The National Institutes of Health announced on Oct. 23 it is launching a $100 million initiative over the next four years to fund research to develop gene-based cures for HIV and sickle cell disease.
The NIH announcement says the initiative will work in collaboration with the Bill and Melinda Gates Foundation, which will also invest $100 million toward gene research for an HIV and sickle cell disease cure.
“Dramatic advances in genetics over the last decade have made effective gene-based treatments a reality, including new treatments for blindness and certain types of leukemia,” an NIH statement says.
“The collaboration between the NIH and the Gates Foundation sets out a bold goal of advancing 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 ten years,” the statement says.
Dr. Anthony Fauci, Director of the National Institute of Allergies and Infectious Diseases, which is an arm of NIH, told the Blade the new initiative is aimed at taking gene research to a new level that has yet to be developed. According to Fauci, the goal is to develop a means of genetically altering disease fighting cells in the body to enable them to protect against HIV through a single injection.
He noted that the current line of research, which is nearing the stage of clinical trials on humans, involves withdrawing blood from the body, extracting disease fighting T-cells from the blood in a laboratory, genetically changing the cells to enable them to successfully kill HIV and prevent someone from being infected, and then to “reinfuse” the altered cells back into the person’s body.
This is a highly expensive process that requires hospitalization, Fauci said, making it difficult to put in place for the large number of people who need it, especially populations in developing countries in Africa.
“We’re talking about something that is highly aspirational with an extremely high reward,” he said. “That is to develop a delivery system that you can essentially with one injection give it to a person and have that delivery system bring the appropriate gene editing tools to whatever cells you want to bring them to,” he said.
Fauci said this system, if it can be technologically achieved, would provide a genetic cure for HIV that could become available on a massive scale in the U.S. and developing countries throughout the world.
“Whether or not we’re going to succeed, we don’t know,” he said. “But we’re going to try.”
He said the combined $200 million from NIH and the Gates Foundation would be made available to qualified independent researchers or researchers affiliated with universities or with private companies working on gene editing technology.
One such company, American Gene Technologies of Rockville, Md., has developed a gene editing process for a possible HIV cure that it believes is ready for human testing. In an announcement last month, the company said it has submitted an Investigational New Drug document, or IND, to the U.S. Food and Drug Administration to obtain final approval for clinical trials on humans.
The company’s gene editing process involves extracting cells from the body, genetically editing the cells in a laboratory, and reinfusing them into the person’s body. Although this requires a hospital or clinic visit and isn’t the more advanced process that Fauci says NIH is now working toward, if successful, it would be the first-ever full cure for HIV through gene therapy.
“We are pleased to see the scientific community backing a strategy American Gene Technologies has been pursuing for many years,” said AGT spokesperson Norman Rogers. “Like the NIH, we believe a cure deliverable to developing nations is a critical goal, especially in the landscape of gene and cell therapy where the efficiencies are more than doubling every year as the costs halve,” he said. “In that environment, anything is possible.”