New Drug Cuts Relapse Risk 74% in Rare Eye Disease

Scientists just announced breakthrough results for treating neuromyelitis optica spectrum disorder, a devastating condition that attacks the eyes and spinal cord in about 22,000 Americans.

The disease mainly affects women and causes recurring episodes of vision loss and paralysis. Each relapse can leave patients with permanent disability, making effective treatment critical.

Researchers tested daratumumab, a drug already approved for blood cancer, in 135 patients across three years. Those receiving the treatment were 74% less likely to experience relapses than those on placebo. Nearly 70% of patients on daratumumab stayed relapse-free compared to just 15% on placebo.

Dr. Michael Levy from Harvard Medical School says the drug works differently than existing treatments. It targets CD38, a protein on the immune cells that produce the harmful antibodies attacking the nervous system. No current therapies use this approach.

The results showed dramatic differences in disability progression too. Only 6% of daratumumab patients got worse compared to 36% on placebo. Side effects were similar between both groups, suggesting the treatment is well tolerated.

The Bright Side

What makes this especially hopeful is that it offers an option for patients who haven't responded to current treatments or can't access them. Dr. Fu-Dong Shi from Beijing Tiantan Hospital notes the drug more selectively targets problem immune cells, potentially making it safer than alternatives.

The treatment requires IV infusions every two weeks initially, then monthly for maintenance. All patients also received low-dose steroids as a safety measure during the trial.

Dr. Benjamin Greenberg from UT Southwestern, who wasn't involved in the research, emphasized that more treatment options are needed for this rare disease. While several FDA-approved therapies exist, patients respond differently and some need alternatives.

The trial ran from November 2022 through March 2025, with patients followed for up to three years. Most common relapses during the study affected the spinal cord and optic nerves, the hallmark targets of the disease.

Researchers say they still need to determine ideal treatment duration and whether patients might eventually stop therapy while staying relapse-free.

For thousands of people living with this unpredictable disease, having another proven option represents real hope for preventing the attacks that steal their vision and mobility.