravenous and intra-arterial administration of various types of stem cells can improve neurologic outcome.
Although some integration of neural stem cells into the peri-infarct brain has been shown, the main mechanisms underlying
recovery of function are likely secretion of trophic factors and immunomodulation that enhance endogenous plasticity and
recovery. These native repair processes include axonal sprouting, dendritic branching, neovascularization, neurogenesis,
gliogenesis, synaptogenesis and decreased inlammation. To date 11 trials of cellular therapy for stroke have been completed
and more than 15 additional trials are underway. This talk will briely review the experimental basis leading to the clinical trials,
and focus on summarizing the clinical results of published and ongoing stroke trials using neural, bone marrow derived and
other stem cells. Unresolved issues will be discussed, including the best cell source and type, optimal route of delivery, timing of
transplantation and patient selection.
Final Program SALT LAKE CITY, UTAH May 15–18, 2013
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