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Hope, But Cost Worries

New gene therapy approaches are increasingly being explored to treat sickle cell disease, but Nature News writes that many new gene therapies have come with price tags that may be unaffordable for many patients, especially in regions of Africa, the Caribbean, and India where the condition is more common.

It adds that there are a number of clinical trials underway that are, for instance, introducing copies of modified fetal hemoglobin, blocking the switch that usually turns fetal hemoglobin off after birth, or providing a β-globin gene that has been altered to resist sickling. Early results from some of these efforts have been promising, Nature News notes.

However, it says that these treatments will likely be expensive, pointing out that a gene therapy for for β-thalassaemia costs about $1.8 million without accounting for hospital stays and related expenses. While an analyst tells Nature News that that price tag is likely lower than what standard treatments would cost during a patient's lifetime, it still is out of reach for many patients in the US as well as in other parts of the world. 

To that end, Nature News adds that there are some researchers like Cincinnati Children's Hospital's Punam Mali who are exploring ways to make treatment more affordable, such as by tweaking the approach to make the associated hospital stay shorter and cheaper.