The combination of two clinically approved drugs (Viagra and Prixafor) can quickly and effectively regulate the blood stem cells in the bone marrow of mice to enter the blood. This strategy is almost as effective as the current standard protocol for hematopoietic stem cell mobilization.
"Given that both of these drugs are approved by the U.S. Food and Drug Administration, they can be tested on human volunteers relatively quickly," said Camilla Forsberg, a stem cell biologist and corresponding author at the University of California, Santa Cruz. "If it is proven safe and effective in clinical studies, doctors can consider these findings."
Hematopoietic stem cell transplantation is a method to treat a variety of blood and immune diseases by replacing abnormal hematopoietic stem cells with healthy cells. This process involves mobilizing hematopoietic stem cells in the bone marrow into the blood, and then collecting these cells for transplantation, which can be transplanted to the same person or to the recipient. However, the main obstacles for hematopoietic stem cell transplantation are the limited supply of donor cells and the lack of effective means of obtaining them.
Currently, the standard protocol for the mobilization of hematopoietic stem and progenitor cells includes the injection of granulocyte colony stimulating factor (G-CSF) for several days. Although G-CSF is effective in most cases, it is expensive and may cause side effects such as nausea, fatigue and bone pain, and is often unsuccessful for cancer patients undergoing chemotherapy.
Another drug that can activate hematopoietic stem cells is Prixafor, but it is not very effective as a single drug. "A better harvest plan will significantly increase the success rate of current indications and open treatment hematopoietic cell therapy." Forsberg said.
Recent experiments have shown that increasing vascular permeability helps mobilize hematopoietic stem cells from the bone marrow into the bloodstream. Based on this, the Forsberg team tested whether Viagra can be a viable option for hematopoietic stem cell transplantation.
Researchers found that Viagra itself is ineffective, which shows that people who currently take Viagra for other reasons do not have to worry about inadvertently mobilizing hematopoietic stem cells.
At present, the number of hematopoietic cells in mice using Prixafor alone has increased by nearly 3 times compared with control mice. But the combination therapy is better-mice that took a single dose of Viagra and injected prisafox had approximately 2500 hematopoietic stem cells in their blood within 2 hours, which was 7.5 times that of control mice. Oral Viagra for 3 days and a single injection of Prixafor had a better effect, about 2800 more hematopoietic stem cells, which was 8.4 times that of control mice. In addition, the effectiveness of this method is comparable to 4 days of G-CSF treatment.
However, the researchers said that more studies on humans are needed to test the safety and effectiveness of this method.