Sickle cell disease is a leading cause of stroke in children. Dahn’s 18-year-old daughter had her first stroke in second grade, causing neurocognitive and academic issues. She’s had more than 10 surgeries, including a brain surgery. Like her older sister, Dahn’s 14-year-old also suffered learning challenges after a series of ministrokes and is slated for the same brain surgery later this month. Both rely on monthly blood transfusions to prevent future strokes.

Dahn, who founded the nonprofit patient advocacy group MTS Sickle Cell Foundation, also known as “My Three Sicklers,” said she wants Georgia to participate in the model.

Her oldest daughter isn’t currently eligible for the therapy because she is high risk. But Dahn is optimistic for her two younger daughters.

“The hope of it was overshadowed by the access to it,” Dahn said about the new gene therapy treatments. “The bulk of our patient community relies on [Medicaid] for payment. So, I think this is wonderful that they’re testing out this model to not only make it possible for patients to access, but in the long run to somehow make it more affordable.”

Tabatha McGee, executive director of the Sickle Cell Foundation of Georgia, was part of a group that advised federal officials developing the program.

“We’re very excited because it is a step in the right direction,” she said. “This is something we absolutely want to come to fruition. … We’ve never had a singular focus on sickle cell disease to help improve the inefficiencies, to help improve the inequality, inside of the health care system.”

In a statement, the Georgia Department of Community Health told Stateline it is reviewing the framework.

Expanding access

Dr. Lewis Hsu, chief medical officer of the Sickle Cell Disease Association of America, said the gene therapies are an innovative option that don’t come with the same risks as a bone marrow transplant — the only procedure that may be able to “cure” sickle cell disease for some patients — since the treatment uses the body’s own stem cells and doesn’t require a match from another person.

“Having gene therapy available for sickle cell disease is just very exciting,” Hsu said.

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