Over the next four years, the National Institutes of Health and the Bill and Melinda Gates Foundation plan to each invest $100 million towards the development of gene therapy-based cures for sickle cell disease and HIV. In particular, they are focusing on making these treatments available in sub-Saharan Africa, where they are needed most.
According to NIH Director Dr. Francis Collins, “This unprecedented collaboration focuses from the get-go on access, scalability and affordability of advanced gene-based strategies for sickle cell disease and HIV to make sure everybody, everywhere has the opportunity to be cured, not just those in high-income countries...We aim to go big or go home (Ducharme: 2019).”
Gene therapy research has made some significant advances in recent years. Already, it is being used to treat several diseases, from inherited blindness to neuromuscular disease and leukemia. And now, both animal studies and some clinical trials have suggested that gene therapy technology may be able to treat two highly prevalent diseases in Africa: sickle cell disease and HIV (Cohen: 2019).
To treat sickle cell disease, the collaboration aims to use in vivo gene therapy to deliver a therapeutic gene or gene-editing tools directly into the body via vectors, such as inactive viruses of nanoparticles. According to Collins, the procedure should resemble a simple blood transfusion.
Although studies are already underway investigating ways that viral vectors can be used to deliver healthy genes to various tissues in the body, so far, in vivo gene therapy has only been used to modify blood stem cells in animal models of certain diseases. Thus, the collaboration aims to build upon this research and build ways for this technology to work in people too.
As for HIV, the collaboration hopes to quicken the development of direct injections of gene editing components to target the CCR5 gene in white blood cells. With two people already having been cured of the disease via this treatment, in crippling the CCR5 gene, HIV is unable to enter new host cells and thus has been shown to naturally peter out. According to Steven Deeks, a leading HIV cure researcher at the University of California, San Francisco, “The potential beauty of in vivo gene editing is that it might be given ultimately as a single shot, curing everyone in a scalable manner (Ibid.).”
Ducharme, Jamie: Time
Cohen, Jon et al.: Science