Developing A New Treatment For A Rare Pediatric Disease–Retinopathy Of Prematurity

Although any premature infant can develop ROP, those at the highest risk are those with birth weights (BWs) <1500 grams or under 2.5 pounds. This translates into about 60,000 US infants/year. Because over 90% of U.S. premature infants with very low birth weights now survive, the incidence of ROP is rising. Over 20,000 infants around the world are become blinded from ROP each year. The successful musician, Stevie Wonder is an example of a famous celebrity who is blind from ROP.

Most of the time, ROP regresses on its own and does not require any treatment. However, for infants who do require treatment, laser surgery has been used for decades. Laser therapy is very effective in preventing blindness by preventing the infant’s retina from detaching. However, complications from laser therapy are common. The laser surgery can cause nearsightedness, crossed eyes, and/or visual field constrictions. The anti-VEGF drug has largely replaced laser surgery for severe cases of ROP. In 2017, the Ophthalmic Technology Assessment Committee (OTAC) of the American Academy of Ophthalmology determined that anti-VEGF drugs are as effective as laser surgery for treating acute ROP. However, it is not known whether the escape of anti-VEGF drugs from the eye into the rest of the body can affect an infant’s growth and development. Because of its macro-molecular size, anti-VEGF drugs need to be injected into the eye. They are too big to be able to cross the cornea. Therefore, they cannot be made into an eye drop.

It goes without saying that there is a definite need for new safe and effective drugs for ROP, especially ones that will be easy-to-administer. Zietchick Research Institute is taking on this challenge. We are developing a new drug that works by blocking excess hormones in the eye. The hormone is needed for normal eye development. But when hormone levels become too high, they can cause the formation of pathologic blood vessels that pull on the back of the eye. Our drug will work by reducing hormone levels to physiologic levels. Very exciting is our plan to formulate it as an eye drop—no injections in the eye will be needed. We are proud to report that we are now certified (in the State of Michigan) as a social-mission company—an L3C—That means that our highest priority is to prevent blindness.