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URMC / Clinical & Translational Science Institute / Stories / August 2017 / Do the Drugs that Keep HIV Patients Alive Damage Their Brains?

Do the Drugs that Keep HIV Patients Alive Damage Their Brains?

Graphic of a man with a lot on his mindResearchers from the University of Rochester Medical Center recently set out to understand whether drugs used to keep HIV patients alive could be damaging their brains.  On the contrary, early results from their clinical study showed that short-term use of combination anti-retroviral therapy (cART) improved mental function in HIV-infected individuals. 

Giovanni Schifitto, M.D., M.S., professor of Neurology at the University of Rochester Medical Center, is leading the study to better understand the short and long term effects of combination antiretroviral therapy on HIV patients’ brains. At 12 weeks, the therapy appears to improve mental performance and functional connectivity in the brain.

HIV patients often experience mental decline ranging from mild impairment to full-blown dementia. Experts have long debated the cause of that mental decline: HIV itself, or the drug used to combat it.

Some of the first HIV drugs were known to cause damage to peripheral nerves. Newer anti-retroviral drugs are believed to be safer, but patients taking these drugs continue to experience mental impairment - even when their viral load is extremely low.  In fact, some studies have shown improvement in HIV patients’ mental function when they stop using cART.

“But those studies were very indirect,” said Schifitto, who is also the director of the Clinical Research Center and function leader for Participant and Clinical Interactions at the CTSI. “They studied cohorts of people who were already on medications, which makes it very hard to pull apart whether the virus or the drug is to blame for effects in the brain.”

Schifitto’s clinical study, on the other hand, followed 17 HIV-infected individuals who had not received any treatment prior to the study. These patients scored worse on mental function tests and brain imaging revealed fewer connections in their brains than the HIV-negative control group.

After receiving cART for 12 weeks, the HIV patients’ mental performance and functional brain connectivity improved nearly to the level of the HIV-negative group. This not only suggests that short term cART use does not damage the brain, but that the virus is the culprit for early mental impairment in HIV-infected patients.

However, this is just a first step of the study, which will enroll and follow over 150 participants for two years. It is possible that cART will cause mental decline after prolonged use and the team want to track if and when that happens.  They are also monitoring sleep, mood, and several other factors that can impact mental function in HIV patients taking cART. 

In the end, the outcomes of the short and long term studies may help health care providers tailor cART cocktails and treatment schedules to individual patients’ needs. The results could also have implications for preventative use of cART in individuals who are at high risk for contracting HIV, a practice called pre-exposure prophylaxis (PrEP).

The study started with just a single site at the University of Rochester Medical Center, but now includes sites at Cornell Medical Imaging Center, Gay Men’s Health Crisis, SUNY Upstate Medical, University at Buffalo, and University of Texas Health Science Center at Houston.  It also utilized the CTSI’s Clinical Research Center, a dedicated space for researchers to conduct safe and controlled clinical studies with the support of highly trained clinical research staff.


The Clinical Research Center is supported by the University of Rochester CTSA award number UL1TR002001 from the National Center for Advancing Translational Sciences of the National Institutes of Health.

Read the full study, here.

Michael Hazard | 8/31/2017

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