Both organizations will invest $100 million to develop and advance candidate gene-based cures for HIV and sickle cell disease to clinical trials within 10 years.
Advances in genetics research have led to the development of effective gene-based treatments for conditions including blindness and certain types of leukemia. Now, the National Institutes of Health (NIH) and the Bill & Melinda Gates Foundation have announced that $200 million will be invested over the next 4 years towards developing gene-based cures for both HIV and sickle cell disease.
Each organization will invest $100 million with the intention of making effective and affordable cures globally available, including in low-resource settings.
“This collaboration is an ambitious step forward, harnessing the most cutting-edge scientific tools and NIH’s sizable global HIV research infrastructure to one day deliver a cure and end the global HIV pandemic,” said Anthony S. Fauci, MD, director of the National Institute of Allergy and Infectious Diseases, part of the NIH, in a press release. “We are taking into account those with the greatest need at the foundation of this effort, to ensure that, if realized, this exceptional public health achievement will be made accessible to all.”
The collaboration will focus on 2 primary areas of coordination. The first area is to identify potential candidate cures for both diseases for pre-clinical and clinical evaluation, which will be co-funded by the 2 groups. The second area of collaboration is to define long-term opportunities to work with African partners in advancing viable options to late-phase clinical trials, for which funding will be determined based on progress.
According to the NIH, the projects will require “new delivery systems that can get prospective therapies to the right places in the body and optimize treatments to target the cells involved in the respective diseases efficiently and specifically.”
Particularly for HIV, this will require a system that targets the reservoir of proviral DNA that continues to exist in a small number of cells, even in patients with a history of effective antiviral therapy. One potential approach is to locate the reservoir of infected cells that harbor integrated HIV genomes after treatment and target DNA sequences with gene-editing technology.
“Such treatments that happen entirely within the body, known as in vivo treatments, would be a major step forward from current treatments, which apply genetic therapies to cells taken outside the body (ex vivo) and then reinfused,” according to the statement.
One of the largest issues with gene-based cures is that the complexity and cost of the treatment is so high that administration is mostly limited to hospitals in wealthy countries. However, both HIV and sickle cell disease disproportionately affect individuals living in Africa.
Approximately 95% of the 38 million people living with HIV globally are in low-resource areas of the developing world, with 67% in sub-Saharan Africa, half of whom are not receiving treatment. Therefore, this investment will focus on developing curative therapies that are safe, effective, and affordable to be used in low-resource settings.
Both organizations will also continue investing in other research efforts towards developing cures for both sickle cell disease and HIV.