FINAL PROGRAM - American Society of Gene & Cell Therapy

13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 55Program ScheduleLilia Bi, PhDRecovery and Reduction Costs for Cell-based Gene Therapy ProductsThis presentation will discuss the recovery and reduction costs for cell-based gene therapy products from following aspects:What is the purpose of cost recovery and where does it apply?Cost recovery regulations and rulesWhat can be charged for under cost recovery?Process for requesting cost recovery and what information sponsors need to providePotential cost reduction by following the step-wise approach to implementing cGMP’s and product characterizationCarl H. June, MDGenetically Engineered T Cells: Can We Afford Not To Do This?A key concern with adoptive cell transfer therapy is a financial issue related to the potential costs of a lymphocyte infusions. Factors that relate to the eventual pricing ofa personalized therapy consisting of autologous T cells are numerous and include issues such as the magnitude of efficacy, durability of responses, safety and tolerability.In this regard, it is instructive to consider allogeneic stem cell transplantation procedures that have potent efficacy and considerable toxicity, and whether adoptive T celltransfer therapy could supplant and extend this procedure.Jeffrey Schlom, PhDRecombinant Cancer Vaccines as Monotherapy and in Combination TherapyRecombinant poxviral vaccines have been developed that contain the transgenes for one or more tumor associated antigens and three T-cell costimulation molecules(TRICOM). Preclinical studies have shown that these vaccines have the ability to break tolerance and induce therapeutic activity in several models. Randomized clinicaltrials in patients with prostate cancer have shown statistical improvements in survival. Preclinical studies have shown that radiation and or chemotherapy can alter the phenotypeof tumor cells and render them more susceptible to T-cell lysis; combination therapy studies with vaccine are ongoing. Recently results have shown that moleculesinvolved in the Epithelial to Mesenchymal Transition (EMT) metastatic process can be targeted by vaccine therapy.Scientific Symposium 31110:30 am - 12:30 pmRoom: Marriott Ballroom Salon 1Stem Cells and MalignancyChairEvan Y. Snyder, MD, PhD, FAAPSpeakersInder M. Verma, PhDCancer Stem Cells: Lessons from GlioblastomasWe have generated novel mouse models of human gliomas using lentiviral vectors containing oncogenes or shRNA to suppressor genes. The resulting tumors had stemcell properties. We will discuss the implications of these results.Roger J. Packer, MDChildhood Brain Tumors: Opportunities for Biologic-Based TherapiesBrain tumors are the most common solid malignancy of childhood and are the leading cancer related cause of morbidity and mortality in the pediatric-aged patients.Progress has been made in the management of childhood brain tumors, but lack a clear understanding of the molecular pathogenesis of these tumors and ways to exploitthis molecular pathogenesis for treatment has slowed progress. Over the past decade, there have been tremendous advances in the understanding of the molecularunderpinnings of a variety of different childhood brain tumors including the most common malignant brain tumor, the medulloblastoma, and the most common benigntumor, the low grade-glioma. Molecular targets have been identified and a host of biologic agents including antiangiogenesis drugs, tyrosine kinase growth inhibitors, andagents interfering with intracellular signaling are in clinical trials. To date, their use has not been associated with improvements in survival but there is great enthusiasm incontinuing their study with the hope of developing more effective and safer therapies.Friday, May 21 st