MINNEAPOLIS — A documentary that just premiered during the Minneapolis St. Paul International Film Festival marks the latest twist in a medical journey that has spanned thousands of miles and last several years for the family of a young girl from Alabama.
"We've been so excited and waiting on this," said Gary Hamlett as he walked into the premiere of "Sequencing Hope" with his 7-year-old daughter Celia Grace Hamlett.
KARE 11 first connected with Celia Grace and her family nearly three years ago, when she and her family traveled from their home in Alabama to undergoing an experimental gene therapy treatment at M Health Fairview Masonic Children's Hospital in 2021 that offered her a chance at a normal life.
"(MLD) affects the brain, it affects the nerves, it's progressive and it's lethal," said Dr. Paul Orchard, the Pediatric Blood and Marrow Transplant Physician at M Health Fairview who petitioned the FDA to make Celia Grace the first patient to receive gene therapy to treat MLD in the United States. "I think it's really an opportunity to move the field forward and to better treat the kids."
The film documents how Celia Grace has done exactly that. It follows her and her family through the long journey to that therapy, and her life in the years since.
"She is doing so well, you would never be able to tell she was sick," said Kassie Hamlett, Celia Grace's mother. "Everything is the same as it was, so it has not progressed any at all, so just praying it continues to stay that way."
The history made by Celia Grace came too late for Pat Lang. Her daughter, Maddie Lang, was diagnosed with MLD long before gene therapy was an option.
The documentary also followed Pat's care for Maddie before she died from MLD in 2022.
"The fact there is now something that can save them is a beautiful thing," Pat Lang said. "Am I jealous? You bet. But is it a great thing? You bet."
Just before the documentary premiered, another great thing happened. The FDA approved the gene therapy treatment, opening the door to many others.
"It's a very exciting thing to be able to be able to offer this now and it's not just MLD, it's sickle cell disease, and a number of other inherited diseases," Dr. Orchard said. "They are going to have the same type of opportunity, so this is really where the field is going to go."
"I want people to see our documentary and not give up hope," Kassie said. "The road is rough, but the end is very rewarding."
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