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District Communications - News Room

20

Burke High Student Undergoes Gene Therapy

 



Fifteen-year-old Molly Troxel is a typical Burke High sophomore. She enjoys sports, drama, singing, getting together with friends, shopping and thinking about life after high school. What sets her apart from other students; however, is a rare genetic disease – Leber congenital amaurosis (LCA) – she inherited from her parents. LCA, which impacts the retina, is an impairment that begins in childhood and can progress to near or total blindness. Her mother, Laura Troxel, first noticed something was wrong when Molly was just an infant.

“I suspected something was wrong when she was between one or two months old,” said Troxel. “She didn’t focus on faces or objects. She would look around us and try to find the nearest, brightest light.” That prompted her to have her daughter seen by a local physician, who in turn, referred her to a specialist at the University of Iowa. There, she was diagnosed her with Leber’s. Although the diagnosis was distressing for her parents, her father, Ryan Troxel, said it never held Molly back.

“Her nickname is the Mollinator,” said Troxel. “The name certainly fits her. Over the years, she participated in rock climbing, snow skiing, paddle boarding, zip lining, playing goal ball and has recently learned scuba-diving. She is fearless.” In August of 2013, Troxel, with the support of her family, underwent twin eye surgeries. Doctors at the University of Iowa Hospitals and Clinics injected her eyes with a virus loaded with the correct copy of her faulty gene. It was a clinical trial of a new gene therapy and it was risky. “We were nervous,” said Ryan Troxel. “Molly never hesitated. She wanted the opportunity to see.”

The therapy, called Luxturna, was developed by a Philadelphia-based biotech startup called Spark Therapeutics and was recommended for approval in October by the Food and Drug Administration. A final decision is expected as early as next year.  Prior to the surgery, Troxel’s vision was 20/400 with glasses. It is now 20/200 with correction. “She still has trouble making out facial expressions,” said Ryan Troxel. “But she can now recognize faces. The best part of the progression has been watching her become more confident and outgoing.” Molly and her family are hoping the therapy will stop the disease’s progression. After four years, the prognosis looks good, but even if it doesn’t, Troxel is determined to continue to conquer new frontiers. “She would like to pursue the medical field during her post-secondary education,” said Ryan Troxel. “Maybe as a nurse practitioner.”  Troxel and her family were featured in a PBS film called “The Gene Doctors” that can be seen at http://www.pbs.org/the-gene-doctors/home/.
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