Americans have begun the first human trials of gene therapy to protect against infections such as HIV, for which no effective vaccine has yet been developed, writes the New York Times.
Research is conducted in several centers in the United States. Scientists have already had successful animal experiments demonstrating that artificial genes, when injected into the body, can produce antibodies to protect against infection. The first human trials will take several months, and the results will probably be known later this year.
The Scripps Research Institute researchers say they tested the gene therapy to protect against HIV in mice. They did this by using a harmless virus as a “genetic taxi”, which they injected into the muscles of rodents. The microorganism contained the gene for the antibody, it integrated into the DNA of the muscle cell and began to produce it. Mice became resistant to HIV infection.
Lead author of this study, immunologist Dr. Michael Farzan, said synthetic genes could be used to protect against various infections such as malaria, ebola, influenza and viral hepatitis. They do not modify the immune system, but are complementary to it.
The genes smuggled into the body produce antibodies on their own, while vaccines stimulate the immune system to produce them. They contain dead or inactivated microorganisms or only the appropriate proteins stimulate immune cells to function.
Dr. Farzan calls the new method immunoprophylaxis. He claims that it can provide long-term immunity to diseases against which, despite many attempts, no effective vaccine has yet been developed. It is mainly about HIV and malaria.
The 1975 Nobel Prize winner Dr. David Baltimore, a Caltech virologist, and Gary W. Ketner, a microbiologist at the Johns Hopkins Bloomberg School of Public Health, have already obtained a protein to protect against mosquito-borne malaria. In August 2014, both researchers reported that when the gene that encoded them, they injected mice with the virus, as much as 80 percent of them. these animals were immune to infection. Previously, this could not be done with any conventional vaccine.
Dr. Gary J. Nabel, research supervisor at the pharmaceutical company Sanofi, believes the new method could even revolutionize germ protection. It would be especially useful when some new epidemic breaks out.
Other researchers, however, are more reserved. They believe that the method must first be shown to be effective and safe in humans. The human immune system can attack viruses that smuggle artificial genes, as well as the proteins they produce. And even if they don’t, muscle cells may not be making enough of them.
University of Pennsylvania virologist Dr. Philip R. Johnson tested the new method in monkeys. He showed that by introducing a gene encoding an appropriate protein into their muscles, they become resistant to the SIV virus (related to HIV, which attacks people). It was produced in sufficient quantities to prevent contamination.
At the same university, Dr. James M. Wilson conducts similar research. Initially, he dealt with the use of gene therapy in the treatment of cystic fibrosis, a genetic disease manifested, among others, by excessive production of sticky mucus in the lungs. Then he started using this method in infections of the upper respiratory tract.
An American specialist has developed synthetic genes encoding proteins that protect against the influenza virus. They effectively protected mice and ferrets against various strains of this microorganism. Now he’s working on gene therapy to protect against the Ebola virus.
Dr. Johnson began his first clinical trials in February 2015. His primary goal is to demonstrate that HIV-protective gene therapy is safe in humans. The first phase of these tests will last until June this year. We are optimists, full of hope – emphasizes the specialist. (PAP)