First Patient Treated in NKX101 Trial for AML, MDS


Recently, biopharmaceutical company Nkarta announced the dosing of the first patient in its Phase 1 clinical trial. Within the trial, researchers will evaluate and analyze the company’s investigational therapy, NKX101, for patients with acute myeloid leukemia or myelodysplastic syndromes. Nkarta will share data from the trial, and the design, at the 2020 American Society of Hematology Annual Meeting. Learn more about the presentation (here).


Developed by Nkarta, NKX101 is an immunotherapy therapy treatment engineered to better identify and target tumor cells. It is created from healthy donor blood. The company sourced natural killer (NK) cells, then engineered them to include membrane-bound interleukin 15 and a chimeric antigen receptor (CAR). According to the British Society for Immunology, NK cells:

are lymphocytes in the same family as T and B cells, [and] are best known for killing virally infected cells, and detecting and controlling early signs of cancer. NK cells were first noticed for their ability to kill tumour cells without any priming or prior activation, [and] secrete cytokines such as IFNγ and TNFα, which act on other immune cells like Macrophage and Dendritic cells to enhance the immune response.

So, the specially engineered NK cells in NKX101 are designed to more efficiently and effectively combat tumor cells. In particular, NKX101 targets NKG2D ligands on cancer cells. The Phase 1 clinical trial will analyze the therapy’s safety, efficacy, pharmacokinetics, and anti-tumor activity. 27 patients enrolled. Patients will receive 3 infusions of NKX101 weekly. 

Myelodysplastic Syndromes (MDS)

There are five subtypes of myelodysplastic syndromes (MDS), which are progressive conditions that stop healthy blood cells from developing in bone marrow. Altogether, these subtypes are refractory anemia, refractory anemia with sideroblasts, refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, and chronic myelomonocytic leukemia. To determine which type of MDS a patient has, doctors must closely examine the bone marrow.

Admittedly, researchers are not quite sure what causes MDS. However, genetics, chemical exposure, and chemotherapy are thought to play a role. MDS typically affects patients more than 60 years old. Males are impacted more frequently than females. In about half of all cases, MDS progresses to become AML. Symptoms include:

  • Anemia (low red blood cell count)
  • Thrombocytopenia (low platelet count)
  • Neutropenia (low white blood cell count)
  • Frequent infections
  • Easy bruising and bleeding
  • Fatigue
  • Heart palpitations
  • Chest pain
  • Pallor (pale skin)
  • Shortness of breath or difficulty breathing

Acute Myeloid Leukemia (AML)

Also sometimes referred to as acute myelogenous leukemia, acute myeloid leukemia (AML) is a blood and bone marrow cancer. AML results from DNA damage in developing cells. As a result, the bone marrow creates immature white blood cells called myeloblasts. These grow in number, crowding out healthy blood cells and causing damage. Symptoms include:

  • Fever
  • Pallor
  • Breathlessness
  • Fatigue
  • Frequent infections
  • Unintended weight loss
  • Easy bruising and bleeding
  • Bone pain
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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