Philanthropy-funded research could improve life for thousands suffering from retinal disease

Shortly after his arrival in late 2021, Assistant Professor of Ophthalmology, Michael Telias, PhD, published an important discovery related to vision restoration.

In many blinding diseases, such as age-related macular degeneration and other hereditary retinal conditions, light gathering photoreceptor cells die – often over the course of years. This loss sets off a chain of events where the intact cells that normally transmit visual signals from the photoreceptors to the brain – called retinal ganglion cells (RGCs) – change. When RCGs undergo this transformation, they become less efficient at stimulating the brain. This accelerates vision loss. Instead of behaving as they should, the RCGs become hyperactive. This creates unwanted “noise” or “static” that limits visual information – still coming from surviving photoreceptors – from being received by the brain.

Telias discovered that if he could disrupt the remodeling process by inhibiting a common compound called retinoic acid (RA), the intact photoreceptors have a better chance of getting clear signals to the brain, thereby improving vision. To achieve this, he used Disulfiram, an already approved drug that is excellent in inhibiting RA. Better known as Antabuse, it is often prescribed in treating alcohol use disorder.

“To our delight, it proved effective,” Telias said. “But testing the drug to see if it can improve vision in people, is different. We needed further resources to design and conduct clinical experiments. It is a big jump going from the laboratory to the bedside.”

Telias presented his work to a number of local interests, including some of FEI’s Advisory board. This resulted in securing one-hundred thousand dollars each from the Glover-Crask Charitable Trust and an anonymous source. With funding in hand, Telias began a clinical study, which is still recruiting candidates.

“We are specifically looking for patients who are diagnosed with alcohol use disorder and have vision problems that are characterized by photoreceptor loss, like AMD,” Telias said. “This way we do not have to change the indication for the drug, which is alcoholism, so that we do not get slowed down by the regulatory process. The first patient has already been consented and is taking the drug.”

Success will be determined by measuring baseline best corrected visual acuity (BCVA). During the 120-day treatment, BCVA will be measured at intervals with investigators looking for improvement along the way. Telias said that this is not a complete clinical trial because study subjects and investigators both know that the patient is receiving the drug. In a true clinical trial, a number of patients would receive a placebo, and the investigators would not know which ones did until the end of the study.

“Still, it’s important,” Telias continued. “If we can generate data that shows an effect, then we can proceed to a full-blown clinical trial. This could be a game changer since the drug is well tolerated and already approved for other uses. It is not a cure for vision loss, but it could be a bridge to maintaining vision and improving peoples’ lives while we search for ways to restore sight.”

Read more about the study here.

1/6/2025