Oral vs IV Steroids for Acute Optic Neuritis

MedicalResearch.com Interview with:

Sarah A. Morrow MD, MS, FRCPC Associate Professor of Neurology Department of Clinical Neurological Sciences University of Western Ontario (Western)

Dr. Morrow

Sarah A. Morrow MD, MS, FRCPC
Associate Professor of Neurology
Department of Clinical Neurological Sciences
University of Western Ontario (Western)

MedicalResearch.com: What is the background for this study?

Response: Acute demyelinating optic neuritis, which presents with loss of vision and painful eye movements, is common in multiple sclerosis (MS) occurring 50% of persons with MS. High dose (≥ 1g) corticosteroids administered through an IV became the standard of practice after the landmark Optic Neuritis Treatment Trial as IV administration. However, in that study the IV dose of corticosteroids was much higher (1 gram daily) than the oral dose (1 mg/kg). Thus, it is not clear if IV administration is still better if equivalent doses are used orally. Oral administration is much more convenient for patients and less expensive, and previous studies showed that it is preferred by patients.

In this study, we asked the following question: are high dose (≥ 1000mg) IV corticosteroids superior to equivalent doses of oral corticosteroids for the acute treatment of optic neuritis?

We randomly assigned fifty-five cases of acute optic neuritis to 1000mg IV methylprednisolone or 1250mg oral prednisone daily for three days and compared recovery of their vision over the next 6 months.  Continue reading

Genetic Link Between Corneal Thickness and Risk of Glaucoma

MedicalResearch.com Interview with:

Eldon E. Geisert, PhD Professor of Ophthalmology Emory School of Medicine

Dr. Geisert

Eldon E. Geisert, PhD
Professor of Ophthalmology
Emory School of Medicine

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: In the late 1990s a group of doctors began a study of glaucoma patients to determine if there were phenotypes that are predictive for developing glaucoma.

In this Ocular Hypertension Treatment Study (OHTS) one of the highly correlated ocular traits was central corneal thickness (CCT). The early clinical studies found that people with thinner corneas were at a higher risk of developing glaucoma. In two large studies, examining thousands of people a number of genes were identified that were risk factors for glaucoma or that controlled CCT in humans. In both cases the identified genes accounted for less than 10% of the genetic risk for glaucoma and less than for 10% of the genetic control for CCT. There was little data linking the genetic control of CCT to the glaucoma risk.

Our group has taken an indirect approach to the question, using well-defined mouse genetic system to identify genes modulating CCT and then interrogating human glaucoma data to determine if these genes are associated with glaucoma risk.   Continue reading

Unnecessary Routine Preoperative Cataract Testing Costs Medicare Millions

MedicalResearch.com Interview with:
Catherine L. Chen, MD, MPH

Assistant Professor
UCSF Department of Anesthesia & Perioperative Care

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Routine preoperative medical testing (such as common laboratory tests looking at a patient’s blood cell counts and kidney function, or cardiac tests like an EKG) are not recommended in patients undergoing cataract surgery, but these tests still occur quite frequently among Medicare cataract surgery patients because these patients tend to be older and sicker than the general population. In the past, researchers have used a 30-day window counting backwards from the date of surgery to determine whether a given test should be categorized as a routine preoperative test. However, we know that testing often takes place outside this window and therefore, the frequency and cost of routine preoperative medical testing has generally been underreported.

In our study, we used a new method to figure out how to determine the start of the routine preoperative testing period. In cataract patients, ocular biometry is a diagnostic test that is performed in anticipation of cataract surgery, and this test is only performed in cataract patients who will be having cataract surgery in the near future. For each patient, we calculated the elapsed time between the ocular biometry and cataract surgery dates to get a better idea of when to start looking for unnecessary routine preoperative testing. Our goal was to identify all the routine preoperative medical testing that occurs once the decision has been made to operate and better estimate the cost to Medicare of this unnecessary testing.

In a previous study that we published in the New England Journal of Medicine, we reported a significant spike in the rate of routine preoperative medical testing that occurs in the 30 days before surgery compared to the baseline rate of testing. In our current study, we discovered that there is a second spike in testing that occurs in the 30 days after ocular biometry. In fact, even if you exclude the testing that takes place during the 30 days before surgery, there is still a 41% increase in testing rates during the interval between ocular biometry and cataract surgery over the baseline rate of testing. In addition, we found that the cost of routine preoperative testing was 47% higher when looking at the entire biometry to surgery timeframe compared to testing that occurs just in the 30 days before surgery.

We estimate that the cost to Medicare of all of this unnecessary testing approaches $45.4 million annually. Continue reading

Tunable Lens Allows Detailed Imaging of Entire Eye

MedicalResearch.com Interview with:

Ireneusz Grulkowski, PhD Assistant Professor Bio-Optics & Optical Engineering Lab Institute of Physics Nicolaus Copernicus University

Dr. Grulkowski

Ireneusz Grulkowski, PhD
Assistant Professor
Bio-Optics & Optical Engineering Lab
Institute of Physics
Nicolaus Copernicus University

MedicalResearch.com: What is the background for this study?

Response: The ophthalmic diagnostics has undergone a revolution over the last 30 years. The access to new modalities allowed to understand the process of development of different eye diseases of the retina and the anterior segment. In particular, optical coherence tomography (OCT) demonstrated the feasibility in visualization of microarchitecture of the ocular tissues. However, most of the ophthalmic equipment is dedicated either to imaging the anterior segment of the eye (e.g. the cornea) or to retinal imaging. This is due to the fact that the eye is composed of the elements, such as the cornea and the lens, that refract the light.

In this report, we wanted to address that challenge. We compensated the refractive power of the eye by the application of the tunable lens. The focus tunable lens is the example of active optical element that changes its focal distance with the applied electric current.

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LUXTURNA Proves Effectiveness of Single Gene Therapy To Cure Rare Cause of Blindness

MedicalResearch.com Interview with:

Dr. Stephen M. Rose, PhD Chief Research Officer Foundation Fighting Blindness

Foundation Fighting Blindness

Dr. Stephen Rose PhD
Chief Research Officer
Foundation Fighting Blindness (FFB)

Dr. Rose comments on the announcement of the FDA approval of voretigene neparvovec (LUXTURNA™) gene therapy for inherited blindness due to mutations in the RPE65 gene.

What is the background for this announcement? What were the main findings from the study?

Response: While it has been 30 years since the RPE65 gene was identified as causing Leber’s Congenital Amaurosis, this shows that it is possible to have an effective gene therapy for an inherited disease. As the first gene therapy for the eye or for an inherited disease, LUXTURNA is a historic milestone in the search for cures for all inherited retinal diseases (IRDs). As a one-time gene therapy, LUXTURNA will not only be life-changing for patients with vision loss due to mutations in the RPE65 gene, it also provides critical momentum for gene therapies – for the eye and other diseases – now in the clinic.  Continue reading

Retinopathy in Premature Infants: Low Dose Ranibizumab May Be Effective Without Systemic Side Effects

MedicalResearch.com Interview with:

Prof. Dr. Andreas Stahl
Geschäftsführender Oberarzt
Leiter Arbeitsgruppe Angiogenese
Universitätsaugenklinik Freiburg | University Eye Hospital Freiburg
Freiburg, Germany

MedicalResearch.com: What is the background for this study?

Response: Retinopathy of prematurity (ROP) is a sight-threatening disease and one of the main reasons for irrreversible bilateral blindness in children. Particularly infants born at very early gestational ages or with very low birth weight are affected. In these infants, vascularization of the retina is unfinished at the time of birth. Severeal weeks into the life of these very prematuerly born infants, angiogenic growth factors, mainly vascular endothelial growth factor (VEGF), become upregulated in the avascular parts of the retina, leading to a re-activation of physiologic vascular growth. If all goes well, these re-activated retinal blood vessels progress towards the periphery and lead to a fully vascularized and functional retina. If, however, the vascular activation by VEGF is too strong, then vascular growth becomes disorganized and vessels are redirected away from the retina and into the vitreous. If left untreated, these eyes can then proceed towards tractional retinal detachment and blindness.

Since the 1990s, the standard method of treating ROP has been laser photocoagulation of avascular parts of the retina. This treatment is sensible because VEGF as the main angiogenic driver of pathologic blood vessel growth is expressed in these avascular parts of the retina. The downside of laser treatment, however, is that treated retinal areas are turned into functionless scar tissue and are lost for visual function. In addition, infants treated with laser need to be under general anesthesia for hours during treatment which can be troublesome in very young and fragile preterm infants. And in the long run, infants treated with laser have a high risk of developing high myopia in later life.

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Does Restasis Work For Dry Eye Disease?

MedicalResearch.com Interview with:
Steven Woloshin, MD MS

Steven Woloshin, MD Professor of The Dartmouth Institute Professor of Medicine Professor of Community and Family Medicine

Dr. Steven Woloshin

Professor of The Dartmouth Institute
Professor of Medicine
Professor of Community and Family Medicine
The Center for Medicine in the Media
Dartmouth Institute for Health Policy and Clinical Practice
Lebanon, New Hampshire

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: There has been a lot of debate about the legal maneuvers (ie, transferring patents to the Mohawk Indians) Allergan has employed to delay marketing of generic alternatives to Restasis (cyclosporine ophthalmic emulsion 0.05%).   But there is a more fundamental question that has received little attention:  Does Restasis work?  It is not approved in the European Union, Australia or New Zealand where registration applications were “withdrawn prior to approval due to insufficient evidence of efficacy” in 2001.   Although Canada approved Restasis, its national health technology assessment unit, unconvinced of meaningful benefit, recommended Canada not pay for it – according to our research, no Canadian provincial or federal drug plan currently does.   Nevertheless, Americans have spent $8.8 billion in total sales between 2009 and 2015 on Restasis, including over $2.9 billion in public monies through Medicare Part D.

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Antibiotic Resistance Common In Infections After Ocular Surgery

MedicalResearch.com Interview with:

Dr-Penny Asbell

Dr. Asbell

Penny Asbell, MD
Icahn School of Medicine
Mt. Sinai, New York City.

MedicalResearch.com: What is the background for this study?

─     Bacterial endophthalmitis is a serious, although infrequent, complication of ocular surgery, typically caused by perioperative introduction of bacterial flora from the patient’s own conjunctiva and skin.

─     Prophylactic measures such as perioperative antibiotic treatment may minimize the risk for endophthalmitis, but can be complicated by antibiotic resistant bacteria.

─     The ongoing Antibiotic Resistance Monitoring in Ocular micRoorganisms (ARMOR) study is the only nationwide antibiotic resistance surveillance program specific to ocular pathogens.

─     The purpose of this presentation is to report on the antibiotic susceptibility profiles of bacterial isolates from the vitreous and aqueous humor collected in the ARMOR study expanding upon earlier findings.

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Deep Learning System Can Screen For Diabetic Retinopathy, Glaucoma and Macular Degeneration

MedicalResearch.com Interview with:

Blausen.com staff (2014). "Medical gallery of Blausen Medical 2014". WikiJournal of Medicine 1 (2). DOI:10.15347/wjm/2014.010. ISSN 2002-4436. Illustration depicting diabetic retinopathy

Illustration depicting diabetic retinopathy

Dr. Tien Yin Wong MD PhD
Singapore Eye Research Institute, Singapore National Eye Center,
Duke-NUS Medical School, National University of Singapore
Singapore

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Currently, annual screening for diabetic retinopathy (DR) is a universally accepted practice and recommended by American Diabetes Association and the International Council of Ophthalmology (ICO) to prevent vision loss. However, implementation of diabetic retinopathy screening programs across the world require human assessors (ophthalmologists, optometrists or professional technicians trained to read retinal photographs). Such screening programs are thus challenged by issues related to a need for significant human resources and long-term financial sustainability.

To address these challenges, we developed an AI-based software using a deep learning, a new machine learning technology. This deep learning system (DLS) utilizes representation-learning methods to process large data and extract meaningful patterns. In our study, we developed and validated this using about 500,000 retinal images in a “real world screening program” and 10 external datasets from global populations. The results suggest excellent accuracy of the deep learning system with sensitivity of 90.5% and specificity of 91.6%, for detecting referable levels of DR and 100% sensitivity and 91.1% specificity for vision-threatening levels of DR (which require urgent referral and should not be missed). In addition, the performance of the deep learning system was also high for detecting referable glaucoma suspects and referable age-related macular degeneration (which also require referral if detected).

The deep learning system was tested in 10 external datasets comprising different ethnic groups: Caucasian whites, African-Americans, Hispanics, Chinese, Indians and Malaysians

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Aravind Eye Care System Reduces Waste and Carbon Footprint From Cataract Surgeries

MedicalResearch.com Interview with:

Cassandra Thiel, PhD Assistant Professor in the Departments of Population Health and Opthamology at NYU Langone Health, and Assistant Professor at NYU Wagner and NYU Tandon School of Engineering

Dr. Thiel

Cassandra Thiel, PhD
Assistant Professor in the Departments of Population Health and Opthamology at NYU Langone Health, and Assistant Professor at NYU Wagner and
NYU Tandon School of Engineering

MedicalResearch.com: What is the background for this study?

Response: Everyone is concerned about the health impacts of climate change, from the United Nations to the Lancet. While other industries are trying to monitor and minimize their environmental footprint, healthcare services have been largely overlooked. Yet, the US healthcare sector emits 10% of the US’s total greenhouse gases.

Cataract surgery is one of the most commonly performed procedures in the world. In the US, these surgeries generate large quantities of waste due to the use of single-use, disposable materials and supplies. However, at Aravind Eye Care System in southern India, the outcomes for this procedure are the same as in the US, but the materials they use are mostly reusable. This study assessed the environmental footprint of Aravind’s surgical process, to determine how their process design and material selection affected their emissions.

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miLOOP Uses Minimal Energy To Encapsulate and Remove Cataracts

MedicalResearch.com Interview with:

Sean Ianchulev, MD MPH Chief Medical Officer VP of Medical Affairs and Business Development Transcend Medical 

Dr. Ianchulev

Sean Ianchulev, MD MPH
Chief Medical Officer
VP of Medical Affairs and Business Development
Transcend Medical 

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Similar to MIGS stent technology for glaucoma, MiLOOP is a an application of micro-interventional technology for cataract surgery – allows the fragmentation of the lens with simple micro-interventional pen-like device which does not require complex, capital-intense phaco-emulsification…also it does not require vibrational energy

MedicalResearch.com: What should readers take away from your report?

 Iantech introduces a micro-­interventional devices designed to deliver energy-­free endocapsular lens fragmentationResponse: That after 50 years of conventional phacoemulsification cataract technology, new breakthrough micro-interventional approaches are on the horizon.

MedicalResearch.com: What recommendations do you have for future research as a result of this study?

Response: Future studies will evaluate whether micro-interventional approaches for cataract surgery can replace phacoemulsification equipment altogether.

Any disclosures?

I am the founder and Chairman of the company.

MedicalResearch.com: Thank you for your contribution to the MedicalResearch.com community.

Citation: Ianchulev S. Microinterventional cataract surgery. Presented at American Academy of Ophthalmology annual meeting; Nov. 11-14, 2017; New Orleans. 

 

Note: Content is Not intended as medical advice. Please consult your health care provider regarding your specific medical condition and questions.

 

 

 

 

 

FDA Advisory Committee Recommended Approval of First Gene Therapy For Inherited Eye Disease

MedicalResearch.com Interview with:
Dr. Stephen M. Rose, PhD Chief Research Officer Foundation Fighting BlindnessDr. Stephen M. Rose, PhD
Chief Research Officer
Foundation Fighting Blindness

Dr. Rose discusses the FDA advisory panel unanimously recommended approval of Spark Therapeutics’ Gene Therapy Luxturna  for the treatment of patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal dystrophies, a group of rare blinding conditions caused by one of more than 220 different genes.

MedicalResearch.com: Would you tell us a little about IRD? Whom does it affect and how?  How common is this disorder?

Response: The retina at the back of the eye is responsible for collecting light and turning it into signals that are transmitted to the brain and interpreted as vision. Think of the retina as the film in a camera, or more recently the sensor at the back of a digital camera. Inherited rare retinal degenerations are when the retina at the back of the eye deteriorates and loses its ability to capture light, thereby leading to blindness.

iRDs can affect anyone, no matter race or ethnicity. These are inherited conditions that are passed down from parents to children, if a parent or both parents are either affected already or are carriers for a variant in any of the over 250 genes responsible for retinal degeneration.

There are over 15 different types of iRDs, with retinitis pigmentosa being the most common with a US affected population around 100,000. The rest of the iRDs make up another approximately 100,000 affected individuals in the US, so there are about 200,000 total affected individuals in the US. Worldwide these iRDs affect somewhere around one to two million individuals.

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Cataract Surgery Linked To Improved Health and Survival, As Well As Sight

MedicalResearch.com Interview with:

Anne L. Coleman, MD, PhD Center for Community Outreach and Policy, Stein Eye Institute David Geffen School of Medicine Director, UCLA Mobile Eye Clinic Department of Epidemiology, Fielding School of Public Health UCLA

Dr. Coleman

Anne L. Coleman, MD, PhD
Center for Community Outreach and Policy, Stein Eye Institute
David Geffen School of Medicine
Director, UCLA Mobile Eye Clinic
Department of Epidemiology, Fielding School of Public Health
UCLA

MedicalResearch.com: What is the background for this study?

Response: Cataracts are a leading cause of vision loss worldwide, and cataract surgery is an intervention that is known to be extremely effective to address the vision loss related to cataract. However, it is unclear if there are benefits of cataract surgery beyond vision improvement in people with cataracts. Previous studies have suggested that in addition to improving vision, cataract surgery may decrease the risk of fractures and accidents, improve mental health, and improve overall quality of life. The purpose of the present study was to further investigate the potential benefits of cataract surgery and to determine if cataract surgery was associated with increased survival in people with cataracts.

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Nerf Guns Can Cause Serious Eye Bleeding

Helgi Halldórsson from Reykjavík, Iceland Wikipedia image

A man aiming an N-Strike Stampede ECS
Wikipedia image

MedicalResearch.com Interview with:
Dr. Mukhtar Bizrah
Accident & Emergency Department,
Moorfields Eye Hospita
NHS Foundation Trust
London, UK

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: We noticed a number of patients presenting to the accident and emergency department at our hospital following ‘Nerf gun’ injuries. We decided to perform this study because an online literature search revealed that currently there was no published work on this topic. It was worth doing because it is a public health issue. A number of doctors in A&E commented that they have seen a number of patients present with Nerf gun injuries. I personally saw a patient which an inflamed eye and damage to the iris following a ‘Nerf gun’ injury.

We decided to write about three patients with bleeding in the eye (hyphema) because most journals have a cap on the number of patients in a case series. Also, bleeding in the eye following trauma is known to be associated with serious ocular injury and long term repercussions.

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Patients With Glaucoma Can Be Taught To Measure Their Own Intraocular Pressure

MedicalResearch.com Interview with:

Andrew Tatham Consultant Ophthalmologist Princess Alexandra Eye Pavilion and Department of Ophthalmology University of Edinburgh, Scotland

Dr. Tatham

Andrew Tatham, FRCOphth
Consultant Ophthalmologist
Princess Alexandra Eye Pavilion and Department of Ophthalmology
University of Edinburgh, Scotland 

MedicalResearch.com: What is the background for this study?

Response: Raised intraocular pressure (IOP) is the major risk factor for the development of glaucoma, the most common cause of irreversible blindness, with lowering IOP the only proven treatment.

Until recently the only way to measure IOP was for patients to visit their clinician  meaning it was only possible to obtain a limited number of measurements. This is problematic given that IOP fluctuates and that 75% of individuals have peak IOP outside office hours. If patients could measure their own IOP it would allow far more measurements to be obtained and result in better understanding of the variation and peaks in IOP. This could improve the detection of glaucoma and determine if patients are adequately controlled with medication.

Recently, a patient-operated, home IOP monitoring device (iCare HOME) has become available. The patient holds the device close to their eye and the device automatically determines if it is in the correct position to take a measurement. The tonometer then deploys a small probe which gently bounces off the surface of the eye to determine IOP. As the probe is only in contact with the surface of the eye for a few milliseconds no anesthetic is needed.

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Risky Behaviors Linked To Contact Lens–Related Eye Infections Among Adults and Adolescents

MedicalResearch.com Interview with:

Dr. Jennifer R. Cope MD Medical Officer Division of Foodborne, Waterborne, and Environmental Diseases National Center for Emerging and Zoonotic Infectious Diseases CDC

Dr. Cope

Dr. Jennifer R. Cope MD
Medical Officer
Division of Foodborne, Waterborne, and Environmental Diseases
National Center for Emerging and Zoonotic Infectious Diseases
CDC

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Wearing contact lenses can increase your chances of getting a severe eye infection. Eye infections can lead to serious problems, including blindness. All contact lens wearers can help prevent serious eye infections by correctly wearing and caring for their contact lenses.

Eighty-one percent of young adults, 85% of adolescents, and 88% of older adults regularly did at least one risky behavior related to their contact lenses. The most frequently reported risk behaviors in adolescents were not visiting an eye doctor as least annually, sleeping or napping in lenses, and swimming in lenses.

Among young adults and older adults, the most frequently reported risk behaviors were replacing lenses at intervals longer than those prescribed, replacing lens storage cases at intervals longer than those recommended, swimming in lenses, and sleeping or napping in lenses.

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Does Maternal Preeclampsia Increase Risk of Retinopathy in Premature Infants?

MedicalResearch.com Interview with:

Mary Elizabeth Hartnett, MD, FACS, FARVO Professor of Ophthalmology, Vitreoretinal Service and Surgery Principal Investigator Retinal Angiogenesis Laboratory Director of Pediatric Retina, Adjunct Professor of Pediatrics John A. Moran Eye Center Salt Lake City UT 84132

Dr. Hartnett

Mary Elizabeth Hartnett, MD, FACS, FARVO
Professor of Ophthalmology, Vitreoretinal Service and Surgery
Principal Investigator Retinal Angiogenesis Laboratory
Director of Pediatric Retina, Adjunct Professor of Pediatrics
John A. Moran Eye Center
Salt Lake City UT 84132

On behalf of the co-authors: Julia Shulman, Cindy Weng, Jacob Wilkes, Tom Greene, M. Elizabeth Hartnett

MedicalResearch.com: What is the background for this study?

Response: Maternal preeclampsia causes morbidity to mothers and infants worldwide. Retinopathy of prematurity (ROP) is a leading cause of childhood blindness worldwide. This study was done to gain insight into the effects of preeclampsia on ROP in a clinical population.

The literature is mixed with some reports that preeclampsia increases risk of Retinopathy of prematurity, whereas others suggest preeclampsia is protective or has no effect. The presence of circulating anti-angiogenic factors in preeclamptic mothers that can enter the fetal circulation lends biologic plausibility to the notion that maternal preeclampsia might interfere with developing vascular beds in the fetus, such as the retina, and potentially lead to severe ROP. However, a report using an experimental model provided evidence that uteroplacental insufficiency, a characteristic of preeclampsia, led to protective mechanisms in the offspring that reduced oxygen-induced retinopathy and promoted overall growth.

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Diabetic Retinopathy May Impact Daily Work and Activities

MedicalResearch.com Interview with:

Jeffrey R. Willis MD, PhD 

Dr. Willis

Jeffrey R. Willis MD, PhD
UC Davis Eye Center
University of California, Davis
Sacramento California

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Diabetic retinopathy is one of the leading causes of blindness in the United States.  Yet there is limited national level data on the impact of worsening DR on quality of life and visual function.

Our study aimed to address this knowledge gap by evaluating the functional burden of DR across severity levels, utilizing data from the National Health and Nutrition Examination Survey (NHANES).

We found that one-half of US adults with severe non-proliferative diabetic retinopathy (NPDR) or proliferative diabetic retinopathy (PDR) had difficulty with ≥ 1 visual function task, possibly impacting their daily work/activities.  These patients reported a significantly greater vision-related functional burden relative to those with less severe forms of DR.

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Iris Freckles Are A Potential Biomarker for Chronic Sun Damage

MedicalResearch.com Interview with:

Iris Freckles Credit: © Africa Studio / Fotolia

Iris Freckles
Credit: © Africa Studio / Fotolia

Dr.med.univ. Christoph Schwab
Departement of Ophthalmology
Medical University of Graz
Graz, Austria 

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Knowledge about risk factors and/or pathways involved in pathogenesis is from special importance in order of preventing diseases.

The role of sunlight in several eye diseases is unclear. In our study we found a close relation between sun light exposure – evaluated by a full body skin examination and a personal questionnaire – and iris freckles. Therefore we suggest the presence of iris freckles as a novel biomarker indicating high ocular sun exposure.

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One Time Injection With Spark’s Gene Therapy LUXTURNA Demonstrated Lasting Visual Improvement

MedicalResearch.com Interview with:

Stephen R. Russell, MD Dina J Schrage Professor of Macular Degeneration Research Service Director, Vitreoretinal Diseases and Surgery Professor of Ophthalmology and Visual Sciences The University of Iowa

Dr. Russell

Stephen R. Russell, MD
Dina J Schrage Professor of Macular Degeneration Research
Service Director, Vitreoretinal Diseases and Surgery
Professor of Ophthalmology and Visual Sciences
The University of Iowa

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: This study examines the efficacy (and safety) of treating children and adults with a form of retinitis pigmentosa known as RPE65-associated Lebers congenital amaurosis, with an adeno-associated viral vector(AAV) delivered RPE65 construct.  Building on successful phase 1/2b trials from multiple centers, the AAV-hRPE65v2 agent now designated as voretigene neparvovec, contains a highly optimized enhancing sequence and promoter.

The main findings were an improvement on a multiple light level mobility test (MLMT) and multiple additional supportive secondary endpoints which included improvements in full-field light sensitivity, Goldmann visual field, and others.

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Eyedrop Kallikrein Inhibitor Shows Promise for Diabetic Macular Edema

MedicalResearch.com Interview with:
Dr. David Kita, PhD Founder and Head of R&D Verseon CorporationDr. David Kita, PhD
Founder and Head of R&D
Verseon Corporation

MedicalResearch.com: What is the background for this study?

Dr. Kita: The preclinical data presented at the 2017 BIO International Conference provided details about Verseon’s plasma kallikrein inhibitors for the treatment of diabetic macular edema (DME).

DME affects millions of people worldwide and is a major cause of vision loss in patients with diabetes mellitus. Upregulation of the kallikrein-kinin system in response to diabetes can result in retinal vascular permeability, which can damage the retina and eventually lead to the central vision loss associated with DME.

The current treatment options for DME include intravitreal injections of anti-VEGF agents or corticosteroids into the eye and surgical laser treatments. Long-term use of intravitreal injections is associated with side effects such as inflammation, infections, and cataracts. For anti-VEGF drugs in particular, there is also a growing concern about geographic atrophy. In addition, about 50% of patients reported at most moderate vision improvements following anti-VEGF therapy in clinical trials. This highlights the need for a new treatment that can serve as a monotherapy or as an adjuvant to current therapies.

At Verseon, we are working on inhibitors of the serine protease plasma kallikrein (KLKB1) that can be administered either topically or orally. Verseon’s unique computer-driven drug discovery platform allows us to design potent, selective drug candidates that are unlikely to be found using traditional approaches. We have generated a number of chemically distinct series of KLKB1 inhibitors and optimized multiple lead candidates, which show good activity, permeability, and solubility.

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New Gene Mutation Found to Cause Retinitis Pigmentosa in SW USA Hispanics

MedicalResearch.com Interview with:

Stephen P. Daiger, PhD TS Matney Professor of Environmental and Genetic Sciences Human Genetics Center, School of Public Health and Mary Farish Johnston Distinguished Chair of Ophthalmology Ruiz Dept. of Ophthalmology and Visual Science The Univ. of Texas HSC at Houston

Dr. Daiger

Stephen P. Daiger, PhD
Professor, Human Genetics Center
Thomas Stull Matney, Ph.D. Professor in Environmental and Genetic Sciences
Mary Farish Johnston Distinguished Chair in Ophthalmology
The University of Texas Health Science Center at Houston

 

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Thanks for your questions about our research.  My research group and I have a long-term interest in finding genes and mutations causing inherited retinal diseases.  Our main focus is on retinitis pigmentosa (RP) and, more specifically, the autosomal dominant form of RP.

Inherited retinal diseases are progressive, degenerative diseases of the retina.  Onset can be very early in life, even at birth, or much later in life.  As the degeneration develops an affected person may first experienced limited loss of vision, progressing to severe loss of vision, ending, in many cases, in legal or complete blindness.  About 300,000 Americans are affected by inherited retinal disease and 50% of these have RP.  RP, like most hereditary conditions, can be inherited in an autosomal dominant, autosomal recessive or X-linked fashion.

One of the surprising, and in some sense, disturbing findings in studying  retinitis pigmentosa is that mutations in many different genes can cause this disease.  We now know that mutations in more than 80 genes can cause RP and thousands of different mutations have been found in these genes.  With next-generations sequencing it is possible to find the cause of RP in from 50% to 80% of cases, depending on the underlying mode of inheritance.For example, in our research we can find the disease-causing mutation in about 75% of families with autosomal dominant RP.  Needless to say, a primary aim of our research is to find the cause in the remaining 25%.

In looking for the cause of retinitis pigmentosa in the remaining 25%, that is, those in whom mutations were not detected by earlier methods, we found a potential dominant-acting mutation in the arrestin-1 gene (gene symbol “SAG”) using whole-genome sequencing.  Molecular modeling suggests this mutation is damaging.  This was unexpected because previously-reported mutations in this gene were associated with Oguchi disease, a recessive retinal disease with symptoms distinct from RP.  On further testing our cohort of patients with autosomal dominant RP, we found this mutation in nearly 4% of families.  Even more surprisingly, when we looked closely at the affected families, and worked with our collaborators to test other patients, we discovered that all of the families with the dominant-acting SAG mutation — 12 total — were of Hispanic origin.  By interviewing informative family members we learned that these families have their roots in the Southwestern United States.  Historically, the mutation may have arisen hundreds of years ago, consistent with genetic variation tracking with the mutation.

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Lithium Use in Pregnancy and Risk of Fetal Cardiac Malformations

MedicalResearch.com Interview with:

Elisabetta Patorno, MD, DrPH Assistant Professor of Medicine, Harvard Medical School Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital

Dr. Patorno

Elisabetta Patorno, MD, DrPH
Assistant Professor of Medicine, Harvard Medical School
Division of Pharmacoepidemiology and Pharmacoeconomics,
Department of Medicine, Brigham and Women’s Hospital

MedicalResearch.com: What is the background for this study?

Response: Lithium, a widely used medicine to treat bipolar disorder, has been associated with a 400 fold increased risk of Ebstein’s anomaly, a congenital malformation of the heart, and a 5 fold increased risk of cardiac defects overall in infants when taken early in pregnancy, based on the results from the International Register of Lithium Babies in the 1970’s. Beyond this data, most of the information on the safety of lithium during pregnancy accumulated in the last 40 years is based on case reports and small studies with conflicting results. Despite these concerns and the limited information, lithium remains a first-line treatment for the 1% of women of reproductive age with bipolar disorder in the U.S. population, due to its recognized efficacy during pregnancy and the postpartum period, and due to the presence of a larger body of evidence showing increased risk of congenital malformations for other mood stabilizers, such as valproate.

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Phase I Study Shows IV Gene Therapy May Improve Macular Degeneration

MedicalResearch.com Interview with:

Prof Peter A Campochiaro MD Director, Retinal Cell and Molecular Laboratory Professor of Ophthalmology Johns Hopkins University School of Medicine Baltimore, MD

Dr. Campochiaro

Prof Peter A Campochiaro MD
Director, Retinal Cell and Molecular Laboratory
Professor of Ophthalmology
Johns Hopkins University School of Medicine
Baltimore, MD

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: Patients with wet age-related macular degeneration (AMD) have increased levels of vascular endothelial growth factor (VEGF) in their eyes resulting in growth of abnormal blood vessels that leak fluid into the retina and reduce vision. The current treatment is to inject proteins that block VEGF which initially provides a very good effect, but repeated injections are needed.

Patients sometimes are unable to keep up the frequency of visits and injections needed to keep the disease quiet and over time there is often gradual loss of vision. The aim of this study was to test a new approach through which a viral vector is injected into the eye resulting in production of a protein that block VEGF in the eye reducing the need for repeated injections.

These are the major findings:

1) Intravitreous injection of an AAV2 vector expressing a protein that blocks vascular endothelial growth factor (VEGF) was safe and well-tolerated.

(2) 5 of 10 patients injected with the highest dose (2 × 10¹⁰ vector genomes) had measurable levels of the therapeutic protein in samples removed from the front of the eye- all of these patients had no or very low levels of anti-AAV2 serum antibodies and 4 of the 5 patients who did not show expression had high anti-AAV2 serum antibodies

(3) Eleven patients had fluid in or under the retina before vector injection and 6 of them showed substantial reduction of the fluid which is the desired outcome.

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New Technology May Allow Topical Delivery of Anti-VEGF Drugs For Macular Degeneration

MedicalResearch.com Interview with:

Dr Felicity de Cogan PhD</strong> Institute of Inflammation and Ageing University of Birmingham

Dr Felicity de Cogan

Dr Felicity de Cogan PhD
Institute of Inflammation and Ageing
University of Birmingham

MedicalResearch.com: What is the background for this study?

Response: The University of Birmingham has a unique approach to developing technologies. By locating chemists, engineers, biologists and clinicians in the same department it revolutionised the way research problems are solved.

Initially, Felicity de Cogan was researching cell penetrating peptides (CPP) and their uses in microbiology. However, after joining forces with Neuroscientists, Dr Lisa Hill and Professor Ann Logan at the National Institute for Health Research Surgical Reconstruction and Microbiology Research Centre (NIHR SRMRC) together with the clinicians and Vision Scientists, Dr Mei Chen and Professor Heping Xu at the Queen’s University Belfast it became evident that there was huge potential to deliver drugs in the eye. This was the start of the project and it developed rapidly from there.

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