Background: Several treatment options are available for patients with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML); however, the majority of patients will fail treatment or relapse. Predicting response is imperative in determining the right treatment regimen for each patient but currently no validated method exists.
Background: HMAs (e.g., azacitidine (AZA), decitabine (DAC)) are approved agents for the treatment of patients with MDS. Despite their widespread use, only 50% of patients respond to HMA without reliable biomarkers of response despite knowing the patient’s genomic and clinical (non-genomic) characteristics prior to treatment.
Introduction: Pts with R/R AML have few effective treatment options. Len has activity in myeloid malignancies including AML, and may have additive properties with aza. Based on encouraging data using sequential aza with high dose len in untreated elderly AML pts, we designed a phase 2 pilot study exploring this therapy (tx) in pts with R/R AML.
Background: Bruton’s tyrosine kinase (BTK), a mediator of B cell receptor signaling, has been effectively exploited as a therapeutic target in Chronic lymphocytic leukemia (CLL). Ibrutinib is a BTK inhibitor that has shown excellent efficacy in treatment-naïve, relapsed/refractory, and high-risk CLL.
Background: Early T-cell precursor acute lymphoblastic leukemia (ETP-ALL) is an aggressive hematological malignancy with poor prognosis after chemotherapy compared to other ALLs. ETP-ALL cases typically display a high burden of chromosomal copy number variations (CNVs) and myeloid gene mutations.
Background: MM is a highly refractory disease that is rarely cured. While introduction of novel therapies has improved survival and overall response rates (ORR), complete remission (CR) rates remain low (30%) and relapse is still frequent.
ETP-ALL, an orphan disease, is a subtype of T-ALL with poor prognosis and high risk of relapse after standard of care (SOC) chemotherapy. ETP-ALL is a genomically heterogeneous disease with many cases harboring complex karyotype and genetic mutations typically seen in myeloid neoplasias, such as acute myeloid leukemia (AML).
ETP-ALL is a high-risk subtype of T-ALL with poor prognosis and high risk of relapse in patients treated with standard of care (SOC). ETP-ALL patients have more complex cytogenetic profiles and a unique immunophenotype compared to other T-ALL cases. ETP-ALL is characterized by the expression of myeloid and hematopoietic stem cell (HSC) surface proteins, as well as gene expression profiles similar to myeloid progenitors and HSCs, including frequent mutations in myeloid oncogenes.
Introduction: Dysregulated B-cell receptor (BCR) signaling drives proliferation of Waldenstrom macroglobulinemia (WM) cells, which characteristically secrete excessive IgM. To maintain IgM production and preserve cellular homeostasis, WM cells rely on optimally functioning proteasomes to degrade and recycle proteins.
Background: Treatment of acute myeloid leukemia (AML) remains a challenge due to short-lived responses and high rates of relapse. AML cells often possess mutations in kinases (FLT3, JAK2, etc.), resulting in uncontrolled proliferation of neoplastic cells.