View the complete Missouri Regional Life Sciences Summit supplement from the Kansas City and St. Louis Business Journals. Click here to view.
Experts from basic, translational and clinical research, industry and private practice will discuss how the partnership of academic and business driven research creates novel and unique opportunities for translational medicine in the areas of diagnosis and therapy development for chronic disease of the eye and brain.
Peter Koulen, professor and Felix and Carmen Sabates Missouri Endowed Chair in Vision Research, University of Missouri-Kansas City
Nelson Sabates, chairman, Department of Ophthalmology, University of Missouri-Kansas City School of Medicine; president, Vision Research Foundation of Kansas City; president and chief executive officer of Sabates Eye Centers P.C.
Integrating academic research, clinical research and private practice: a unique opportunity for translational medicine: Degeneration or acute damage of nerve cells in the retina due to age-related macular degeneration (AMD), diabetic retinopathy and glaucoma are the major causes of visual loss and blindness in the United States and worldwide. It is estimated that over 9 million people over the age of 40 have early signs of AMD - the most common cause of decreased vision in people over 65. For almost 90% of these patients no effective treatment is available. Diabetic retinopathy also affects millions of individuals and the numbers are rising rapidly due to an increase in the incidence of juvenile and type 2 diabetes, similar to glaucoma, the most common cause of decreased vision in African-Americans. The determination of the function and effectiveness of new neuroprotective drugs and surgical treatment options has the potential to produce therapy approaches for these chronic degenerative diseases of the eye that focuses on the nerve cells of the retina and that has the potential to be both preventative and therapeutic in nature. As neurodegeneration of the retina affects significant and increasing portions of the U.S. population including minorities affected by disparities in health care delivery, determining causes, mechanisms of action and subsequently potential treatment strategies will contribute to improving health care, health and performance requiring visual tasks. With the formation of the new interdisciplinary Vision Research Center Kansas City is well on its way to becoming a national center for eye research where these ocular diseases and others are being researched to develop new therapy approaches urgently needed by physicians in the US and worldwide, transferring basic science findings seamlessly into practical use with patients. Collaboration between UMKC, companies, foundations supporting eye research and Kansas City Medical Centers offers an unprecedented interdisciplinary synergy with a unified goal: to better diagnose, prevent, and treat eye disease and vision disorders in order to make a difference in the lives of tens of millions of people worldwide. The Vision Research Center also provides significant opportunities for students and doctors in training at UMKC to work on innovative research studies and to receive state-of-the-art training in new therapy approaches for chronic neurodegenerative diseases of the eye.
Ashim K. Mitra, vice provost for interdisciplinary research, University of Missouri Curators' Professor of Pharmacy and chairman, Division of Pharmaceutical Sciences, School of Pharmacy, University of Missouri-Kansas City
Translating novel drug delivery technologies to ophthalmic products- Unique opportunity for collaboration between academia and pharmaceutical industry: To improve human health, scientific discoveries must be translated into clinical applications. Such discoveries typically begin at "the bench" with basic research — in which scientists study disease at a molecular or cellular level — then progress to the preclinical arena and ultimately to the patient's "bedside." Translational research has proven to be a powerful process that drives the clinical research engine. A strong collaboration among basic scientists in academia and the pharmaceutical industry is absolutely essential for the development of new ophthalmic products. Instillation of topical eye drops is the preferred and most convenient route of drug administration for treating ocular diseases. However, formulating mits the feasibility of producing aqueous formulation concentrations sufficient twater insoluble drugs for topical applications is challenging. Hydrophobicity lio achieve therapeutic levels in the posterior ocular tissues. To overcome these problems, a novel nanomicellar formulation of voclosporin, a calcineurin inhibitor, has been developed to treat dry eye syndrome in association with Lux Biosciences Inc. Moreover this mixed micellar formulation has the ability to deliver the drugs to posterior segment of eye. This formulation underwent Phase I and Phase II trials. This unique nanomicellar drug delivery platform presents potential opportunities for topical administration of additional hydrophobic drugs and the ability to non-invasively target retinal and other posterior segment diseases i.e., age related macular degeneration, diabetic retinopathy, diabetic macular edema and posterior uveitis.
Russell H. Swerdlow, professor of neurology, molecular and integrative physiology; director, KUMC neurodegenerative disorders program; University of Kansas School of Medicine; Landon Center on Aging
Of Mice and Men and the Search for an Alzheimer's Disease Treatment: Alzheimer's disease is a common affliction that disrupts the lives of millions and costs the country hundreds of millions of dollars. The federal government, state governments, philanthropic foundations, and industry have all committed resources to solving the Alzheimer's disease problem. Clinical and basic science investigators with a wide range of backgrounds have dedicated themselves to this disease. This talk will review the current state of Alzheimer's disease research and discuss where we stand in terms of a cure. This talk will try to predict where Alzheimer's disease research will go in the near future by considering where it's been in the recent past.
Daniel Lindgren, president, Ocuscience LLC, Kansas City
Comparative Ophthalmology: scientific discovery and innovation create synergies for veterinary and human medicine: Comparative ophthalmology is the study similarities and differences between humans and various animal species. Our understanding of these comparisons has yielded many breakthroughs in eye and neurological research of diseases and other causes of vision loss. Many technological innovations have been made which give doctors and scientists unprecedented insights to mechanisms of the eye and effects of treatment in animal models and humans. This presentation will briefly touch on recent discoveries in comparative ophthalmology. Some examples of new technologies being developed for commercialization in Missouri will be highlighted. Specifically, advancements in our ability to observe cellular structure and function of the eye and visual nervous system are moving from basic research to patient care.
Ann Smith, division of molecular biology and biochemistry, School of Biological Sciences, University of Missouri-Kansas City
Biochemistry and Neuroscience: Why basic science is critical for translational research and the life science industry (AKA Neuroprotective role of hemopexin): In the USA, every year more than ~800,000 people experience a stroke and one person has a stroke every 40 seconds on average (source: the American Heart Association). Ischemic stroke accounts for the vast majority of all strokes. Therefore, providing a better understanding of how to prevent the debilitating damage following ischemic stroke is important. It is even more vital to develop novel therapeutic avenues for the prevention and treatment of stroke. Combining basic science with pre-clinical models of stroke has already led to novel findings on the protection of the brain by the heme-binding protein hemopexin in ischemia-reperfusion and provided evidence for the first time that heme has a causal role in stroke damage. By further defining the mechanism whereby brain cells are protected from heme-related toxicity by hemopexin, we hope to develop molecules and/or tools that interact with or mimic components of the hemopexin system to protect brain cells and minimize brain injury in acute ischemic neurological disorders including stroke. Heme oxygenases are biological catalysts that break down heme, which is a biological form of iron, released when cells experience various types of stress (e.g., a clot or poor blood flow that lowers oxygen - termed ischemic injury). This damages neurons, other brain cells and red blood cells. We have provided evidence that heme oxygenases play an important role in protecting cells of the brain from heme toxicity in stroke as well as hemorrhage. Oxidative stress is produced as blood flow is restored to injured regions of the brain and elevated heme oxygenase-1 provides protection, which was shown using various models of injured neurons and a pre-clinical model of ischemic stroke in mice. Heme also contributes to damage from the inflammation that accompanies injury. Hemopexin is the main heme-binding protein that tightly binds heme in a manner that neutralizes its chemical activity. Consequently, hemopexin plays an important role as an anti-oxidant by controlling the levels of free heme outside cells. In addition, hemopexin has the unique ability to transport heme into cells by a process termed endocytosis that occurs when heme-hemopexin complexes bind to specific receptors at the cell surface. This heme is the substrate for heme oxygenases, and uptake of heme-hemopexin induces heme oxygenase-1 in neurons. Furthermore, unlike neurons cultured from the brains of normal mice those cultured from mice lacking heme oxygenase-1 are not protected by hemopexin from heme toxicity and from free radical damage. Thus, hemopexin plays a significant role in protecting neurons via heme oxygenase-1. Essentially very little is known about the regulation of heme uptake by cells and its degradation by the various cells of the brain or the role of extracellular heme-binding proteins for neuroprotection. Our recently published data obtained from hemopexin knock out- mice show that hemopexin protects the brain from heme toxicity and inflammation in an ischemia-reperfusion model of stroke. Our overall goal is to determine how hemopexin controls heme toxicity in ischemia-reperfusion injury of the brain in stroke and then to determine ways to develop novel beneficial therapies.
Dennis O'Brien, chancellor's chair in comparative neurology, College of Veterinary Medicine, University of Missouri-Columbia
Canine Neurodegenerative Diseases: A Bridge from Bench to Health for Humans and their Companions: Spontaneous neurologic diseases are common in dogs and frequently there is a genetic predisposition. Advances in canine genomics and the nature of pure-bred dog populations make it possible to efficiently map and sequence the genes responsible. In contrast to transgenic rodent models, investigations into spontaneous canine disease start with a recognized disease process and work from there to the contribution of genetics to that disease. Thus there is never a question whether the findings will be relevant to real world disease since that is where we start. Once a disease has been characterized and the genes responsible identified, either the clinical population or a research colony can be utilized to investigate the pathogenesis of disease or to conduct therapeutic trials. The larger size and complexity of the brain in dogs compared with rodents can make the canine model more predictive of outcome in human trials in modalities such as gene, stem cell, or enzyme replacement therapies. The Comparative Neurology Program at the University of Missouri, College of Veterinary Medicine has applied this approach to identify the genes responsible for numerous developmental and degenerative diseases of the nervous system. These include seizures disorders, inborn errors of metabolism, amyotrophic lateral sclerosis, and parkinsonism; and we continue to identify new diseases regularly through our clinical practice and our relationship with other veterinary neurologist and neurosurgeons throughout the world. We have partnered with industry and other institutions to translate these findings into improved diagnostic and therapeutic approaches that will improve both human and animal health.