FINAL PROGRAM - American Society of Gene & Cell Therapy

13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 43Program ScheduleMarkus Grompe, MDHepatobiliary Stem/Progenitor CellsCarolyn Lutzko, PhDDeveloping Stem Cell Based Therapies for Lung Disease: Bringing Genes to Lung Stem Cells and Stem Cells to the LungThis presentation will focus on different strategies for developing stem cell based therapies for lung diseases. One strategy that will be discussed is the genetic modificationof endogenous lung progenitor cells during development. This presentation will also discuss progress on the differentiation of lung progenitor cells from pluripotent stemcells in vitro.Mervin C. Yoder, MDAre Endothelial Progenitor Cells Derived from the Endothelial Lining of Blood Vessels?Circulating endothelial progenitor cells (EPC) have been reported to play important roles in vascular repair and regeneration and are altered in concentration in patientswith arthritis, diabetes, cancer, and cardiovascular disease. No unique marker exists to identify this cell. Indeed, evidence suggests that numerous cell types participate inneoangiogenesis under the term EPC. We will review recent literature that suggests the local endothelial lining to be a major site of vascular repair.Scientific Symposium 21210:30 am - 12:30 pmRoom: Virginia SuiteCell and Gene Therapy Approaches for Genetic Diseases: Progress and ChallengesChairCharles P. Venditti, MD, PhDSpeakersNathalie Cartier-Lacave, MDGene Therapy for AdrenoleukodystrophyWe report results of the first clinical trial using an HIV vector to correct hematopoietic stem cells (HSC) in adrenoleukodystrophy, a severe degenerative disease of thecentral nervous system. Three children received autologous corrected HSC after full myeloablative conditionning. Up to 36 months after treatment, stable expression wasdemonstrated in peripheral blood leukocytes and bone marrow CD34+ cells, with polyclonal integration of the lentiviral vector. Neurological outcome was comparable tothat observed after allogeneic transplantation. These results demonstrate that lentiviral vectors are promising tools for gene transfer into hematopoietic stem cells in theabsence of selective advantage.Alessandra Biffi, MDHSC Gene Therapy for Metachromatic and Globoid Leukodystrophies: The Next Trials for Inherited Neurological DisordersWe are implementing a gene therapy approach based on the transplantation of gene corrected hematopoietic stem cells (HSC) for the treatment of severe forms ofLysosomal Storage Disorders (LSD) lacking efficacious and safe alternative therapeutic opportunities. To this goal, we exploit the unique features of lentiviral vectors (LV),which are prime candidates for HSC gene transfer. Indeed, by using LV for HSC gene correction, we proved the therapeutic potential and safety of HSC gene therapy in themurine model of metachromatic leukodystrophy (MLD), a severe dysmyelinating LSD. According to these promising results, HSC gene therapy for MLD has now reachedclinical testing. Similarly, an advanced LV design allowed rendering HSC gene therapy a feasible and efficacious approach to be further developed also for globoid cellleukodystrophy, on which we obtained very promising evidences in the disease model.Ronald D.G. McKay, PhDRobert D. Steiner, MDCNS Transplantation of Neural Stem Cells in Neuronal Ceroid Lipofuscinoses: Phase I Trial ResultsInfantile and late-infantile neuronal ceroid lipofuscinosis (INCL and LINCL) are universally fatal neurodegenerative lysosomal storage disorders caused by palmitoyl proteinthioesterase and tripeptidyl peptidase deficiency, respectively. A Phase I trial of human central nervous system stem cells (HuCNS-SC®) was conducted in subjects withINCL and LINCL. This study represents the first US FDA authorized use of human neural stem cells for clinical testing. The primary goal of the trial was to evaluate thesafety of direct CNS delivery of HuCNS-SC, the immunosuppression regimen, and the surgical technique. Six children underwent HuCNS-SC transplantation;. the studyresults to be presented will include adverse events as well as the neurological, seizure, EEG, radiological, neuropsychological, functional, and quality-of-life measuresassessed at frequent intervals post-transplant, as well as evidence of engraftment.Thursday, May 20 th