Montana Boy, 5, Inspires $3M Push for Gene Therapy Cure

Five-year-old Loic Blackford loves Spider-Man, American flags, and making people smile. But behind his big personality, a rare genetic disease is slowly stealing away his abilities.

Loic has pantothenate kinase-associated neurodegeneration, or PKAN. The terminal disorder affects only one to three children in every million, progressively robbing them of physical and cognitive function.

When Loic's mom Sherine discovered her son's diagnosis at age 2½, the prognosis was devastating. "It's coming up on the screen that it's terminal, there is no treatment, there is no cure," she said.

Before symptoms accelerated last October, Loic was hitting milestones. He ran, walked, counted, and learned his colors. Then those hard-won abilities began disappearing.

But the Blackford family refused to accept that future. They launched the Loving Loic Foundation to fund research into treatments and a potential cure.

The foundation partnered with the University of Massachusetts Chan Medical School to develop gene therapy for PKAN. For Sherine, this isn't theoretical anymore. "It exists. It's no longer just a theory, it's something that is already in this world," she said.

The Ripple Effect

The Blackfords have raised $3 million of their $5 million goal. That money is funding real science that could save Loic and other children fighting PKAN.

But Sherine sees something bigger. "If we are able to do this in this disorder, it creates a pipeline for doing it in others," she explained. Success here could open doors for treating other rare genetic diseases.

The Bozeman community has rallied around the family in extraordinary ways. Loic started kindergarten this year with teachers and classmates who embrace him. Strangers have become supporters.

"This community is amazing and there are so many people who are just willing to help you, even strangers," Sherine said.

Despite countless surgeries and declining abilities, Loic keeps showing up with his trademark smile. His fighting spirit fuels his family's race against time.

The Loving Loic Foundation will hold its third annual gala October 9 at the Armory in Bozeman, bringing the community together to push toward that $5 million finish line and the gene therapy that could change everything.