Ground-breaking medical procedure is given for the first time in Florida
The first gene therapy surgery ever given to a patient in need has become news just recently, as Creed Pettit, a blind 9-year-old boy from Mount Dora, Florida, became the beneficiary at the Bascom Palmer Eye Institute, part of UHealth - University of Miami Health System and the Miller School of Medicine.
Dr. Audina Berrocal, M.D., pediatric retinal surgeon and professor of clinical ophthalmology performed the surgery on Creed's right eye. The procedure lasted one hour and the specialist used a super thin needle to inject the medication directly under the retina. Creed is scheduled for surgery on his left eye very soon at Bascom Palmer.
Most patients affected by this condition lose vision before the age of 10 due to degeneration of the retina. Luxterna, a product developed by Spark Therapeutics, is the first gene therapy medicine for the eyes that was approved by the FDA in December 2017. It is a viral vector that makes the photoreceptor cells in the retina produce the protein that these people with the RPE65 mutation lack.
“Genetic therapy is the beginning of treatment for hereditary problems of the eyes," said Berrocal. "The treatment combines technology, sub-specialized care and advances in science." Dr. Byron Lam, M.D., neuro-ophthalmologist, vision scientist and genetic therapy expert was the one who diagnosed Creed years ago at Bacom Palmer. According to the especialist the FDA approval of this drug sets a precedent for gene therapy as a viable option to restore vision to people with inherited eye diseases. The Bascom Palmer has an extensive genetic therapy program that includes ophthalmologists, surgeons, genetic counselors and research scientists who study the different forms of inherited eye diseases.
Creed Pettit was diagnosed at the age of 2 years with Leber congenital amaurosis, which is caused by the genetic mutation RPE65. His family, especially his mother, Sarah St. Pierre Pettit, constantly sought after treatment for the condition of her son, a state that obstructs his vision under low light conditions. Even though the child can draw, play the piano and enjoy other activities during the day, at night Creed was completely blind. The surgery performed recently can completely change his perspectives, his vision and his life
