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“Modulating Iron Metabolism to Treat Myelodysplastic Syndrome” In Myelodysplastic Syndrome red blood cell formation is impaired, leading to severe progressive anemia. Mutation of SF3B1 often causes Myelodysplastic Syndrome, and is associated with the appearance of iron-loaded immature red blood cells. We propose to use genetic tools to repair this altered iron metabolism, restoring red blood […]

“Targeting SF3B1 in myelodysplastic syndrome” Myelodysplastic syndromes (MDS) are a group of pre-leukemia blood disorders with a high risk of progression to aggressive leukemia, SF3B1 mutations are frequent, early events in MDS pathogenesis and ideal therapeutic targets. The proposed studies will evaluate how SF3B1 mutations cause MDS and test novel agents in SF3B1-mutant cells.

“Engineering more potent T cells to target lymphoma associated antigens” We have developed strategies to grow a patient’s own immune cells in the laboratory and then re-inject them into the patient to fight lymphoma. In a clinical trial at our center, 3 of 7 patients with active lymphoma responded to this treatment without the need […]

“Investigating the Impact of Oncologic Therapy on Clonal Hematopiesis and Subsequent Risk of Developing Therapy Related Leukemia” Dr. Papammanuil will study the key risk factors that lead to the therapy related leukemia development in cancer patient survivors. We will combine clinical, molecular and treatment information in a cohort of 45,000 patients to study disease biology […]

Mind-body Medicine for Adolescents and Young Adults (AYA) Coping with Hodgkin Lymphoma  Dr. Milbury will conduct a pilot randomized controlled trial to test the feasibility and initial efficacy of a mind-body intervention targeting the psychosocial needs of adolescents and young adults (AYAs; ages 15-39) with Hodgkin Lymphoma.  AYAs with a new Hodgkin Lymphoma diagnosis will complete […]

Investigating the roles of microRNAs in lymphomagenesis A microRNA is a small RNA that can simultaneously modulate the levels of tens or even hundreds of different proteins. We propose to use multiple experimental approaches to investigate the functions of two families of miRNAs that are suspected to play a key role in lymphomas. To learn […]

Genomic characterizations of myeloproliferative neoplasms The Levine Lab studies chronic leukemias which manifest as an excess of red blood cells, platelets, and white blood cells, respectively. Importantly, these patients are at high risk for developing bone marrow failure (due to increased scar tissue in their bone marrow) or transformation to acute leukemia; when patients develop […]

The Development of STAT Inhibitors as Novel Anti-Leukemia Agents: Use of Semi-Random Synthesis Technology In recent years, tremendous advances have been made toward understanding the molecular abnormalities occurring in tumor cells. During this time, however, relatively little progress has been made in the treatment of leukemias and other forms of cancer. The challenge, therefore, is […]

Targeted Nanotherapeutics for Treatment of Acute Leukemia Dr. Carnes’ research employs materials developed using state-of-the-art nanotechnology combined with cutting-edge cell recognition systems to create microscopic suitcases which carry drugs directly to cancer cells. Control over simple properties such as the size and appearance of these tiny packages will allow us to deliver virtually any combination […]

Applying synthetic lethality to eradicate high risk NF1-deficient B-cell acute lymphoblastic leukemia Acute lymphoblastic leukemia (ALL) is a leading cause of cancer-related pediatric death. Many ALL patients are cured but specific sub-types of ALL are lethal. We have developed a new therapy that exploits a common feature of these poor prognosis pediatric leukemias and propose […]

Targeting ‘undruggable’ initiating events in AML Acute myeloid leukemia is a devastating illness with approximately 600 pediatric and 13,000 adult diagnoses, and 10,000 deaths, yearly. In this proposal we will develop prototype drugs that destroy the aberrant components, or proteins, within the cell that drive the malignant phenotype laying the groundwork for development of first-in-class […]

MYC-dependent replication stress as a pathogenic driver and therapeutic target in aggressive B cell lymphomas B cell lymphomas with MYC gene alterations are very aggressive and not curable using current therapies. Targeting MYC would thus be an ideal therapeutic strategy in these patients. Here, we describe a cellular process representing a unique vulnerability in MYC-driven […]