When Rebekah and Evan Lockard’s daughter, Naomi, was diagnosed with a devastating ultra-rare genetic disease, they didn’t know where to turn. Then they found Terry Pirovolakis, an IT professional who had made a gene therapy for his son with the same disease. But the process of getting Naomi treated has been an uphill battle, full of financial and logistical obstacles. The Lockard’s story flips the question we’ve been asking all season on its head. Instead of wondering, "if we could do something, should we," we're now asking, "if we can do something that helps patients, should we do it at any cost?" And this question isn’t for scientists or researchers, it’s for the rest of us.

Credits

Host: Alexis Pedrick
Executive Producer: Mariel Carr
Producer: Rigoberto Hernandez
Associate Producer: Sarah Kaplan
Audio Engineer: Samia Bouzid
Music by Blue Dot Sessions

Resource List

Mast, Jason. "A dad built a gene therapy for his son. Can he save other kids, too?" STAT News.

Elpida Therapeutics.

"Battling SPG50 and changing the world." August 17, 2022. The Jackson Laboratory.

Brent, Jonathan R. and Deng, Han-Xiang. "Paving a way to treat spastic paraplegia 50."

"AAV gene therapy for hereditary spastic paraplegia type 50: a phase 1 trial in a single patient." June 28, 2004.

"Colorado family pushes for more funding, awareness around rare neurological disorder." August 12, 2024. CBS News Colorado.

Naomi's Corner.