A Phase I Study of VOR33 Cellular Therapy and Mylotarg in People with Acute Myeloid Leukemia at High Risk of Relapse after Stem Cell Transplantation


Full Title

A First-In-Human, Open-Label, Multicenter Study of VOR33 in Patients with Acute Myeloid Leukemia who are at High-Risk for Leukemia Relapse following Hematopoietic Cell Transplantation


In this study, researchers want to see if the cellular therapy VOR33 safe and effective for people with acute myeloid leukemia (AML) who will be receiving a stem cell transplant from a donor and have a high risk of relapse. Investigators will also see if it is safe to give a standard leukemia drug called Mylotarg to participants after they receive VOR33.

VOR33 is made in a laboratory by genetically modifying stem cells from the donor by removing the gene that makes a protein called CD33. When patients receive VOR33 cells during the transplant, these cells will grow into healthy blood cells and platelets that do not contain CD33. Patients will then receive Mylotarg, a drug that targets and kills cancer cells with CD33 on them.

Researchers think that giving VOR33 cells followed by Mylotarg may help protect healthy donor stem cells while killing any remaining cancer cells. The treatments used in this study are given intravenously (by vein).


To be eligible for this study, patients must meet several requirements, including:

  • Participants must have AML and achieved remission or are near remission following chemotherapy.
  • Patients must have a high risk of AML relapse and be candidates for a stem cell transplant from a donor.
  • Patients must be physically well enough that they are able to be mobile, take care of themselves, and engage in all but physically strenuous activities. For example, they must be well enough that they could carry out office work or light housework.
  • This study is for people ages 18-70.


For more information and to ask about eligibility for this study, please contact the office of Dr. Christina Cho at 646-608-3785.





Disease Status

Relapsed or Refractory


Cho, Christina, MD