Thomas Cluzeau, MD, PhD

Thomas Cluzeau, MD, PhD, head of hematology department, Central University Hospital of Nice, in Nice, France, discusses the rationale for evaluating azacitidine in combination with APR-246 as treatment of patients with 

-mutant myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML).

 mutations occur in about 5% to 10% of patients with MDS and AML, and it is more in common in AML, says Cluzeau. These patients tend to have a very poor prognosis with a low rate of complete responses with azacitidine alone, as well as a short median overall survival of 6 to 9 months.

. The TP53 pathway becomes functional, and apoptosis could be used in this cell after treatment with APR-246. In vitro and in vivo analyses have demonstrated a synergistic effect with the combination of azacitidine and APR-246, which is why investigators evaluated this treatment in a phase 2 study.

<< View more from this interview with Cluzeau

Videos

Thomas Cluzeau, MD, PhD, discusses the rationale for evaluating azacitidine in combination with APR-246 in patients with TP53-mutant myelodysplastic syndrome and acute myeloid leukemia.

Thomas Cluzeau, MD, PhD | Authors

Articles

Azacitidine Plus APR-246 Appears Promising as Treatment of TP53-Mutant MDS and AML