Author Interviews, Emory, Genetic Research, JAMA, Ophthalmology / 24.01.2018

MedicalResearch.com Interview with: 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.   (more…)
Author Interviews, Cost of Health Care, JAMA, Ophthalmology, Surgical Research, UCSF / 23.01.2018

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. (more…)
Author Interviews, FDA, Genetic Research, Ophthalmology / 15.01.2018

MedicalResearch.com Interview with: 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.  (more…)
Author Interviews, JAMA, Ophthalmology, Pediatrics / 14.01.2018

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. (more…)
Allergan, Author Interviews, JAMA, Ophthalmology / 05.01.2018

MedicalResearch.com Interview with: Steven Woloshin, MD MS 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. (more…)
Author Interviews, Infections, Ophthalmology, Surgical Research / 14.12.2017

MedicalResearch.com Interview with: 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. (more…)
Author Interviews, Diabetes, JAMA, Ophthalmology, Technology / 13.12.2017

MedicalResearch.com Interview with: 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 (more…)
Author Interviews, Environmental Risks, NYU, Ophthalmology / 08.12.2017

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 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. (more…)
Author Interviews, JAMA, Surgical Research / 26.11.2017

MedicalResearch.com Interview with: Brian C. Stagg, MD Department of Ophthalmology and Visual Sciences University of Michigan Medical School National Clinician Scholars Program University of Michigan Institute for Healthcare Policy and Innovation Joshua D. Stein, MD, MS Associate Professor University of Michigan Department of Ophthalmology and Visual Sciences Director, Center for Eye Policy and Innovation Ann Arbor  MedicalResearch.com: What is the background for this study? What are the main findings? Response: Cataract surgery is one of the most common surgeries in the US. It is typically performed at either hospital outpatient departments (HOPDs) or ambulatory surgery centers (ASCs). ASCs are cheaper and more efficient, but some people believe that HOPDs may be safer for people with co-morbid medical conditions. We conducted this study to evaluate how the use of ambulatory surgery centers for cataract and other ocular surgeries has changed since 2001. We also wanted to see what factors influenced whether or not a patient had cataract surgery at an ASC (versus a HOPD), and to compare ASC use for cataract surgery with ASC use for other common eye surgeries (glaucoma, cornea, retina, strabismus). (more…)
Author Interviews, Genetic Research, Ophthalmology / 07.11.2017

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. (more…)
Author Interviews, BMJ, Ophthalmology / 19.09.2017

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. (more…)
Author Interviews, JAMA, Ophthalmology / 06.09.2017

MedicalResearch.com Interview with: 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. (more…)
Author Interviews, CDC, Infections, Ophthalmology, Pediatrics / 18.08.2017

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 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. (more…)
Author Interviews, JAMA, Ophthalmology, Pediatrics / 16.08.2017

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 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. (more…)
Author Interviews, Diabetes, JAMA, Ophthalmology, UC Davis / 30.07.2017

MedicalResearch.com Interview with: 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. (more…)
Author Interviews, Cancer Research, Dermatology, Ophthalmology / 24.07.2017

MedicalResearch.com Interview with: 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. (more…)
Author Interviews, Genetic Research, Lancet, Ophthalmology / 17.07.2017

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 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. (more…)
Author Interviews, Diabetes, Ophthalmology / 07.07.2017

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. (more…)
Author Interviews, Lancet, Macular Degeneration, Ophthalmology / 18.05.2017

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 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. (more…)
Author Interviews, Macular Degeneration, Ophthalmology, Technology / 17.05.2017

MedicalResearch.com Interview with: 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. (more…)
Author Interviews, Genetic Research, Ophthalmology / 15.05.2017

MedicalResearch.com Interview with: Zheng-Rong Lu, Ph.D. M. Frank Rudy and Margaret Domiter Rudy Professor of Biomedical Engineering Department of Biomedical Engineering Case Western Reserve University Cleveland, OH 44106 MedicalResearch.com: What is the background for this study? What are the main findings? Response: Genetic vision disorders are a major cause of severe vision loss and blindness, especially in children and young adults. Currently, there are no approved therapies to treat these types of disorders. This study focused on one such disease known as Leber’s congenital amaurosis type 2 (LCA2). Patients with LCA2 are born with some degree of vision loss, and are often legally blind by early adulthood. LCA2 is a recessive disease caused by a mutation in one of the genes responsible for visual processing. LCA2 is a good candidate for gene therapy, and clinical trials underway to test viral vectors that deliver a healthy copy of the mutated gene into the eye have demonstrated considerable therapeutic efficacy. These trials have validated the feasibility of gene therapy to treat this disease, however viral vectors are limited by potential safety issues, complex preparation methods, and limitations on the size of genes that can be delivered. In this study, we successfully treated LCA2 in mice for 120 days by delivering the gene responsible for LCA2 in a synthetic lipid nanoparticle instead of a viral vector. Our delivery system, called ECO, specifically targets the cells in the retinal pigmented epithelium, where the mutation behind LCA2 occurs. Our nanoparticle delivery system is easy to produce, safe, and has unlimited cargo capacity. Most important, our nanoparticle gene delivery system is a platform that can be used to deliver any gene into the retina, opening the door for safe and effective gene therapy for any genetic vision disorder. (more…)
Author Interviews, JAMA, Ophthalmology / 08.05.2017

MedicalResearch.com Interview with: John H Kempen, MD, PhD Protocol Chair, MUST Trial Follow-up Study; Vice Chair, MUST Research Group Director of Epidemiology for Ophthalmology, Massachusetts Eye and Ear Harvard Medical School Editor-in-Chief, Ophthalmic Epidemiology President, Sight for Souls MedicalResearch.com: What is the background for this study? Response: Uveitis is about the fifth leading cause of blindness in the united states.  Among types of uveitis, intermediate, posterior and panuveitis are the leading causes of blindness.  Before 2005, systemic therapy with corticosteroids—supplemented when indicated with immunosuppressive drugs (most of the time)—was the primary treatment recommended for these conditions.  With approval of the fluocinolone acetonide implant in 2005 for intermediate, posterior and panuveitis, it became unclear which of the alternative treatment approaches should be the treatment of choice. The multicenter uveitis steroid treatment (must) trial was initiated in 2005 to directly compare the alternative treatments.  Systemic therapy was administered using high dose prednisone followed by tapering of corticosteroids to maintenance doses of 10 mg/day or less (generally 7.5 mg/day or less) or to zero; this was supplemented by immunosuppressive corticosteroid-sparing drugs in 88% of participants.  Implant therapy was done by initial quieting of the anterior chamber of the eye with topical, injected or systemic corticosteroids followed by surgical implant placement within 28 days (first eye) and 56 days (second eye if it was indicated).  After this, systemic corticosteroids and immunosuppressive drugs were tapered off. (more…)
Author Interviews, JAMA, Ophthalmology, Pediatrics / 05.05.2017

MedicalResearch.com Interview with: Rohit Varma, MD, MPH Executive Director - USC Roski Eye Institute and Dean of the Keck School of Medicine of USC MedicalResearch.com: What is the background for this study? What are the main findings? Response: Visual impairment in early childhood has profound impact on a child’s development. It can significantly impair development of visual, motor, and cognitive function1-3 and lead to adverse psychosocial consequences. There has been a lack of accurate data characterizing the current and expected numbers of visual impairment cases among preschool children in the United States from 2015 to 2060. The number of preschool children with visual impairment is projected to increase by 26% in 2060. And 69% of these visual impairment will result from simple uncorrected refractive error such hyperopia and myopia, which can be prevented or treated by low-cost refractive correction. (more…)
Author Interviews, JAMA, Macular Degeneration, Ophthalmology, Primary Care / 01.05.2017

MedicalResearch.com Interview with: David C Neely, MD The University of Alabama at Birmingham MedicalResearch.com: What is the background for this study? What are the main findings? Response: This study examined the prevalence of eyes with age-related macular degeneration (AMD) in patients seen in primary eye care clinics who purportedly have normal macular health. Approximately 25.0% of eyes deemed to be normal based on dilated eye examination by primary eye care providers had macular characteristics that indicated age-related macular degeneration. (more…)
Author Interviews, Ophthalmology / 23.04.2017

MedicalResearch.com Interview with: Kang Zhang, M.D., Ph.D. Professor of Ophthalmology Chief, Ophthalmic Genetics Founding Director, Institute for Genomic Medicine Co-Director, Biomaterials and Tissue Engineering, Institute for Engineering in Medicine Board Certification in Ophthalmology Fellowship in Vitreoretinal Disease and Surgery Guangzhou Women and Children's Medical Center Guangzhou Medical University Guangzhou China MedicalResearch.com: What is the background for this study? What are the main findings? Response: Retinitis pigmentosa is a common blinding condition characterized by mutations in rod photoreceptor specific genes, night blindness and tunnel visual with eventual loss of day vision. Since it can be caused by numerous different mutations in many genes therefore it has been difficult to provide treatment benefits to a majority of patients. Traditional gene therapy has been in a piece-meal fashion, meaning to create a therapy for a particular gene or mutation. In this paper, we describe a universal gene therapy approach using the latest gene editing technology CRISPR/CAS9 to reprogram rod photoreceptors to cone photoreceptors with reversal of RP and restoration of vision. (more…)
Addiction, Author Interviews, Infections, JAMA, Ophthalmology / 22.04.2017

MedicalResearch.com Interview with: Aubrey Tirpack, PGY3 New England Eye Center Tufts Medical Center  MedicalResearch.com: What is the background for this study? What are the main findings? Response: Intravenous drug abuse is a known risk factor for the development of endogenous fungal endophthalmitis (EFE), a severe intraocular infection cause by the seeding of mycotic organisms to the eye. Our institution noted a marked increase in cases of EFE beginning in May 2014, which correlates to increasing rates of opioid abuse throughout the New England region. Ten patients were found to have intravenous drug abuse related EFE over the two year time period studied. The most common presenting symptoms were floaters, decreased vision, and pain. All patients were treated with systemic antifungals and nine patients underwent intravitreal antifungal injection. All patients were ambulatory at presentation and the majority were without systemic signs of infection. (more…)
Author Interviews, Inflammation, JAMA / 20.04.2017

MedicalResearch.com Interview with: Andrew Dick FRSB FMedSci Professor of Ophthalmology Bristol Eye Hospital, University of Bristol, Bristol, England National Institute for Health Research Biomedical Research Centre, Moorfields Eye Hospital, Institute of Ophthalmology, University College London, London, England and John Sheppard, MD President, Virginia Eye Consultants Professor of Ophthalmology, Eastern Virginia Medical School MedicalResearch.com: What should readers take away from your report? Dr. Andrew Dick: These findings demonstrate that adalimumab is associated with clinically meaningful improvements in visual functioning for patients with non-infectious intermediate uveitis, posterior uveitis, and panuveitis. The emphasis of this work is that for the first time in uveitis we have seen patient reported outcome benefit of a biologic treatment. This analysis supports the use of adalimumab as a promising new treatment option, having demonstrated improvements in both clinical and visual functioning outcomes in patients with active and inactive uveitis. Dr. John Sheppard: Uveitis has a substantial effect on individuals’ physical, professional, psychological, avocational and social functioning in day-to-day life. Adalimumab, an anti-inflammatory drug that binds to tumor necrosis factor, was recently approved for the treatment of non-infectious intermediate uveitis, posterior uveitis, and panuveitis. It is the first systemic therapy specifically approved for uveitis.  The analyses in this study provide evidence that patients with noninfectious uveitis treated with adalimumab experience significant and clinically meaningful improvements in vision-related quality of life, compared with those who received placebo. (more…)
Author Interviews, Diabetes, NEJM, Ophthalmology, Personalized Medicine / 20.04.2017

MedicalResearch.com Interview with: John M. Lachin, Sc.D. Research Professor of Biostatistics and of Epidemiology, and of Statistics The George Washington University Biostatistics Center and David Matthew Nathan, M.D. Professor of Medicine, Diabetes Unit Massachusetts General Hospital  MedicalResearch.com: What is the background for this study? Response: Traditional guidelines for screening for retinopathy, based on indirect evidence, call for annual examinations. The automatic annual screening for retinopathy, without considering potential risk factors for progression,  appears excessive based on the slow rate of progression through sub-clinical states of retinopathy. (more…)
Addiction, Author Interviews, BMJ, Ophthalmology / 17.04.2017

MedicalResearch.com Interview with: Dr. Rebecca Rewbury Sussex Eye Hospital Brighton and Sussex University Hospitals Trust Brighton, UK MedicalResearch.com: What is the background for this study? What are the main findings? Response: ‘Poppers’ are recreational drugs which are illegal to sell for human ingestion, but are sold under the guise of household cleaning products. Inhalation leads to a brief sense of euphoria, enhanced sexual arousal and smooth muscle relaxation. The Psychoactive Substances Act 2016 was due to outlaw poppers, but they were excluded on the basis that they do not act directly on the central nervous system. The main constituent of poppers, isopropyl nitrite, replaced isobutyl nitrite when the latter was classified as a carcinogen in 2006. Since then, there have been several case reports of ‘poppers maculopathy.’ We noted an increase in patients presenting with central visual disturbances after using poppers and describe 12 such cases. They all demonstrated similar disruption of the photoreceptor layer on retinal imaging. Onset of symptoms was frequently linked to specific brands of poppers, with 3 people having used poppers for many years and only developing side effects on changing brand. Chemical analysis showed that these products contained isopropyl nitrite. One brand of poppers, used without side effects by one patient, contained amyl nitrite, 2-methyl butyl nitrite and isobutyl alcohol, but no isopropyl nitrite. The outcome of poppers maculopathy varied, but following abstention, visual disturbances and retinal damage tended to improve over months, if not fully resolve. Although in some cases, symptoms and/or imaging findings were prolonged. Ongoing use of implicated brands led to persistent, but not worsening maculopathy, whereas one patient that switched back to another brand showed full recovery. (more…)