Sylvester Comprehensive Cancer Center

John Goldberg, M.D.

John Goldberg, M.D.

Assistant Professor of Clinical Pediatrics

Description of Research

Dr. Goldberg's research focuses on the translation of laboratory discoveries into therapies for pediatric cancer and for cancers that overlap between adults and adolescents. This work has two main clinical thrusts: One is to develop targeted treatments for liquid and solid cancers based on work from laboratories here in Miami. The other is to develop immunotherapy based on work from the Dodson Interdisciplinary Immunotherapy Institute, including a DC vaccine program.

Dr. Goldberg's programmatic goal is to continue to develop a Phase I pediatric cancer clinical trials organization within the University of Miami Sylvester Comprehensive Cancer Center, and to provide leadership to nascent efforts across Florida to link pediatric cancer centers in a state-wide clinical trials group. In addition to the emphasis on Florida based developmental therapeutics, Dr. Goldberg is collaborating with HGG Immuno, based in Belgium, to develop the DC vaccines for brain tumors and sarcoma, and received a K12 award last year to expand this DC work and incorporate aptamers into pediatric cancer immunotherapy.

Previously, Dr. Goldberg led a vaccine protocol and a c-Met inhibitor protocol in patients with unusual sarcomas based on transcription factor biology. The c-Met inhibitor trial was opened at UM/Sylvester to treat both adults and children with these unusual cancers. He seeks to take his therapeutic ideas into the clinic through UM, and to bring these ideas to the national groups to make his novel ideas available to children everywhere. Additionally, because of group memberships, he gains access for the children of Florida to more novel treatments from investigators across the world.

In hematological malignancies, Dr. Goldberg is the national chair of a protocol through TACL studying panobinostat. Panobinostat is a novel HDAC inhibitor, more potent than currently approved HDAC inhibitors, found to be synergistic with chemotherapy against ALL. He has initiated an AMP kinase activator trial in relapsed pediatric acute lymphoblastic leukemia through the Sunshine Project. By speeding drug development in the pediatric setting, he takes advantage of laboratory findings to get the compound earlier for children who desperately need new treatments. Both of these protocols are based on work from Dr. Barredo's laboratory.

In solid tumors, he has grant funding to study DC vaccines in patients with recurrent high grade glioma as well as to study DC vaccines in conjunction with standard chemotherapy in patients with relapsed sarcoma. The DC vaccine technique is based on science first described in murine systems by UM’s Eli Gilboa. Dr. Gilboa removed PGE2 from the maturation cocktail of the DCs and found that by substituting topical imiquimod treatment for PGE2, DC were mature but less tolerogenic. His collaborator, Stefaan Van Gool, took this technique into the clinic with remarkable results in brain tumor patients. He is now pursuing an international study of the DC technique, with other new advances from the Dodson mixed into the program. Dr. Goldberg recently began enrolling subjects and treating them on these immunotherapy protocols.


  • Initiated and oversaw Phase I clinical trial of vaccine in pediatric and adult patients in Boston
  • Lead investigator and initiating investigator for ARQ 197-204, clinical trial of novel c-Met inhibitor in the MiT tumor patients
  • SARC / TACL / Sunshine Project / HGG Immuno PI at the University of Miami
  • Director, Pediatric Oncology Early Phase Clinical Trials Program at UM/Sylvester Comprehensive Cancer Center
  • Has now initiated DC vaccination of patients with brain tumors and sarcoma at UM/Sylvester in collaboration with tumor immunology researchers
  • Member of the TACL Steering Committee and the Sarcoma Foundation of America Medical Advisory Board

Selected Cancer-Related Publications

  • Shah AH, Bregy A, Heros DO, Komotar RJ, Goldberg J. Dendritic Cell Vaccine for Recurrent High Grade Gliomas in Pediatric and Adult Subjects: Clinical Trial Protocol. Neurosurgery :,2013 Read more »
  • Bregy A, Wong TM, Shah AH, Goldberg JM, Komotar RJ. Active immunotherapy using dendritic cells in the treatment of glioblastoma multiforme. Cancer Treat Rev :,2013 Read more »
  • Lindsey SF, Sanchez MI, Elgart GW, Milikowski C, Civantos FJ, Goldberg J, Grichnik JM. Malignant melanoma from a nevus of Ota in a pediatric patient with fatal outcome. J Am Acad Dermatol 69:e195-7,2013 Read more »
  • Goldberg JM. Immunotherapy of sarcomas. Curr Opin Oncol 25:390-7,2013 Read more »
  • Wagner AJ, Goldberg JM, Dubois SG, Choy E, Rosen L, Pappo A, Geller J, Judson I, Hogg D, Senzer N, Davis IJ, Chai F, Waghorne C, Schwartz B, Demetri GD. 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 Read more »
  • Goldberg J, Furman WL. Management of colorectal carcinoma in children and young adults. J Pediatr Hematol Oncol 34 Suppl 2:S76-9,2012 Read more »
  • Dillmon M, Goldberg JM, Ramalingam SS, Mayer RJ, Loehrer P, Van Poznak C. Clinical practice guidelines for cancer care: utilization and expectations of the practicing oncologist. J Oncol Pract 8:350-3,2 p following 353,2012 Read more »
  • Goldberg JM, Scully RE, Sallan SE, Lipshultz SE. Cardiac failure 30 years after treatment containing anthracycline for childhood acute lymphoblastic leukemia. J Pediatr Hematol Oncol 34:395-7,2012 Read more »

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