A YEAR SINCE OBAMA'S LIFTING OF BAN ON STEM CELL FUNDING, BOSTON TEAM READY TO DISTRIBUTE LINES WORLDWIDE BUT OBSTACLES REMAIN. NEW WEBSITE TO END PUBLIC CONFUSION AND PUSH SCIENCE FORWARD

Researchers at Children's Hospital Boston Aim to Demystify Science and Share Potential for Treatments and Cures on Range of Diseases from Diabetes to Parkinson's

StemCell.ChildrensHospital.org

MARCH 9, 2010 [Boston] - Exactly one year to the day after President Obama's historic decision to reverse an eight-year restriction on federally funded embryonic stem cell research, the Children's Hospital Boston Stem Cell Program today announced the development of a unique, comprehensive web site and released a candid video message from its top researchers on the current state of stem cell research to introduce the new site:stemcell.childrenshospital.org.

Designed as an engaging and accessible resource, the new site seeks to demystify the science, clear up misperceptions and illuminate the public about the power and value of different types of stem cells to create cost effective treatments and potential cures for a range of diseases--including Type I diabetes, Parkinson's, leukemia and other blood cancers, Huntington's, sickle cell anemia, neurological disorders, and more.

Beginning today, visitors to stemcell.childrenshospital.org can access an introductory video, featuring Drs. Leonard Zon and George Daley, along with Stem Cell Task Force leader President of the Kraft group and the New England Patriots, Jonathan Kraft, --who together cite the promise and ongoing challenges in this field and call for new support. Visitors can also sign up to receive updates leading up to the official launch of the website--which will be fully operational on April 26, 2010.

The timing of the April 26th date is also significant in the field of stem cell research, because it marks the 5th anniversary of the release of guidelines from the US National Academy of Sciences (NAS) for the responsible conduct of embryonic stem cell research. Children's was among the first to adopt those guidelines, and in the five years since the guidelines were released, the Stem Cell Program at Children's Hospital has emerged as an internationally recognized leader in stem cell research.

Despite operating under significant constraints over the past eight years, Children's made remarkable progress on multiple fronts:

• Children's created an aggressive business plan, their roadmap for bringing research from the bench to the bedside.
• Children's produced 11 of the first 13 hESC lines approved by the US National Institutes of Health (NIH) for federal funding in December, 2009. Children's is receiving requests for these lines from scientists across the U.S. who are studying diseases from diabetes to Parkinson's.
• Drs. Daley and Zon have been awarded major grants from the NIH for the creation of human models of disease and to study whether stem cells reprogrammed from adult stem cells (iPS) cells are equivalent to those derived from human embryos, one of the field's most pressing questions.
• Dr. Zon's research has already been rapidly translated from the laboratory to the clinic: studies on blood formation in the zebrafish--a model organism pioneered by the Zon lab--led to the discovery of a drug that expands blood stem cells, which is now being tested in patients undergoing umbilical cord blood transplants.
• Children's Researchers were the first to isolate lung stem cells, with implications for treating lung cancer, cystic fibrosis and pulmonary diseases.
• Science Magazine cited Dr. Daley's creation of disease-specific stem cells from patients in its 2008 "Breakthrough of the Year" issue.

"Stem cell research is an incredibly exciting field and this is an extraordinary time to be part of it," said Leonard Zon, MD, Director of Children's Hospital's Stem Cell Program. "In just the five short years since the release of the NAS guidelines, we have seen major break-throughs in the science and in the techniques we can use to investigate potential therapies for a wide range of diseases. It's absolutely essential that we continue that forward momentum."

The Human Face of Stem Cell Research

Now, the stem cell program at Children's Hospital Boston, the principal pediatric teaching hospital for Harvard Medical School, invites the public to become part of the dialogue and the progress. The new web site will clarify misconceptions about stem cell research and provide a broad range of easily accessible information about the history and science of stem cells, current research and issues, a primer on the ethical debates surrounding stem cell research, and much more. Special areas on the site will be devoted to some of the specific diseases mentioned above--and will show individuals how they can help. The newsroom portion will feature the latest breakthroughs in the field, and will also enable visitors to opt in to receive the latest updates via Twitter and Facebook.

The site will also give people a chance to experience the human face of stem cell therapy through inspiring videos and interviews with families and patients who have been treated by stem cell transplants or have donated their own cell lines to research in the hope of one day helping to bring about breakthroughs and cures:

• A teenaged sickle cell patient and gifted student who spent his summer helping out by working in Dr. George Daley's stem cell lab.
• An avid cyclist paralyzed in an accident five years ago, whose own tissues have been made into stem cells by Children's researchers, who has organized major support for new studies that could one day benefit patients with spinal cord injuries.
• A family whose decision to conceive a baby daughter using IVF enabled life-saving stem cells from her umbilical cord to save the life of her infant brother, who was born with a fatal genetic defect.

"Over the past year, improvements in governmental policy and breakthroughs in the lab have advanced the field of stem cell research at a more rapid pace the than ever before, but the public dialogue suggests that people want to learn more," said George Daley, MD, PhD, Director of Stem Cell Transplantation at Children's Hospital Boston. "It's critical for people to have a clear understanding of the science, and to appreciate the promise and the limitations of this research. I think this website will help us achieve that."

CONTACT:
Eileen Campion
Dera, Roslan + Campion Public Relations
212-966-4600 / Eileen@drcpublicrelations.com

Organization for 'promoting useful knowledge' honors Dr. Jordan Orange

		IMAGE: Jordan S. Orange, M.D., Ph.D., is a pediatric immunologist at the Children's Hospital of Philadelphia. Click here for more information.

A pediatric immunologist at The Children's Hospital of Philadelphia has received a prestigious annual award from the American Philosophical Society, an organization founded in 1743 by Benjamin Franklin. Jordan S. Orange, M.D., Ph.D., received the Society's Judson Daland Prize on Nov. 13 for his contributions to research and treatment of inherited immune deficiency diseases.

The Judson Daland Prize for Clinical Investigation recognizes outstanding achievements in patient-oriented research. Honorees are nominated by chairs of clinical departments at a U.S. medical school or hospital and selected by a distinguished committee of biomedical researchers.

Dr. Orange's work involves the innate immune system, the body's first defense against life-threatening infections and diseases. His particular research focus is natural killer cells, a major component of the innate immune system, which have an inherent ability to destroy cancer or virus-infected cells. While in graduate school at Brown University, he discovered that natural killer cells produce cytokines, which are secreted immune signaling molecules, to participate in defending the body against viruses.

		IMAGE: At the Nov. 13 presentation of the Daland Prize were, from left, Dr. Baruch Blumberg, APS President; Dr. Mary Patterson McPherson, APS Executive Officer; Dr. Clyde F. Barker, APS Vice... Click here for more information.

Dr. Orange has continued his research in natural killer cells as a pediatrician, defining the field of human diseases caused by inherent deficiencies of natural killer cells. These include diseases in which only natural killer cells are defective, or where they are defective along with other components of immunity.

One example is Wiskott-Aldrich syndrome, a difficult to treat, life-threatening, immune-deficiency disease. Dr. Orange discovered a natural killer cell defect and its mechanism in these patients, which make them especially vulnerable to herpesvirus infections and blood cell cancers. He used this knowledge to develop a novel therapy that bypasses the cellular defect, and has begun a unique clinical trial of the treatment at The Children's Hospital of Philadelphia.

In addition to his laboratory research at Children's Hospital, Dr. Orange evaluates and cares for children with primary immunodeficiency diseases, a varied and complex group of disorders resulting from a congenital defect in some component of the immune system. Though it results in recurrent or severe infections, the child's underlying disease may go undiscovered for years until he or she receives an accurate diagnosis and appropriate treatment. Establishing an accurate diagnosis and providing therapy can dramatically improve outcome in these diseases and often provides a child with a relatively normal childhood and adult life.

Dr. Orange received the Daland Prize, including a $10,000 honorarium, during an award presentation on Nov. 13, at the American Philosophical Society's headquarters in center city Philadelphia. The prize commemorates Judson Daland, a prominent Philadelphia physician and medical researcher, who left a bequest to the Society to support clinical research. As part of that fund, the Society has awarded Judson Daland Fellowships since 1938, and established the annual prize in 2001.

About The Children's Hospital of Philadelphia: The Children's Hospital of Philadelphia was founded in 1855 as the nation's first pediatric hospital. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals and pioneering major research initiatives, Children's Hospital has fostered many discoveries that have benefited children worldwide. Its pediatric research program is among the largest in the country, ranking third in National Institutes of Health funding. In addition, its unique family-centered care and public service programs have brought the 441-bed hospital recognition as a leading advocate for children and adolescents. For more information, visithttp://www.chop.edu.

About the American Philosophical Society: The American Philosophical Society, the oldest learned society in the United States, was founded in 1743 by Benjamin Franklin for the purpose of "promoting useful knowledge." In the 21st century the Society sustains this mission in three principal ways. It honors and engages distinguished scientists, humanists, social scientists, and leaders in civic and cultural affairs through elected membership and opportunities for interdisciplinary, intellectual fellowship, particularly in the semi-annual Meetings in Philadelphia. It supports research and discovery through grants and fellowships, lectures, publications, prizes, exhibitions, and public education. It serves scholars through a research library of manuscripts and other collections internationally recognized for their enduring historic value. The American Philosophical Society's current activities reflect the founder's spirit of inquiry, provide a forum for the free exchange of ideas, and convey the conviction of its members that intellectual inquiry and critical thought are inherently in the public interest.