Target intelligence / Profile preview

Myeloablation

01

Overview

Myeloablation is a clinical state or procedure characterized by the severe and permanent depletion of the bone marrow's hematopoietic stem and progenitor cells. It is not a molecular target but rather a pharmacological or radiological outcome intended as a conditioning regimen prior to hematopoietic stem cell transplantation (HSCT) to eliminate malignant cells or dysfunctional immune systems (1, 2). The process serves three primary goals: to eradicate underlying disease (such as leukemia), to suppress the recipient's immune system to prevent graft rejection, and to create physical space in the marrow niche for donor stem cell engraftment (2, 4). Historically, myeloablation is achieved through high-dose non-specific chemotherapy, such as busulfan or cyclophosphamide, or total body irradiation (TBI), which are associated with significant systemic toxicities (4). Modern therapeutic developments are moving toward "targeted myeloablation," which utilizes monoclonal antibodies or radioimmunoconjugates against specific hematopoietic markers like CD45 or CD117 (c-Kit) to achieve the same marrow-clearing effect with reduced off-target damage to non-hematopoietic tissues (3). Without the subsequent infusion of healthy stem cells (rescue), a myeloablative state is fatal due to the body's inability to produce essential blood components (2). Citations: (1) National Cancer Institute (NCI). NCI Dictionary of Cancer Terms: Myeloablation. (2) StatPearls. Hematopoietic Stem Cell Transplantation (HSCT) Conditioning. (3) Majeti R, et al. (2022). Targeted conditioning for hematopoietic stem cell transplantation. (4) Gyurkocza B, Sandmaier BM. (2014). Conditioning regimens for hematopoietic cell transplantation. Blood.

Other names
Bone marrow ablationMarrow-ablative conditioningHematopoietic ablationAblative therapyTotal marrow ablation
02

Mechanism of action

Induction of severe or complete destruction of hematopoietic stem cells and progenitor cells in the bone marrow through high-dose alkylating agents, antimetabolites, total body irradiation, or targeted monoclonal antibodies.

03

Biological functions

Cell deathHematopoiesis inhibitionApoptosisDNA damage response
04

Disease associations

LeukemiaLymphomaMultiple myelomaSickle cell diseaseBeta thalassemiaSevere combined immunodeficiency (SCID)
05

Safety considerations

Severe pancytopeniaOpportunistic infectionsSepsisVeno-occlusive disease (VOD)MucositisGraft-versus-host disease (GvHD)InfertilitySecondary malignancies
06

Interacting drugs

Busulfan

7 more in the full profile.

07

Biomarkers

Absolute neutrophil count (ANC)Platelet countCD34+ hematopoietic stem cell countBone marrow cellularityHemoglobin level

Beyond the preview

Go deeper on Myeloablation.

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Drug pipeline

Full profile access

Explore the programs pursuing this target and their development progress.

  • Drug candidates
  • Developers
  • Development stage

Clinical trials

Full profile access

Follow the clinical studies evaluating therapies directed at this target.

  • Trial design
  • Status
  • Readouts

Competitive landscape

Full profile access

Compare approaches across drug candidates, modalities, and indications.

  • Programs
  • Modalities
  • Indications

Literature & evidence

Full profile access

Investigate the research and source evidence behind target biology and development.

  • Publications
  • Sources
  • Analysis

Patents

Full profile access

Explore patent activity around therapies and technologies addressing this target.

  • Patents
  • Assignees
  • Technologies

Research & analysis

Full profile access

Connect target biology, drug development, and emerging evidence in your research.

  • Biology
  • Development news
  • Analysis

Bring the full picture into focus.

See how Gosset can support your research on Myeloablation.

Explore the full profile

Gosset Free

Get started with Gosset.

Enter your work email and we’ll be in touch with next steps.

Work email preferred.

Book a call