In what could be a roadmap to the invention of the HIV virus, scientists have for the first time successfully eliminated HIV from the entire genome of lab mice using a slow-acting drug and gene-editing. The promising cure which could be the first giant step toward a cure for the nearly 37 million people living with the virus was revealed in Nature Communications on Tuesday.
In the study, researchers from Temple University's Lewis Katz School of Medicine and the University of Nebraska Medical Center (UNMC) coupled genome editing technology with a slow-release virus suppression drug to eliminate HIV cells entirely from some infected mice. A team spearheaded by an HIV expert in Nebraska and a gene-editing expert in Philadelphia has presented the unprecedented fruits of a five-year project: using a slow-acting drug called LASER ART that corners the virus, followed by CRISPR Cas9 gene-editing that blitzes it.
'We didn't believe it,' Dr Howard E Gendelman, Director of the Center for Neurodegenerative Diseases at the University of Nebraska Medical Center, told DailyMail.com.
'We thought it was a fluke, a problem with the graphs; that the cells carrying HIV had died; that our assay system was wrong.
'It was only after we repeated it a couple of different times,' he says, that they accepted they had hit the veritable jackpot.
What's more, journals didn't believe it.
'After we got it right, we submitted it for publication and it was rejected from many different journals,' Dr Gendelman said.
'They had a hard time believing HIV could be cured.'
He and his co-author (and 'best bud') Dr Kamel Khalili, of Temple University in Philadelphia, added no less than 20 supplemental figures to their paper - far more than usual - to prove that their results were not a fluke, finally getting the green light from Nature Communications.
'There was a lot of frustration, self-introspection, denials, reaffirmation, and just laborious day by day activities to prove it,' Dr Gendelman said.
HIV is so hard to obliterate because it is a virus that infects the genome. And while standard ART is a soluble drug that dissolves into the blood and gets to work, the drugs in LASER ART are packaged inside nanocrystals, which the immune system recognizes as foreign and carries it up to tissues where HIV hides.
In 2008, Timothy Ray Brown is an American considered to be the first person cured of HIV/AIDS. Hopes then swelled in March when a second person was announced to have been cured of the virus after a stem cell transplant successfully eliminated any trace of the virus from his blood.
The team now hopes to start the clinical trial in macaque monkeys in summer 2020. This is not yet a cure, but Dr Khalili and Dr Gendelman are bursting with relief and excitement.
'At the very least it's a proof of concept,' Dr Gendelman said. 'It shows it's possible that HIV could be cured.'