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BEDFORD RESEARCH FOUNDATION Massachusetts 501(c)(3) not for profit organization P: 617-281-7902 E: [email protected] W: www.bedfordresearch.org

Four Years of the Spinal Cord Workshop At the Frontier of Stem Cell Research and Spinal Cord Injury

are easily accessible at the Foundation’s website. For more information about this year’s spinal cord workshop, www.SpinalCordWorkshop.org to read an interview with Dr. Kiessling about what she learned this year, and what you can do (in addition to donating) to help.

Help Us Reach Our Goal

As of November 2012, the Foundation The 2009 Workshop in partnership with University of Georgia and the Shepherd Center has raised $375,000 towards the testis stem cell project. We need another $185,000 to reach the total of $560,000 Since 2008, the Spinal Cord Workshop has devastating, effects only 11,000 new patients to complete the study. Please help us brought together international leaders in every year. Therefore, most corporations are pursue this promising research. surgery and basic science to debate and unwilling to invest in SCI treatments, even if develop a list of the challenges to cures discoveries could cascade to help a host of Your donation makes a difference! for spinal cord injury. The goal of the other diseases in the future. workshop is to develop a “white paper,” Participants have expressed the importance listing the obstacles. of intimate workshops like this one, to The workshop has brought to light such allow for a space to share not only problems as how researchers use animal scientific discoveries, but also failures. models to test theories, how to measure Much of scientific progress is failure and in outcomes from studies (i.e. what defines the current “success story only” success?), and the distribution of funds. environment, scientists and physicians don’t have the chance to share the side of Additionally, the researchers have pointed the research that leads to many important out a significant lack of corporate discoveries: what doesn’t work in the lab. involvement because of the limited market size, considering that spinal cord injury, while The reports from these clinician scientists

MORE NEWS INSIDE FALL 2011 - WINTER 2011 Developing The First A New Branch of Testis Project Update The 2013 Activated Prostate Cancer Circadian Incubator Medicine: A Letter Thanks to private Egg Symposium Research Update Inside the Foundation’s From the Director donations, scientists are Dr. will Dr. Robert Eyre and Dr. new Videomicroscope. Translating discoveries in Phase 3 of the testis keynote the tenth annual Joseph Ciccone, work into therapies. stem cell project. Symposium. with BSCRF scientists. Testis Stem Cell Project The 2013 Activated Egg Symposium Thanks to private donations, BSCRF scientists Nov 8, 2013: Dr. Mario Capecchi will in any desired gene. The power of this are in Phase 3 of the testis stem cell project. keynote the tenth annual AES. Dr. technology is that the investigator Testis tissue may be a readily Capecchi is a Distinguished Professor of chooses both which gene to mutate and available source of patient- Human Genetics & at the how to mutate it. This allows scientists to specific stem cells for men, and University of Utah School of Medicine, evaluate in detail the function of any may take less time to develop an Investigator with the Howard Hughes gene of the mouse. and characterize than the iPS Medical Institute, and a Nobel Laureate cells Professor Yamanaka for & Medicine, 2007. received the for this year. Bedford Research scientists Dr. Capecchi is best known for his are taking advantage of their more pioneering work on the development of than two decades of experience "gene targeting" in mouse embryonic with testis tissues to collaborate stem (ES) cells. This technology allows with Dr. Martin Dym, Georgetown scientists to create mice with mutations 2013 Keynote: Mario Capecchi, PhD University, who reported deriving pluripotent stem cells from testis biopsies in 2009.

Dr. Dym has generously shared some of the cryopreserved tissues used in his original First Circadian Incubator Videomicroscope report so Bedford Research scientists can improve the stem cell derivation process.

Three years ago Bedford Research Phase 4 will use fresh biopsies from male Foundation scientists discovered that volunteers for this study to establish the circadian rhythm genes may be important success rate. Phase 4 will begin in 2013, if for stem cells. If true, new ways of culturing funds are available. them need to be developed. The question is: what circadian signals to 2012 New Jersey Stem Cell test? A signal common to all mammals is a Symposium Keynote daily fluctuation in body temperature. Dr. Kiessling was the Keynote Speaker BRF scientists are taking advantage of a at the Symposium on September 19, mouse strain that has been engineered to 2012, “Totipotency, Pluripotency and glow when the circadian rhythm genes are Mouse developing heart cells actually beating Growth Factors.” The sixth annual in culture. See the video on our website. active. BRF scientists derived two symposium lines from this mouse attracted over determine if circadian temperature strain, and are conducting studies to 240 scientists. oscillations play a role in stem cell growth and tissue SPAR Presented at HIV development. Forum in Washington, D.C. Mouse = A characteristic of embryonic A roundtable, “Safe Conception and + stem cells is the ability to Reproductive Options for HIV-Infected Firefly aggregate together and Individuals” organized by the Forum for form embryonic muscle Collaborative HIV Research, UC Berkeley cells of the heart. Work is in The PER2Luc mouse has circadian genes coupled with firefly School of Public Health, featured a “Luciferase” genes. When the circadian genes turn “on” they progress to see if this presentation about SPAR by Dr. Kiessling. glow, dimly, like a firefly. process involves expression The Foundation’s specialized HIV- of circadian rhythm genes. infertility program now has over 170 healthy babies, www.SemenTesting.org.

2 THE BEDFORD STEM CELL RESEARCH FOUNDATION Prostate Cancer Research We are now nearly a decade A New Branch and a half into that era, with Project Update Of Medicine astounding scientific progress being made all over the LETTER FROM THE DIRECTOR ABOUT world, but little in the way of RESEARCH AT THE FOUNDATION therapies. Why? Fifty years ago, John B. Gurdon discovered frogs could develop from the genetic What are the obstacles to translating material of a single adult cell transplanted laboratory discoveries into off-the-shelf Dr. Robert Eyre Dr. Joseph Ciccone Clinton McCabe into a frog egg, the first cloned animal. The therapies? Is it the way the experiments are Dr. Robert Eyre, and his colleague, Dr. experiment was done not to clone frogs, but designed in the laboratories that does not Joseph Ciccone, collaborate with Bedford to ask a question: do adult cells contain all translate into the safety studies required by Research scientists to develop improved the genetic information needed for the Food and Drug Administration? Is it early detection tests for prostate cancer. embryonic development? unnecessary bureaucracy? Is it lack of Following up work Dr. Eyre reported at the coordination among the groups needed to go ’s experiment from bench to bedside? New England chapter of the American answered the question, Urologic Association, the team is recruiting genetic information is not men needing prostate biopsy for cancer lost in adult tissues, it is detection to also provide specimens for the selectively turned off or on. new Bedford Research screening test. This heralded a new era of cell discovery The test under development will screen that took 50 years for the Nobel Committee semen specimens, half of which are fluids to recognize and award Professor Gurdon 19 identical albino frogs created by nuclear and cells from the prostate gland, for the the Nobel Prize in Physiology and Medicine transplantation into unfertilized eggs. presence of genes that are over-expressed this year. For the next 34 years, however, it and under-expressed specifically in was believed that only frogs, not mammals, One known barrier to stem cell therapy is prostate cancer. could be cloned from adult cells, a viewpoint the need for transplanted cells to not be Recent concerns about the lack of specificity astoundingly reversed by the successful rejected as “foreign.” Cell rejection would of the PSA test, currently the only available of Dolly the sheep in 1996. be avoided if stem cells could be derived screening test for prostate cancer besides from the patient’s own cells, rather than prostate biopsy, have increased the Embryonic stem cells were derived from from a stem cell bank. Toward this end, importance of this study. The goal is to mouse embryos in the early 1980’s and from discovered in 2007 a new provide physicians and their patients with a human embryos in 1998. The remarkable method to re-program adult cells into more accurate screening test for cancer, that capacity of embryonic stem cells to multiply pluripotent cells, thus replacing eggs. The also distinguishes aggressive cancers from in culture, and become every cell type in the discovery so captured the attention of the slow-growing tumors. The research is being body, heralded a new era of regenerative global scientific community that coordinated at BSCRF by Clinton McCabe. medicine. laboratories all over the world quickly confirmed Yamanaka’s findings, thus lifting

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Board Of Trustees Ethics Advisory Board Alan S. Geismer, Esq. Chairman John D. Lee, MEd, Extension Education Arthur Applbaum, PhD, Chairman Robert D. Truog, MD Sugarman, Rogers, Barshak & Cohen Prof. of Ethics and Public Policy Prof of Medical Ethics and Pediatrics, Alan Mayer, AIA Kennedy School of Government Children's Hospital, Harvard Medical School Jose Cibelli, DVM, PhD Principal Architect, Mayer & Associates Prof. of Animal Biotechnology, Stanley J. Bodner, MD Daniel Wikler, PhD, Susan L. Moss, JD, PhD Michigan State University Infectious Disease Specialist Prof. of Ethics and Public Health, Consultant on Equity & Diversity Harvard School of Public Health Lawrence R. Jones Kenneth A. Burry, MD, Anil Purohit, PhD CEO, Jones Marketing Services Prof of Obstetrics and Gynocology Programs for AIDS Testing and Awareness Oregon Health Sciences University Scientific Committee Robert L. Kaufmann, MD Victoria Staebler, CPA Reproductive Endocrinologist Norman Daniels, PhD, Carol M. Warner, PhD, Chairman Senior Financial Advisor, Merrill Lynch Sean Kealy, Esq. Prof. of Ethics & Public Health, Prof. of Biology, Emeritus, Northeastern Boston University School of Law Margaret S. Wray, BSN, Psychiatric Nurse Harvard School of Public Health University

WWW.BEDFORDRESEARCH.ORG 3 BEDFORD STEM CELL Nonprofit Organization Postage and Fees Paid RESEARCH FOUNDATION Bedford Research Foundation PO Box 1028 Zip Code 01730 Bedford, MA 01730 Permit No. 25

News Inside

1 4 Years of the Spinal Cord Workshop

Developing the First Circadian 2 Incubator Videomicroscope

3 The 2013 Activated Egg Symposium 4 Update on Stem Cells from Testis 5 Prostate Cancer Screening Project

BEDFORD STEM CELL RESEARCH FOUNDATION continued from page 3 also apply to other diseases, such as heart My frustration about how much more disease, diabetes, Parkinson’s Disease, BSCRF could accomplish each day with stroke, multiple sclerosis, AIDS, and cancer. more people, more money, is balanced by pride in our ability to do more with less, and We are witnessing the by gratitude to the supporters and development of a new branch Dr. Yamanaka’s discovery of induced pluripotent committee members who believe in our of medicine, with all its stem cells may eliminate the need for embryos. mission. Our administrative costs are low, growing pains. our ratio of new information/research dollar stem cell research out of the controversy At this time, there is no consensus on the spent is high, our goals are lofty. associated with human embryos. Dr. best source of stem cells, the best way to Yamanaka shared the Nobel prize this year With gratitude for your support, program pluripotent stem cells into the with Professor Gurdon. tissue needed, and how best to guard Ann A. Kiessling, Ph.D. The prospect of regenerating damaged against unwanted growth of stem cells that tissues and organs is arguably the most could lead to cancer. Bedford Research exciting development of modern medicine. Foundation scientists remain focused on Spinal cord injury seems especially suited how to improve the efficiency of deriving for regenerative medicine because the patient-specific stem cells from naturally injury is usually a well-defined lesion in an pluripotent sources, including unfertilized otherwise healthy adult. This year’s 2012 eggs and testis, and whether or not the Spinal Cord Workshop is a frank discussion discovery by BRF scientists a few years ago Dr. Steven L. Stice, Dr. Hans Keirstead, among leaders in the field to pinpoint, and that circadian rhythms may play a role is true. Dr. Ann A. Kiessling and Dr. Wise Young at the hence correct, the principal barriers to cure first Spinal Cord Workshop in 2008. SCI. Once identified, those barriers could

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