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AHS grad wins contest, has eye on patent for treatment for eye disease

An Albemarle County student has her eye on potentially patenting a treatment for a currently untreatable eye disease.

Meenakshi Ambati, 17, just graduated from Albemarle High School and is already working to patent a potentially groundbreaking treatment for a degenerative eye disease called dry macular degeneration.

The disease affects the retina and can lead to irreversible blindness. Over 200 million people worldwide have the disease and there’s no FDA approved treatment.

“This is a huge unmet medical need. And so here at the lab, I made the surprising discovery that Prozac, which is an antidepressant, could actually be repurposed to treat this disease,” Ambati said.

She published her findings in the Proceedings of the National Academy of Sciences medical journal last year.

“I have several patents on this technology and my process and we’re planning to start clinical trials and are working with a biotech company to get those started soon,” Ambati said.

Ambati recently received first place in the Eye On The Future national competition by the National Eye Institute for her research. She will receive a $2,000 stipend and has been invited to tour the institute’s facilities later this month and meet with scientists.

“It’s really exciting,” Ambati said.

Ambati has been working with Dr. Bradley Gelfand, an assistant professor of ophthalmology at the University of Virginia, since she was just a freshman in high school. She decided to reach out to him because she was inspired by his work in finding a cure for age-related macular degeneration.

“We don’t yet know the full significance of this finding. But what we have now is the potential for something truly groundbreaking and significant,” Gelfand said.

Ambati discovered her love for science as a fourth-grader when she did her first science fair project.

“I’ve always been interested in humans and how we work. That really led me down the path of medical research,” she said. “In middle school, I learned about dry macular degeneration. And I was absolutely shocked that there are people around the world who have to face these trials of slowly going blind over several years.”

She said she was ‘devastated’ by the disease’s impact on people and that there was no treatment.

“Almost every American family has at least one person in their family with this disease. That is absolutely shocking and I wanted to do something to help contribute to solving this problem,” she said.

Ambati came up with the idea to test Prozac as a treatment for dry macular degeneration after studying the chemical makeup of the antidepressant.

“I was looking at the various chemical structures of different diseases and various chemical structures of various treatments for different diseases. So I wanted to first find a treatment that many people are already taking,” Ambati said.

“I was looking at all the antidepressants, and I found that Prozac has a similar chemical structure to a known inhibitor of various pathology related to this disease,” she said.

Praveen Yerramothu, a research scientist at UVa who has been working at Ambati, said he has been shocked and inspired by her findings.

“It is definitely not normal for a person at that particular age to come up with breakthroughs like this. I’ve been working in science research for about 15 years now and I have not seen anyone at this particular stage in their lives to make a discovery like this. So I think this is fantastic,” he said.

Gelfand said Ambati has the skills of a seasoned professional while bringing youthful curiosity to her work.

“She has sort of an unusual and exceptional combination, where she has the energy and vibrancy of a young person matched with the demeanor, professionalism, and intelligence of a full time researcher,” he said.

Gelfand also said it’s important to understand while Ambati’s finding is significant, it is not an approved treatment yet.

“It’s really important to understand that this is preliminary. There’s just not enough evidence to recommend that people start taking it for this disease,” he said. “What I recommend is for people who are interested in looking at new treatments for their retinal disease to contact UVa Ophthalmology. There are a lot of experimental treatments out there and they may qualify for as part of a clinical trial.”

In terms of balancing her research with regular high school classes and being a teenager, Ambati said she spends all her spare time in the lab at UVa. However, she loves it so much that it doesn’t feel like work.

“It’s certainly a passion project. I absolutely love doing it, I loved coming up with the hypotheses for this project and coming up with the experimental variables. That was awesome,” Ambati said.

As for Ambati’s next steps, she was accepted to both Yale University and Harvard University. She ultimately decided to go to Yale after she was awarded a research scholarship that is only given to 10 members of the incoming freshman class.

“I was really excited that they showed a vested interest in performing research, and were willing to fund me and support me all the way from freshman year,” Ambati said.

She said she wants to pursue her PhD and continue to make discoveries.

“I’m going to keep working on science. I’m going to try and work with other outstanding researchers to come up with new ideas, testing them out, and hopefully to contribute to the scientific community,” Ambati said


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