Plerixafor

Plerixafor (rINN and USAN, also known as MOZOBIL, JM 3100 and AMD3100) is a macrocyclic compound and potential fusion inhibitor. It is an antagonist (or perhaps more accurately a partial agonist) of the alpha-chemokine receptor CXCR4.

HIV
It was initially developed for potential use in the treatment of HIV, for its role in the blocking of CXCR4, a chemokine receptor which acts as a co-receptor for certain strains of HIV (along with the virus's main cellular receptor, CD4). However, clinical trials in patients with HIV-AIDS have to date shown relatively little useful anti-viral activity.

Mobilization of hematopoietic stem cells
However, the CXCR4 alpha-chemokine receptor and its ligand SDF-1 are also important in hematopoietic stem cell homing to the bone marrow and in hematopoietic stem cell quiescence. Plerixafor has been found to be a strong inducer of "mobilization" of hematopoietic stem cells from the bone marrow to the bloodstream as peripheral blood stem cells.

Peripheral blood stem cell mobilization, which has become extremely important as a source of hematopoietic stem cells for transplantation over the past 10 to 15 years, is generally performed using the cytokine drug G-CSF, but is ineffective in around 15 to 20% of patients. AMD3100 offers clinical promise as a drug for peripheral blood stem cell mobilization, and has recently completed Phase 3 clinical trials. It is not yet in routine clinical use.

Small molecule cancer therapy
AMD3100 was seen to decrease metastasis in mice in several studies.