Earlier this month, we learned 22-year-old Daniel Cressy would become the first person in Louisiana to receive a gene therapy that promises to provide what patients and their families have long dreamed of — a cure for sickle cell disease.
Cressy’s journey, as documented by reporter Emily Woodruff, is the fruit of decades of research that will benefit thousands.
In Louisiana, there is a high prevalence of the disease per capita, and it disproportionately affects Blacks.
The life-changing nature of this breakthrough is evident when you know a little about the disease and patients like Cressy who have lived with it since birth. Sickle cell disease gets its name from a genetic mutation that causes red blood cells, which are normally round, to be shaped like a crescent, or sickle. As these mutated cells move through vessels, they can become stuck, causing episodes of debilitating pain. For patients, this can mean weeks of not being to work, attend school or perform the essential functions of life.
It can mean they have to give up on their dreams. Cressy, who aspires to become a licensed pilot, was denied medical clearance by the Federal Aviation Administration due to his sickle cell disease. He's hoping that will change after he completes the gene therapy.
It’s no easy road. Just getting the green light to receive the therapy took 18 months. Then, there are potential risks and side effects to confront. The therapy itself requires blood transfusions, stem cell collections and chemotherapy over several months.
Cressy is undergoing the process at Manning Family Children’s, and Woodruff’s reporting put a spotlight on the incredible teamwork of the medical staff there. After Cressy’s stem cells were collected, they were set to be shipped off to Scotland, where, in a process that seems like science fiction, scientists will be able to edit his genes to address the mutation. The idea is that when the edited cells are returned to his body, they will help rebuild healthy cells.
We must not overlook that robust federal funding for basic science research is why we see breakthroughs like this. NIH-funded studies of the human genome led to the development of CRISPR, the gene-editing technology used here. While cuts to the National Institutes of Health may save money in the short run, improving lives like Cressy’s in the long run is worth far more.
And lastly, we must face the tremendous cost of this therapy, more than $1 million for a single patient, not counting hospital stays and other costs. Louisiana is lined up to get up to support from a new federal program called the Cell and Gene Therapy Access Model to help Medicaid patients get these therapies.
With roughly 3,000 Medicaid patients in the state who have sickle cell disease, clearly, more will need to be done. But for now, we are glad to see patients like Cressy and their loved ones finally have a reason to hope.