REGENERA PHARMA NAION STUDY

Non-arteritic Anterior Ischemic Optic Neuropathy (NAION) Treatment Study

Non-arteritic anterior ischemic optic neuropathy (NAION) is the most common cause of optic nerve-related, sudden and permanent loss of vision in individuals 50 years or older.  Currently there is no treatment that can improve vision after an NAION attack.  NORDIC, Regenera Pharma, Ltd, and Emmes Corp. have partnered to design a treatment trial, called the Regenera NAION Study, at 12 clinical sites across the US. The study is evaluating whether a drug made from purified plant sap is safe and whether it improves vision in people who have NAION.  The plant sap, called Gum Mastic, which already is used in some medicines and food, will be given by self-injection into the body, similar to an insulin injections for diabetes.  It will be injected at the beginning of the study and twice a week for 26 weeks. Below are some details about the study.

If you or someone you know has had fairly sudden, painless, vision loss in one eye, it could be NAION.  If you know you have had NAION, reading further may help you better understand the disorder.  This page will also give you more information about the Regenera treatment trial that is currently recruiting patients with prior NAION in one or both eyes and have remaining vision loss.

What is NAION?

NAION is caused by temporary, reduced blood flow to the optic nerve.  It is not caused by inflammation (e.g. optic neuritis) or compression (e.g. tumor).  Vision loss due to NAION can appear as blurring in the center, lower, or upper portion of your vision, or your entire vision while covering the better eye and viewing with the affected eye. Commonly, patients will complain that they can’t see objects below their straight-ahead line of sight. The vision loss can worsen over days to a week.

You can think of the optic nerve as the video cable connecting your eye, which is like an ultra-sophisticated camera, to your brain, which processes the signals from the eye to create your vision.  NAION interrupts those signals resulting in defects in your visual field.  The poor blood flow to the optic nerve is temporary so unlike a brain stroke, there is no blockage of arteries.  That means that treatments to restore blood flow, like those used for strokes, do not work for NAION.

What is the outlook if I have NAION?

Vision recovery is unlikely for eyes affected with NAION.  Permanent visual impairment persists in nearly all NAION cases.  When doctors look at images of an eye with NAION, the optic nerve eventually appears pale, which is called optic atrophy.  Also, major areas of regular and peripheral can become blurred or blocked.  Up to 50% of patients have diminished eye site, as well as significant visual field loss in at least one eye.  There is no proven treatment for the recovery of vision in patients with previous NAION, meaning patients with optic nerve damage that occurred months to years earlier from an attack of NAION.

Figure 1: Example of a visual field test from someone with NAION.  This test shows portions of the person’s visual field where they cannot see.

 

Figure 2: An image of an optic nerve from someone with NAION.  This image shows pale coloration of the optic nerve, which is called optic atrophy.

 

What is the Regenera NAION Study?

The study will involve twice weekly subcutaneous injections (under the skin with a small needle) of an experimental drug over a six month period of time.  The study will monitor the visual function in participants with prior NAION to determine whether the drug is effective.

What is the drug being used in the Regenera NAION Study?

The name of the experimental drug in this study is RPh201.  It is a proprietary, isolated fraction of Gum Mastic, which is a commercially available resin exudate from the Pistacia lentiscus tree. There are extensive data of human use of the Gum Mastic itself, and extracts of this gum have been in human use extensively for many generations in both traditional and non-traditional medicine.  It possesses anti-carcinogenic, antibacterial, antioxidant, analgesic and anti-inflammatory properties.  Based on its large number of potentially beneficial effects, Gum Mastic and its extracts currently are in use in health and care products and in the food industry.  For example, mastic has previously been reported in studies treating gastrointestinal conditions, including duodenal and peptic ulcer, functional dyspepsia, wounds and Crohn’s Disease. RPh drug substrate (RPh-DS) is a naturally derived botanical composition.  The safety and tolerability of RPh has been tested in healthy participants following single ascending and multiple ascending subcutaneous doses of RPh201.  Experimental animal models of brain and optic nerve injury show the ability of RPh201 to improve function in these animals.

What are the requirements to participate in the study?

  • Records must be available indicating definite diagnosis of NAION (including optic disc swelling observed by an ophthalmologist at the onset of visual loss)
  • Patient must have been at least 50 years old when NAION occurred
  • NAION must have occurred at least 1 year and not more than 5 years earlier
  • The participant must be able to read at least 20 letters but not more than 60 letters with the affected eye on an eye chart with best correction
  • The eye with NAION must have disc pallor present
  • The eye with NAION must have stable vision
  • The patient must be able to understand and sign a form called an Informed Consent that explains the study

How do I participate?

If you think you or an individual you know fit the criteria and are interested in finding out more, you should see a doctor to be evaluated.  On the following website, under “Contacts and Locations,” you can look for the contact information of a doctor nearest you:  https://clinicaltrials.gov/ct2/show/NCT03547206

Clinical sites participating in this study:

Bascom Palmer Eye Institute, Miami, FL

Bethesda Neurology, LLC, North Bethesda, MD

Byers Eye Institute at Stanford University, Palo Alto, CA

Charleston Neuroscience Institute, Ladson, SC

Doheny Eye Center UCLA, Pasadena, CA

Massachusetts Eye and Ear Infirmary, Boston, MA

Neuro-Eye Clinical Trials, Inc., Houston, TX

New York Eye and Ear Infirmary of Mount Sinai, New York, NY

NorthShore University Health System, Glenview, IL

The Eye Care Group, Orange, CT

The Eye Care Group, Southbury, CT

University of Virginia, Charlottesville, VA

Washington University in St. Louis School of Medicine, St. Louis, MO