HIV drug lamivudine holds potential for patients with DME, researchers find

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Researchers have discovered that a long-approved HIV drug has the potential to improve vision in patients with diabetic macular edema (DME).¹ Researcher Jayakrishna Ambati, MD, founding director of UVA Health’s Center for Advanced Vision Science, alongside collaborators at Brazil’s Universidade Federal de São Paulo led by Dr. Felipe Pereira and Dr. Eduardo Buchele Rodrigues, found that lamivudine could provide an oral option to more effectively treat DME at a lower cost to patients, according to a news release.

“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 Ambati in the release.

Researchers enrolled 24 participants with DME in a small randomized clinical trial, which randomly assigned said participants to receive either lamivudine or placebo twice daily, in addition to eye injections of drug bevacizumab starting after 4 weeks of treatment. Those who received lamivudine showed significant vision improvements in those first 4 weeks before their first eye injections. These patients’ ability to read letters on an eye chart improved by 9.8 letters at 4 weeks, while patients receiving placebo saw their ability decrease by 1.8 letters. Additionally, a month after receiving bevacizumab injections, lamivudine recipients improved by 16.9 letters, with the placebo group increasing by 5.3. Thus, results from the study suggest that lamivudine may work both alone and in conjunction with bevacizumab injections.¹

While larger studies that follow patients for longer than 8 weeks will be needed to reinforce these results, Ambati said in the release that lamivudine alone could make a lifechanging impact for patients in areas of the world that have limited access to specialty doctors or who are unable to afford or 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 in the release.

Researchers hypothesize that lamivudine was effective in the clinical trial against DME because the drug blocks the activity of inflammasomes, which can be implicated in the development of DME.¹

“The mechanism of action of lamivudine is different from that of existing treatments, so we could also develop combination therapies. This trial demonstrates that blocking inflammasomes can improve vision in DME. We have developed a safer version of lamivudine called K9, which blocks inflammasomes without the potential side effects of lamivudine,” said Ambati in the release. “So, we are excited by the ongoing and planned clinical trials of K9 in DME as well.”

The trial was supported by grants from the National Institutes of Health’s National Eye Institute and National Institute on Aging.¹

Reference:

1. Drug improves sight for diabetic macular edema patients. University of Virginia Health. May 27, 2025. Accessed June 10, 2025. https://newsroom.uvahealth.com/2025/05/27/drug-improves-sight-for-diabetic-macular-edema-patients/