Latest AML therapies: novel preparations and first in class agents
Sep 14, 2020
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Guests Thomas Cluzeau, Naval Daver, and Andrew Wei discuss the latest AML therapies, including the use of CPX351 as a potential cure for certain patients, the safety and efficacy of Magrola MAB in combination with azacitidine, the high response rates and safety profile of treatment for TP53 AML patients, and updates on Venetoclax plus low dose Cytarabine study in previously untreated older AML patients.
CPX351, a dual drug liposomal encapsulation of Cytarrabine and Darna Ruberson, has shown benefits in remission rates and overall survival for poor risk acute myeloid leukemia patients.
The Phase 1B study of Magrola MAB, an anti-CD47 antibody, in combination with azocytidine, shows promise in patients with myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) by allowing macrophages to phagocytize tumor cells, leading to high response rates and manageable side effects.
Deep dives
Therapies for Poor Risk Acute Myeloid Leukemia
One therapy discussed for poor risk acute myeloid leukemia is CPX351, a dual drug liposomal encapsulation of Cytarrabine and Darna Ruberson. This therapy has shown benefits in remission rates as well as an increase in overall survival. The analysis of CPX351 treated patients in France confirms the data published by Geoffrey Ransette, and the long-term follow-up of the phase-two study shows continued improvement in overall survival after five years.
Phase 1B Study of Magrola MAB
The Phase 1B study of Magrola MAB, an anti-CD47 antibody, in combination with azocytidine, shows promise in patients with myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). By blocking CD47, the drug allows macrophages to phagocytize tumor cells, leading to high response rates. The safety profile of Magrola MAB is encouraging, with manageable side effects such as early-onset anemia. The study focuses on frontline AML and MDS patients, especially those with TP53 mutations, and shows high response rates in this population.
Phase Three Study of Venetoclax Plus Low Dose Cytarrabine
The Phase Three study of Venetoclax plus low dose Cytarrabine (VLEC) in previously untreated older patients with acute myeloid leukemia (AML) demonstrates improved survival rates compared to low dose Cytarrabine alone. The study shows an overall improvement in survival, a statistically superior response rate, and an improvement in event-free survival. Notably, the patients with nuclear Fosmon mutation positivity had a particularly promising response rate. Venetoclax is now considered an important medication for older AML patients who are considered unfit for intensive chemotherapy.
Acute myeloid leukemia (AML) is the most common type of acute leukemia among adults with almost 20,000 new cases reported in the US in 2020. With the identification of recurrent mutations and the development of novel agents, stratification has allowed for improvements in patient outcomes. In this podcast, Thomas Cluzeau, MD, PhD, of the Central University Hospital of Nice, Nice, France, Naval Daver, MD, of the University of Texas MD Anderson Cancer Center, Houston, TX, and Andrew Wei, MBBS, PhD, of Alfred Hospital and Monash University, Melbourne, Australia, discuss key updates in the AML field presented at EHA 2020.