CD117 antibody to prepare people with AML or MDS for donor stem-cell transplant

CD117 Antibody-Based HCT Conditioning for Myeloid Malignancies

NIH-funded research Stanford University · NIH-11197509

A CD117-targeting antibody is used to clear bone marrow cells so people with AML or MDS can receive a less toxic donor stem-cell transplant.

Quick facts

Grant typeP01 program project
Study typeNIH-funded research
Funding institutionStanford University NIH-funded
Lab location1 site (Stanford, United States)
Project IDNIH-11197509 on NIH RePORTER

What this research studies

This approach uses an antibody that binds CD117 (c-Kit) on blood stem and progenitor cells to selectively remove those cells from the bone marrow before an allogeneic stem-cell transplant. The antibody can target disease-initiating myeloid cells and has been tested together with low-dose radiation or the drug 5-azacytidine to increase killing of malignant clones. Early work at Stanford showed the antibody was safe in older patients when used as part of transplant conditioning. The overall aim is to replace harsh, non-specific chemotherapy with targeted conditioning that lowers toxicity and helps donor cells engraft.

Who could benefit from this research

Good fit: Ideal candidates are adults with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) who are eligible for allogeneic stem-cell transplantation, particularly those needing reduced-toxicity conditioning.

Not a fit: People without AML/MDS, patients who are not transplant candidates, or those whose leukemia lacks CD117 expression are unlikely to benefit from this approach.

Why it matters

Potential benefit: If successful, this could make donor stem-cell transplants safer and better tolerated, especially for older or frail patients with AML or MDS.

How similar studies have performed: Preclinical studies showed CD117 antibodies can deplete malignant myeloid clones and an early clinical trial combining the antibody with low-dose radiation reported acceptable safety, so the approach is promising but still relatively new.

Where this research is happening

Stanford, United States

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.