Mozobil, a novel small molecule CXCR4 chemokine receptor antagonist, has been shown in multiple earlier studies to rapidly and effectively increase the number of stem cells in circulation in the blood in patients with non-Hodgkin’s lymphoma and multiple myeloma. Once circulating in the blood, stem cells can be collected for use in an autologous stem cell transplant. While G-CSF is the standard mobilizing agent for PBSC donors, it is associated with some significant side effects and requires a multi-day dosing regimen. The other cytokine approved for stem cell mobilization, granulocyte-macrophage colony stimulating factor (GM-CSF), alters graft composition and may reduce the development of graft-versus-host disease (GVHD), but a significant minority of donors fails to provide sufficient CD34+ cells with GM-CSF and some experience unacceptable toxicity. Mobizil may be more effective and is better tolelrated but it is usually sued with G-CSF and toxicity advantages may not be clinically relevant. Patients treated with G-CSF and Mozobil were three times more likely than patients treated with G-CSF alone to reach the target threshold for collection with four or fewer days apheresis.
On December 15, 2008, U.S. Food and Drug Administration has granted marketing approval for Mozobil (plerixafor injection), a drug intended to be used in combination with granulocyte-colony stimulating factor (G-CSF) to mobilize hematopoietic stem cells to the bloodstream for collection and subsequent autologous transplantation in patients with non-Hodgkin’s lymphoma (NHL) and multiple myeloma (MM). The product has also been granted orphan drug designation. Mozobil is intended to be used in combination with granulocyte-colony stimulating factor (G-CSF) to mobilize hematopoietic stem cells for collection and subsequent autologous transplantation in patients with non-Hodgkin’s lymphoma and multiple myeloma.
Use of Mozobil in other diagnoses is off-label. A number of ongoing trials are exploring various facets of Mozobil therapies but do not include a specific trial for neuroblastoma. While it would be reasonable to conclude that Mozobil is safe and effective in codnitions other than indicated, this has not been explored and is not known.
Inpharma, Volume 1, Number 1600, 2007-08-11 , pp. 6-6(1)
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