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Yale Cancer Center study shows first randomized trial using checkpoint inhibitors promising for patients with myeloid cancers

December 09, 2019

New findings by Yale Cancer Center (YCC) and Smilow Cancer Hospital researchers show promising results using checkpoint inhibitors for patients with myeloid cancers. The study was presented today at the 61st American Society of Hematology (ASH) meeting in Orlando, Florida. The annual conference is attended by an international audience of more than 25,000 hematology professionals.

Amer Zeidan, MBBS, MHS, associate professor of medicine (hematology) at YCC, is leading the research study using the chemotherapy drug azacitidine. Azacitidine is a standard treatment for patients with acute myeloid leukemia (AML) who are unfit for intensive chemotherapy as well as those with higher risk myelodysplastic syndromes (MDS). Pre-clinical research had suggested that azacitidine might prove more effective when combined with a checkpoint inhibitor drug such as durvalumab, which inhibits the PD-L1 protein that cancer cells may use to hold off immune system attacks. This theory was tested by Zeidan and colleagues in a large, international randomized phase 2 clinical trial of first-line treatments for both groups of patients, which compared azacitidine alone versus azacitidine combined with durvalumab.

“The final results showed no significant improvement in response rate or survival of patients with the combination therapy in either cohort,” said Zeidan. “But this study was the first randomized trial of immune checkpoint inhibition, a form of immune therapy which provided impressive results in patients with some forms of advanced solid tumors, in patients with myeloid malignancies.” Zeidan added the trial is also expected to yield additional laboratory results that improve the understanding of immune changes in these blood cancers and might inform the design of future trials with these agents.

Submitted by Anne Doerr on December 08, 2019