Research

Ofelia Alvarez, M.D.

Dr. Ofelia Alvarez is a Professor of Clinical Pediatrics. Her research interests are in sickle cell disease and global health.  In the sickle cell disease field, she has performed clinical research in sickle cell nephropathy, demonstrating the importance of cystatin C as a marker of renal function, and evaluating markers of acute kidney injury.  She has participated in several federally funded hydroxyurea and stroke prevention studies, including BABY HUG, STOP, STOP2, POST STOP, SWiTCH, TWiTCH, and DISPLACE.  In addition, she has been center investigator for new drug safety and efficacy trials (placebo-controlled clinical trial for crizanlizumab and voxelotor among others), which have resulted in new FDA-approved drugs for the management of patients with sickle cell disease. Over the last 5 years, Dr. Alvarez has established partnerships with pediatricians in Haiti and started a sickle cell newborn screening program, validating a point-of-care test for the early identification of newborns with sickle cell disease.  Currently, she has an NIH grant to compare populations of children with sickle cell disease in four Haitian sites with the University of Miami cohort, with the implementation of newborn screening and transcranial Doppler ultrasound screening for children with sickle cell anemia, who may be at risk of developing a stroke.

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Warren Alperstein, M.D.

Dr. Warren Alperstein is an Assistant Professor and his area of focus includes bone marrow transplant and cellular therapies with an interest in graft vs host disease (GVHD) and haplo-identical transplants. His expertise also includes post-transplant lymphoproliferative disorder (PTLD), and he is a member of the Sylvester Comprehensive Cancer Center’s Adolescent and Young Adult (AYA) Program and conducts clinics at SCCC Alex’s Place and SCCC at Deerfield Beach.  He was awarded the 2019 Hyundai Hope Scholar Grant for collaborative work on High-throughput drug screen for pediatric sarcomas and he is involved in clinical trials through the PTCTC, in addition to investigator driven protocols.  He participates on immunotherapy clinical trials with engineered cytotoxic T cells (CTLs) against EBV positive PTLD/lymphoma, and he is an active educator with the Miller School of Medicine.  

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Julio Barredo, M.D.

Dr. Julio Barredo is a Professor of Pediatrics, Medicine, and Biochemistry and Molecular Biology and performs basic, translational and clinical research. As a full member of the Sylvester Comprehensive Cancer Center, he leads the cancer center efforts in the Adolescent and Young Adult (AYA) Program. He continues to mentor PhD investigators and physician scientists, and as a member of the graduate school teaches students enrolled in the Cancer Biology Program. His laboratory and clinical research are funded by federal, state, national and international foundations and industry. His laboratory and translational research focus in in understanding the role of energy metabolism in resistant/refractory acute leukemia and his lab uncovered the role of AMP-activated protein kinase (AMPK) as a therapeutic target in acute leukemia and other cancers. His clinical research focuses on refractory/resistant acute lymphoblastic leukemia (ALL) and has developed national protocols for the Children’s Oncology Group (COG), TACL, the Sunshine Project, as well as investigator-initiated studies (IIS) for the treatment of relapsed/refractory acute leukemia.

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David Crawford, M.D., Ph.D.

David Crawford is an Associate Professor with diverse clinical interests in hematopoietic stem cell transplantation. One of his major focuses is the use of transplant to treat many different debilitating or lethal non-malignant conditions such as the mucopolysaccharidoses, hemoglobinopathies, conditions with defective or dysregulated immunity, and other genetic disorders. Another focus has been the prevention and treatment of transplant-related complications such as sinusoidal obstruction syndrome, transplant-associated thrombotic microangiopathy, life-threatening infections, and graft-versus-host disease. Finally, the development of CAR-T and other cellular therapies as well as gene therapy for genetic disorders will be a focus in coming years.

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Aditi Dhir, M.D.

Dr. Dhir is an Assistant Professor of Pediatrics and her primary focus is sarcomas, cancer genetics and epigenetics. She is a member of the Cancer Epigenetics program at SCCC and is also a part of the adolescent and young adults (AYA) Sarcoma program. She is actively involved in the Phase I clinical trial program at University of Miami and represents UM as the vice-PI (Primary Investigator) for the multi-institutional TACL and Sunshine project collaborations. She is also the institutional PI for a multi-institutional ‘COVID-19 registry in pediatric, adolescents and young adults with malignancies’ study, which provides real-time feedback to clinicians. Her research and clinical interests include better identification of germline predispositions to improve cancer surveillance as well as discovery of new molecular targets in sarcomas to develop more individualized therapy in this era of precision oncology.

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Taumoha Ghosh, M.D., MS

Dr. Ghosh is an Assistant Professor of Pediatrics and her primary clinical focus is in solid tumors, retinoblastoma, and survivorship. She is a member of the Cancer Control program at Sylvester Comprehensive Cancer Center (SCCC) and is the Children's Oncology Group (COG) Cancer Control Responsible Investigator for the University of Miami (UM) Miller School of Medicine - Sylvester Cancer Center. Her clinical research has included collaborations within COG looking at the impact of obesity on pediatric cancers with a particular interest in associated disparities, for which she has was awarded an Emerging Scientist Award from the Children’s Cancer Research Fund (CCRF). Currently, she is involved in a Childhood Cancer Survivor Study (CCSS) looking at lung cancers as a subsequent malignant neoplasm in survivors of childhood cancer and is developing a retinoblastoma survivorship program at UM. Her clinical and research focus remains within cancer control and survivorship to improve long term outcomes of survivors of childhood cancer. She is also an enthusiastic educator with the Miller School of Medicine.

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Fernando Corrales-Medina, M.D.

Dr. Fernando Corrales-Medina is an Assistant Professor of Pediatrics and the national principal investigator of several investigator-initiated clinical studies and industry-sponsored phase III clinical trials. His work focuses in the use of point-of-care ultrasound to evaluate the presence and progression of joint disease in pediatric patients with hemophilia and women with bleeding disorders. Several industry-sponsored phase III clinical trials are available for patients with hemophilia evaluating the efficacy and safety of new factor VIII recombinant concentrates as well as other novel hemophilia non-factor replacement therapies, including the use of humanized, recombinant bispecific immunoglobulin G4 monoclonal antibody and small interfering RNA (siRNA) thrombin inhibitor therapy and gene therapy in the treatment of hemophilia.  Dr. Corrales-Medina is also the on-site principal investigator for several phase III randomized clinical studies evaluating the efficacy and safety of novel direct oral anticoagulants (DOACs) in children with acute thrombosis and those in need of thromboprophylaxis.

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Joanna A. Davis, M.D.

Dr. Joanna Davis is an Associate Professor of Pediatrics and established the Pediatric Hemophilia Treatment Center in 1987 and has a particular interest in women with bleeding disorders.  She is committed to education and outreach to the patient community as well as to health care providers and has established several educational initiatives which are nationally implemented. Dr. Davis sees pediatric patients with bleeding disorders at the Sylvester Comprehensive Cancer Center Alex’s Place and, monthly, at the UHealth facility in Plantation.

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Asha Pillai, M.D.

Dr. Asha Pillai is an Associate Professor of Pediatrics and Microbiology & Immunology, and Director of the Federation of Clinical Immunology Societies (FOCIS) Center of Excellence in Human Immunology at the University of Miami Miller School of Medicine.  Her areas of research focus are in the pre-clinical elucidation of innate immune strategies for transplantation tolerance and anti-tumor immunotherapy and in development of novel conditioning strategies to induce transplant immune tolerance across histocompatibility barriers. These studies are extending the application of curative haplo-identical hematopoietic stem cell transplantation (HSCT) for non-malignant disorders with high tolerance barriers, including severe aplastic anemia (SAA) and hemoglobinopathies. 

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Winsome S. Thompson, Ph.D.

Dr. Winsome Thompson is an Assistant Professor and the Pediatric Psychologist who provides services to pediatric hematology-oncology and BMT patients. Dr. Thompson’s research focus is on the cognitive and emotional effects of cancer and bleeding disorders. She is currently a co-investigator on an intervention study to improve academic achievement in children with sickle cell disease who are at risk for central nervous system impairment. This study received funding support from the National Heart Lung and Blood Institute.

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Edward Ziga, M.D.

Dr. Edward Ziga is an Assistant Professor and his clinical research focuses on improving the options for pediatric and adolescent patients who need hematopoietic stem cell transplantation (HSCT) by developing new regimens that use HLA mismatched first-degree relatives as donors. His team has demonstrated the safety of this therapy through an initial pilot study of five patients who remain asymptomatic and disease free for a median of 12 months follow up since HSCT. Following this success, they have developed a phase I/II trial that is pending IRB approval to start enrollment. He collaborates with industry and is the site principal investigator for an ongoing chimeric antibody receptor (CAR) T-cell therapy study to treat children and adolescents with relapsed or refractory leukemia and lymphoma.

Our division laboratories are funded by federal and state agencies (National Cancer Institute, National Heart Lung Blood Institute, State of Florida  Live Like Bella), and by several nationally and internationally recognized foundations (Leukemia Lymphoma Society. St. Baldrick’s Foundation, Micah Batchelor Foundation, National Pediatric Cancer Foundation, Sebastian Strong Foundation, and others).

Dr. Andreansky Lab

Dr. Samita Andreansky’s lab centers on the development of cancer vaccines. Joining the Miller School of Medicine from the University of Arizona, Dr. Andreansky has extensive experience with viral oncology and tumor immunology. Her work is currently funded by the American Cancer Society.

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Dr. Barredo Lab 

Dr. Barredo’s work focuses on investigating how tumor metabolism leads to drug resistance in acute leukemia and development of new therapies for childhood leukemia. Dr. Barredo’s work has led to the discovery of AMP-activated protein kinase (AMPK) as a target for acute leukemia and has resulted in the translation into three IIS for patients with relapsed/refractory acute leukemia, including pediatric and AYA patients. His lab uncovered that acute lymphoblastic leukemia cells are exquisitely vulnerable to ER stress secondary to metabolic and proteotoxic stress. He has also uncovered novel a role for AMPK, which differs from its canonical role, in which responses to metabolic/energy stress are mediated via binding of AMPK to chromatin leading to changes in gene expression as an adaptive/survival mechanism. 

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Dr. Pillai Lab

Dr. Pillai’s lab elucidated pathways for in vivo expansion of Treg across major donor-recipient histocompatibility barriers and recently published novel reduced-toxicity HSCT preparative regimens harnessing these immune mechanisms in a preclinical mouse model of beta-thalassemia and in clinical series for pediatric patients with SAA. These findings have now been extended to a clinical trial using haploidentical donors to cure sickle cell disease (SCD) and other non-malignant conditions (in preparation).  Additionally, her group developed a robust and reproducible protocol for ex vivo expansion of human invariant natural killer T (iNKT) cells and defined mechanisms to augment their cytolytic and regulatory capacity for peri-transplant and non-transplant cancer immunotherapy. 

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The Division of Pediatric Hematology/Oncology participates in clinical trials for children, adolescents and young adult (AYA) cancers and blood disorders. The Early Phase Clinical Trials Program brings experimental agents to pediatric/young adult oncology patients in the state of Florida in a collaborative method of care. Our faculty develops novel institutional investigator-initiated studies (IIS) based on our own laboratory discoveries and participates in the development of national clinical trials through our membership on national clinical trial consortia. 

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Leukemias & Lymphomas

We are a full member of Therapeutic Advances in Childhood Leukemia and Lymphoma . As a TACL member, phase I/II trials are available through our group, and dovetail with very high-risk leukemia patients who are under consideration for hematopoietic stem cell transplant through our designated Children’s Oncology Group Bone Marrow Transplant institute. Our participation and leadership on national consortia have led to the completion of several IIS using novel agents for the treatment of relapsed/refractory leukemia in pediatric and AYA patients.

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Sarcomas & Brain Tumor

We have a major focus on developing tumor vaccines for solid tumors and brain tumors of childhood, as well as clinical trials for adolescent and young adult patients. As member of Sarcoma Alliance for Research through Collaboration and the National Pediatric Cancer Foundation Sunshine Project, we have the opportunity to bring clinical trials for sarcomas to patients in south Florida.

Through our involvement in national groups, we are able to develop and lead clinical trials for children across the country based on the work of our own laboratory investigators. Currently there are multiple open pediatric Phase I/II protocols for childhood leukemia and solid tumors. 

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Hemophilia Treatment Center (HTC)

The University of Miami - Hemophilia Treatment Center (HTC) was established in 1987. It is the only federally funded 501(c)(3) nonprofit, combined Adult and Pediatric hemophilia program in South Florida. Our families may participate in the American Thrombosis and Hemostasis Network (ATHN) project. Availability to the Centers for Disease Control Community Counts database.  Patients with bleeding disorders have access to several phase II-III clinical studies to investigate novel factor replacement concentrates as well as to novel, non-factor interventions for patients with hemophilia A and B, including phase 1 gene therapy clinical trials. The HTC also offers several investigator-initiated clinical trials focusing in the use of point-of-care ultrasound to evaluate the presence and progression of joint disease in pediatric patients with hemophilia and women with bleeding disorders.   

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Sickle Cell Disease Program

The Sickle Cell Disease Program is funded by the National Heart Lung and Blood Institute and is one of only ten national Basic and Translational Research Programs in Sickle Cell Disease. We participate in several NIH sponsored, industry and investigator-initiated studies. 

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Pediatric Neuro-Oncology Program

The Pediatric Neuro-Oncology Program is led by the only Pediatric Oncologist certified in Neuro-Oncology in the state of Florida, Antonello Podda, M.D., and is comprised of a multidisciplinary team of professionals who deliver the necessary, comprehensive care to children affected by brain and spinal cord tumors. Using the most sophisticated of imaging techniques including MR spectroscopy and PET-CT as well as the most advanced radiation therapy methodologies like IMRT and gamma knife, the multi-specialty team delivers complex care covering all aspects from diagnosis and multimodal therapy to physical and neuro-cognitive rehabilitation.

Care for children with brain and spinal cord tumors is further enhanced by pediatric rehabilitation medicine with inpatient and outpatient facilities, audiologists and speech therapists, child neuropsychologists, social workers, teachers and child life specialists. The children are followed from the day of diagnosis and therapy through the time of reentry to school and society, to early adulthood for long-term surveillance.