Graves' Disease Therapy Found to Safely Reduce Symptom Severity in Mid-Stage Study

One million people diagnosed with Graves’s eye disease, also known as thyroid eye disease (TED), could soon have some relief for their condition.

Researchers from the University of Michigan Kellogg Eye Center conducted a randomized, multi-center clinical trial where they tested the benefits of a drug called teprotumumab against placebo on 88 patients diagnosed with TED.

TED is a rare disorder associated with Graves’ disease, an autoimmune condition that induces hyperthyroidism. Symptoms can include eyelid swelling or retraction, double vision, bulging eyes, and constant stare.

“The eyes are particularly susceptible to Graves’ disease because the autoimmune attack often targets the eye muscles and connective tissue surrounding the eye,” Raymond Douglas, M.D., Ph.D., a Graves’ eye disease specialist and an oculoplastics surgeons, who helped lead this study, said in a statement. “This occurs as a consequence of these tissues containing proteins that are shared with the thyroid gland.”

Investigators administered the drug to the control group through intravenous injections over 24 weeks.

Results indicated that 69 percent of the study patients receiving these infusions once every three weeks experienced reduced eye bulging, improved vision, and increased quality of life compared to 20 percent in the placebo arm.

Also, a reported 43 percent of these patients started showing improvements within six weeks of receiving treatment versus four percent taking placebo.

The Food and Drug Administration originally granted teprotumumab a breakthrough designation as a treatment for cancer.

It’s a monoclonal antibody that inhibits the function of an insulin-like growth factor 1 receptor (IGF-1), which the researchers feel plays an integral role in Graves’ disease and other autoimmune disorders.

“Specifically, we have found that there is an unusually high presence of IGF-1 receptors on cells surrounding the eye in TED,” said lead study author and endocrinologist Terry Smith, M.D., in a statement.

“The findings have implications for other autoimmune diseases in which IGF-1 reception has already been implicated such as rheumatoid arthritis,” continued Smith.

The results provide the first step in establishing a therapy to safely minimize the severity of the eye condition, but the drug cannot be prescribed to TED patients unless they are participating in a clinical trial. A follow-up trial is underway though and is expected to be completed in 2018.

These study results were published in the New England Journal of Medicine.