Mount Sinai is a leading research center for hematology and medical oncology. The close association between clinicians working in the Derald H. Ruttenberg Treatment Center, many of whom are physician-scientists who also conduct research, and basic scientists keeps all research focused on one goal, improving outcomes for patients with cancer and hematological disorders. The division is particularly well known for its leadership in mylodysplastic syndrome and for the development of 5-azacytidine, the first drug approved by the FDA for this disorder.

Sickle cell anemia and hemophilia have been major areas of focus for the division for many years. Several new therapies for these disorders were developed at Mount Sinai. Ongoing studies are focused on understanding the molecular basis of these and other blood diseases.

Mount Sinai researchers were involved in development of the first gene therapy for blood disorders and they continue to be leaders in the development of this treatment modality. Using recombinant adeno-associated virus vectors, new gene therapies, including use of suicide gene therapy, are in both basic and clinical phases of development. A Mount Sinai developed immunotherapy using gene transfer therapy for advanced colorectal and breast cancers is currently in clinical trials. Gene therapy is also being pursued as a new approach to treating pain associated with cancer.

In both clinical and basic research, investigators continue to seek means of improving therapeutics for a variety of disorders including breast, liver, and lung cancers, myelodysplastic syndrome leukemia, myelosuppression, and severe chronic neutropenia.

A new technique for bone marrow transplantation, known as mini-bone marrow transplantation was refined at Mount Sinai. Because this technique does not require intensive preparative conditioning that leads to the destruction of the patient's existing bone marrow, so it makes this life saving treatment available to those with complicating conditions and those too frail to tolerate traditional bone marrow transplantation.

In the laboratory, researchers are studying molecular pathways to understand the steps that are necessary for the transformation of a normal cell to a cancer cell. They are then using this knowledge to identify ways to interfere with the ability of the cancer cell to survive and divide. In addition, researchers are looking at cancer cells to discover what traits allow them to metastasize in the host.

• Louis M. Aledort, M.D.
• George F. Atweh, M.D.
• Margaret H. Baron, M.D., Ph.D.
• Andreas S. Beutler, M.D.
• Vladimir Bogdanov, Ph.D.
• A. Philippe Chahinian, M.D.
• Hengjun Chao, M.D.
• Janet Cuttner, M.D.
• Paul S. Frenette, M.D.
• Janice L. Gabrilove, M.D.
• Doris A. Germain, Ph.D.
• Tony E. Godfrey, Ph.D.
• James F. Holland, M.D.
• Luis M. Isola, M.D.
• Yongkui Jing, Ph.D.
• Jacqueline Mason
• Rafael Mira-Y-Lopez, M.D., Ph.D.
• Takao Ohnuma, M.D., Ph.D.
• Liliana Ossowski, Ph.D.
• Beatriz G. Pogo, M.D.
• John Roboz, Ph.D.
• Jonathan D. Schwartz, M.D.
• Lewis R. Silverman, M.D.
• David W. Sternberg, M.D., Ph.D.
• Max W. Sung, M.D.
• Christopher E. Walsh, M.D., Ph.D.
• Samuel Waxman, M.D.

Arsenic Trioxide and Acute Myeloid Leukemia
Primary Investigator: Yongkui Jing
Funding Agency: NATIONAL CANCER INSTITUTE
Project Period: 15-Jul-02 - 30-Jun-07

In vivo Sickle Cell Vaso-occlusion: Adhesion Mechanisms
Primary Investigator: Paul S. Frenette
Funding Agency: NATIONAL HEART, LUNG & BLOOD INSTITUTE
Project Period: 30-Sep-01 - 31-Jul-06

Therapeutic Strategies in Disorders of Hematopoiesis
Primary Investigator: Janice Gabrilove
Funding Agency: NATIONAL CANCER INSTITUTE
Project Period: 23-Jun-03 - 31-May-08

Intrathecal Gene Transfer for Chronic Cancer Pain
Primary Investigator: Andreas Beutler
Funding Agency: NATIONAL INSTITUTE NEUROLOGICAL DISORD & STROKE
Project Period: 01-Feb-04 - 31-Jan-09

Reversal of Epigenetic Silencing of the G-Globin Gene
Primary Investigator: George Atweh
Funding Agency: NATIONAL HEART, LUNG & BLOOD INSTITUTE
Project Period: 01-Apr-03 - 31-Mar-07

Gene Therapy for the Hemophilias
Primary Investigator: Christophe Walsh
Funding Agency: NATIONAL HEART, LUNG & BLOOD INSTITUTE
Project Period: 01-Jul-02 - 30-Jun-07

Gene Transfer of Hematopoetic Stem Cells
Primary Investigator: Christophe Walsh
Funding Agency: NATIONAL HEART, LUNG & BLOOD INSTITUTE
Project Period: 01-Aug-01 - 31-Jul-07

Expression of Alternatively Spliced Human Tissue Factor
Primary Investigator: Vladimir Bogdanov
Funding Agency: NATIONAL INSTITUTE OF DIABETES, DIGESTIVE.& KIDNEY DISEASES
Project Period: 07-Feb-05 - 31-Jan-09

Methods of Modulation of Hemato-Vascular Development
Primary Investigator: Margaret Baron
Funding Agency: NATIONAL INSTITUTE OF BIOMED IMAGING/BIOENG
Project Period: 20-Sep-03 - 31-Jul-08

Microtubule-Targeting Therapy for Prostate Cancer
Primary Investigator: George Atweh
Funding Agency: DEPARTMENT OF DEFENSE, UNITED STATES ARMY
Project Period: 16-Jan-04 - 15-Jan-07

FIX Tolerance in Hemophilia Gene Therapy
Primary Investigator: Hengjun Chao
Funding Agency: NATIONAL HEART, LUNG & BLOOD INSTITUTE
Project Period: 16-Apr-04 - 31-Mar-08

Role of Skp2 & Skp2B in Breast Cancers
Primary Investigator: Doris Germain
Funding Agency: NATIONAL CANCER INSTITUTE
Project Period: 30-Sep-05 - 30-Jun-10

Modulation of Apoptosis by KLF6 in Liver Cancer
Primary Investigator: Michaela Banck
Funding Agency: NATIONAL INSTITUTE OF DIABETES, DIGESTIVE.& KIDNEY DISEASES
Project Period: 01-Jul-05 - 30-Jun-10

Intractable Pain From Cancer Such as Multiple Myeloma
Primary Investigator: Andreas Beutler
Funding Agency: THE G&P FOUNDATION FOR CANCER RESEARCH
Project Period: 01-Apr-04 - 31-Mar-07

Tracking the Fate of Aberrant Clone in Myelodysplastic
Primary Investigator: Lewis Silverman
Funding Agency: DEPARTMENT OF DEFENSE, UNITED STATES ARMY
Project Period: 20-Apr-05 - 19-Apr-07

Transcriptional Regulation of Mesoderm Differentiation
Primary Investigator: Hailan Zhang
Funding Agency: NATIONAL INSTITUTE OF DIABETES, DIGESTIVE.& KIDNEY DISEASES
Project Period: 01-Jun-05 - 31-May-08

Molecular Mechanisms of KLF6 in Prostate Cancer
Primary Investigator: Michaela Banck
Funding Agency: DEPARTMENT OF DEFENSE, UNITED STATES ARMY
Project Period: 28-Dec-05 - 27-Dec-09

Detection of Occult Metastases in Lung Cancer
Primary Investigator: Tony Godfrey
Funding Agency: NATIONAL CANCER INSTITUTE
Project Period: 08-Jul-02 - 30-Jun-07

Regulation of Metastasis by Retinoic Acid
Primary Investigator: Rafael Mira-y-Lopez
Funding Agency: FOGARTY INTERNATIONAL CENTER
Project Period: 01-Apr-05 - 31-Mar-08

FVIII Immunity & Tolerance in AAV-Based Gene Transfer
Primary Investigator: Hengjun Chao
Funding Agency: NATIONAL HEMOPHILIA FOUNDATION
Project Period: 01-Jul-06 - 30-Jun-10

Molecular Staging of Lymph Nodes in Head and Neck Cancer
Primary Investigator: Tony Godfrey
Funding Agency: NATIONAL CANCER INSTITUTE
Project Period:27-Jun-05 - 31-May-08

Oncogenes and Retinoid Chenoprevention
Primary Investigator: Rafael Mira-y-Lopez
Funding Agency: NATIONAL CANCER INSTITUTE
Project Period: 09-Aug-06 - 31-May-10

Specification of Hematopoietic Mesoderm
Primary Investigator: Joan Isern
Funding Agency: COOLEY'S ANEMIA FOUNDATION
Project Period: 01-Jul-05 - 30-Jun-07

Repair of cWF Gene Defects by Targeted RNA Transsplicing
Primary Investigator: Yanping Yang
Funding Agency: HEMOPHILIA OF GEORGIA, INC
Project Period: 01-Jul-05 - 30-Jun-07

HTS Assay Development for Inhibitors/Sickle Cell Vaso.
Primary Investigator: Paul S. Frenette
Funding Agency: NATIONAL HUMAN GENOME RESEARCH INSTITUTE
Project Period: 23-Sep-05 - 31-Aug-07

Real Time Contributions of Platelets and Procoagulants
Primary Investigator: Paul S. Frenette
Funding Agency: AMERICAN HEART ASSOCIATION - NATIONAL
Project Period: 01-Jan-06 - 31-Dec-10

Translational Regulation Gene Expression Acute Leukemia
Primary Investigator: David Sternberg
Funding Agency: G&P FOUNDATION FOR CANCER RESEARCH
Project Period: 01-Apr-06 - 31-Mar-09

Cellular Retinol-Binding Protein Silencing Breast Cancer
Primary Investigator: Rafael Mira-y-Lopez
Funding Agency: CHEMOTHERAPY FOUNDATION
Project Period: 01-Jan-06 - 31-Dec-07

Rituximab for Treatment of Congenital Hemophilia A
Primary Investigator: Christopher Walsh
Funding Agency: NEW ENGLAND RESEARCH INSTITUTE
Project Period: 27-Mar-06 - 26-Mar-08

The Function of Faconi Anemia Genes in Hematopoiesis
Primary Investigator: Beiqing Pan
Funding Agency: FANCONI'S ANEMIA RESEARCH FUND,INC.
Project Period: 01-Aug-06 - 31-Jul-07

Regional Comprehensive Hemophilia Diagnostic & Treatment
Primary Investigator: Christophe Walsh
Funding Agency: HEALTH RESOURCES & SERVICES ADMINISTRATN
Project Period: 30-Sep-96 - 31-May-08

Prevention of the Complications of Hemophila through HTC
Primary Investigator: Christopher Walsh
Funding Agency: CENTER FOR DISEASE CONTROL (ATLANTA)
Project Period: 30-Sep-96 - 29-Sep-11

Prevention of the Complications of Hemophila through HTC
Primary Investigator: Christopher Walsh
Funding Agency: CENTER FOR DISEASE CONTROL (ATLANTA)
Project Period: 30-Sep-96 - 29-Sep-06

Aberrant Retinoid Signaling in Breast Cancer
Primary Investigator: Rafael Mira-y-Lopez
Funding Agency: NATIONAL CANCER INSTITUTE
Project Period: 01-Apr-91 - 30-Nov-10

Factors Involved in Malignant Behavior of Human Tumors
Primary Investigator: Liliana Ossowski
Funding Agency: NATIONAL CANCER INSTITUTE
Project Period: 01-Aug-95 - 31-Mar-08

HLA Association in Leukemia in Lymphoma
Primary Investigator: Janet Cuttner
Funding Agency: NEW YORK COMMUNITY TRUST
Project Period: 01-Oct-91 - 30-Sep-07

SCOR: Tissue Factor, Thrombosis and the Arterial Wall
Primary Investigator: Yale Nemerson
Funding Agency: NATIONAL HEART, LUNG & BLOOD INSTITUTE
Project Period: 01-Mar-96 - 31-Jan-07

Induction of Hematopoiesis by Bone Morphogenetic Protein
Primary Investigator: Margaret Baron
Funding Agency: NATIONAL INSTITUTE OF DIABETES, DIGESTIVE.& KIDNEY DISEASES
Project Period: 01-Jan-98 - 30-Nov-07

Activation of Hematopoiesis in the Mouse Embryo
Primary Investigator: Margaret Baron
Funding Agency: NATIONAL HEART, LUNG & BLOOD INSTITUTE
Project Period: 01-Jul-00 - 30-Apr-07

Strategic Curriculum for Clinical Research (SCCR)
Primary Investigator: Janice Gabrilove
Funding Agency: NATIONAL CENTER FOR RESEARCH RESOURCES
Project Period: 01-Jun-99 - 31-May-10

Adhesion Pathways of Blood/Endothelial Progenitors
Primary Investigator: Paul S. Frenette
Funding Agency: NATIONAL INSTITUTE OF DIABETES, DIGESTIVE.& KIDNEY DISEASES
Project Period: 01-Mar-00 - 31-Jul-10