Acute Myeloid Leukemia - Healing Genes

Acute Myeloid Leukemia

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Genetically Modified T-cell Immunotherapy in Treating Patients With Relapsed/Refractory Acute Myeloid Leukemia and Persistent/Recurrent Blastic Plasmacytoid Dendritic Cell Neoplasm

Phase I Study of Cellular Immunotherapy Using T Cells Lentivirally Transduced to Express a CD123-Specific, Hinge-Optimized, CD28-Costimulatory Chimeric Antigen Receptor and a Truncated EGFR for Patients With CD123+ Relapsed/Refractory Acute Myeloid Leukemia and Persistent/Recurrent Blastic Plasmacytoid Dendritic Cell Neoplasm

Phase 1


Doctors at the City of Hope Medical Center seek to enroll patients in a clinical trial to treat the following cancers:

  • Adult Acute Myeloid Leukemia in Remission
  • Acute Biphenotypic Leukemia
  • Early Relapse of Acute Myeloid Leukemia
  • Late Relapse of Acute Myeloid Leukemia
  • Recurrent Adult Acute Myeloid Leukemia
  • Secondary Acute Myeloid Leukemia
  • Blastic Plasmacytoid Dendritic Cell Neoplasm
  • Acute Myeloid Leukemia
  • Adult Acute Lymphoblastic Leukemia
  • Interleukin-3 Receptor Subunit Alpha Positive
  • Minimal Residual Disease
  • Refractory Acute Myeloid Leukemia
  • Untreated Adult Acute Myeloid Leukemia

The investigatory treatment, a gene-modified immunotherapy, can treat the listed cancers by withdrawing immune T-cells from the patient and, utilizing gene editing, the white blood cells will be trained to target their cancer cells. Then the modified T-cells will be infused to the patient after a course of chemotherapy. If their disease has not responded after 28 days, they can receive a second infusion of the T-cells. Follow-up to track the progress of the tumors through lab assessments, MRI, and physical assessments will be required for ~6 weeks, and less frequent follow up will be required for up to 15 years.


  • Be 12 years of age and older
  • Be diagnosed with one of the eligible cancers, with DNA tests to confirm subtype required
  • Have a life expectancy of at least 16 weeks at time of enrollment
  • Agree to contraception for at least 6 months after the immunotherapy
  • Be HIV negative


  1. Prescreening tests to confirm eligibility of the patient to participate.
  2. Have blood drawn, immune cells separated and blood cells returned (leukapheresis)
  3. After lab modification time, the patient will be admitted to the hospital and receive chemotherapy over 3 to 5 days.
  4. A few days later, the gene-edited immune cells will be infused and drugs to help promote their growth injected.
  5. Recovery in the hospital.
  6. A second infusion at 28 weeks if no disease response.
  7. After completion of study treatment, patients are followed up at 24 hours, then every 2 days for up to 14 days, every week for 1 month, every month for 1 year and then yearly for 15 years.


The study site is at the City of Hope Medical Center in Duarte, California. Map.

Contact: Lihua E. Budde  |  800-826-4673  |  [email protected]



The National Cancer Institute (NCI)


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