Leukemia Research

As one of the world's largest cancer research centers, MD Anderson’s leukemia research experts are leading the effort to better diagnose and treat leukemia. We’re combining excellent patient care with cutting-edge leukemia research through our comprehensive clinical trials portfolio, which offers patients with diverse and innovative treatment strategies for every stage of this disease.

Immunotherapy has emerged as a powerful tool for treating several advanced-stage solid tumor types. Our research experts are working to extend the benefits of immunotherapy to patients with hematologic malignancies. Checkpoint inhibitors are a type of immunotherapy that unleash the anti-tumor activity of powerful cancer-killing immune cells. Based on preclinical research, we’re initiating clinical trials to investigate the effectiveness of PD1 and CTLA4 checkpoint inhibitor drugs in combination with standard antileukemic agents, with additional checkpoint inhibitors to be evaluated in near future.

Despite the progress in treatment of acute myeloid leukemia (AML) in younger patients, the prognosis for elderly patients (60-65 years or over) has not significantly improved. Maintenance therapy seems to benefit AML patients that are in remission but have a high risk for relapse, but the ideal drug to achieve this has not been identified. We are currently investigating whether nivolumab effectively controls AML progression in such individuals. 

Another approach of great interest in older AML patients is the use of combinatorial treatment strategies. Combinatorial therapies that have already shown promising results for elderly patients including:

• Combining venetoclax, a targeted therapy drug, with azacytidine, which has shown very high response rates in these patients.
• Vosaroxin in combination with decitabine, which demonstrated a response rate of 75% in older patients with AML who were not candidates for high-dose chemotherapy. This is more than double the expected response rate of 25-35% with decitabine alone in a similar patient population. With these promising results, the combination will be evaluated further in a larger multicenter trial.

Our researchers are also focused on better understanding and developing novel treatment options for blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare leukemia most prominent in men. Preclinical studies determined that BPDCN cells are coated with a molecule called CD123, which can be targeted with a drug called SL-401. With this information in hand, we initiated the first-ever clinical trial of patients with BPDCNs to test the effectiveness of SL-401.

This ongoing multicenter trial has already uncovered that a multiple-cycle treatment with this drug is a safe and feasible treatment option for patients with BPDCN. The goal of these efforts is to determine if SL-401 can be used instead of or in conjunction with stem cell transplantation to improve patients’ survival outcomes.