National Institutes of Health lab photo (Courtesy of NIH)
National Institutes of Health lab photo (Courtesy of NIH)

The National Institutes of Health (NIH) plans to invest at least $100 million over the next four years toward developing affordable, gene-based cures for sickle cell disease (SCD) and HIV.

The Bill & Melinda Gates Foundation will also invest $100 million toward this goal. The intention is for these cures to be made globally available, including in low-resource settings.

The collaboration between the NIH and the Gates Foundation sets forth a mission to advance safe, effective and durable gene-based cures to clinical trials in the United States and relevant countries in sub-Saharan Africa within the next seven to 10 years. The ultimate goal is to scale and implement these treatments globally in areas hardest hit by these diseases.

“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,” said NIH Director Francis S. Collins. “We aim to go big or go home.”

“In recent years, gene-based treatments have been groundbreaking for rare genetic disorders and infectious diseases,” said Dr. Trevor Mundel, president of the foundation’s Global Health Program. “While these treatments are exciting, people in low- and middle-income countries do not have access to these breakthroughs. By working with the NIH and scientists across Africa, we aim to ensure these approaches will improve the lives of those most in need and bring the incredible promise of gene therapy to the world of public health.”

SCD and HIV are major burdens on health in low-resource communities around the world. Approximately 95 percent of the 38 million people living with HIV globally are in the developing world, with 6 percent in sub-Saharan Africa, half of whom are living untreated. Fifteen million babies will be born with SCD globally over the next 30 years, with about 75 percent of those births occurring in sub- Saharan Africa. An estimated 50-90% of infants born with SCD in low-income countries will die before their 5th birthday and SCD is identified as the underlying cause of about 1 in 12 newborn deaths in sub-Saharan Africa.

The collaboration will focus on two areas of coordination. First, identify potential candidate cures for SCD and HIV for pre-clinical and clinical evaluation, co-funded by the NIH and Gates Foundation. Second, define long-term opportunities to work together and with African partners on advancing promising candidates to late-phase clinical trials, with funding to be determined as candidates progress.

Though SCD, a genetically inherited disease, and HIV, an acquired infectious disease, present significantly different scientific challenges, gene-based treatments hold promise for both, and many of the technical challenges for gene-based cures are expected to be common to both diseases.

“Our excitement around this partnership rests not only in its ability to leverage the expertise in two organizations to reduce childhood mortality rates in low resource countries, but to bring curative therapies for sickle cell disease and HIV to communities that have been severely burdened by these diseases for generations,” said Dr. Gary H. Gibbons, director of the NIH’s National Heart, Lung and Blood Institute. “A person’s health should not be limited by their geographic location, whether rural America or sub-Saharan Africa; harnessing the power of science is needed to transcend borders to improve health for all.”

Brenda Siler is an award-winning journalist and public relations strategist. Her communications career began in college as an advertising copywriter, a news reporter, public affairs producer/host and a...

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