In a bold new approach ultimately aimed at trying to cure AIDS, scientists used genetic engineering in six patients to develop blood cells that are resistant to HIV, the virus that causes the disease.
It’s far too early to know whether this scientific first will prove to be a cure or even a new treatment. The research was meant to show only that, so far, it seems feasible and safe.
The concept was based on the astonishing case of an AIDS patient who seems to be cured after getting blood cells from a donor with natural immunity to HIV nearly four years ago in Berlin. Researchers are seeking a more practical way to achieve similar immunity using patients’ own blood cells.
The results announced Monday at a conference in Boston left experts cautiously excited.
“For the first time, people are beginning to think about a cure” as a real possibility, said Dr. John Zaia, head of the government panel that oversees gene therapy experiments. Even if the new approach doesn’t get rid of HIV completely, it may repair patients’ immune systems enough that they can control the virus and not need AIDS medicines — “what is called a functional cure,” he said.
Carl Dieffenbach, AIDS chief at the National Institute of Allergy and Infectious Diseases, agreed.
“We’re hopeful that this is sufficient to give the level of immune reconstitution similar to what was seen with the patient from Germany,” he said.
This is the first time researchers have deleted a human gene permanently and infused the altered cells back into patients. Other gene therapy attempts tried to add a gene or muffle the activity of one and have not worked against HIV.
The virus can damage the immune system for years before people develop symptoms and are said to have AIDS — acquired immune deficiency syndrome. The virus targets special immune system soldiers called T-cells. It usually enters these cells through a protein receptor, or “docking station,” called CCR5.
Some people (about 1 percent of whites, fewer of minorities) lack both copies of the CCR5 gene and are naturally resistant to HIV. One such person donated blood stem cells in 2007 to an American man living in Berlin who had leukemia and HIV.
The cell transplant appears to have cured both problems, but finding such donors for everyone with HIV is impossible, and transplants are medically risky.
So scientists wondered: Could a patient’s own cells be used to knock out the CCR5 gene and create resistance to HIV?
A California biotechnology company, Sangamo BioSciences Inc., makes a treatment that can cut DNA at precise locations and permanently “edit out” a gene.
Dr. Jacob Lalezari, director of Quest Clinical Research of San Francisco, led the first test of this procedure with the company and colleagues at the University of California in San Francisco and Los Angeles.
He warned that it would be “way overstated” to suggest that the results so far are a possible cure.