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Proteomics allows researchers to identify existing drugs to treat rare eye disease

By identifying abnormally expressed proteins in the eye, a Stanford-led team matched existing drugs with these proteins to quell patients' symptoms.

In the past, treatment for a very rare genetic eye condition called neovascular inflammatory vitreoretinopathy, or NIV, was trial-and-error, and usually not very successful. For example, if a patient experienced an internal eye bleed, or hemorrhage, which totally blocks vision, clinicians tried surgery, the customary way to resolve a hemorrhage. But in NIV patients, the surgery causes scarring and inflammation.

Then, the next symptom would pop up.

So, a team led by Stanford ophthalmologist Vinit Mahajan, MD, PhD, decided to try something different. "This constant uphill battle to save the vision of NIV patients made us determined to find the molecules active inside the eye that can lead us to better therapies," he said.

After extracting a small amount of liquid from the eyes of patients with NIV and several non-NIV eyes, the Mahajan and his colleagues catalogued 200 immune-signaling proteins that could be involved in inflammatory processes. In eyes affected by NIV, they identified 64 that were different, usually more abundant.

In a paper that appears in JCI Insight, the team chronicles how they paired these overexpressed proteins with existing drugs known to lessen their effects. For the NIV patients, all from the same family, the approach helped, although it didn't cure the disease.

But the applications for this proteomics-based approach are much broader, first author Gabriel Velez, a MD-PhD student at the University of Iowa and a visiting researcher at Stanford, explained in a recent news release:

Patients with rare diseases often have few therapeutic options, and many conventional therapies fail them. Proteomic profiling allows for clinicians to analyze a patient's diseases tissue in real time and identify proteins that are targeted by already-approved drugs.

That could clinicians to diagnose diseases before symptoms develop, or to tailor treatments to each individual's unique stage of disease, Mahajan said.

Previously: Precision health: a special report from Stanford Medicine magazine, Aim higher: Dean Lloyd Minor calls for widespread embrace of precision health and The quest to restore sight at Stanford
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