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ASCAT 2023 | Gene therapy for the treatment of SCD: benefits and considerations
Andrew Campbell, MD, Children’s National Medical Center, George Washington University School of Medicine and Health Sciences, Washington, DC, speaks on the potential of gene therapy to be a widely-available curative treatment option for sickle cell disease (SCD) and the impact this would have on patients with the disease. Dr Campbell also considers the challenges which must be overcome for widespread implementation of the therapy to be possible; especially in developing countries where the greatest number of barriers exist and disease burden is greatest.
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Transcript
Obviously it’s the gene therapy, I think, which is a potential game changer. Right now, unfortunately, the access is in developed countries and high-income countries. So I’m very excited about that because if it shows it’s safe and effective, and it needs to be studied long term, it will really change how we think about sickle cell disease management and access to sickle cell disease management and it will change the potential outlook of a child who’s born with sickle cell disease and as they think about their future, that can significantly change what they can do...
Obviously it’s the gene therapy, I think, which is a potential game changer. Right now, unfortunately, the access is in developed countries and high-income countries. So I’m very excited about that because if it shows it’s safe and effective, and it needs to be studied long term, it will really change how we think about sickle cell disease management and access to sickle cell disease management and it will change the potential outlook of a child who’s born with sickle cell disease and as they think about their future, that can significantly change what they can do. We’ve experienced it because we have also performed gene therapy at our hospital in Washington, DC, and we’ve seen the impact that is made in a patient that’s already received this.
The concern, obviously, is the cost of this gene therapy, and we are concerned about that and hopefully over time the cost will come down. And that’s what we are concerned about, especially in low- to middle-income countries, because that’s where the greatest burden is, especially in sub-Saharan Africa. So we would like to improve the access of these curative therapies, most notably gene therapy, because with the gene therapy you don’t need a bone marrow transplant donor, they use your own stem cells so you’re not dependent on someone else on that.
But I also don’t want it to overshadow the need for disease modifying therapy access, because gene therapy at this time, at this moment, is more likely to go to the severe patients. But as the technology is improved, I do believe that the access to these curative like therapies will go to low- to middle-income countries, and we just hope that it is done in a safe way. And also that the monitoring after the transplant, after the gene therapy, is established wherever the therapy is offered, because that’s something you have to do, a long term follow-up to make sure there’s no long term effects from a health standpoint for the sickle cell patient. But yeah, we’re very excited. In the United States we’re reviewing it on December 8th and 20th- two gene therapy options coming up that the FDA will review as potential treatment options for sickle cell patients in the United States.
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