HIV Drug Shows Promise for Diabetes Vision Loss

An inexpensive HIV medication that costs as little as $20 per month could offer new hope for millions of people with diabetic eye disease, according to results from a clinical trial in Brazil.

The drug lamivudine improved vision in patients with diabetic macular edema—a leading cause of blindness in diabetes—more effectively than existing treatments that can cost up to $2,000 monthly and require painful injections directly into the eye.

The condition affects approximately 1 in 14 people with diabetes, threatening the sight of millions among the 37 million American adults living with the disease. Current treatments involve monthly eye injections that carry risks of infection and retinal detachment.

Oral Alternative to Eye Injections

“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 researcher Jayakrishna Ambati, MD, founding director of UVA Health’s Center for Advanced Vision Science. “The mechanism of action of lamivudine is also different from that of existing treatments, so we could also develop combination therapies.”

Diabetic macular edema occurs when fluid builds up in the retina, the light-sensitive tissue at the back of the eye. This swelling distorts vision and can lead to permanent blindness if left untreated.

The randomized clinical trial, conducted at Brazil’s Universidade Federal de São Paulo, enrolled 24 adults with the condition. Participants received either lamivudine or a placebo pill for eight weeks, with eye injections of the drug bevacizumab added after four weeks.

Dramatic Vision Improvements

The results were striking. Patients taking lamivudine alone improved their ability to read letters on an eye chart by 9.8 letters—about two full lines—after just four weeks. Meanwhile, those receiving placebo actually lost 1.8 letters of vision during the same period.

When combined with bevacizumab injections, the improvements became even more pronounced. After eight weeks, lamivudine recipients had gained 16.9 letters of vision—more than three lines on the eye chart. The placebo group receiving only bevacizumab injections improved by just 5.3 letters.

These improvements suggest lamivudine works both independently and in combination with existing treatments, though larger studies will be needed to confirm the findings.

Global Health Game Changer

The cost difference could be transformative for healthcare systems worldwide. Current anti-VEGF treatments like aflibercept and ranibizumab rank among the top five drugs in Medicare spending, with monthly costs reaching $2,000 per injection.

“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 noted.

The implications extend far beyond cost savings. Many patients in developing countries lack access to ophthalmologists capable of performing monthly eye injections. An oral medication could make treatment accessible to millions more patients globally.

Key Study Results:

• Lamivudine alone improved vision by 9.8 letters after 4 weeks
• Combined with bevacizumab, improvement reached 16.9 letters after 8 weeks
• No significant difference in adverse events between treatment groups
• Monthly cost potential of $20 versus $2,000 for current treatments
• Oral administration eliminates injection-related complications

The Inflammasome Connection

What makes this discovery particularly intriguing is how lamivudine works differently from existing treatments. While current therapies target vascular leakage by blocking growth factors, lamivudine appears to protect retinal neurons and block inflammasomes—protein complexes in the immune system that normally detect infections but have been implicated in diabetic eye disease.

This dual mechanism addressing both neuronal dysfunction and vascular problems in diabetic retinopathy represents a fundamentally different therapeutic approach. The drug blocks purinergic receptor P2X7 pore formation and pathways that activate specific inflammasomes, all of which contribute to diabetic retinopathy progression.

The synthetic control analysis revealed another crucial finding: lamivudine performed as well as expensive treatments like aflibercept and significantly better than bevacizumab or ranibizumab when compared to historical data from major clinical trials. This suggests the HIV drug could match or exceed the performance of standard care at a fraction of the cost.

Beyond Diabetes

The discovery builds on Ambati’s previous research suggesting HIV drugs may have broader medical applications. His team recently found that these medications, called nucleoside reverse transcriptase inhibitors, can substantially reduce the risk of Alzheimer’s disease and may also lower diabetes and macular degeneration risk.

“We have developed a safer version of lamivudine called K9, which blocks inflammasomes without the potential side effects of lamivudine,” said Ambati, who serves as the DuPont Guerry III Professor in UVA’s Department of Ophthalmology. “So, we are excited by the ongoing and planned clinical trials of K9 in DME as well.”

This research exemplifies what Ambati calls “Big Data Archeology”—mining large healthcare databases to discover new uses for existing drugs. The approach has already yielded multiple promising leads for repurposing established medications.

What’s Next?

While the initial results are encouraging, researchers acknowledge limitations in their study. The trial was small and lasted only eight weeks, following patients for a relatively short period. Future studies will need to enroll larger numbers of participants and track them for longer periods to fully establish lamivudine’s safety and efficacy.

However, the researchers found encouraging signs that lamivudine’s benefits extended beyond the typical four-week window when most DME improvement occurs. Vision continued improving during weeks five through eight, suggesting sustained therapeutic effects.

For the millions of people facing progressive vision loss from diabetic eye disease, lamivudine represents more than just another treatment option—it could democratize access to effective therapy regardless of geography or economic status. Whether this HIV drug will ultimately transform diabetes care remains to be seen, but the early evidence suggests we may be witnessing the beginning of a new chapter in treating one of diabetes’s most devastating complications.