Dr. Pemmaraju on the Challenges of Targeting TP53 Mutations in MDS/AML

Video

Naveen Pemmaraju, MD, discusses the challenges of targeting TP53-mutant myelodysplastic syndrome and acute myeloid leukemia.

Naveen Pemmaraju, MD, associate professor in the Department of Leukemia of the Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center, discusses the challenges of targeting TP53-mutant myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML).

TP53 mutations are present in up to half of patients with solid and liquid tumors, says Pemmaraju. However, the aberrations have been historically difficult to target.

Although some success has been made in indirectly targeting the TP53 axis, these mutations continue to be associated with poor prognosis across hematologic malignancies, explains Pemmaraju.

On January 30, 2020, the FDA granted a breakthrough therapy designation to the combination of APR-246 and the hypomethylating agent azacitidine for the treatment of patients with MDS with a susceptible TP53 mutation. Moreover, a phase 3 clinical trial evaluating the combination in TP53-mutant patients with MDS completed accrual on June 3, 2020.

While the data are still early, the combination appears promising in targeting TP53 mutations in this high-risk patient population, concludes Pemmaraju.

Related Videos
Emmanuel Antonarakis, MD, associate director, Translational Research, Masonic Cancer Center, University of Minnesota, Clark Endowed Professor of Medicine, University of Minnesota Medical School
Gautam Jha, MD, medical director, M Health Fairview Masonic Cancer Clinic and the Advanced Treatment Center at the M Health Fairview Clinics and Surgery Center—Minneapolis, chair, cancer committee, M Health Fairview Ridges Hospital
Ricardo D. Parrondo, MD, hematologist/oncologist, Mayo Clinic
Ilyas Sahin, MD
Raj Singh, MD
Jaime R. Merchán, MD, professor, co-leader, Translational and Clinical Oncology Research Program, director, Phase 1 Clinical Trials Program, Department of Medicine, Division of Medical Oncology, the University of Miami Miller School of Medicine, Sylvester Comprehensive Cancer Center
Saad J. Kenderian, MB, CHB
Tycel Phillips, MD
Minesh Mehta, MD
Shivaani Kummar, MBBS, FACP, Margaret and Lester DeArmond Endowed Chair of Cancer Research, Professor and Division Head, Division of Hematology/Medical Oncology, Oregon Health & Science University School of Medicine; co-director, Center for Experimental Therapeutics, co-deputy director, Knight Cancer Institute