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Exploiting Novel Therapeutic Vulnerabilities in Chronic Myelomonocytic Leukemia
The overall objective of this project is to identify novel pathways that may be targeted for therapeutic benefit in CMML. We have identified abnormal inflammation mediated by RSK1 in CMML patient cells, and we hypothesize that RSK1 drives CMML disease development. We thus propose studies to determine how RSK1 contributes to CMML pathogenesis, and to evaluate the therapeutic potential of RSK1 inhibition for CMML patients.Doxorubicin
Doxorubicin is FDA approved to treat people who have some kinds of blood cancer, including acute lymphoblastic leukemia, acute myeloblastic leukemia, and Hodgkin and non-Hodgkin lymphoma.
Topotecan
Topotecan is an FDA-approved cancer drug that is used to treat people who have certain types of cancer including acute myeloid leukemia and acute lymphoblastic leukemia. This drug may cause a temporary loss of hair in some people. After treatment with topotecan has ended, normal hair growth should return.
A phase 1b/2 study targeting apoptotic and signaling pathways in T-acute lymphoblastic leukemia
T-acute lymphoblastic leukemia (T-ALL) is an aggressive leukemia with limited treatment options after first-line chemotherapy. Our preclinical work in animal models of T-ALL demonstrated the activity of a novel-novel combination treatment strategy, which includes LP-118 (activator of suicide pathways within leukemic cells) and tyrosine kinase inhibitors (inhibiting growth-promoting LCK and ACK1 signaling pathways).Understanding Mutations to Treat MDS
Benjamin Ebert is researching what genetic mutations cause myelodysplastic syndromes (MDS), a group of disorders in which the bone marrow fails to produce sufficient blood cells, and how that information can be used to determine prognosis and therapy. MDS frequently progresses to an acute leukemia.
Rituximab
is FDA approved for the treatment of:

Clinical Trials: Why They Matter and How to Make Them More Accessible
Cancer treatments can be lifesaving. As an organization, The Leukemia & Lymphoma Society (LLS) has invested more than $1.7 billion in blood cancer research since its inception in 1949—all with the hope that this research leads to scientific breakthroughs that improve and save lives.
But to make sure this research leads to safe and innovative treatments, we have to thoroughly test it.

A Source of Hope: Understanding Bone Marrow and Stem Cell Transplants
Imagine a community filled with families, friends, and healthcare professionals united to transform the meaning of living with blood cancer. This diverse group of people—including researchers, doctors, care partners, and healthcare teams—are bound together by hope, resilience, and a shared goal: to overcome the challenges and live longer better lives.
Ibrutinib
Ibrutinib has been FDA-approved to treat:
- Patients with chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL).
- Patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (SLL) with 17p deletion.
- Patients with Waldenström macroglobulinemia (WM).
- Adult and pediatric patients age 1 year and older with chronic graft versus host disease (cGVHD) after failure of one or more lines of systemic therapy.
Interferon alfa-2a
Interferons are substances naturally produced by cells in the body to help fight infections and tumors. They may also be synthetic versions of these substances. Interferon alfa-2a is FDA approved to treat people who have hairy cell leukemia and Philadelphia chromosome positive chronic myeloid leukemia who are minimally pretreated (within 1 year of diagnosis). It may cause a temporary loss of hair. After treatment has ended, normal hair growth should return.
Midostaurin
Rydapt is FDA approved for the treatment of adult patients with:
- Newly diagnosed acute myeloid leukemia (AML) that is FLT3 mutation positive as detected by an FDA-approved test, in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation.
Limitations of Use:
RYDAPT is not indicated as a single-agent induction therapy for the treatment of patients with AML. - Aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia (MCL).

People with CLL are living longer than ever before, and cures are on the horizon
People with the most common type of blood cancer, chronic lymphocytic leukemia (CLL), are living longer than ever before without their disease progressing. While cures for CLL patients are still rare, survival rates have improved steadily over the last 50 years, with nearly 90 percent of people diagnosed with CLL today surviving at least five years, and most for many years longer.
Investigating the dependency for protein synthesis in Venetoclax/Azacitidine-resistant acute myeloid leukemia
Relapsed and/or refractory acute myeloid leukemia (AML) display resistance to Venetoclax and Azacitidine (Ven/Aza) with approximately one third of patients demonstrating upregulated protein synthesis. This proposal will investigate the mechanism(s) underlying the dependence of Ven/Aza-resistant AML on protein synthesis as well as the functional consequences of targeting this pathway. Successful completion of these studies will provide novel insights into Ven/Aza resistance mechanisms.Precision Targeting of Hairy Cell Leukemia using Chimeric Antigen Receptor T cells
Though effective treatments in hairy cell leukemia and variant (HCLv) exist, they are associated with profound immunosuppression; thus, more targeted, non-toxic therapies are warranted. In order to specifically target leukemic cells while sparing most normal B cells, we will develop a novel chimeric antigen receptor T cell immunotherapy against the IGHV-4-34 B-cell receptor that is found in a significant subset of HCL and associates with poor prognosis.
What’s Next in Blood Cancer: Looking Ahead to 2023
The most important blood cancer scientific meeting, the American Society of Hematology (ASH), is held every December.