Gary Kupfer, MD

Professor of Pediatrics (Hematology / Oncology) and of Pathology; Section Chief, Pediatric Hematology/Oncology; Clinical Program Leader, Pediatric Hematology & Oncology Program, Smilow Cancer Hospital; Disease Aligned Research Team Leader, Pediatric Hematology & Oncology Program, Yale Cancer Center

Research Departments & Organizations

Pediatrics: Kupfer Lab | Pediatric Hematology & Oncology

Pathology

Cancer Genetics

Molecular Virology: Virology Laboratories

Yale Cancer Center: Radiobiology & Radiotherapy

Yale Stem Cell Center

Office of Cooperative Research

Research Interests

Fanconi Anemia; Genomic Instability; Hematology; Medical Oncology; Neoplasms by Histologic Type; Pediatrics; Therapeutics

Research Summary

The Kupfer lab works on the relationship of genomic instability and the propensity towards development of cancer. Specifically, we focus on the genetic syndrome Fanconi anemia (FA). Interestingly, children with FA are born with congenital anomalies and develop aplastic anemia and an assortment of leukemias and other cancers. Based on our interest on marrow failure and genomic instability, we have also started working on 3 related projects. First, we have begun to purify the protein complexes containing gene products that are defective in 2 additional hematopoietic failure syndromes, Diamond-Blackfan anemia (DBA) and congenital dyserythropoietic anemia (CDA). As in FA, the proteins (RPS19 for DBA, codanin for CDA) have no known function, and additional genes accounting for additional genetic complementation groups remain to be cloned and identified. Second, we are investigating ways to use our knowledge of genomic instability for improving cancer therapeutics. Finally, we have also started a more clinical project, using mass spectroscopy technology we have used to find FA binding proteins. Again in collaboration with the Semmes laboratory, we have adapted the mass spec to analyze sera from patients with pediatric malignancies in order to identify unique protein markers of disease. These markers could then be used for diagnosis, prognosis, staging, and tracking of minimum residual disease in patients. In addition, our goal is to identify interesting proteins for further analysis in our laboratory.

Specialized Terms: Genomic instability; Development of cancer; Genetic syndrome Fanconi anemia (FA); Viral protein enhancement of cancer therapeutics

Extensive Research Description

See:

kupferlab.org

Clinical Trials

Conditions Study Title
Brain and Nervous System A Phase 2 Study of Veliparib (ABT-888) and Local Irradiation, Followed by Maintenance Veliparib and Temozolomide, in Patients With Newly Diagnosed High-Grade Glioma (HGG) Without H3 K27M or BRAFV600E Mutations
Brain and Nervous System A Phase 3 Study of 131I-Metaiodobenzylguanidine (131I-MIBG) or Crizotinib Added to Intensive Therapy for Children With Newly Diagnosed High-Risk Neuroblastoma (NBL)
Bones and Joints, Kidney, Soft Tissue, Pediatrics Phase 3 Accelerated BEP: A Randomised Phase 3 Trial of Accelerated Versus Standard BEP Chemotherapy for Patients With Intermediate and Poor-risk Metastatic Germ Cell Tumors
Other Hematopoietic, Pediatrics A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Non-Malignant Disease Using a Reduced-Intensity Preparatory Regime
Pediatrics A Phase 1/2 Study of VX15/2503 in Children, Adolescents, or Young Adults With Recurrent or Relapsed Solid Tumors
Bones and Joints, Brain and Nervous System, Leukemia, not otherwise specified, Leukemia, other, Lymphoid Leukemia NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) Screening Protocol
Lymphoid Leukemia A Phase 2 Study of the JAK1/JAK2 Inhibitor Ruxolitinib With Chemotherapy in Children With De Novo High-Risk CRLF2-Rearranged and/or JAK Pathway-Mutant Acute Lymphoblastic Leukemia
Hodgkin's Lymphoma, Pediatrics Risk-based, Response-adapted, Phase II Open-label Trial of Nivolumab + Brentuximab Vedotin (N + Bv) for Children, Adolescents, and Young Adults With Relapsed/Refractory (R/R) CD30 + Classic Hodgkin Lymphoma (cHL) After Failure of First-line Therapy, Followed by Brentuximab + Bendamustine (Bv + B) for Participants With a Suboptimal Response (CheckMate 744: CHECKpoint Pathway and Nivolumab Clinical Trial Evaluation)
Anus, Breast - Female, Breast - Male, Colon, Corpus Uteri, Esophagus, Eye and Orbit, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung, Melanoma, skin, Other Digestive Organ, Other Endocrine System, Other Female Genital, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Ovary, Pancreas, Prostate, Rectum, Small Intestine, Soft Tissue, Stomach, Thyroid, Pediatrics, Cervix, Urinary Bladder A Randomized Phase 3 Study of Vincristine, Dactinomycin, Cyclophosphamide (VAC) Alternating With Vincristine and Irinotecan (VI) Versus VAC/VI Plus Temsirolimus (TORI, Torisel, NSC# 683864) in Patients With Intermediate Risk (IR) Rhabdomyosarcoma (RMS)
Other Male Genital, Ovary Active Surveillance, Bleomycin, Carboplatin, Etoposide, or Cisplatin in Treating Pediatric and Adult Patients With Germ Cell Tumors
Anus, Bones and Joints, Brain and Nervous System, Breast - Female, Breast - Male, Colon, Corpus Uteri, Esophagus, Eye and Orbit, Hodgkin's Lymphoma, Kaposi's sarcoma, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung, Melanoma, skin, Mycosis Fungoides, Non-Hodgkin's Lymphoma, Other Digestive Organ, Other Endocrine System, Other Female Genital, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Ovary, Pancreas, Prostate, Rectum, Small Intestine, Soft Tissue, Stomach, Thyroid, Pediatrics, Cervix, Urinary Bladder A Phase I/II Study of Pembrolizumab (MK-3475) in Children With Advanced Melanoma or a PD-L1 Positive Advanced, Relapsed or Refractory Solid Tumor or Lymphoma (KEYNOTE-051)
Anus, Bones and Joints, Brain and Nervous System, Breast - Female, Breast - Male, Colon, Corpus Uteri, Esophagus, Eye and Orbit, Hodgkin's Lymphoma, Kaposi's sarcoma, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung, Lymphoid Leukemia, Melanoma, skin, Non-Hodgkin's Lymphoma, Other Digestive Organ, Other Endocrine System, Other Female Genital, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Ovary, Pancreas, Prostate, Rectum, Small Intestine, Soft Tissue, Stomach, Thyroid, Cervix, Urinary Bladder NCI-MATCH: Targeted Therapy Directed by Genetic Testing in Treating Patients With Advanced Refractory Solid Tumors or Lymphomas
Brain and Nervous System, Pediatrics A Clinical and Molecular Risk-Directed Therapy for Newly Diagnosed Medulloblastoma
Bones and Joints, Brain and Nervous System, Eye and Orbit, Hodgkin's Lymphoma, Kidney, Leukemia, other, Liver, Lymphoid Leukemia, Myeloid and Monocytic Leukemia, Non-Hodgkin's Lymphoma, Other Digestive Organ, Other Endocrine System, Other Female Genital, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Ovary, Small Intestine, Soft Tissue, Pediatrics The Project: Every Child Protocol: A Registry, Eligibility Screening, Biology and Outcome Study
Other Respiratory and Intrathoracic Organs, Pediatrics Effects of Dexrazoxane Hydrochloride on Biomarkers Associated With Cardiomyopathy and Heart Failure After Cancer Treatment (HEART)
Brain and Nervous System, Pediatrics Response and Biology-Based Risk Factor-Guided Therapy in Treating Younger Patients With Non-high Risk Neuroblastoma
Brain and Nervous System, Pediatrics Maintenance Chemotherapy or Observation Following Induction Chemotherapy and Radiation Therapy in Treating Younger Patients With Newly Diagnosed Ependymoma
Brain and Nervous System, Pediatrics Biomarkers in Tumor Tissue Samples From Patients With Newly Diagnosed Neuroblastoma or Ganglioneuroblastoma
Lymphoid Leukemia, Pediatrics Neuropsychological and Behavioral Testing in Younger Patients With Cancer
Leukemia, not otherwise specified, Pediatrics Risk-Stratified Randomized Phase III Testing of Blinatumomab in First Relapse of Childhood B-Lymphoblastic Leukemia (B-ALL)

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