Site Editor

Rebecca Olin, MD, MS


TCT 2021: Natural Killer Cell Therapy Under Study in Selected Patients With AML

By: Vanessa A. Carter, BS
Posted: Friday, February 26, 2021

James McCloskey, MD, of Hackensack University Medical Center, New Jersey, and colleagues evaluated the use of CYNK-001—a cryopreserved allogeneic, off-the-shelf natural killer cell investigational product derived from placental CD34-positive cells—in select patients with acute myeloid leukemia (AML). At the 2021 Transplantation & Cellular Therapy Meetings (TCT), these investigators presented their overview of the ongoing CYNK-001-AML-001 trial (Abstract 173).

In a previous phase I trial, PNK-007, a fresh formulation comparable to CYNK-001 was administered as a single infusion of up to 106 cells/kg to patients with relapsed or refractory AML. The treatment was deemed safe, with one treatable event of cytokine-release syndrome, and a clinical response was achieved in 2 of 10 patients.

The ongoing trial will enroll up to 16 patients who are in morphologic complete remission (with or without hematologic recovery) and have minimal residual disease (MRD). Placental CD34-positive cells have been cultivated in the presence of cytokines to generate CYNK-001. Following 300 mg/m2 of lymphodepletion with cyclophosphamide and 25 mg/m2 of fludarabine, participants will be administered three infusions of either 600 million, 1.2 billion, or 1.8 billion cells using a 3+3 design. Transplant-eligible patients may receive their transplant as indicated once the 28-day dose-limiting toxicity period is complete and their MRD status is identified.

Safety will be evaluated by the frequency and severity of adverse events using a non–weight-based dose of CYNK-001. Efficacy will be determined by the duration of and time to MRD response, MRD conversion rate, duration of morphologic complete remission, and progression-free and overall survival.

At the time of abstract presentation, one MRD-positive patient with intermediate-risk AML in morphologic complete remission had received three doses of 600 million cells following consolidation/induction with azacitidine and venetoclax. This patient is currently in follow-up and is reported to have tolerated CYNK-001 well, without dose-limiting toxicities.


Disclosure: For full disclosures of the study authors, visit

By continuing to browse this site you permit us and our partners to place identification cookies on your browser and agree to our use of cookies to identify you for marketing. Read our Privacy Policy to learn more.