RESEARCH AT MD ANDERSON HOUSTON, TX
Philanthropic support is pivotal in moving the needle and we are grateful for your interest in Dr. Pemmaraju’s AML research. The first two articles below are two clinical studies from Craig's physician that credit SagerStrong in providing the funding to make the trial happen.
Dr. Pemmaraju is currently engaged in other clinical trials with descriptions further down the page. This is what Stacy and the SagerStrong Foundation have worked tirelessly the past two years doing and are so proud of the results.
Erythroleukemia-historical perspectives and recent advances in diagnosis and management
Acute erythroleukemia is a rare form of acute myeloid leukemia recognized by its distinct phenotypic attribute of erythroblastic proliferation. After a century of its descriptive history, many diagnostic, prognostic, and therapeutic implications relating to this unique leukemia subset remain uncertain. The rarity of the disease and the simultaneous involvement of its associated myeloid compartment have complicated in vitro studies of human erythroleukemia cell lines. Although murine and cell line erythroleukemia models have provided valuable insights into pathophysiology, translation of these concepts into treatment are not forthcoming. Integration of knowledge gained through a careful study of these models with more recent data emerging from molecular characterization will help elucidate key mechanistic pathways and provide a much needed framework that accounts for erythroid lineage-specific attributes. In this article, we discuss the evolving diagnostic concept of erythroleukemia, translational aspects of its pathophysiology, and promising therapeutic targets through an appraisal of the current literature.Continue Reading
Glioblastoma and acute myeloid leukemia: malignancies with striking similarities
Acute myeloid leukemia (AML) and glioblastoma (GB) are two malignancies associated with high incidence of treatment refractoriness and generally, uniformly poor survival outcomes. While the former is a hematologic (i.e. a “liquid”) malignancy and the latter a solid tumor, the two diseases share both clinical and biochemical characteristics. Both diseases exist predominantly in primary (de novo) forms, with only a small subset of each progressing from precursor disease states like the myelodysplastic syndromes or diffuse glioma. More importantly, the primary and secondary forms of each disease are characterized by common sets of mutations and gene expression abnormalities. The primary versions of AML and GB are characterized by aberrant RAS pathway, matrix metalloproteinase 9, and Bcl-2 expression, and their secondary counterparts share abnormalities in TP53, isocitrate dehydrogenase, ATRX, inhibitor of apoptosis proteins, and surviving that both influence the course of the diseases themselves and their progression from precursor disease. An understanding of these shared
features is important, as it can be used to guide both the research about and treatment of each.
Patients with AML and BPDCN:
CD123-targeted clinical trial with SL-401. PI: Marina Konopleva, M.D., Co-PI: Naveen Pemmaraju, M.D. We have identified a marker present on many AML blasts called CD123 (or IL3R alpha). This trial is offered to patients with either AML or a rare cancer called Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN), which usually expresses high levels of CD123. This trial is open and enrolling currently.View This Trial
Patients with Myelofibrosis (MF):
Treatment with a Smac Mimetic, LCL-161. PI: Naveen Pemmaraju, M.D., Co-PI: Srdan Verstovsek, M.D., Ph.D. We will treat patients with a very new type of targeted therapy that aims to promote cell death (apoptosis) in cancer cells that overexpress certain proteins that prevent them from dying normally. I will collaborate with a senior scientist, Bing Carter, M.D., to test all patient samples for correlative studies of the markers of cell death. This will help us understand the exact nature of the drug targets and the tumor cells. This trial is enrolling patients.View This Trial
Patients with Advanced Myeloproliferative Neoplasms (MPNs):
CMML, HES, SM, MF. Treatment with Targeted Therapy. PI: Naveen Pemmaraju, M.D., Co-PI: Srdan Verstovsek, M.D., Ph.D. This clinical trial is slated to open next month. It is for patients with advanced MPNs who have had one prior line of therapy. We will offer them a new therapy targeted at molecules found/expressed on the blast/stem cells of their tumors.View This Trial