NIH, Gates Foundation Pledge $200 Million to Bring Gene Therapies to Patients Who Need Them Most

Some of the most cutting-edge — and effective — treatments in medicine are unaffordable to the majority of people who need them, with price tags sometimes exceeding $1 million. A new initiative from the National Institutes of Health (NIH) and the Bill & Melinda Gates Foundation is meant to change that unfortunate reality.

Over the next four years, the NIH and the Gates Foundation will each invest $100 million toward developing gene-based cures for sickle cell disease and HIV, with a special focus on making these treatments available to the patients who need them most, the NIH announced Wednesday. Both HIV and sickle cell disease — a group of inherited disorders that lead to abnormal red blood cell shape and function, potentially contributing to a number of possible complications — disproportionately affect those living in lower-income areas, namely Africa, and people of color, who in the U.S. are more likely than white Americans to struggle with poverty.

“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,” NIH Director Dr. Francis Collins said in the NIH statement. “We aim to go big or go home.”

Gene therapies, which alter genes in order to treat or prevent disease, have already yielded breakthroughs for highly difficult-to-treat conditions including blindness and leukemia, but these treatments have been inaccessible to those in lower-resource countries, the statement says. The new initiative will first focus on developing gene-based treatments for sickle cell disease and HIV, then shift attention to accessibility.

In terms of development, researchers have already zeroed in on potential pathways for gene-based treatments. For sickle cell disease, the statement says, a therapy could either correct the gene mutations that cause the disorder in the first place, or help the body achieve normal red blood cell function. For HIV, it could mean targeting infected DNA that lives inside a person’s cells, even if they are taking antiretroviral treatment.

Researchers are already working on potential cures for both sickle cell disease and HIV, but the new initiative could mean any breakthroughs that are achieved reach a much broader swath of the population. That said, the challenges of both developing and disseminating gene therapies are vast.

“I’m not going to lie,” Collins told reporters on Wednesday. “This is a bold goal.”