ASCAT 2023 | The barriers to gene therapy implementation for treating SCD in low- and middle-income countries
In this video, Lawrence Faulkner, MD, Cure2Children Foundation, Florence, Italy, addresses the challenges to delivering gene therapy for sickle cell disease (SCD) treatment in low-income countries. The high cost of this treatment is a large barrier to implementation, making bone marrow transplant the most practical treatment option. This interview was held at the 18th Annual Scientific Conference on Sickle Cell and Thalassemia (ASCAT) 2023, held in London, UK.
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Transcript (edited for clarity)
I don’t have any doubt that gene therapy will be the answer eventually, but this may take many years. For now, it is for most low- and middle-income countries totally impractical because the cost is astronomical, we’re talking about millions of dollars. And of course there are technical challenges. Also, presently, if a child has a matched sibling, he’s not even eligible for gene therapy...
I don’t have any doubt that gene therapy will be the answer eventually, but this may take many years. For now, it is for most low- and middle-income countries totally impractical because the cost is astronomical, we’re talking about millions of dollars. And of course there are technical challenges. Also, presently, if a child has a matched sibling, he’s not even eligible for gene therapy. So my personal belief is that bone marrow transplantation, which is also, of course, improving, will probably be the realistic option for cure for many years to come.
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