The Scripps Research Institute on Wednesday published details about the potential drug, touted as a vaccine alternative. Dr. Michael Farzan, a professor of infectious diseases at TSRI’s Florida campus, said he expects his new drug candidate could cure the 35 million people living with HIV worldwide within the next few years.
“Most people looking at data will appreciate the potential of a new drug candidate,” Farzan said.
This discovery is the culmination of scientists’ continuous work on the biochemistry of how HIV enters cells, he said.
An estimated 39 million people have died worldwide from HIV-related causes, and while treatments have improved, a cure has remained elusive for decades.
Farzan said this new study was based on his previous discovery of a co-receptor — the part of a cell that HIV binds to — which produces a protein that hinders the entry of the virus into the cells. He said his team developed a drug based on that protein that can help prevent infection.
They also created a way to inject the drug into muscle tissue and allow the body to produce the new protective protein for years. Farzan is expecting the widespread use of gene therapy to help treat a number of conditions in addition to HIV and AIDS.
But Dr. David Margolis, a UNC professor of medicine and leader of the Collaboratory of AIDS Researchers for Eradication, cast doubt on several of the study’s findings: whether it will be as effective for humans as it was for monkeys, whether the protection will last long enough and whether human cells will react abnormally to the proteins.
Margolis cited an example from UNC’s Gene Therapy Center, where researchers are studying treatments based on similar methods of transmitting protective proteins.
“This is not novel and has been tried in the lab for decades,” he said.
The monkeys used in the clinical test were completely protected from extremely high doses of viruses. Still, Farzan acknowledged that a substantial amount of time would be needed to conduct human trials of his drug.
“Everybody, including ourselves, wants to be sure that it is very safe,” Farzan said.
Margolis said the issue is whether these types of treatments can successfully be used to treat humans.
“I don’t know if it can be put into human trials even, but I hope it becomes successful,” he added.
Farzan said he is working on ensuring the safety of his new drug.
“I have a goal to initiate very basic and very safe human trials of protein alone in early 2016,” he said.