An inexpensive, orally administered drug commonly used too treat HIV, lamivudine, may offer a new approach to managing diabetic macular edema (DME), according to recent research from Brazil. This development could perhaps reduce the need for frequent intravitreal injections, a standard treatment for DME.

Jayakrishna Ambati, MD
Image Credit: UVA Health

Lamivudine: A Repurposed Antiretroviral

Lamivudine, a first-generation nucleoside reverse transcriptase inhibitor (NRTI), received approval for HIV-1 infection in 1995 and for hepatitis B virus in 1998. Since 2002,it has been a primary advice for first- and second-line antiretroviral therapy for both adults and children. The drug is administered orally, reaching maximum serum concentrations within 0.5 to 1.5 hours after dosing.

Expert Insight

Dr. Jayakrishna Ambati, 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, emphasized the potential impact of this finding:

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.
Jayakrishna Ambati, MD, UVA Health

He further noted the potential for combination therapies, stating, The mechanism of action of lamivudine is also diffrent from that of existing treatments, so we could also develop combination therapies.

Clinical Trial Results

The clinical trial involved 24 participants with DME, randomly assigned to receive either lamivudine or a placebo. All participants also received intravitreal injections of bevacizumab after the initial four weeks. Key findings include:

• Patients receiving lamivudine showed significant vision improvement even before the bevacizumab injections.
• At four weeks, the lamivudine group improved by an average of 9.8 letters on an eye chart, equivalent to roughly two lines.
• The placebo group experienced a decrease of 1.8 letters.
• One month after bevacizumab injections, the lamivudine group saw a 16.9 letter improvement, compared to a 5.3 letter improvement in the placebo group.

Investigators suggest that these results indicate lamivudine’s efficacy both as a standalone treatment and in conjunction with bevacizumab. However, they caution that further research is necessary to confirm these findings.

Mechanism of Action

Dr. Ambati and his colleagues believe that lamivudine’s effectiveness against DME stems from its ability to block inflammasome activity. Inflammasomes, while crucial as infection sensors, have been identified as a key factor in the development of DME.

Future Research

The research team acknowledges the need for future trials involving larger patient populations and longer timeframes. Despite these limitations, the initial results are encouraging, notably given that vision improved not only during the first four weeks but also in the subsequent four weeks.

Potential Impact

Regardless of its potential in combination therapy, lamivudine alone could significantly improve the lives of patients in areas with limited access to specialized medical care or those who cannot attend monthly eye appointments.

Dr. Ambati highlighted the potential cost-effectiveness of lamivudine:

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.
Jayakrishna Ambati, MD, UVA Health

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