Maryland researchers study whether HIV cure can come from infusing patients with genetically modified ‘super T cells’

A week ago, a Washington, D.C., man in his 30s with HIV became the first person to be infused with a heaping load of his own genetically modified white blood cells that a Maryland biotech firm believes one day could lead to the elusive cure for the disease.

The company, Rockville-based American Gene Technologies, announced the infusion Thursday, the first of an initial six people who will receive the so-called “super T cells” in coming months.


It’s an early step in a trial primarily aimed to test for safety. But officials say it marks the next phase in years of study building on their own and others’ research into how to keep the virus from killing T cells, a type of white blood cell that normally fight off infections.

“There is no guarantee, of course,” said Jeff Galvin, the company’s founder and CEO. “But there are a lot of things happening to give people hope that this product may be the functional cure or a subsequent product may be the cure.”


Almost 38 million people globally and 1.1 million Americans live with HIV, or human immunodeficiency virus, which if left untreated can lead to AIDS, or acquired immunodeficiency syndrome, and a host of health maladies.

There are dozens of studies underway across the globe deploying a variety of technologies to cure or better control chronic HIV infections without people having to take daily antiretroviral drugs that can come with side effects and fear of uncontrolled illness.

American Gene Technologies’ method involves taking T cells out of a person’s blood and genetically modifying them in the lab to resist infection before they are reinfused.

The company’s chief science officer, C. David Pauza, is a former professor and researcher at the University of Maryland School of Medicine’s Institute for Human Virology.

He said the company’s beginning point — fixing T cells— isn’t new. Past efforts were not powerful enough or long-lasting enough to be considered a functional cure, where people can live disease-free indefinitely with medications even if the virus still lingers in their bodies.

Pauza said researchers enhanced the process of modifying the T cells to make them better able to fend off HIV. The researchers also sought a way to enhance the cells so the virus couldn’t return and successfully attack the cells again later.

In a photo provided by NIAID, a colored scanning electron micrograph of a human T-cell, blue and green, infected with H.I.V., yellow.  (NIAID via The New York Times)

The company’s researchers worked with federal infectious disease researchers to cooperatively assess the method before the U.S. Food and Drug Administration allowed the first human study to start.

“HIV shuts off the immune response; it’s insidious,” Pauza said. “We amped up the response massively. That’s really the new twist.”


The FDA calls for participants to be followed for 15 years, with the first check for safety in July. If the cells don’t cause any concerns, the participants could enroll sometime after that in another study phase where they stop taking their medications to see whether the cells actually stop infections.

The first patient was infused May 19 at a northeast D.C. treatment center called the Washington Health Institute. Dr. Jose Bordon, the study’s principal investigator at the site, said the man has shown no side effects.

The Institute for Human Virology in Baltimore is evaluating whether to enter the study, confirmed its co-founder and director, Dr. Robert Gallo, who is internationally regarded for his role in discovering HIV and developing a blood test to detect it.

Gallo said many people are working toward the same goals, though he cautioned that HIV research has been a tough road with several promising but disappointing outcomes.

“Ultimately we want something we can deliver globally, simply, inexpensively and will have no side effects,” he said.

“Those are ideals,” Gallo added. “There are numerous approaches throughout many places around the world. This is a new, rational and interesting concept.”


It’s not possible to say how well any method will work based on early studies, said Dr. David Margolis, an HIV researcher not associated with the company ahead of Thursday’s announcement.

“HIV Cure research is now extremely broad and deep,” said Margolis, head of University of North Carolina’s HIV Cure Center. “But the timeline for success is likely to be many more years, like the search for cures for cancer and other challenging diseases.”