Local
Nationwide Children’s gene therapy offers new option for Michigan boy with Sanfilippo syndrome
A gene therapy developed at Nationwide Children’s Hospital has been approved for Sanfilippo syndrome type A, the condition affecting 6-year-old Clark Willmore. His Michigan family is now working with the Columbus hospital to determine how he can begin treatment.
COLUMBUS, Ohio — A newly approved gene therapy developed at Nationwide Children’s Hospital is giving families facing Sanfilippo syndrome type A a treatment option for the first time, including the family of 6-year-old Clark Willmore, who lives in Michigan.
Clark’s parents, Japheth and Cloey Willmore, said they are working to find out what he needs to start treatment at the Columbus hospital. Nationwide Children’s is one of a small number of U.S. centers able to administer the therapy and is the closest such center to the family, they said.
Sanfilippo syndrome is a rare genetic disorder that causes progressive loss of cognitive function. The disease is sometimes called childhood dementia, and patients often die in their teens. Clark has type A, the form targeted by the newly approved gene therapy.
Before the approval, families searching for a treatment had no FDA-approved option, Cloey Willmore said. The news brought both excitement and emotion for the family, Japheth Willmore said.
Dr. Kevin Flanigan, who led the therapy’s clinical trial, said results indicate the treatment is slowing cognitive decline. He described the findings as highly promising and said the therapy may have a meaningful effect for children with the disease. He also cautioned that the treatment does not amount to a cure; the information provided about the approval does not specify how much patients’ decline may be slowed or the treatment’s long-term outcomes.
Ohio State football has offered the Willmores a welcome routine amid medical travel and the strain of Clark’s diagnosis. Though Clark may not understand the game as a typical 6-year-old would, his father said he is drawn to the television when a Buckeyes game is on. The family returns home from medical trips in Minnesota in time to watch some games, Cloey Willmore said.
The therapy’s development at Nationwide Children’s gives the family another reason to look toward Columbus. Flanigan said progress on this treatment could also help researchers advance work on other devastating diseases.