A Bright Future for Stem Cell Research in New York State

Dr. David Guzick, M.D., Ph.D.

October 11, 2007

It is not uncommon for legislative bodies to "craft" (i.e., negotiate) budgets right up until the last minutes of a fiscal-year deadline. Often, the nature of the political process leads to a budget containing provisions that are illogical or unfathomable to the general public, or to no approved budget at all. This year, however, on the issue of funding for stem cell research, the New York State legislature got it right.

On March 31, 2007, minutes before the start of the state's 2008 fiscal year, NYS legislators passed a budget that includes $100 million for stem cell research this year. The money will be administered by a new entity set up within the state health department. In addition, for the next 10 years, the state will provide up to $50 million annually for stem cell research from a fund created from the sale of state-run insurance plans (notably the Health Insurance Plan (HIP) of New York) to private entities. Moreover, there is reason to be optimistic that the state legislature will appropriate an additional $50 million a year over the next 10 years from general sources. Thus, in round figures, it is possible that the State will allocate up to $1 billion for stem cell research over the next decade.

For a number of years, and spurred by the 2004 passage of Proposition 71 in California (see Dean's Newsletters Nov 18, 2004 and Nov 24, 2004), NYS legislators have been trying to pass a funding initiative for stem cell research. There had been strong support from the State Assembly, but the matter would be tabled by leaders of the State Senate and by former Governor Pataki. This year, substantial funding from the state was finally authorized. Whether this was effective advocacy, as described below, or simply a change in the governor's office, the time for NYS funding of stem-cell research is here.

The agreed-upon Enacted Budget sets a broad definition for stem cells as "stem or progenitor cells that divide and are capable of generating one or more different types of progeny." This includes human embryonic stem cells and umbilical cord cells in addition to adult stem cells. While the bill excludes funding for stem cell research for human reproductive cloning, it makes no direct stipulation against funding for somatic cell nuclear transfer.

Our university played major roles in the events leading up to this legislation, both in advocacy and in crafting the provisions and scientific language contained in the bill. President Seligman spearheaded the advocacy efforts of University Presidents and Chancellors, working with our scientists and governmental relations staff to write a key White Paper that was released on Feb 6, 2006. (The white paper, "New York and Stem Cell Research: A Scientific, Therapeutic, Economic, and Policy Analysis," can be downloaded from http://www.rochester.edu/news/pdfs/stemcellwhitepaper.pdf.) This document, signed by the Presidents and Chancellors of 17 NYS universities and other institutions with substantial biomedical and life sciences research programs, details the competitive research environment that has emerged in the past several years and its implications for the state's biomedical research community and economy.

The Rochester White Paper makes the following case in support of NYS funding of stem cell research: Federal funding restrictions on human embryonic stem cell research have prompted several states to establish state-based research funds aimed at capturing the scientific and commercial potential of this new field of medicine. While New York's research institutions are widely acknowledged to possess the scientific talent that would enable the state to be a major leader internationally in stem cell research, there is significant risk that researchers in New York will be recruited away to institutions in other states where they would have access to more resources to pursue their research. The loss of these scientists will have a significant negative ripple effect on a university's entire research enterprise as research grants, junior scientists, biotech companies, and venture capital will similarly migrate to those institutions that are perceived to be on the cutting edge of biomedical research. A decline in the fortunes of New York's biomedical research community in turn would have significant economic consequences for the entire state. New York's State universities, teaching hospitals, and research laboratories contribute significantly to the state's economy through employment, through spending and through the development of innovative products and concepts for the biotechnology and pharmaceutical industries.

To give some quantitative impact, the academic medical community contributes an estimated $30 billion per year to the state's economy and generates more than 459,000 jobs. The biotech and pharmaceutical sectors are responsible for $18.1 billion in economic activity and 110,000 jobs.

Critical to the advocacy efforts was NYAMR, a coalition of patient advocacy groups, faith-affiliated organizations, medical colleges and biotechnology companies that have a shared interest in encouraging and funding embryonic stem-cell research in New York State. Led tirelessly by Robin Elliot, Executive Director of the Parkinson's Disease Foundation and Chair of NYAMR, this 46-member coalition includes the Parkinson's Disease Foundation, Juvenile Diabetes Research Foundation, Hadassah, the Christopher Reeve Paralysis Foundation, the Michael J. Fox Foundation, the Biotechnology Association of New York, and numerous other stakeholders. Among the scientific community, a key member of NYAMR is our own Mark Noble, PhD, Professor of Biomedical Genetics. Mark played the role of lead scientist in this coalition, working constantly with coalition members over the last three years to keep them informed about the scientific issues while making sure that all members of the coalition understood the specific value of each of the populations of stem cells currently under examination in different laboratories. He was also one of a small number of coalition members on the phone when the governor and state legislature were working out the final language of the proposal. In the last several months before the legislation was finalized, Craig Jordan, Ph.D, Associate Professor of Medicine in the Wilmot Cancer Center, joined the advocacy effort, also serving as an effective voice. Indeed, the final language defining stem cells, which embraced the full spectrum of relevant research, was the outcome of efforts by Mark, Craig, and Sally Temple (then of Albany Medical College).

Dr. Noble sums up the NYS initiative this way: "Stem cell science is going to prove as important a medical advance as vaccination and antibiotics. Here, in NYS, we have a community of stem cell scientists engaged in this work at every level, including learning how to use stem cells to repair tissue, learning how their behavior is disrupted in the developmental maladies that afflict millions of children, and discovering means of killing those cancer stem cells that make it so difficult to cure cancer. This is currently one of the top stem cell communities in the world. If New York becomes the only state with such a high level of scientific activity that is not actively promoting this work, then it is inevitable that many of our best people will be attracted elsewhere. If the NYAMR initiative is funded, in contrast, New York will be one of the great places in which to conduct this research."

As part of the oversight process, the Governor appoints a Board, which in turn includes two committees – the Funding Committee and Ethics Committee. The Empire State Stem Cell Board will be chaired by the Commissioner of Health, Richard F. Daines, M.D., who will also act as a member on each committee. Twelve members are appointed by the Governor with six of those members recommended by legislative leaders. The Funding Committee will recommend awarding grants to the Commissioner of Health, based on the analysis and recommendations of an independent scientific peer review process. The Ethics Committee will make recommendations regarding scientific, medical and ethical standards. I am pleased to report that Brad Berk, MD, PhD, our URMC CEO, will be a member of the Empire State Stem Cell Board. Brad was nominated by Senator Malcolm Smith, the Democratic leader in the state Senate.

Whether the result of effective advocacy or simply the fact that we had a change of parties in the Governor's office, we find ourselves at a point where we can look forward to the potential (i.e., subject to peer review) of funding important stem cell research at SMD. The breadth of scientific investigation in the field of stem cell medicine at the medical school is very impressive, with almost 40 faculty and more than 250 staff engaged in one way or another in this area of research. Some examples of the ongoing efforts with direct clinical applications are as follows:

Steve Goldman, M.D.,Ph.D.

Steve Goldman, M.D.,Ph.D., Dean Zutes Professor of Neurology and Co-Director of the newly established Center for Translational Neuroscience, leads a pioneering effort in stem cell biology of the CNS that is pointed towards therapeutic application in a variety of conditions. He and his colleagues are playing leading roles in understanding the underlying principles of neurogenesis in the adult nervous system, in the development of cellular therapies for pediatric disorders in which normal myelination is compromised, for adult disorders such as Parkinson's disease and Huntingdon's disease and also for treatment of spinal cord injury. Dr. Goldman and his colleagues are also working in the field of cancer research to provide new insights into the cellular origin and potential treatment of tumors of the CNS.

Craig Jordan, Ph.D.

By identifying vital differences in their biological properties, Craig Jordan, Ph.D. and colleagues are developing strategies to eradicate stem cells that become damaged and lead to cancer. Their current efforts are focused on the blood cancer leukemia, which is known to arise from a leukemia stem cell. Exciting recent findings have shown that a naturally occurring compound found in medicinal plants can act as a powerful drug to selectively destroy leukemia stem cells. Building on this discovery, Dr. Jordan and his collaborators have nearly completed the development of a pharmaceutical grade of the drug, which is expected to enter clinical trials within the next few months. These studies represent the first therapeutic effort ever designed to specifically target a cancer stem cell.

Chris Proschel, Mark Noble, Ph.D., and Margot Mayer-Proschel

Mark Noble, Ph.D. and colleagues Margot Mayer-Proschel and Chris Proschel are engaged in a broad-ranging effort to extend the boundaries of stem cell medicine in the central nervous system. Along with playing a leading role in the discovery of new precursor cells of the CNS, they have been defining a wide range of developmental disorders that appear to be caused by disruption of the normal function of stem and progenitor cells. These include conditions caused by thyroid hormone deficiency, iron deficiency, and Vanishing White Matter disease. Their discoveries of the vital role of the redox state in regulating progenitor cell function have led to discoveries of molecular mechanisms that explain the effects of exposure to environmentally relevant levels of chemically diverse toxicants known to compromise normal CNS development. In addition, their work provides new insights into the biological basis for the adverse effects of systemic chemotherapy on the CNS, potential means of preventing such damage, and a range of novel insights into the biology of gliomas. As Co-Director of the NYS Center of Research Excellence in Spinal Cord Injury, Dr. Noble is also part the URMC team (along with Margot and Chris) developing new means of repairing damaged spinal cord by transplantation of astrocytes, the major support cells of the CNS and of understanding the cellular foundations underlying the generation of chronic pain syndromes following spinal cord injury.

Jim Palis, M.D.

Jim Palis, M.D. and his colleagues are investigating the ontogeny of the blood forming system, reasoning that a better understanding of embryonic organ development will lead to novel insights into tissue repair and organ replacement therapies. Studies of the emergence of the mammalian embryo's earliest blood-forming progenitors has recently led to the discovery of an erythroid precursor containing extensive self renewal capacity while retaining the ability to fully mature into red cells. This work raises the possibility that alternative sources of universal red cells can be derived from embryonic stem cells to safely meet the ever-expanding clinical need for transfusion therapy.

Ed Puzas, Ph.D.

Regis O'Keefe, M.D., Ph.D.

Ed Puzas, Ph.D., Regis O'Keefe, M.D., Ph.D., and their colleagues in the Center for Musculoskeletal Diseases are engaged in several efforts related to the development and function of the stem and progenitor cells involved in bone formation. Their investigations range from analysis of the loss of stem cell function during aging, to adverse effects of lead exposure on bone repair, to the application of treatments in which endogenous stem or progenitor cells are recruited to provide new treatments for osteoporosis and to accelerate fracture healing. Related work investigates how bone cells regulate hematopoietic stem cell function in the bone marrow. This has great potential to improve bone marrow transplantation methods in cancer patients and others.

In the beginning, there is the stem cell. It is the origin of an organism's life, a single cell that can give rise to progeny that differentiate into any of our specialized cells. Now stem cells hold the promise for a day in which the effects of many catastrophic diseases can be reversed. Can the damaged cells of Alzheimer's disease patients be restored? Can spinal cords severed from trauma be rejoined? Can damaged organs—liver, pancreas, heart--be rebuilt? In thinking about these tantalizing questions, perhaps it is fitting to end with a quote from Stuart Orkin (Nature Medicine, November 2000, Vol. 6, Number 11, p. 1212): "The ability to turn blood into liver would be the envy of the alchemists of former times. Turning stem cells into 'therapeutic gold' will probably rest on our ability to identify the mechanisms by which tissue-derived stem cells respond to environmental cues and execute new developmental decisions." It is our hope that NYS scientists, including those at the University of Rochester, can take advantage of this extraordinary funding opportunity to accomplish the goal of turning stem cells into "therapeutic gold."

Meliora,

David S. Guzick, MD, PhD
Dean, School of Medicine and Dentistry
University of Rochester

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