Sylvester Comprehensive Cancer Center

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John M. Goldberg, M.D.

General Information

John M. Goldberg, M.D.


  • Fax: 305-325-8387


  • English


  • American Board of Pediatrics-Pediatric Hematology-Oncology


  • Pediatrics
  • Pediatric Hematology-Oncology (Pediatrics)


  • Assistant Professor of Clinical Pediatrics
  • Director, Pediatric Oncology Early Phase Clinical Trials Program

Clinical Interests


Recurrent or refractory leukemia and lymphoma

Immunotherpay in sarcoma and brain tumors

Developmental therapeutics and pediatric Phase I clinical trials

Adolescent and Young Adult Oncology

Research Interests

My research focuses on the translation of laboratory discoveries into new therapies for patients. My particular interests include immunotherapy, new treatments for leukemia and lymphoma, new treatments for patients with sarcoma and brain tumors, and the development of HDAC inhibitors for children with cancer. I trained at the Dana-Farber Cancer Institute in the laboratory of David E. Fisher, where I developed a background in molecular oncology, and carried out immunotherapy and targeted trials with the adult melanoma and sarcoma groups. Through our Phase I program, we bring homegrown science from the Sylvester Comprehensive Cancer Center to patients. Our clinical trials dovetail with my interests and passion for helping patients with the most difficult cancers to treat, such as recurrent brain tumors and sarcoma and refractory leukemia.

Pediatric Phase I Program

The pediatric Phase I program, or Early Phase Clinical Trials Program, is closely integrated into the research enterprise both of the Sylvester and the Department of Pediatrics. The program is part of Therapeutic Advances in Childhood Leukemia and Lymphoma (TACL), an international group dedicated to finding new treatments for children with leukemia and lymphoma. I lead a national trial through TACL, T2009-012, A Phase I Dose Finding Study Of Panobinostat In Children With Refractory Hematologic Malignancies, and am a member of the Steering and Prioritization Committee for the group, as well as the principal investigator at the University of Miami for TACL. I lead a national trial through the Sunshine Project, a Florida based Phase I group dedicated to carrying out early phase clinical trials in children with cancer, which combines metformin with chemotherapy for childhood leukemia. Both of these clinical trials are based on collaboration with the laboratory of Julio Barredo, the division chief of pediatric hematology oncology at the University of Miami, and Director of Children’s Cancer Programs for Sylvester. I am member of the executive committee for the Sunshine Project. Through these groups, I seek to build the evidence, based both on pre-clinical laboratory work of collaborators and through initial safety data from Phase I trials, to bring new drugs such as HDAC inhibitors into combination therapy for children with cancer.

Immunotherapy Program

Cancer and cancer therapy can impair the immune response against tumors, and tumors take advantage of this to evade destruction. We seek to turn the immune response back on, to rid the patient of cancer. Within our program, we carry out several immunotherapy trials based on vaccination that can accrue both children and adults. These trials are based on the principal of “in situ maturation” of dendritic cells described by the University of Miami’s Eli Gilboa, and represent collaboration with Stefaan Van Gool, of the Catholic University of Leuven in Belgium, who put this methodology into practice in patients. Currently, children and adults with brain tumors and sarcoma are recruited to three trials; our intention is to expand this program and to combine newer therapies with vaccination to achieve synergy. We collaborate with the Interdisciplinary Stem Cell Institute, as well as the University of Miami Brain Tumor Initiative, led by Ricardo Komotar.

Aptamer immunotherapy

Based on collaboration with Eli Gilboa’s laboratory in the Dodson Interdisciplinary Immunotherapy and Institute, we are developing aptamers, oligonucleotide based drugs, to stimulate the immune system to attack cancer. Aptamers can help focus a lackluster immune attack on tumors, and can overcome the cancer’s attempt to turn off the immune response and act as a so called immune checkpoint inhibitor. The potential targets for aptamer based immunotherapy are nearly limitless.


Research profile


  • Weiss, A, Gill J, J, Goldberg, J, Lagmay, J, Spraker-Perlman, H, Venkatramani, R and Reed, D. Advances in Therapy for Pediatric Sarcomas. Curr Oncol Rep 16:395 (2014).
  • Goldberg, JM, Panoff, J. Pediatric Oncology Drug Development: A Case Report and Pathways Forward. Journal of Pediatrics and Therapeutics (2014).
  • Wilky, B.A. & Goldberg, J.M. Immunotherapy in sarcoma: a new frontier. Discovery medicine 17, 201-206 (2014).
  • Goldberg, J.M., Gavcovich, T., Saigal, G., Goldman, J.W. & Rosen, L.S. Extended Progression-Free Survival in Two Patients With Alveolar Soft Part Sarcoma Exposed to Tivantinib. Journal of clinical oncology : official journal of the American Society of Clinical Oncology (2014).
  • Shah, A.H., Bregy, A., Heros, D.O., Komotar, R.J. & Goldberg, J. Dendritic cell vaccine for recurrent high-grade gliomas in pediatric and adult subjects: clinical trial protocol. Neurosurgery 73, 863-867 (2013).
  • Lindsey, S.F., Sanchez, M.I., Elgart, G.W., Milikowski, C., Civantos, F.J., Goldberg, J. & Grichnik, J.M. Malignant melanoma from a nevus of Ota in a pediatric patient with fatal outcome. Journal of the American Academy of Dermatology 69, e195-197 (2013).
  • Goldberg, J.M. Immunotherapy of sarcomas. Current opinion in oncology 25, 390-397 (2013).
  • Dillmon, M., Goldberg, J.M., Ramalingam, S.S., Mayer, R.J., Loehrer, P. & Van Poznak, C. Clinical practice guidelines for cancer care: utilization and expectations of the practicing oncologist. Journal of oncology practice / American Society of Clinical Oncology 8, 350-353, 352 p following 353 (2012).
  • Wagner, A.J., Goldberg, J.M., Dubois, S.G., Choy, E., Rosen, L., Pappo, A., Geller, J., Judson, I., Hogg, D., Senzer, N., Davis, I.J., Chai, F., Waghorne, C., Schwartz, B. & Demetri, G.D. Tivantinib (ARQ 197), a selective inhibitor of MET, in patients with microphthalmia transcription factor-associated tumors: Results of a multicenter phase 2 trial. Cancer (2012).
  • Goldberg, J. & Furman, W.L. Management of colorectal carcinoma in children and young adults. Journal of pediatric hematology/oncology 34 Suppl 2, S76-79 (2012).
  • Goldberg, J.M., Scully, R.E., Sallan, S.E. & Lipshultz, S.E. Cardiac failure 30 years after treatment containing anthracycline for childhood acute lymphoblastic leukemia. Journal of pediatric hematology/oncology 34, 395-397 (2012).
  • Wittman, B., Horan, J., Baxter, J., Goldberg, J., Felgar, R., Baylor, E., Cromwell, B., Cross, N. & Bennett, J.M. A 2-year-old with atypical CML with a t(5;12)(q33;p13) treated successfully with imatinib mesylate. Leukemia research 28 Suppl 1, S65-69 (2004).
  • Goldberg, J.M., Silverman, L.B., Levy, D.E., Dalton, V.K., Gelber, R.D., Lehmann, L., Cohen, H.J., Sallan, S.E. & Asselin, B.L. Childhood T-cell acute lymphoblastic leukemia: the Dana-Farber Cancer Institute acute lymphoblastic leukemia consortium experience. Journal of clinical oncology : official journal of the American Society of Clinical Oncology 21, 3616-3622 (2003)

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