Viagra Enables Efficient, Single-Day Hematopoietic Stem Cell Mobilization

Summary Hematopoietic stem cell (HSC) transplantation is a curative treatment for a variety of blood and immune disorders. Currently available methods to obtain donor HSCs are suboptimal, and the limited supply of donor HSCs hampers the success and availability of HSC transplantation therapies. We recently showed that manipulation of vascular integrity can be employed to induce HSC mobilization from the bone marrow to the blood stream, facilitating non-invasive collection of HSCs. Here, we tested whether FDA-approved vasodilators are capable of mobilizing HSCs. We found that a rapid, 2-h regimen of a single oral dose of Viagra (sildenafil citrate) combined with a single injection of the CXCR4 antagonist AMD3100 leads to efficient HSC mobilization at levels rivaling the standard-of-care 5-day regimen of granulocyte-colony stimulating factor (G-CSF/Filgrastim/Neupogen). Our findings solidify vascular integrity as an essential regulator of HSC trafficking and provide an attractive, single-day regimen for HSC mobilization using already FDA-approved drugs.
Graphical Abstract
Highlights • Viagra enhances AMD3100-mediated HSC mobilization • Extremely rapid and efficient HSC mobilization with two FDA-approved drugs • Vascular integrity regulates HSC trafficking
In this article, Forsberg and colleagues demonstrate that Viagra, a widely used vasodilator, enhances Plerixafor-mediated hematopoietic stem cell (HSC) mobilization from the bone marrow to the blood stream. These findings solidify vascular integrity as an essential regulator of HSC trafficking and provide an attractive, single-day regimen for HSC mobilization using already FDA-approved drugs.