A race to find the best treatment for premature babies at risk for retinopathy of prematurity (ROP) the leading cause of childhood vision loss and blindness has spurred both controversy and research.
Anti-vascular endothelial growth factor (VEGF) therapies, currently in clinical trials, curb ROP by inhibiting abnormal blood vessel growth in the eye. The problem: VEGF also plays an important role in the development of the retina, and the infant.
"Although clinical trials are testing lower doses of neutralizing VEGF antibodies in severe ROP, a safe and effective dose remains elusive," explains John A. Moran Eye Center's Mary Elizabeth Hartnett, MD, whose work is funded by the National Institutes of Health and Research to Prevent Blindness.
Now, new findings from Hartnett and Moran researchers Silke Becker, PhD, and Haibo Wang, PhD, suggest the viability of a different, more selective approach.
The researchers used gene therapy to knockdown overexpressed VEGF in Müller cells in the rat model of oxygen-induced retinopathy the most representative model of human ROP. Their findings show targeting VEGF-164 may be effective without being as harmful to the retina as inhibiting VEGF completely.
Scientific Reports published the research, Targeted Knockdown of Overexpressed VEGFA or VEGF164 in Müller Cells Maintains Retinal Function by Triggering Different Signaling Mechanisms, on Jan. 31, 2018.
The findings are significant as rates of ROP increase along with survival rates for premature infants.
"Additional research must be done, but knowledge like this is getting us closer to better and safer therapies for these babies," said Hartnett.
In addition to Hartnett, the study was conducted by Moran researchers Silke Becker, PhD, Haibo Wang, PhD, and Aaron B. Simmons, PhD. Additional collaborators included Thipparat Suwanmanee, PhD, and Tal Kafri, MD, PhD, from the University of North Carolina Chapel Hill, and University of Utah Department of Internal Medicine Adjunct Associate Professor Gregory J. Stoddard, MD.