New LPR Drug? Trial to Assess a Drug that Targets Pepsin

Letzte Aktualisierung:
8. June 2024

pepsin lpr medication trial

Update September 21: The crowdfunding campaign has been closed (work on the trial is continuing) .

A new trial is on its way to find a new medication for LPR.

Laryngopharyngeal reflux (LPR), also known as silent reflux, is a type of reflux that affects the throat and airways. It is characterized by irritation and inflammation and leads to respiratory symptoms such as hoarseness, coughing, and asthma-like symptoms.

Currently, there are no medications for LPR that are both safe and effective. Many patients feel like they are left on their own.

Often, LPR patients are prescribed the same medications as for heartburn, so-called proton-pump inhibitors (PPI). However, studies have shown that PPIs don’t work better than a placebo against LPR. In other words: taking a PPI does not make sense for LPR.1

Targeting Pepsin

Unlike heartburn, LPR symptoms are not mainly caused by acid but by the stomach enzyme pepsin.2 Pepsin helps to break down protein from food in the stomach. Outside the stomach, pepsin causes inflammation. The result is the typical LPR respiratory symptoms.

Unfortunately, there is currently no drug available to LPR patients that specifically targets pepsin. That is what this new trial is about: to find a medication to block pepsin and relieve LPR symptoms.

Dr Nikki Johnston

Medical researcher Dr. Nikki Johnston is spearheading this new trial. In recent years, Dr. Johnston has published a number of studies on LPR and pepsin that have advanced the knowledge about the disease. Now it’s time to study potential drugs for LPR.

The drug at the center of the trial is not new. Instead, Dr. Johnston researched existing medication that could be repurposed for the treatment of LPR. She found out that some of the drugs used for HIV treatment also happen to inhibit pepsin.

Existing data supports the hypothesis that HIV medication could be an effective treatment for LPR. According to data provided by the Medical College of Wisconsin, of 2062 patients with HIV who take an HIV inhibitor, only five (0.2%) have LPR. In comparison, it is suspected that 10 to 34.4% of the general population have LPR.3, 4 This supports the hypothesis that HIV medication could be an effective treatment for LPR.

Dr. Johnston’s research will initially focus on an oral application of the medication: a pill. HIV inhibitors are safe and considered to have a low side-effect profile. However, the goal would be to develop a topical variant. Patients could apply the drug using a nasal spray or inhaler. This could reduce potential side effects.

How You Can Help – Crowdfunding Campaign

Dr. Johnston is currently raising money for this research project through crowdfunding. She will need $700,000 to fund the trial. While it might at first look like a large sum, it’s tiny if even just a fraction of LPR sufferers chip in.

Here is the link if you want to add a donation:

Link Removed as the crowdfunding campaign has been closed (work on the trial is continuing)

Important: You will need to manually select “Other” for the designation of your donation. Then copy-paste “New Therapeutic for Reflux Disease” (without the quotation marks) in the field at the bottom. This is important to make sure your money is added to the fund for Dr. Johnston’s trial. Choose the amount you want to donate, click on “Add donation,” and fill out your billing information.

Here is a screenshot of what the correct information in the field looks like:

New Therapeutic for Reflux Disease

If you have questions or would like more information about the study, you can visit this Facebook group about the trial that LPR patients have set up:

In case Dr. Johnston is looking for more people to participate in the study, we might send an email to Refluxgate’s email subscribers. If you want to add yourself to the email list, click here to open the sign-up form.


1. Liu C, Wang H, Liu K. Meta-analysis of the efficacy of proton pump inhibitors for the symptoms of laryngopharyngeal reflux. Braz J Med Biol Res. Jul 4 2016;49(7)doi:10.1590/1414-431X20165149

2. Johnston N, Dettmar PW, Bishwokarma B, Lively MO, Koufman JA. Activity/stability of human pepsin: implications for reflux attributed laryngeal disease. Laryngoscope. Jun 2007;117(6):1036-9. doi:10.1097/MLG.0b013e31804154c3

3. Kamani T, Penney S, Mitra I, Pothula V. The prevalence of laryngopharyngeal reflux in the English population. Eur Arch Otorhinolaryngol. Oct 2012;269(10):2219-25. doi:10.1007/s00405-012-2028-1

4. Lowden M, McGlashan JA, Steel A, Strugala V, Dettmar PW. Prevalence of symptoms suggestive of extra-oesophageal reflux in a general practice population in the UK. Logoped Phoniatr Vocol. 2009;34(1):32-5. doi:10.1080/14015430902735847

About the author 

Gerrit Sonnabend

Gerrit is a German data scientist & medical publisher. His formal education is in qualitative research. He had severe reflux himself. Read more about him here.