New Approach to Reflux Treatment Could Revolutionize Care for LPR and GERD

For millions of people worldwide, everyday activities like eating or speaking can be a struggle. Chronic heartburn, hoarseness, and persistent cough are just a few symptoms of Laryngopharyngeal Reflux (LPR) and Gastroesophageal Reflux Disease (GERD).

Though common, these conditions have long lacked consistently effective treatments. Nikki Johnston, PhD, a professor of otolaryngology and communication sciences and microbiology and immunology at the Medical College of Wisconsin (MCW), is working to change that. Her groundbreaking research offers a new approach that could transform countless lives and treat reflux long-term.

Reflux occurs when stomach contents flow back into the esophagus and sometimes all the way up to the throat. For decades, treatment has centered on Proton Pump Inhibitors (PPIs), medications that reduce stomach acid.

While effective for many GERD patients, PPIs often fall short. In fact, 40% of GERD sufferers and 86% of those with LPR don’t respond to them.

“By the time the reflux reaches the throat, it’s often not acidic anymore. That’s why acid-suppressing medications like PPIs don’t help these patients,” says Dr. Johnston. “We needed to look beyond acid to find a better solution.”

A New Target: Pepsin

Dr. Johnston’s research has uncovered a crucial culprit in reflux disease: pepsin, a digestive enzyme produced in the stomach.

While essential for breaking down food, pepsin causes inflammation and tissue damage when it reaches the throat. Dr. Johnston’s studies have confirmed the presence of pepsin in patients with reflux symptoms and linked it directly to airway and throat damage.

“Pepsin is the missing piece of the puzzle,” says Dr. Johnston. “We’ve shown that it causes inflammation and damage, even when the reflux isn’t acidic. That’s why we’re targeting pepsin as a new therapeutic approach.”

Instead of developing a new drug from the ground up, Dr. Johnston and her team took an innovative approach by repurposing Fosamprenavir, an older HIV protease inhibitor with a strong safety record. Crucially, it also binds to and inhibits pepsin.

“Fosamprenavir is a great candidate because it’s already been used in humans, so we know it’s safe,” says. Dr. Johnston. “Plus, it targets a foreign virus, so it’s less likely to cause side effects.”

Through her company, N-Zyme Biomedical, Dr. Johnston has developed a new formulation of Fosamprenavir specifically as an anti-pepsin treatment. Now patented, two Phase 2 clinical trials – one for LPR and one for GERD – are slated for launch in 2025.

A Growing Demand for Better Reflux Treatments

The demand for better reflux treatments is overwhelming. In the U.S. alone, more than 60 million people live with GERD, and another 80 million are affected by LPR. For many, current options, such as PPIs, lifestyle changes, or surgery, fall short. Dr. Johnston’s work offers new hope for those left without effective solutions.

“There’s a huge unmet need,” says Dr. Johnston. “Patients with LPR, in particular, have been underserved. They often go undiagnosed or are told their symptoms are in their head. But we know this is a real condition, and we’re working to give these patients a better option.”

The response to her upcoming clinical trials underscores that urgency. Her LPR study, which requires 104 participants, has already drawn more than 400 volunteers.

“It’s incredible to see how many people are eager to take part,” Dr. Johnston says. “It speaks volumes about how much this treatment is needed.”

From Lab to Biotech Startup Success

The need for better treatments propelled Dr. Johnston from the lab to the launch of a biotech venture. After years of academic research, she co-founded N-Zyme Biomedical with business partner Franco Vigile, who suffers from LPR. Together, they’ve raised nearly $3 million in funding and secured key patents to move the treatment forward.

“I never thought I’d be running a company,” Dr. Johnston admits. “But when we realized the potential of this research, it made sense to take the leap.”

The support of MCW has also been instrumental.

“From the Office of Technology Development to my colleagues, everyone has been behind this project,” Dr. Johnston says. “It’s been a challenging journey, but I’m so glad we’ve taken it.”

By targeting pepsin – the root cause rather than just the symptoms of the condition – Dr. Johnston’s work offers the promise of lasting relief for millions living with reflux. With clinical trials on the horizon, a safe, effective treatment is now within reach.

“This research has consumed my life, but it’s worth it,” she says. “I hear from people every day – teachers, singers, anyone who depends on their voice. It’s incredibly rewarding to know we may finally have a solution.”