ARVO 2014: LCA Gene Therapy Recipient Featured During Keynote
15-year-old Yannick Duwe transfixed the audience, describing how the treatment enabled him to use a computer instead of Braille, and work much easier and faster at school.
Gene-therapy pioneer Jean Bennett, M.D., Ph.D., may have been the keynote speaker for the closing session at the annual meeting of the Association for Research in Vision and Ophthalmology (ARVO), but Yannick Duwe, a 15-year-old patient with Leber congenital amaurosis (LCA) who was treated in her clinical trial six years ago, stole the show. He and his father, Tony, were part of a panel which also included members of Dr. Bennett’s research team.
Yannick was understandably nervous to be in a vast auditorium under bright lights before hundreds of scientists, but the audience was transfixed by the teenager, who said that the treatment enabled him to use a computer instead of Braille, and work much easier and faster at school.
Yannick’s father explained that the therapy has given his son independence that he never had. “We were searching for six years for a cure for my son. He had become completely blind,” said Tony in response to a question from Dr. Bennett. “But then we met you, and now he sees much more.”
The loudest applause came after Dr. Bennett asked Yannick what advice he’d give other kids who were considering entering a gene therapy clinical, and he replied, “Just do it.”
Before Yannick and his father spoke, Dr. Bennett invited the other panelists to discuss the challenges and rewards that have come over the past 15 years in moving the LCA gene therapy from canine studies to humans. Much of that research was made possible by funding from the Foundation Fighting Blindness.
The Phase III clinical trial for the treatment is currently being conducted by Spark Therapeutics with study sites at the Children’s Hospital of Philadelphia and the University of Iowa. If all goes well, the investigators could apply for approval for the therapy from the U.S. Food and Drug Administration in about two years.
Arlene Drack, M.D., a clinical investigator at the University of Iowa, is impressed by the confidence and enthusiasm of the Phase III pediatric trial participants. “The kids have been very eager for the second eye [to be treated]. They’re a little concerned about the first eye, but they want the second eye done,” she explains. “That, to me, says that we’re really doing something helpful.”