Grant Update 2019

In 2019, the Foundation’s Medical Advisory Board selected eight of the nation’s top junior investigators to receive our three-year, $225,000 Medical Research Award. We are so proud to support these innovative projects and look forward to sharing updates about them throughout the year!

Clockwise from top left: Kathrin Milbury, PhD, MD Anderson; Bilal Omer, MD, Texas Children’s Hospital; Esther Obeng, MD, PhD, St. Jude Children’s Research Hospital; Richard Possemato, PhD, NYU; Ann Mullally, MD, Dana-Farber Cancer Institute; Daniel Herranz, PhD, Rutgers Cancer Institute of New Jersey; Elli Papaemmanuil, PhD, Memorial Sloan Kettering Cancer Center; Jeffrey Bednarski, MD, PhD, Washington University in St. Louis

Kathrin Milbury, PhD– MD Anderson Cancer Center

“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 baseline measures of psychological distress and cancer symptoms and blood draws for biomarker analyses. Then, they will be randomized to the intervention or an attention control group.  Both study arms will be delivered in a group setting (5 sessions total; 75 min each) via videoconference.  All participants will be reassessed 6 and 12-weeks later.  

Bilal Omer, MD– Texas Children’s Hospital Cancer Center 

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 for chemotherapy.  Dr. Omer’s research will now plan to genetically modify these immune cells to make them more powerful. 

Esther Obeng, MD, PhD– St. Jude Children’s Research Hospital 

“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. 

Richard Possemato, PhD– New York University 

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 cell production 

Ann Mullally, MD– Dana-Farber Cancer Institute 

“Biology and Therapeutic Targeting of Mutant Calreticulin in Myeloproliferative Neoplasms” 

Somatic mutations in celreticulin (CALR)are frequent and disease-initiating in myeloproliferative neoplasms (MPN). Dr. Mullally’s laboratory recently elucidated the mechanism by which mutant CALR is oncogenic and induces MPN. Building on this work and guided by strong preliminary data they have generated, she will now identify unique molecular vulnerabilities in CALR-mutant MPN that arise as a result of (i) the pathogenic binding interaction of mutant CALR with MPL and (ii) the differential protein-protein binding interactions of mutant CALR. Dr. Mullally will exploit this information to target unique molecular vulnerabilities in CALR-mutant MPN stem cells to achieve definitive cure. 

Daniel Herranz Benito, PhD– Rutgers Cancer Institute of New Jersey 

“The role of SIRT1 in the pathogenesis and treatment of T-Cell Acute Lymphoblastic Leukemia” 

T-Cell Acute Lymphoblastic Leukemia (T-ALL) is an aggressive blood cancer where 20-50% of patients relapse, and there are few therapeutic options at this stage. Notch1 activating mutations are the main driver in T-ALL. However, responses observed with targeted therapies against NOTCH1 have been limited, such that the identification of novel targets and combination therapies is one of the most urgent goals in T-ALL. In this context, we have identified Sirt1 as a critical mediator of resistance to NOTCH1 inhibition. Thus, we propose to dissect the role of Sirt1 in the pathogenesis of T-ALL and as a therapeutic target in vivo.

Jeffrey Bednarski. MD, PhD– Washington University St. Louis 

“B Cell Specific DNA Damage Responses Prevent Leukemic Transformation” 

Pediatric acute lymphoblastic leukemia occurs as a result of genetic mutations that block normal development of immune cells and promote continued expansion of cancer cells. The goals of Dr. Bednarksi’s project is to understand how cells prevent generation of these mutations and how cells are eliminated if mutations do occur. 

Elli Papammanuil, PhD– Memorial Sloan Kettering Cancer Center 

“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 and inform guideline development aimed to prevent this aggressive disease.