Interview with Wayne Fuchs, MD, Co-Investigator, PXE Clinical Trial
Wayne Fuchs, MD, is currently a Clinical Professor of Ophthalmology at The Mount Sinai Hospital in New York. He's been a long-time friend of PXE International, serving on our Professional Advisory Board since its inception, and presenting on PXE and the Eye at our biennial conferences.
More on the PXE magnesium supplement clinical trial.
Terry Mac Dermaid: Dr. Fuchs, thank you for all your service to the PXE community! What is your role in the current clinical trial of magnesium supplementation for treatment of PXE?
Wayne Fuchs: As an ophthalmologist, my role in this clinical trial is to document the ophthalmologic manifestations and assess the rate of progression of ocular complications of PXE. My clinical practice has been limited to diseases of the retina with a special interest in PXE since 1984, and it is a pleasure to serve as a co-investigator in this study.
Terry: Why is the PXE clinical trial important? What excites you about this trial?
Wayne: This trial is critical as we are searching for a treatment to prevent the complications PXE. The major risk factor in the eye is choroidal neovascularization, which can lead to leakage of fluid, bleeding and ultimately a scar with loss of central vision. Fortunately, we have excellent medications to treat patients who have already progressed to neovascularization and reduce their risk of vision loss, but are seeking a method to prevent the early stages of disease.
I am excited that magnesium may slow the rate of elastic tissue calcification and possibly reduce the risk of early clinical manifestations of PXE. We currently have no therapy that prevents features such as peau d’orange or angioid streaks from developing, or to treat patients who exhibit progressive atrophy of the pigment epithelium.
Terry: What are you looking for in the eye to determine effectiveness of the treatment?
Wayne: Each patient enrolled in the study undergoes a complete ophthalmologic evaluation at baseline, and at months 12 and 24. This includes visual acuity testing using the ETDRS chart, a detailed retina exam with fundus drawing, High Definition Optical Coherence Tomography (HD-OCT) with recording of the central thickness of the macula, photography (color, red-free, and auto-fluorescence), and angiography (indocyanine green or fluorescein). Changes in visual acuity will be recorded and a gain or loss of 15 letters will be considered significant. The presence and degree of peau d’orange, angioid streaks, blood, optic nerve drusen, subretinal fluid, lipid, retinal pigment atrophy, and central retinal thickness will be documented at the start of the study and at subsequent visits. We are hoping for a reduction in the progression of ophthalmologic complications in the individuals receiving the magnesium supplements.
Terry: In your opinion, what motivates PXEers to participate in this trial?
Wayne: PXEers are concerned about their general health and the well-being of their families. Many are motivated by a fear of vision loss, but most are simply eager to assist researchers working diligently in search of a cure or better treatments to reduce the complications of this inherited disease.
Terry: If this trial does show that magnesium is an effective treatment for PXE, what happens next?
Wayne: If magnesium supplementation is shown to be effective, a larger clinical trial will probably be launched to confirm the results prior to recommending that all PXEers begin treatment. Additional studies may investigate whether magnesium plays a role in Age-related Macular Degeneration, a disease with similar features.
Terry: Any other comments you'd like to share?
Wayne: Unfortunately, there are limitations to this study, which include but are not limited to the small sample size. There were no specific ocular exclusion criteria for enrollment in the study, and many patients entered the clinical trial with advanced ocular complications such as central scarring, and some had already received anti-VEGF injections for choroidal neovascularization. If magnesium supplementation shows promise, perhaps a larger trial would enroll patients at an earlier stage of their disease process, when treatment is more likely to be of benefit.
It is my pleasure to thank all the patients who have devoted their time and energy to participate in this clinical trial and I look forward to sharing in their care over the next two years.
Terry: Thank you so much for your time, Dr. Fuchs!