W. Scott Goebel, M.D, Ph.D.
Assistant Professor of Pediatrics
705 Riley Hospital Drive, ROC 4340
Indianapolis, IN 46202
- Pediatric Stem Cell Transplantation
- Pediatric Hematology Oncology
Areas of Interest
My clinical/translational research interests include cellular therapies to enhance hematopoietic stem cell transplantation and reduce regimen-related toxicities, and gene therapy for hematopoietic diseases.
I have been on the faculty at Indiana University School of Medicine since 2001. As a clinical stem cell transplant physician I take care of children with a variety of malignant and non-malignant diseases, and I served as the Interim Director of the Pediatric Stem Cell Transplantation Program from 2012-2014. With my background in transplantation, I have a particular interest in cellular therapeutics and gene therapy. My laboratory background is in gene therapy, submyeloablative conditioning for stem cell transplantation, and mesenchymal stem cell (MSC) biology. In my laboratory I have studied the impairment of stem cell function following ex vivo gene transfer, and potential means to enhance engraftment of transduced hematopoietic stem cells in submyeloablated hosts, laying the foundation for clinical gene therapy studies. I continue to MSC biology, particularly how to isolate, expand and cryopreserve mesenchymal stem cells from various tissue sources in a cGMP-compliant manner for clinical use. My clinical and translational research interests currently include development of cellular therapies to enhance hematopoietic stem cell transplantation and reduce regimen-related toxicities, and development of clinical gene therapy protocols.
|1995||MD Indiana University School of Medicine|
|1995||PhD Indiana University School of Medicine|
|1998||Residency St Louis Children's Hospital|
|2001||Fellowship Indiana University School of Medicine|
Honors & Awards
- 2000-2002 William Kennedy Research Fellow of the National Childhood Cancer Foundation
- 2007 Indiana University School of Medicine Trustee Teaching Award
- 2012 Plenary Session abstract presentation, AABB Annual Meeting
- 2013, 2014 Grant reviewer, St. Baldrick’s Foundation
Riley Outpatient Center
705 Riley Hospital Drive
Indianapolis, IN 46202
Research & Grants
Two major projects are in progress. The first is a clinical Phase I/II gene therapy study using a novel vector and envelope system to treat bone marrow failure in children and young adults with bone marrow failure due to Fanconi anemia. The lentiviral vector and foamyvirus envelope in the protocol was developed by researchers here at IU. Patients will have bone marrow harvested, transduced with the vector-envelope combination in vitro, and have their cells re-infused the following day. Patients will be followed over several years for the correction of their marrow failure and any toxicities. The protocol has been submitted to the FDA, and a grant to fund the project is under review. The second project involves the use of MSC to enhance engraftment and prevent graft-vs.-host disease in stem cell transplant recipients. We have successfully treated one patient to date with MSC generated at Cook General BioTechnology LLC, a local biotech company, with plans to treat a second patient this summer. Our first patient engrafted promptly, had no graft-vs.-host disease, and is clinically well 2 ½ years after transplant (Goebel WS et al., Transfusion, vol. 52 supplement, page 13A, abstract P6-030A; manuscript in preparation). In collaboration with Cook General BioTechnology, we intend to capitalize on this experience to develop larger institutional and multi-center cell therapy trials.
Grants under review to fund a clinical gene therapy protocol for Fanconi anemia patients (PI), and to study the use of mesenchymal stem cells for bone regeneration (collaborator).
Goebel, W.S., N. Pech, J.L. Meyers, E.F. Srour, M.C. Yoder and M.C. Dinauer. A murine model of antimetabolite-based, submyeloablative conditioning for bone marrow trans-plantation: biologic insights and potential applications. Exp. Hematol. 32:1255-64, 2004.
Goebel, W.S., L.A. Mark, S.D. Billings, J.L. Meyers, N. Pech, J.B. Travers, and M.C. Dinauer. Gene Correction Reduces Cutaneous Inflammation and Granuloma Formation in Murine X-Linked Chronic Granulomatous Disease. J. Invest. Dermatol.125:705-10, 2005.
Wyss, B.K., J.L. Meyers, A.L. Sinn, S. Cai, K.E. Pollok and W. S. Goebel. A novel competitive repopulation strategy to quantitate engraftment of ex vivo manipulated murine marrow cells in submyeloablated hosts. Exp. Hematol. 36: 513-521, 2008.
Perry, B.C., D. Zhou, X. Wu, F.C. Yang, M.A. Byers, T.-M.G. Chu, J.J. Hockema, E.J. Woods, and W. S. Goebel. Collection, cryopreservation and characterization of human dental pulp-derived mesenchymal stem cells for banking and clinical use. Tissue Engineering Part C: Methods 14(2): 149-156, 2008
Li, Y., S. Chen, J. Yuan, Y. Yang, J. Li, J. Ma, X. Wu, M. Freund, K. Pollok, H. Hanenberg, W.S. Goebel, and F-C. Yang. Mesenchymal stem/progenitor cells promote the reconstitution of exogenous hematopoietic stem cells in Fancg-/- Mice in vivo. Blood 113:2342-2351, 2009.
Sadat, M.A., S. Dirscherl, L. Sastry, J. Dantzer, N. Pech, S. Griffin, T. Hawkins, Y. Zhao, C.N. Barese, S. Cross, A. Orazi, C. An, W.S. Goebel, M.C. Yoder, X. Li, M. Grez, K. Cornetta, S.D. Mooney and M.C. Dinauer. Retroviral vector integration in post-transplant hematopoiesis in mice conditioned with either submyeloablative or ablative irradiation. Gene Therapy 16(12):1452-64, 2009.
Alge, D.L., D. Zhou, L.L. Adams, B.K. Wyss, M.D. Shadday, E.J. Woods, T.M.G. Chu, and W. S. Goebel. Donor-matched comparison of dental pulp stem cells and bone marrow-derived mesenchymal stem cells in a rat model. J. Tissue Eng. Regen. Med. 4(1):73-81, 2010
Woods, E.J., A. Bagchi, W.S. Goebel, R. Nase and V.D. Vilivalam. Container system for enabling commercial production of cryopreserved cell therapy products. Regen. Med. 5(4):659-67, 2010.
Alge, D.L., W.S. Goebel and T.M.G. Chu. In vitro degradation and cytocompatibility of DCPD cements prepared using the MCPM/HA system reveals rapid conversion to HA as a key mechanism. J Biomed Mater Res B Appl Biomater; 100B:595–602, 2012.
Alge D.L., W.S. Goebel, and T.M.G. Chu. Effects of DCPD cement chemistry on degradation properties and cytocompatibility: comparison of MCPM/?-TCP and MCPM/HA formulations. Biomed Mater. 8(2):025010, 2013.