Repurposed Cancer Drug Shows Promise in Clearing HIV from Brain

According to Tulane University scientists, a cancer drug may hold the key to eliminating HIV from one of its most elusive hiding spots: the brain. Researchers at the Tulane National Primate Research Center have discovered that an experimental drug, originally developed for cancer treatment, significantly reduced levels of SIV (the nonhuman primate equivalent of HIV) in the brains of test subjects.

The study, published in Brain, marks a crucial step toward eradicating HIV from hard-to-reach reservoirs where the virus evades current treatments. Lead author Woong-Ki Kim emphasized the importance of this research, stating, "By specifically targeting the infected cells in the brain, we may be able to clear the virus from these hidden areas, which has been a major challenge in HIV treatment."

While antiretroviral therapy (ART) has transformed HIV from a terminal illness to a manageable condition, it cannot completely eliminate the virus from the body. The brain, protected by the blood-brain barrier, has been particularly challenging to treat, allowing the virus to persist in long-lived macrophages.

The researchers focused on these macrophages, using a small molecule inhibitor called BLZ945 to block a receptor that increases in HIV-infected cells. This approach successfully reduced viral load in the brain, effectively clearing the virus from brain tissue.

The study utilized three groups to model human HIV infection and treatment, with the high-dose treatment group showing a 95-99% decrease in viral DNA loads in the brain. Importantly, the treatment did not significantly impact essential microglia cells or show signs of liver toxicity.