Long-Approved HIV Drug Yields Positive Results in Treating Diabetic Macular Edema

According to recent research from Brazil, lamivudine, an inexpensive and long-approved oral drug for HIV, may effectively mitigate diabetic macular edema (DME), offering an alternative to monthly intravitreal injections.1

Lamivudine is a first-generation nucleoside reverse transcriptase inhibitor (NRTI), approved in 1995 for HIV-1 infection and in 1998 for hepatitis B virus. Since 2002, lamivudine has been the primary recommendation as first- and second-line antiretroviral therapy for adults and children. Lamivudine is administered orally, with maximum serum concentrations reached between 0.5 and 1.5 hours after dosing.2

“An oral drug that improves vision in DME would be a game changer because it would be more convenient for patients than frequent, often monthly, injections into the eye,” said Jayakrishna Ambati, MD, founding director of UVA Health’s Center for Advanced Vision Science and DuPont Guerry III Professor in the UVA School of Medicine’s Department of Ophthalmology. “The mechanism of action of lamivudine is also different from that of existing treatments, so we could also develop combination therapies.”1

A total of 24 participants with DME were included in the trial and randomly assigned to receive either lamivudine or placebo, along with intravitreal injections of bevacizumab after 4 weeks. Over the course of the trial, patients receiving lamivudine exhibited significant vision improvement even before first injection. Their ability to read letters on an eye chart improved by 9.8 letters, or roughly 2 lines, at 4 weeks; participants receiving placebo saw a decrease of 1.8 letters.1

A month after bevacizumab injections, the lamivudine subgroup saw a 16.9 letter improvement, while the placebo subgroup saw a 5.3 letter improvement, attributed to the bevacizumab injections. Investigators stated that these data indicate lamivudine’s efficacy both alone and in conjunction with bevacizumab, although they caution that further research is needed to confirm.1

Ambati and colleagues said they believe that lamivudine is most likely effective against DME because it blocks inflammasome activity. Despite their importance as infection sensors, inflammasomes have been cited as a core cause of DME development.1

The team also notes that future trials will require larger number of patients and a longer timeframe. However, the results are still encouraging, especially given the fact that vision improved not only during the first 4 weeks – when the most improvement is typically seen in DME – but in the following 4 weeks as well.1

Regardless of its potential in combination therapy, findings indicate lamivudine on its own may be lifechanging for patients in areas with limited access to specialty doctors or who cannot travel to monthly eye appointments.1

“A $20-a-month or even cheaper oral pill that improves vision as much as or more than therapy with injections into the eye that cost up to $2,000 per month could be transformative both for patients and the health care system,” Ambati said.1

References

1. University of Virginia Health System. HIV drug can improve vision in patients with common diabetes complication, clinical trial suggests. Eurekalert! May 27, 2025. Accessed June 4, 2025. https://www.eurekalert.org/news-releases/1085125

2. Quercia R, Perno CF, Koteff J, et al. Twenty-Five Years of Lamivudine: Current and Future Use for the Treatment of HIV-1 Infection. J Acquir Immune Defic Syndr. 2018;78(2):125-135. doi:10.1097/QAI.0000000000001660