Not long ago, a biological company called Excision BioTherapeutics publicly announced that in its Phase 1/2 clinical trial of EBT-101, the first subject had officially started receiving HIV gene therapy.
The subject, who was dosed in July 2022, will continue to be monitored for safety and is expected to be eligible for an analytical treatment interruption (ATI) on background antiretroviral therapy (ART) in the evaluation for a potential cure.
The announcement came on the heels of the California Institute for Regenerative Medicine (CIRM) announcing a $6.85 million grant from the biocompany to support the clinical development of EBT-101.
We all know HIV's current treatment: To insist on taking medicine for life.
HIV has long been a well-known disease in the medical world. And the development of antiretroviral therapy (ART), which dates back to the 1860s, has saved countless lives stricken by HIV.
ART makes the diagnosis of AIDS no longer a death sentence. Some infected people can achieve "long-term remission", that is, live like normal people.
HIV borrows the concept of "long-term remission" from leukemia and lymphoma treatment, which is a functional cure. That is to say, the virus is not eradicated, but the virus database is maintained at a low level.
Let the body's immune system clear the remaining virus, or achieve a balance so that the virus does not rebound.
A more informal explanation is that the HIV virus synthesizes DNA by reverse transcription, which is integrated into resting and activated lymphocytes and macrophages.
Viral DNA within cells can stably persist in a dormant-like state under the influence of immune responses and antiretroviral therapy.
Tissues and cells with similar functions are called HIV reservoirs.
It is also because of the existence of the virus reservoir that ART cannot permanently eliminate the virus and cure the disease caused by the viral infection.
HIV-infected individuals may experience a viral rebound in the weeks following ART discontinuation.
If the problem of the virus repository can be solved, this problem may be solved.
Now EBT-101, if it is successful will achieve a one-time cure?
The news of the "AIDS cure" is no stranger to everyone.
From the first "Berlin patient" to the second "London patient", two patients suffering from AIDS and hematological tumors received hematopoietic stem cell transplantation from CCR5-delta32 donors, not only cured their tumors, HIV was no longer found in the blood.
According to the academic point of view, CCR5 is the main receptor for the HIV virus to invade T cells, and people with the CCR-delta32 mutation can be immune to the CCR5-tropic HIV virus.
However, the high cost, high mortality, and strict indications of hematopoietic stem cell transplantation have prevented this method from becoming a mainstream treatment for AIDS.
And EBT-101 seems to represent a more universal possibility:
EBT-101 is a technology that snips out the provirus on the HIV reservoir.
These magic scissors are the CRISPR-Cas 9 gene editing technology. It is an adeno-associated virus (AAV) that delivers the scissors.
Based on long-term nonhuman primate safety data and efficacy data in humanized mice, this therapeutic technique has the potential to cure HIV.
A functional cure for HIV would have a huge impact on these communities and others around the world.
From a longer-term perspective, the research enthusiasm for gene editing technology is a good thing.
However, how to explore compliant new treatments based on existing research is what contemporary clinicians continue to do.
