Basic and Translational Research: Oncology
- WOLF Foundation Leukemia/Lymphoma Laboratory: To understand the molecular mechanisms that promotes and sustains the malignant proliferation and survival of leukemic cells. Adolfo Ferrando (supported by the Leukemia and Lymphoma Society, Stand Up To Cancer Foundation, NIH R01 CA120196, NIH R01 CA129382, NIH R01CA155743)
- Brain and Nervous System Tumor Laboratory: To study the deregulation of the cell cycle and the differentiation in tumors derived from the brain and nervous system; Anna Lasorella (supported by the Brain Tumor Foundation, NIH R01 CA101644, and NIH R01 CA85628)
- Taybanz/ Matthews Family Foundation Solid Tumor Laboratory: To understand the biology of pediatric solid tumors and investigate angiogenesis, as a lens to illuminate the fundamental steps of tumor progression, metastasis, and resistance; Darrell Yamashiro (NIH R01 CA124644).
- Orjuela Laboratory: To investigate etiologic mechanisms underlying development of childhood cancer secondary to environmental exposures and genetic susceptibility, with specific focus on genetic damage associated with pre-natal and early childhood exposures to pro-carcinogenic polyaromatic hydrocarbons and aberrant metabolism of ingested folate pathway nutrients; Manuela Orjuela (NIH U01CA159157, R21CA167833)
- Shan Laboratory : To understand the mechanism of DNA double strand break repair and the pathways that lead to recurrent chromosomal translocations in human cancer; Shan Zha (supported by St. Baldrick Foundation, NIH R01 CA158073)
- Kung Laboratory: Identification of causes of various blood and solid cancers, and development of novel diagnostic and therapeutic strategies; Andrew Kung (supported by the Leukemia and Lymphoma Society, NIH R01 GM083054, NIH R21 CA166805, NIH R03 DA034596)
Clinical Research: Oncology
- Children’s Oncology Group: In addition to being a participating institution of the largest National Cancer Institute supported clinical trials network for childhood cancer research, members of the faculty hold key leadership positions within the organization: Julia Glade Bender (Developmental Therapeutics, Bioethics, Bone Sarcoma, Neuroblastoma), Eduvigis Cruz-Arrieta (Minority Affairs), Kara Kelly (Hodgkin Lymphoma, Cancer Control, Scientific Chairs), Jennifer Levine (Survivorship), Alice Lee (Institution Principal Investigator, Developmental Therapeutics), Manuela Orjuela (Non Hodgkin Lymphoma, Retinoblastoma, Epidemiology), Stephen Sands (Behavioral Sciences, CNS Tumors), Prakash Satwani (Hodgkin Lymphoma), and Darrell Yamashiro (Neuroblastoma, Renal Tumors).
- Dana Farber Cancer Institute Acute Lymphoblastic Leukemia Consortium: Our program is one of nine collaborating institutions who collectively accrue newly diagnosed pediatric patients for treatment of acute lymphoblastic leukemia to help advance research into effective treatments for this disease. This research includes a large prospective observational study evaluating the relationship of the diet with treatment related toxicity; Kara Kelly, Elena Ladas and Manuela Orjuela (American Institute for Cancer Research).
- Pediatric Cancer Foundation Developmental Therapeutics Program: In addition to being one of only 21 elite institutions in the COG Phase I and Pilot Consortium, the Pediatric Cancer Foundation Developmental Therapeutics Program offers clinical trials of new therapeutics as a member of the limited institution, Therapeutic Approaches to Childhood Leukemia (TACL) Consortium and a special member of the New Approaches to Neuroblastoma Therapy (NANT) Consortium, Julia Glade Bender.
- Integrative Therapies-Complementary Medicine: Research initiatives are aimed at providing evidence-based research on the safety and efficacy of complementary therapies primarily for symptom management utilizing conventional research designs. Current studies include a phase I feasibility trial of a probiotic supplement during allogeneic transplantation (Elena Ladas, Monica Bhatia) and an observational study of factors associated with acupuncture use during chemotherapy (Elena Ladas, Kara Kelly), as well as a global health initiative in several Central and South American countries investigating traditional healing practices as a means towards enhancing adherence with conventional cancer therapies (Elena Ladas, Kara Kelly).
- Center for Survivor Wellness: Research focuses on the survivors of childhood cancer, investigating the long-term complications of oncology treatment, conducting outcomes research and developing new programs and methods to provide supportive care to pediatric cancer survivors throughout their childhood and adolescence and into adulthood. A major focus is the initiation of a study within the Children’s Oncology Group network to evaluate the impact of chemotherapy on ovarian reserve, before, during and after treatment; Jennifer Levine (Leukemia and Lymphoma Society).
- Valerie Fund Psycho Social Program: Studies are investigating neuropsychological function and quality of life late effects of treatment for survivors of pediatric brain tumors, leukemia and stem cell transplantation; Stephen Sands.
Basic and Translational Research: Hematology
- High-Tc susceptometer to monitor transfusional iron overload: To evaluate our new high-transition-temperature (high-Tc) superconducting magnetic susceptometer for monitoring iron overload in patients with chronic red blood cell transfusion; Gary M. Brittenham (FDA R01 FD003702)
- Oral iron chelators predicated on desferrithiocin: To develop and evaluate orally active iron-chelating agents based on desferrithiocin for the treatment of iron overload; Gary M. Brittenham (NIH R37 DK049108 subcontract)
- Harmful effects of transfusion of older stored red cells: iron and inflammation: To determine if transfusion of red blood cells after prolonged storage can be harmful; Steven Spitalnik and Gary M. Brittenham (NIH R01 HL098014)
- Malaria and the safety of iron interventions: To examine the effects of acute infection with Plasmodium falciparum on the absorption, pharmacokinetics and metabolism of iron; Gary M. Brittenham (NIH U01 HD061233)
- Neuropathology of severe malaria in Thailand: MRI studies: To determine if axonal injury is responsible for neurological dysfunction in severe and cerebral malaria; Gary M. Brittenham (NIH R21 NS055348)
- Mechanisms of effect of iron status & interventions on malaria & other infections: To examine the factors responsible for the effects of iron deficiency and iron interventions on infections with malaria and other microbial pathogens; Gary M. Brittenham (NIH U01 HD064827).
- ZnPP-fluorometer: noninvasive field measurement of erythrocyte zinc protoporphyrin: To develop a portable, battery-powered instrument for the noninvasive detection of iron deficiency. (Gary M. Brittenham, Nestec Ltd. CU PTAABF5161)
- Vitamin D for sickle-cell respiratory complications: Phase 2: To determine if monthly oral vitamin D reduces the risk of respiratory complications in children with sickle-cell disease; Gary M. Brittenham and Margaret T. Lee (FDA R01 FD003894).
- Pharmacogenetic Prediction of Hydroxyurea Response in Sickle Cell Disease: To identify genetic variations in candidate genes associated with fetal hemoglobin (HbF) levels and HbF response to hydroxyurea in children with sickle cell disease; Nancy S. Green (NIH 3UL1RR024156-04S4)
- Pharmacokinetics of Hydroxyurea in Children with Sickle Cell Anemia: A multi-site study designed to provide key pharmacokinetic information to inform the FDA so as to gain approval for use of hydroxyurea in children with sickle cell disease; Nancy S. Green (Best Pharmaceuticals for Children Act, NICHD contract # HU B01)
Clinical Research: Hematology
- Sickle Cell Stroke Prevention: Our program is one of the sites that contributed to the landmark research studies STOP and STOP II that established the use of Transcranial Doppler (TCD) for assessment of risk of stroke, and treatment with chronic red blood cell transfusion for primary prevention of stroke in children with sickle cell disease. We currently participate in the Post-STOP study (Prevention of Stroke after STOP) to obtain and analyze follow-up information from STOP and STOP II study participants that will provide an update on the status of current sickle cell care related to stroke and provide data for improved evidence-based stroke surveillance guidelines; Margaret T. Lee (subcontract with MUSC).
Our program was also a participant in the recently concluded SWiTCH trial (Stroke with Transfusions Changing to Hydroxyurea) that compared standard red blood cell transfusions with hydroxyurea for secondary prevention of stroke. At present, we are one of the sites in the U.S. and Canada taking part in the TWiTCH trial (Transcranial Doppler (TCD) with Transfusions Changing to Hydroxyurea) to compare standard blood transfusions with hydroxyurea for primary prevention of stroke in children with sickle cell disease; Margaret T. Lee (NIH R01 HL095647).
- Sickle Cell Pain Management: In collaboration with the Departments/Divisions of Pediatric Emergency Medicine and Pediatric Pain Medicine, we have conducted clinical research on the impact of using a clinical pathway for the management of sickle cell vaso-occlusive crisis pain in the emergency department and are currently initiating an investigation of the benefit of incorporating additional strategies/medications into the standard of care; Katherine L. Ender.
- Identifying and Addressing Barriers to Hydroxyurea Use in Children with Sickle Cell Disease: Research studies aimed at improving hydroxyurea use by understanding barriers to use at family and provider levels, testing interventions to improve use, assessing the relationship to alternative treatment with hematopoietic stem cell transplantation are currently ongoing; Nancy S. Green.
- Blood Transfusion: In collaboration with researchers at the New York Blood Center, we are examining immunologic responses to red blood cell transfusions in patients with sickle cell disease and thalassemia; Margaret T. Lee.
- Cardiovascular Complications: Along with the Division of Pediatric Cardiology and the Pulmonary Hypertension Center at CUMC, current research initiatives are focused on developing guidelines for screening and evaluation of cardiac abnormalities in sickle cell disease, including pulmonary hypertension. Our program participated in the Walk-PHaSST trial to determine the safety and efficacy of sildenafil in the treatment of pulmonary hypertension in patients with sickle cell disease, and in a multi-site pilot study to evaluate effects of hydroxyurea on elevated pulmonary artery pressures in children with sickle cell disease; Margaret T. Lee and Nancy S. Green.
- Avascular Necrosis: In collaboration with Pediatric Orthopedic Surgery, we are conducting studies on early screening of AVN in children with sickle cell disease with x-ray and MRI, determination of AVN risk factors, and development of new treatment options for early stage disease; Margaret T. Lee.
- New Therapeutic Agents for Sickle Cell Disease: Expanding on earlier research conducted at Columbia and using the resources of the CTSA/CRC, we are studying the anti-sickling effect of medications not previously used to mediate the complications of sickle cell anemia; Katherine L. Ender and Gary M. Brittenham.
- Sickle Cell Services Transition Program for Young Adults to Adult Medical Care: Five year grant from the New York State Department of Health for the establishment of an integrated, comprehensive program at Columbia affiliate Harlem Hospital Center for the successful transition of young adults with sickle cell disease from pediatric to adult medical care; Katherine L. Ender (NYS DOH #1001120321).
- Newborn Screening Program and Policy: Nationally, newborn screening is overseen by the Maternal and Child Health Bureau of the Health Resources and Services Administration (HRSA). To facilitate national recommendations for current and future activities, we staff the Secretary’s Advisory Committee on Heritable Disorders and Genetic Diseases in Newborns and Children. In New York State, we serve the Registry and Surveillance System for Hemoglobinopathies (RuSH) program, funded by CDC (Nancy S. Green, HRSA contract).
Clinical Research: Stem Cell Transplantation
- Pilot study of Sequential Myeloablative Stem Cell Transplantation and Reduced Intensity Allogeneic Stem Transplantation Followed by Adoptive Cellular Immunotherapy in Patients with Refractory or Recurrent Hodgkin’s Lymphoma and Non–Hodgkin’s disease; Prakash Satwani.
- Allogeneic Stem Cell Transplantation to Induce Mixed Donor Chimerism in Patients with Sickle Cell Disease: Determine the toxicity and incidence of graft failure associated with moderately ablative therapy and allogeneic stem cell transplantation in patients with sickle cell disease; Monica Bhatia.
- A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Non-Malignant Diseases Using a Reduced Intensity Preparatory Regime; Monica Bhatia.
- Unrelated Donor Reduced Intensity Bone Marrow Transplant for Children with Severe Sickle Cell Disease; Monica Bhatia (Bone Marrow Clinical Trials Network - AAAF9482 )
- The Role of Minimal Residual Disease Testing before and after Hematopoietic Cell Transplantation for Pediatric Acute Myeloid Leukemia; Monica Bhatia (Pediatric Blood and Marrow Transplant Consortium (PBMTC) and the Center for International Blood and Marrow Transplant Research (CIBMTR))
- Defibrotide for Patients with Hepatic Veno-Occlusive Disease (VOD): A Treatment IND Study (Under CRF 312.34); Monica Bhatia.
- Expanded Access of Prochymal (Ex-vivo Cultured Adult Human Mesenchymal Stem Cells) Infusion for the Treatment of Pediatric Patients Who Have Failed to Respond to Steroid Treatment for Acute GVHD; Prakash Satwani.