If everything goes according to plan, a gene therapy clinical trial could start later this year, according to McCarty. Six children with Sanfilippo type B and nine children with Sanfilippo type A would get injected with a virus that serves as a vector to deliver new genetic material to their cells, hopefully allowing them to make the enzyme they need to breakdown the buildup of heparin sulfate in their cells.
“We don’t want the patient families to have unrealistic expectations,” researcher Fu said. “We don’t want to say it’s a cure now because it isn’t a cure until after the trial.”
The O’Neills know there’s no guarantee Eliza will get into the clinical trial or that it will work, but said they need to try everything they can for their only daughter.
“We don’t want to be two years from now and say as parents that we left anything on the table,” Glenn O’Neill said.