NIH Aiming to Use ‘Gene Therapy’ to ‘Treat’ Babies’ Immune Systems

In a new video posted to YouTube National Institutes of Health (NIH) Staff Scientist Nisha Nagarsheth describes how the agency aims to use “gene therapy” to modify and then re-implant babies’ stem cells.

In a new video a staff scientist from the NIH—the federal agency encompassing Anthony Fauci’s NIAID, the National Cancer Institute (NCI), etc.—describes how she and her colleagues plan to use “gene therapy” to “treat” babies with defective immune systems. The specifics aren’t clear as of this writing, but the general idea is to take a baby’s stem cells from his or her bone marrow, genetically modify the cells to “fix” immune defects, and then re-insert those modified cells back into the baby.

In the NIH’s “SciBites” video (above) Staff Scientist Nisha Nagarsheth offers a brief overview of how the agency plans to use gene therapy to modify babies’ DNA. Nagarsheth, who graduated from the University of Michigan with her PhD in Immunology in April 2017 and is “excited by the power of the immune system”—begins by talking about how the human immune system is like “an army, ready to fight any invader at any time.” Nagarsheth notes the immune system fights off viruses, bacteria, and even cancer.

Nagarsheth goes on to note, however, that “some babies are born with defects in all or parts of their immune system.” She adds that these babies “can be very sick at a very young age, and can even die of simple infections that most people can easily fend off.”

To solve the problem, Nagarsheth describes “a new treatment for these disorders, called gene therapy.” The Staff Scientist goes on to say that:

“[I]n gene therapy, we first take the baby’s blood stem cells from the blood or bone marrow; and these cells are special because they can morph into all types of immune cells that the baby needs to survive. We then add the normal version of the faulty gene that leads to the defect in the baby’s immune system. Finally, we take those modified stem cells and we re-infuse them into the baby in the hopes that they will transform into healthy immune cells that healthy adults have.”

Unfortunately, Nagarsheth doesn’t go into much detail regarding how, exactly, the NIH is going to modify babies’ genomes. One would assume the agency will deploy CRISPR technology—i.e., they’ll use a bacterium’s genome-editing ability (relying on Cas9 enzymes to do the genetic clipping) that’s usually used to memorize and then defend against viral invaders, inside of human cells—but the method is unclear. (First utilized for modification of human embryos in 2015 by Chinese researchers, CRISPR seems like the most likely candidate for the necessary tech.)

Despite the fact the NIH links to “Nisha’s Lab,” the link actually goes to the lab headed by NIH Senior Investigator Sung-Yun Pai. Pai, who began working at the NIH in 2021 and is with the agency’s Immune Deficiency Cellular Therapy Program, said in an April 8, 2021 tweet that she was looking for post-doctoral fellows “passionate about inborn errors of immunity & their treatment with transplantation & gene therapy.”

In Pai’s tweet, she actually links to a jobs board on the NIH’s Center for Cancer Research (CCR) site. Indeed, in a March, 2018 video outlining “New paths to cure cancer” at the NIH, the AFP News Agency noted that “Doctors [at NIH] have used immunotherapy to target tumors caused by human papillomavirus (HPV).” Dr. Christian Hinrichs, a Lead Investigator at the CCR who’s featured in AFP’s video, is focused on “immunotherapy for HPV+ cancers” and “discovered personalized cellular and gene therapies for HPV+ cancers.”

In the video immediately above Hinrichs describes how he’s developing T-cell therapies for HPV. “We take immune cells from the patient, make them target cancers in the laboratory and return them to the patient to treat the cancer,” Hinrichs says in the video. “This is highly personalized approach in which the cancer treatment is made from the patient’s own cells,” he adds.

Hinrichs says in the video the treatment has already been shown as effective in “certain blood cancers,” although in a study published in the peer-reviewed journal Biologics: Targets & Therapy, author Tafere Mulaw Belete at the University of Gondar in Ethiopia concluded, in part, that “Gene therapy for cancer treatment has good progress in the last three decades, [but with] few drugs approved… .” Gene therapy as a field, perhaps not incidentally, is commonly thought to have begun in the early ’90s.

Feature image: National Institutes of Health (NIH)

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