New hope for those affected by thyroid eye disease

The newly approved drug teprotumumab can offer hope to adults with thyroid eye disease, a rare and potentially blinding condition.

It’s the first treatment specifically approved for thyroid eye disease. The drug was approved Tuesday, January 11, 2020 by the U.S. Food and Drug Administration. It will be marketed under the brand name Tepezza.

“This may be a drug that offers a new approach and a paradigm shift,” said Dr. Raymond Douglas, lead author of a study the FDA considered when making its final decision. “Currently, we use supportive therapy for people with thyroid eye disease, but their quality of life is diminished by their symptoms.

“Teprotumumab was extraordinarily effective. We hope that people with thyroid eye disease won’t have to suffer as they have in the past. With treatment, they’re not going to be blind. They’re not going to be disfigured. They can even watch their kids playing soccer when it’s windy,” Douglas explained. Without treatment, wind could be too drying to the eyes.

Thyroid eye disease causes inflammation in the back of the eye causing the eyes to bulge. The swelling may also squeeze the optic nerve, causing vision problems, said Douglas, a professor of surgery in the division of ophthalmology at Cedars-Sinai Medical Center in Los Angeles.

Thyroid eye disease typically develops in people with an overactive thyroid gland caused by Graves’ disease. Redness, swelling and a gritty feeling in the eyes are also classic symptoms, the American Thyroid Association says. Other conditions that can occur include dry eyes or excessive tearing, double vision and sensitivity to light. The most noticeable symptom, however, is the bulging of the eyes, and it may be difficult to completely close the eyes.

Because thyroid eye disease is rare, the FDA gave this drug “orphan drug” status. This means companies are provided incentives to encourage the development of drugs for rare diseases. The medication was also given the FDA’s priority review, fast track and breakthrough therapy designations to assist the manufacturer in bringing the drug to market.

The drug’s approval was based on results of two studies that included 170 people with active thyroid disease. The drug was administered intravenously once every week for three weeks over a 21-week period. Douglas said the IV infusions take about an hour.

Teprotumumab started working after just two doses, Douglas said. After 24 weeks, 83 percent of people on the drug had a measurable reduction in eye bulge — more than 2 millimeters — versus just 10 percent of those on a placebo.

The overall response rate was 78 percent among those taking the drug compared to 7 percent of people taking a placebo. Quality of life was much higher for people taking the drug, the study found.

Douglas said treatment with the drug reversed double vision as well as surgery does.

Side effects were minimal and well-tolerated, according to Douglas. Some of the more common side effects included muscle cramps, nausea, hair loss and fatigue. The drug should not be used in pregnancy.

Learn more about thyroid eye disease from the American Thyroid Association.

 To read the original article, click here (New drug may stop blindness from thyroid eye disease)

Eye exams may one day predict Alzheimer’s

One day in the not too distant future, it may be possible to screen patients for Alzheimer’s disease using an eye exam.

By using technology similar to what is already found in many eye doctors’ offices, researchers at Washington University School of Medicine in St. Louis have detected evidence suggesting Alzheimer’s in older patients who had no symptoms of the disease.

“This technique has great potential to become a screening tool that helps decide who should undergo more expensive and invasive testing for Alzheimer’s disease prior to the appearance of clinical symptoms,” said the study’s first author, Bliss E. O’Bryhim, MD, PhD,. “Our hope is to use this technique to understand who is accumulating abnormal proteins in the brain that may lead them to develop Alzheimer’s.”

Substantial brain damage from Alzheimer’s disease can occur years before any symptoms such as memory loss and cognitive decline appear. Scientists estimate that Alzheimer’s-related plaques can build up in the brain two decades before the onset of symptoms, so researchers have been looking for ways to detect the disease sooner.

Physicians now use PET scans and lumbar punctures to help diagnose Alzheimer’s, but they are both expensive and invasive.

Previous studies that involved examining the eyes of people who had died from Alzheimer’s had reported that the eyes of such patients showed signs of thinning in the center of the retina and degradation of the optic nerve.

In the new study, the researchers used a noninvasive technique — called optical coherence tomography angiography — to examine the retinas in eyes of 30 study participants with an average age in the mid 70s, none of whom exhibited clinical symptoms of Alzheimer’s.

Those participants were patients in The Memory and Aging Project at Washington University’s Knight Alzheimer’s Disease Research Center

“In the patients with elevated levels of amyloid or tau, we detected significant thinning in the center of the retina,” said one of the researchers “All of us have a small area devoid of blood vessels in the center of our retinas that is responsible for our most precise vision. We found that this zone lacking blood vessels was significantly enlarged in people with preclinical Alzheimer’s disease.”

The eye test used in the study shines light into the eye, allowing a doctor to measure retinal thickness, as well as the thickness of fibers in the optic nerve. A form of that test often is currently available in some optometrist and most ophthalmologist’s offices. In fact, the Mettawa office of Visibly Better Eye Care has the OCT machine needed to perform this test, but as of yet they are not offering this service.

For purpose of this study, however, the researchers added a new component to the more common test: angiography, which allows doctors to distinguish red blood cells from other tissue in the retina.

“The angiography component allows us to look at blood-flow patterns,” said the other co-principal investigator said. “In the patients whose PET scans and cerebrospinal fluid showed preclinical Alzheimer’s, the area at the center of the retina without blood vessels was significantly larger, suggesting less blood flow.”

“The retina and central nervous system are so interconnected that changes in the brain could be reflected in cells in the retina.”

Of the patients studied, 17 had abnormal PET scans and/or lumbar punctures, and all of them also had retinal thinning and significant areas without blood vessels in the centers of their retinas. The retinas appeared normal in the patients whose PET scans and lumbar punctures were within the typical range.

“We know the pathology of Alzheimer’s disease starts to develop years before symptoms appear, but if we could use this eye test to notice when the pathology is beginning, it may be possible one day to start treatments sooner to delay further damage,” one of the researchers said.

To read the original article in its entirety, click here. https://www.sciencedaily.com/releases/2018/08/180823140921.htm