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New York State Stem Cell Science

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Planning Grants for Emerging Opportunities and Consortia Development for Stem Cell Research

RFA No. 0802071100
Sponsoring Institution PI Amount Title
Albert Einstein College Of Medicine Eric Bouhassira $120,000 New York Blood Disease Consortium
Brookhaven National Laboratory Fritz A. Henn $76,800 Planning Collaborative Research between Cold Spring Harbor Laboratory, Stony Brook School of Medicine and Brookhaven National Laboratory
Cold Spring Harbor Laboratory David J. Stewart $118,920 Cold Spring Harbor Stem Cell Training Program
Columbia University - Morningside Gordana Vunjak-Novakovic $119,960 Molecular, Genetic and Biophysical Regulation of Human Stem Cells for Medical Impact
Columbia University Medical Center James E. Goldman $120,000 Stem Cell Biology: Novel Insights into Therapeutic Treatment of Human Disease
Cornell University Alexander Yu Nikitin $120,000 Stem Cells, Microenvironment and Cancer
Montefiore Medical Center Sanjeev Gupta $118,180 Liver Cell Transplantation
Mount Sinai School of Medicine Ihor R. Lemischka $120,000 Systems Biology and Stem Cells: An Integrated Multi-Disciplinary Strategy
New York Medical College Thomas H. Hintze $119,705 Translational Cardiovascular Stem Cell Consortium
The New York Stem Cell Foundation Susan L. Solomon $119,797 Design of a New York Stem Cell Foundation Stem Cell Screening Lab
Ordway Research Institute Stewart Sell $85,409 Stem Cells and Aging
Regenerative Research Foundation Sally Temple $120,000 Retinal Stem Cell Consortium
Roswell Park Cancer Institute Andrei Gudkov $120,000 Pharmacological Targeting of Stem Cells
SUNY Downstate Medical Center Olcay Batuman $120,000 SUNY Downstate Vascular Stem Cell Genome Consortium
SUNY Stony Brook University Ira S. Cohen $118,800 Mechanical and Electrical Regeneration of Heart With Stem Cells
SUNY University at Albany James Fossett $119,948 New York Institute for Ethical Stem Cell Research (NYIESCR)
University of Rochester Mark D. Noble $76,751 Clinical Translation in Stem Cell Medicine: From Principles to Practice
Weill Medical College of Cornell University M. Flint Beal $117,127 Development of a Safe and Effective Stem Cell-Based Therapy of Parkinson's Disease

New York Blood Disease Consortium

Eric Bouhassira, Ph.D.
Albert Einstein College Of Medicine

We propose here to establish a consortium for blood cell disease that will help develop stem cell and blood diseases research in New York State. The general objective of this planning grant is to create a large consortium that will be centered on the creation of three main shared research facilities to provide essential services to all New York stem cell scientists. A planning team will work in the next few months to develop the concepts that are briefly summarized below. The creation of shared facilities will eliminate duplication of services at each university and would foster collaboration between research groups that often compete with each other. The consortium will also provide stem cell scientist with cells, animals and techniques that they currently cannot access because of a lack of specific equipment or expertise. The first facility that the planning team will consider is a stem cell production facility that will produce, bank and distribute stem cells from patients with hereditary blood diseases. This facility will also differentiate the stem cells into terminally differentiated cells and distribute them to investigators. This is an important function because it is often difficult to obtain tissues from rare diseases for study and for development of therapies. The second proposed facility is an animal model facility. Although studying human cells is essential to develop cures to blood diseases, much can be learned by studying rodent models of human diseases. We propose here to create a facility that will help breed these mice and distribute them to all researchers who need them. The planning team will also develop a proposal to create a bioinformatics group that will specialize in blood diseases. The group will maintain a web site to integrate all data related to blood diseases, and will create or maintain tools to help investigators take maximal advantage of the data.

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Planning Collaborative Research Between Cold Spring Harbor Laboratory, Stony Brook School of Medicine and Brookhaven National Laboratory

Fritz A. Henn, M.D., Ph.D.
Brookhaven National Laboratory

This proposal will bring together investigators from Cold Spring Harbor Laboratory, Stony Brook School of Medicine and Brookhaven National Laboratory to plan and implement a program of cross-cutting research and education in the area of stem cell biology. This program will develop a plan to educate both specialized researchers and students across multiple disciplines to allow the development of true stem cell translational research programs. The areas will range from molecular and cellular approaches to understand stem cell function and differentiation, through in vivo transplantation and imaging technology to follow the integration of stem cells into differentiated tissue, and, finally, an analysis of functional changes due to stem cell integration into specific organs.

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Cold Spring Harbor Stem Cell Training Program

David J. Stewart, Ph.D.
Cold Spring Harbor Laboratory

Recent years saw great progress in our understanding of the biology of stem cells. The properties and behaviors of stem cells are currently an area of intense research focus because of the great clinical potential for stem cell therapies in a variety of diseases. The Empire State Stem Cell Board recently identified training and education as a major challenge for both the future of fundamental stem cell research and the development of potential biomedical and biotechnological applications. Building on a long history of biology education, the Stem Cell Training Program at Cold Spring Harbor Laboratory will provide advanced training, education and focused high-level discussion through the development of laboratory courses, training workshops and small discussion meetings. The planning phase of this program will include two planning meetings associated with major international stem cell conferences in the summer and early fall of 2009 intended to foster program design and curriculum development for the proposed course and small meeting series, together with a trial two-day workshop on fundamentals in stem cell biology, which will immediately precede the Cold Spring Harbor fall conference on Control & Regulation of Stem Cells.

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Molecular, Genetic and Biophysical Regulation of Human Stem Cells for Medical Impact

Gordana Vunjak-Novakovic, Ph.D.
Columbia University - Morningside

Perhaps more than any other area of biology or medicine, stem cell research spans a wide range of disciplines and is driven by the urgent need to identify ways to harness these versatile cells, whose ultimate applications may be well beyond what is currently imaginable. There is a pressing need to push the current limits of stem cell research by merging basic stem cell research with cutting-edge technological advances. The NYSTEM Program is responding to this need by supporting the establishment of consortia for interdisciplinary research that can rapidly advance fundamental understanding of stem cells and the much-needed translation of scientific results into new options for treating human disease. We believe that the path towards the application of stem cells in regenerative medicine (to improve human life), studies of disease (to expedite drug discovery), and fundamental research (to further our understanding of the unique properties of stem cells) leads through an interdisciplinary and integrated effort. This planning grant will support the formation of a consortium for fundamental and translational studies of the molecular, genetic and biophysical regulation of human stem cells. The focus is on human embryonic stem cells, with possible extensions to other cell types of interest, including stem cells from adult organisms and induced pluripotent stem cells. We propose to study human stem cells using a new generation of culture systems and tools that will help to unlock their full biological potential. Our strategic plan for building the research team and linking the partner institutions will be developed through activities with strong educational and training components, and far-reaching effects. Working together, we aim to develop a consortium that will address, in the most efficient way, the current and future needs in stem cell research, clinical translation, training and education of a new generation of stem cell scientists.

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Stem Cell Biology: Novel Insights into Therapeutic Treatment of Human Disease

James E. Goldman, M.D., Ph.D.
Columbia University Medical Center (CUMC)

The interests, expertise and current research of CUMC investigators and our Consortium partners cover broad and deep areas of stem cell biology, relevant to many organ systems and diseases. There are more than 60 laboratories at CUMC with interests in basic and clinical stem cell research, involving all areas of basic science and medicine. Several groups are pursuing "translational" research vigorously, using stem/progenitor cells to intervene in diseases. There are common themes and technologies among these various fields, but we have not had a coordinated structure within which to integrate research efforts and thus make each investigator's work proceed more effectively. With so many investigators in so many fields interested in stem cell work, we critically need an organization to promote cooperation among laboratories, sharing of technology, education and rapid communication of advances. This application is to plan a large Stem Cell Consortium with CUMC and several partners, whom we have chosen preliminarily because of ongoing collaborations and shared scientific interests. These include 1. The New York Neural Stem Cell Institute, 2. Memorial Sloan-Kettering Institute, 3. Harvard University, 4. The New York Stem Cell Foundation, and 5. Project ALS (Amyotrophic Lateral Sclerosis and other diseases of Motor Neurons). We will use planning grant resources over the next year to convene a series of workshops to plan this Consortium to promote inter- and intra-institutional cooperation and provide a means for rapid transmission of technical advances in the stem cell field. The major motivating force is to enhance the work of all investigators in the Consortium. We will also consider additional partners in the Consortium, if appropriate.

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Stem Cells, Microenvironment and Cancer

Alexander Yu Nikitin, M.D., Ph.D.
Cornell University

The Stem Cell Program of Cornell University, Ithaca, New York, the Roswell Park Cancer Institute (RPCI), Buffalo, New York, the Yale Stem Cell Center, Yale University School of Medicine, New Haven, Connecticut, and the Eli and Edythe Broad Center for Regenerative and Stem Cell Research at the University of Southern California (USC), Los Angeles, California will develop a consortium entitled "Stem Cells, Microenvironment and Cancer." This proposal is built on a unique synergistic combination of major strengths of Cornell University, such as strong comparative medicine and unparalleled interface of biomedical sciences with other disciplines, with outstanding capabilities of other participating institutions, such as access to clinical materials and trials, expertise of work with reprogrammed pluripotent cells, and high throughput screening and validation technologies. A number of intra- and inter-institutional collaborations among investigators of participating institutions have already formed and their further development will benefit tremendously from strengthening inter-institutional links. The formation of the consortium is also essential because new exciting opportunities for further collaborations among investigators of participating institutions were identified. Furthermore, there are numerous needs in exchange of services of institutional cores, development of new centralized cores and cross-institutional training in stem cell-related technologies. We will develop a consortium that will leverage the complementary expertise of participating institutions to address the roles of intra- and extracellular cues in regulation of stem cells, their directed differentiation, and prevention of malignant transformation. This will invigorate advancements in stem cell research and more immediate application of the newly gathered knowledge to clinical practice.

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Liver Cell Transplantation

Sanjeev Gupta, M.D.
Montefiore Medical Center

Our planning team will develop this consortium to focus on aspects of stem cell biology, cell therapy, organ transplantation, hepatology, hematology, virology and immunology, which will enhance systematic approaches with stem cells for disease-specific therapies. The liver plays central roles in the body for metabolism, synthesis of essential proteins, removal of waste products and toxins, and disposal of drugs. Changes in liver function due to genetic abnormalities, infections, e.g., chronic viral hepatitis, alcohol, hepatotoxins, drugs, etc., produce diseases that may affect the liver itself or alter the function of other organs. Animal studies over many years provided fundamental new insights into how the liver is repopulated with transplanted healthy cells. In this way, liver-directed cell and gene therapies offer exciting opportunities for making life-altering or life-enhancing impacts in people. For instance, viral hepatitis alone affects approximately 400 million people worldwide, many developing progressive liver disease and fatal liver failure. Similarly, a large number of genetic and other diseases may be curable by replacing healthy cells in the liver with cells that produce molecules that are missing. A notable example is hemophilia A, which was recently cured in mice using liver cell transplantation. Although one may use adult human livers as cell donors, human organs are extremely scarce. This necessitates alternative approaches, such as cell therapy with stem cells. Many types of stem cells have captured our imaginations. Among these, fetal liver cells are of particular interest for early cell transplantation studies in people because these cells exhibit a number of desirable properties, including their ability to restore liver function without requiring manipulations that may induce risks for cancer formation. Better insights into the biological nature of embryonic stem cells and generation of patient-specific stem cells without cancer risks add to the alternatives. We consider that development of cell therapy programs will require cooperative and coordinated efforts of experts in several disciplines, irrespective of the cell types used for transplantation. Therefore, this initial proposal will identify infrastructural and other needs for cell therapy with liver stem cells.

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Systems Biology and Stem Cells: An Integrated Multi-Disciplinary Strategy

Ihor R. Lemischka, Ph.D.
Mount Sinai School of Medicine

Stem cell research will have an impact on medicine in the 21st century as great as antibiotics and radiology in the 20th century. Before scientific breakthroughs in stem cell research are translated into clinical benefits, a complete understanding of the cellular and molecular mechanisms that control stem cells will be required. Such an understanding is required to enable the design of superior diagnostic and therapeutic methodologies, without untoward deleterious complications. To date, much has been learned regarding the basic properties of both tissue-specific and embryonic stem cells. However, a "deep" understanding of stem cell regulatory mechanisms has not been obtained. In our Consortium we will adopt quantitative and rigorous Systems Biology approaches and methodologies to obtain fundamental knowledge about how stem cell decisions are controlled. Our results will provide a sound foundation for the extension of basic aspects of stem cell research into medical applications. We propose to develop a new avenue in stem cell research that will provide a sound and quantitative view of how the biological properties of these cells are regulated. Our overall approach embraces Systems Biology, a relatively new paradigm in biological research. We assembled an outstanding team of investigators from numerous institutions within the State of New York (Mount Sinai School of Medicine, New York University, Rockefeller University, Albert Einstein School of Medicine, Weill Cornell School of Medicine, University of Rochester) and Princeton University in New Jersey.

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Translational Cardiovascular Stem Cell Consortium

Thomas H. Hintze, Ph.D.
New York Medical College

Over the past 10 years there was an explosion of information related to the identification, function and potential therapeutic use of stem cells in medicine in general and cardiovascular medicine in particular. Much of this work centered upon understanding the cell and molecular biology under which these cells operate, with initial studies of medical benefit performed in small rodents including mice and rats. Whereas those studies will show proof of principle, it is absolutely necessary to develop mechanisms more directly related to testing potential therapies that will ultimately be used in humans. The goal of our Translational Cardiovascular Stem Cell Consortium in New York State is to provide that bridge leading directly to the testing of potential therapies for cardiovascular disease in patients. Thus, we began to assemble experts in cardiovascular function and medicine, from cell and molecular biologists to cardiovascular systems biologists to clinicians, well versed in conducting trials of therapies in patients, to establish a platform upon which we will pursue cures for cardiac and vascular disease using stem cells in humans. The goals of our Consortium will be to use cutting-edge science to better understand stem cell biology, to expedite the use of stem cells in the treatment of cardiovascular disease, and to translate the knowledge we gain into limited clinical trials in humans.

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Design of a New York Stem Cell Foundation Stem Cell Screening Lab

Susan L. Solomon, J.D.
The New York Stem Cell Foundation

Developing new stem cell-based therapeutics requires different specialized technologies. One of the most useful relies on the ability to establish cell culture systems that model human diseases faithfully and on the application of these systems to screens that can identify new types of therapeutics. We propose to establish such a screening facility at the New York Stem Cell Foundation (NYSCF). The NYSCF laboratory is a center for stem cell derivation and characterization open to researchers from all institutions. The current New York State initiative to provide funding for stem cell research will generate a wealth of scientific data and potential targets for drug development that require highly specialized screening and informatics analysis. NYSCF proposes to meet this increasing need by configuring a collaborative laboratory that will allow New York State scientists to plan and perform stem cell-based screens to accelerate the discovery and development of new treatments for serious diseases. In this way, NYSCF will provide a comprehensive and self-contained facility for the derivation, differentiation and specialized analysis of disease-specific cell lines that is available to all stem cell researchers. In order to set up a facility that will adequately represent the needs of potential users of such a core screening facility, we will convene a series of meetings around New York State. The objective of the meetings is to assess the needs of the user group and, based on those needs, to estimate the amount and type of equipment, the type and size of small molecule screening libraries, and the number of personnel that will be required over the next one to three years. The overall goal is to produce a plan and a budget for a NYSCF screening lab that will be a resource for New York State stem cell biologists and that will operate in collaboration with the greater pharmaceutical and biotechnology community.

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Stem Cells and Aging

Stewart Sell, M.D.
Ordway Research Institute

Aging is a process that involves all individuals in New York State, and, in fact, the world. As the worldwide population ages, the need to identify strategies that will prolong healthy aging is intensifying. One intervention is the use of tissue stem cells to replenish worn-out, dead or non-functional cells that result in loss of function associated with decreased quality of life in elderly people. In order to use stem cells to restore youthful levels of function, significant and wide-ranging problems that currently prevent translating animal studies into relevant human interventions need to be solved. Solving these problems will require the concerted efforts of researchers with specific expertise who currently are either unaware of or unable to share what they know with others. Much of this lack of communication is due to logistical constraints that are part of the nature of academic science as it is practiced today. The goal of this consortium is to remove these barriers. The activities of this consortium will not only increase the collaboration of senior scientists working on different aspects of aging, but also train the next generation of young investigators. The goal of this application is to have the investigators from participating laboratories cooperate to formulate ways that the different experimental models employed by each can be exploited to expand our knowledge of aging. As part of the planning of the consortium we hope to identify clinical participants in our program to encourage translational application of our findings.

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Retinal Stem Cell Consortium

Sally Temple, Ph.D.
Regenerative Research Foundation

Retinal disease is a significant burden to patients, their families and to the economic health care budget. In the United States, one in five people over age 75 has Age-Related Macular Degeneration (AMD), which affects 11 million individuals and is the leading cause of blindness in the country. As the population ages, the prevalence of AMD is increasing. Diabetic retinopathy is also a highly prevalent disorder that leads to debilitation and blindness. There are several genetic disorders that cause severe loss of vision, such as retinitis pigmentosa and cone dystrophy, and some lead to formation of retinal tumors such as retinoblastoma. Loss of sight is one of the most feared medical conditions in our culture, and as yet there are relatively few treatments that can lead to sight restoration. The retina is part of the central nervous system (brain and spinal cord), and as such the tissue is delicate and complex. Moreover, the retina has only limited powers of repair, so that therapeutics must be developed to either prevent retinal degeneration or to aid in the repair process, for example by finding ways to replace lost and damaged cells. Stem cell research can help achieve both these goals, by providing cells that can be used to study retinal disease in culture dishes and thus identify novel therapeutic agents, or to create a safe cell source for cell replacement therapies. We present a proposal to create a consortium of researchers and scientists whose focus is to generate stem cell-based therapies to treat retinal disease and other causes of blindness. A consortium is needed to accomplish goals that are unachievable by individual scientists, given the complexity and multi-faceted nature of the problem. Our long-term objective is to provide new treatments for blinding retinal diseases. We will bring together individuals with a variety of important skills: leading stem cell scientists, retinal researchers, ophthalmologists and representatives of biotechnology companies with an interest in retinal stem cell research who understand how research can be brought towards commercial application and to the clinic.

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Pharmacological Targeting of Stem Cells

Andrei Gudkov, Ph.D.
Roswell Park Cancer Institute

Successful treatment of cancer in individual patients is a tenuous balance between killing the cancer and killing the patient. Stem cells, both the benign stem cells responsible for maintenance and repopulation of blood components, the intestinal tract and hair follicles, and the cancer stem cells of solid tumors or hematologic malignancies, are now understood to be the critical targets in determining the therapeutic index, the effectiveness of selectively killing the cancer versus killing the organism. The mission of the projected Consortium is to coordinate and facilitate development of new therapeutic agents and approaches for stimulation and protection of normal stem cells and selective killing of tumor progenitor cells. We expect to establish new therapies and agents that improve significantly the therapeutic index of cancer treatment and prophylaxis. This Consortium will integrate the efforts of a multi-investigator, multi-institutional team of researchers, clinicians and facility directors from internationally recognized centers of academic research and translational research, led by Roswell Park Cancer Institute (RPCI). The Consortium institutions include RPCI, The University of Rochester, Cornell University and the State University of New York at Buffalo. Basic and pre-clinical findings at the participating research centers will be used for development of bioactive molecules that target normal or tumor stem cells (for tissue protecting and anticancer applications, respectively). After pharmacological optimization, toxicological assessment and large-scale manufacturing, they will be translated rapidly to clinical trials at our clinical facilities and vetted for commercial potential through allied biotech companies, Cleveland Bio Labs and AndroBioSys, which have received NIH/DOD funding for applied research targeting benign and malignant stem cells. The Consortium will make this drug discovery-development pipeline possible by gathering a critical mass of investigators with complementary expertise, coordinating their effort, providing infrastructural components necessary for successful development of drugs for protection of benign stem cells and selective inactivation/killing of tumor stem cells, and maintaining focus on clinically relevant targets.

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SUNY Downstate Vascular Stem Cell Genome Consortium

Olcay Batuman, M.D.
SUNY Downstate Medical Center

Uncontrolled blood vessel formation is a leading common pathogenic mechanism both in bone marrow cancers such as multiple myeloma, and in eye disorders such as macular degeneration and retinal degenerations resulting from diabetes and sickle cell anemia. Exciting research advances show that targeting uncontrolled vascular growth can have significant beneficial effects in these disorders. Our recent results in genetically engineered mice and in patients with multiple myeloma implicate key similarities in mechanisms responsible for dysregulation of vascular stem cell development that govern these disorders. However, the processes underlying the initiation, inhibition or reversal of vascular stem cell development from earlier (less committed) stem cells, and the role of molecular and genetic alterations accompanying the course of these disorders, remain unclear. Greater knowledge of these mechanisms will inform treatment strategies, both new and current, and will increase our understanding not only of blood cancers and blinding diseases, but also of a vast array of other debilitating disorders that share vascular dysfunction. It is important to note that control of pathological blood vessel formation prevents neuronal cell death in the retina and presumably other tissues. The proposed consortium will develop a comprehensive translational research program that investigates vascular stem cell pathology as it relates to eye diseases and bone marrow cancers. We will proceed by identifying shared and disease-specific vascular stem cell markers; this information will be implemented for repair of the retina in blinding diseases by transplantation of optimized stem cells, as well as to develop new techniques of targeting abnormal vascularization in blood cancers and eye diseases. The planned consortium is envisioned to develop a multi-site synergistic, cooperative and interactive stem cell facility, which, in ways not available now, will benefit patients and create educational and scientific opportunities for upcoming student scientists as well as established researchers and clinicians.

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Mechanical and Electrical Regeneration of Heart With Stem Cells

Ira S. Cohen, M.D., Ph.D.
SUNY Stony Brook University

In 2003, 71.3 million Americans suffered from some form of cardiovascular disease. The single major cause of this is coronary artery disease, which afflicts 13.2 million Americans. Of these, 911,000 (67,700 New Yorkers in 2002) succumbed to their cardiovascular problems, making it the number one cause of death in the United States and New York State. Our consortium aims to develop a plan to determine the mechanisms for successful stem cell therapies for mechanical and electrical regeneration of the heart. This plan will have two objectives. When a billion or more cardiac myocytes die, the heart fails as a mechanical pump. The first objective is to develop stem cell therapy for the treatment of heart disease. Multiple stem cell types have been studied as cardiac myocyte replacement cells, each with limited success. The second objective is to understand the causes of and develop cell-based therapies for arrhythmias. Although treatment for slow heart rates with pacemaker therapy is effective, it is not optimal. Recently, we showed proof of principle of biological pacemakers in animal studies in vivo. However, as with the repair of failed heart, multiple cell types have been employed and no comparisons of their efficacies have been made. We will also expand the stem cell training of young investigators by having them experience both embryonic and adult stem cell biology. This project represents collaboration between investigators at two New York institutions, Columbia University and Stony Brook University. The PI and co-PI have collaborated for 25 years on a program grant from the NHLBI. Thus the basics of the inter-institutional collaboration are already in place. In the proposed consortia, we will be bringing in investigators from Worcester Polytechnic Institute in Massachusetts and the Technion in Israel, thus promoting intrastate, national and international collaborations in stem cell research.

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New York Institute for Ethical Stem Cell Research (NYIESCR)

James Fossett, Ph.D.
SUNY University at Albany

Research on stem cells is designed to gain greater understanding of how the human body grows and repairs itself and to develop medical therapies for diseases resistant to medical treatment. However, stem cell research raises special ethical, legal and social issues. Although stem cells found in adults show tremendous promise, stem cells found in four- or five-day old human embryos remain of major importance to researchers. Ethical issues arise at every stage of the stem cell research process, from the way in which researchers procure immature human eggs for use in research, to questions about who owns stem cell lines (a family of cells derived by duplicating or "cloning" a single cell). To fulfill New York's pledge to conduct stem cell research according to the highest ethical and legal standards, researchers and lawmakers need ready access to experts from across several disciplines for information and training to help them resolve thoughtfully the challenging legal, policy and ethical questions stem cell research raises. Our proposal seeks support for the planning of the New York Institute for Ethical Stem Cell Research (NYIESCR-pronounced "nicer"), a statewide resource with offices in Albany and New York City that will conduct research and provide information and advice on the wide range of ethical, legal, social and educational issues involved in establishing a viable stem cell research infrastructure in New York State. It will address the distinctive ethical, legal and policy challenges that arise from research on human embryonic stem cells. These include the rapidly evolving nature of stem cell science, the complex patent picture surrounding scientific discoveries, the presence of multiple public and private funders of stem cell research with different standards for the research they will support, and the need to establish new public policies around such questions. NYIESCR will establish an authoritative, objective source of information on the ethical, legal, policy, governance and social and religious issues related to human stem cell research, and serve the needs of policy makers, researchers, educators and the public for information, education and training about ethical and social issues surrounding stem cell research.

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Clinical Translation in Stem Cell Medicine: From Principles to Practice

Mark D. Noble, Ph.D.
University of Rochester

The quest to bring the fruits of stem cell medicine to the clinic is often characterized as a race, but it is important to recognize that it is also an obstacle course. Each new clinically-relevant advance made in the laboratory encounters multiple challenges in the transition to clinical practice, as demonstrated by the fact that, even for pharmaceutical agents, translation of successful pre-clinical studies in the laboratory into medical practice occurs in a very small proportion of cases. The most efficient way to overcome the obstacles to the development of a mature field of stem cell medicine is to take potential therapies that have progressed a great distance on the path to clinical implementation and use these as paradigms for solving problems of general relevance. In this way one achieves the dual benefits of advancing those therapies under study closer to clinical use, while at the same time deriving principles that will be broadly relevant to the development of the field. Thus, this consortium is focused on achieving two goals critical to the development of the field of stem cell medicine: bringing promising therapies forward to the clinic and providing paradigms that will facilitate the development of still other therapies. The consortium will focus on near-term clinical opportunities in the repair of the central nervous system and bone. To achieve the goal of bringing promising therapies to the clinic, particular attention will be paid to the development of strategies with application to multiple fields of tissue repair, as well as to the solving of challenges relevant to each particular clinical endeavor. In addition, this consortium will provide mechanisms for the development of other opportunities that show clinical promise and for the discovery of basic paradigms in stem cell biology that will enhance our ability to make more efficient progress in multiple fields of clinically-relevant activity. Finally, this consortium will also include experts in educational outreach who will work with consortium members to provide public education tools (primarily focused on junior high and high school students) that can successfully translate laboratory knowledge into public education.

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Development of a Safe and Effective Stem Cell-Based Therapy of Parkinson's Disease

M. Flint Beal, M.D.
Weill Medical College of Cornell University

Parkinson's Disease is a chronic, progressively debilitating neurodegenerative disorder that affects a significant fraction of the elderly, and whose overall prevalence is increasing as the population ages; its prevalence exceeds one in 1000 in New York City, suggesting that over 20,000 individuals with significant Parkinson's reside in New York State. Although medications are effective in the early to mid-stages of Parkinson's, it is a progressive disease, and most patients fail treatment after a number of years. Surgical strategies such as pallidotomy and deep-brain stimulation are important adjuncts to Parkinson's treatment, but are by no means definitive. In contrast, cell-based strategies offer the possibility of definitive treatment of Parkinson's Disease (PD) by providing the possibility of both sustained and effective functional restoration of midbrain dopaminergic neurons - the brain cell type lost in PD - with cultured replacements. Human embryonic stem cells, and ultimately patient-specific induced pluripotent cells, offer potentially rich and renewable sources of human dopaminergic neurons. However, a combination of knowledge gaps and technical difficulties have limited the development of human ES cells as cell therapeutics, both in PD and otherwise. Our goal in this consortium effort is to advance this strategy, with a multi-institutional, vertically-integrated collaboration intended to achieve the selective induction and isolation, preclinical therapeutic modeling, and initial clinical assessment of human embryonic stem cell-derived nigrostriatal dopaminergic neurons for the treatment of Parkinson's disease.

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