The FDA has recently granted fast-track designation to a new natural killer cell therapy called CYNK-001 for those patients with AML. CYNK-001 is a natural killer (NK) cell therapy that consists of non-genetically modified stem cells. “The majority of patients with AML continue to have poor long-term outcomes, particularly those who suffer relapse or have measurable residual disease (MRD), necessitating the development of novel therapies, including CYNK-001.” According to the Food and Drug Administration (FDA), fast track is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier. Fast Track addresses a broad range of serious conditions.

From MRD Positive to MRD Negative

The ongoing trial of CYNK-001 continues to study those patients who are measurable residual disease (MRD) positive. In June 2021, a patient in this trial converted to MRD negative. Thus, the trial has been expanded to study those patients who can be MRD positive and transition with CYNK-001 cells to MRD negative. This patient initially received 7+3 chemotherapy that failed, and was still MRD positive after 4 months on azacitidine. The patient then received lymphodepletion followed by 1.8 billion CYNK-001 cells on days 0, 7, and 14 (a total of 5.4 billion cells). By day 28, the patient showed MRD negative. CYNK-001 cells were found in both the bone marrow and the blood.

The CYNK-001 Trial

Now, this trial has been expanded and is evaluating 22 patients and their maximum-tolerated dose of CYNK-001. Each patient must have primary or secondary AML, be MRD positive and be between 18-80 years old. Any patient with a significant medical condition, autoimmune disease or inadequate organ function can not participate in this study. Patients are given cyclophosphamide and fludarabine followed by CYNK-001 on days 0,7, and 14. The main endpoints of this study are the number of patients who experience dose-limiting toxicity (DLT), maximum tolerated dose (MTD) and frequent adverse effects.
The frequency of those who go from MRD positive to MRD negative is measured, as well as the duration of the MRD response, progression-free survival time and the length of complete remission (CR).

This study hopes to expand to those patients with relapsed/refractory AML. It intends to increase the CYNK-001 to 9.0 billion cells. “We believe that the unique properties of our cell source, including the ability to proliferate and maintain activity, could be the key to improving response rates and durability for patients. We are pleased to receive this fast-track designation from the FDA for AML, supporting the continued development of our placental-derived NK cell platform.”

Trial specifics and locations can be found on our

Clinical Trial Finder

This study will find the maximum tolerated dose or the maximum planned dose of CYNK-001 which contains natural killer (NK) cells derived from human placental CD34+ cells and culture-expanded. CYNK-001 cells will be given after lymphodepleting chemotherapy. The safety of this treatment will be evaluated, and researchers want to learn if natural killer cells will help in treating AML.