Research Abstract
Dr. Ghobrial's research and clinical efforts focus on early detection and interception to prevent progression in Multiple Myeloma. She has a broad background in the biology of multiple myeloma and the bone marrow niche, specifically focusing on MGUS and Smoldering myeloma (SMM) and defining both cell-autonomous and bone marrow niche-dependent mechanisms of disease progression. Her group examines genetic and epigenetic alterations that regulate clonal progression and the role of the bone marrow niche in disease progression from early precursor stages (MGUS and SMM) to active Multiple Myeloma. She is the PI of PROMISE and PCROWD, observational cohort studies that are screening 30,000 individuals and following over 3,000 patients with MGUS and SMM. She has led several clinical trials in high-risk smoldering myeloma including immunotherapeutic precision interventions. All of these pursuits are focused on understanding and preventing the development of myeloma and other hematological malignancies.
Dr. Ghobrial's laboratory research is focused on understanding how early precursor conditions like MGUS and SMM progress to overt MM. We examine the genetic, epigenetic, and immune alterations that clonal progression and the role of the bone marrow niche in disease progression to active Multiple Myeloma. We work to understand the biological changes that occur during progression and develop therapeutic strategies to stop progression in its tracks. In addition, her research has been translated to innovative investigator-initiated clinical trials. She has led over 15 phase I and II clinical trials, and studies on the bone marrow and immune microenvironments are currently being translated into immunotherapy trials in high-risk precursor patients.
She is the co-leader of the Center for Prevention of Progression of Blood Cancers (CPOP), where patients with precursor conditions such as MGUS, early Myelodysplastic Syndrome (MDS), and early Chronic Lymphocytic Leukemia (CLL) are monitored prospectively for clonal evolution during disease progression. CPOP coordinates clinical trials and research efforts for patients with these conditions to develop therapeutic agents that can prevent or delay disease progression in these early malignant conditions. The PROMISE study, which is screening healthy people who may be at risk for precursor conditions and MM, and the PCROWD study, a tissue bank study collecting samples from patients with precursor conditions, are part of this effort.
