The National Institutes of Health (NIH) and the Bill & Melinda Gates Foundation have each committed $100 million to advance cures for HIV and sickle cell disease. The goal is to start testing in vivo gene-based treatments for the diseases in humans within 10 years and ultimately to make them available at a cost that enables global use.
President Donald Trump moved the eradication of HIV in the U.S. up the agenda earlier this year, leading to 2020 budget proposals that carve out money for tackling the disease. The NIH has responded to the call to wipe out HIV by committing to spend $100 million over the next four years and enlisting the Gates Foundation to match the commitment.
The plan set out by the NIH and Gates Foundation expands on Trump’s proposal, most notably by adding sickle cell disease to the agenda. Sickle cell differs from HIV in many ways, not least the fact that it is a genetic, not infectious, disease, but the NIH sees important overlaps.
In both diseases, the NIH and Gates Foundation plan to pursue the same gene-based strategies. The NIH listed vector tropism and efficiency, gene targeting and in vivo delivery as the shared strategies.
The focus on in vivo delivery is key to the NIH and Gates Foundation’s long-term plans. Vertex Pharmaceuticals and CRISPR Therapeutics already have a gene-based treatment for sickle cell in the clinic. However, the gene editing takes place outside of the body, meaning cells must be taken from a patient, modified and readministered back into the same individual.
That is an expensive, logistically challenging process in the U.S. At best, the resulting therapies would be too expensive to deploy across sub-Saharan Africa, as the NIH and Gates Foundation envisage. At worst, it would be logistically impossible to produce the therapies in sub-Saharan Africa at all.
Those factors make in vivo treatments a better choice, but the science of editing DNA within the body is immature. Armed with $200 million, the NIH and Gates Foundation aim to change that, for example by identifying ways to selectively modify genes in hematopoietic stem cells.
The money will fund efforts to identify potential cures for both diseases for preclinical and clinical testing. Beyond that, the partners want to ally with organizations in Africa to collaborate on getting drugs into late-phase development. The NIH wants trials to be underway in the U.S and sub-Saharan Africa within seven to 10 years.