Allegheny General Hospital (AGH) has been awarded a $1.7 million grant from the National Institutes of Health (NIH) to study gene therapy using ultrasound for the treatment of xerostomia.
Called ultrasound-assisted gene transfer, the technique will be explored by the AGH team in an animal model study to determine its efficacy in the treatment of xerostomia, a high-incidence complication of radiation therapy for head and neck cancer in which the salivary glands are damaged, according to a hospital press release.
Ultrasound-assisted gene transfer uses sonoporation to modify the permeability of cell membranes and allow DNA to pass through. After mixing a gene drug with a solution of microbubbles and infusing it into the treatment site, a very powerful, low-frequency ultrasound beam is directed into the area. The resulting implosion creates a shock wave that briefly opens the cell membrane and allows the gene drug to enter.
The AGH study will use sonoporation in an attempt to restore the function of damaged or destroyed salivary glands by infusing a genetic drug called aquaporin-1 (AQP1) into the cells of the gland. Using a syringe, the gene/microbubble solution is delivered directly into the salivary gland through its opening in the mouth. The ultrasound beam is then administered for just a few minutes. There is no need for surgery or anesthesia.
Several preclinical studies have already demonstrated the potential of AQP1 gene therapy to provide substantial relief from xerostomia.
The NIH award is one of the largest grants ever received by AGH and will be distributed over the course of four years. The project is being led by Michael Passineau, PhD, director of the Gene Therapy Program at Allegheny Singer Research Institute.
