25 Years of Improving Lives Through Discovery: 1986 – 2011

NIAMS 25th Anniversary Scientific Symposium

Improving Lives Through Discovery

The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) celebrated its 25th anniversary in June 2011 with a scientific symposium, "Improving Lives Through Discovery," at Lipsett Amphitheater on the campus of the National Institutes of Health (NIH) in Bethesda, Maryland. These videos contain the presentations and panel discussions of the day's featured speakers, including NIH Director Dr. Francis S. Collins, NIAMS Director Dr. Stephen I. Katz, and Research!America Chairman John Edward Porter, as well as patients, scientists, and clinicians from across the NIAMS community who came together for the special day.
8:30 8:45 a.m.
Introduction and Overview Stephen I. Katz, M. D., Ph.D.
Director, National Institute of Arthritis and Musculoskeletal and Skin Diseases

Dr. Stephen Katz thanks the nearly 300 friends of the NIAMS who are present for joining in the day’s festivities, emphasizing the role of the NIAMS’ many partners that make its work possible, including patients, the public, and the professional and patient groups that make up the NIAMS Coalition. Noting that much of the work honored by the symposium is really the result of trans-NIH partnerships, he says, “These collaborations are really how the NIH works, and how the NIH works best.” Dr. Katz directs the audience’s attention to the NIAMS 25th Anniversary Milestone Banner, which highlights a representative snapshot of NIAMS-supported research over the years, as well as the Institute’s interactions with researchers, clinicians, patients, and the public. Dr. Katz thanks the NIAMS staff, calling them “one of our Institute’s most valuable resources,” and discusses the accomplishments of his predecessors, the late Dr. Lawrence Shulman, a world-renowned rheumatologist who served as the first NIAMS Director, from 1986 to 1994, and Dr. Michael Lockshin, who led the NIAMS as its Acting Director following Dr. Shulman’s retirement.

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8:45 9:00 a.m.
Welcoming Address Francis S. Collins, M.D., Ph.D.
Director, National Institutes of Health

Calling the NIAMS’ 25th anniversary “a chance to reflect on where we have come from,” as well as an opportunity “to think about the future,” Dr. Francis Collins praises the Institute’s leadership for anticipating scientific opportunities, for strong corporate citizenship within the NIH, for tackling diseases that are “common and chronic and costly and crippling,” and for supporting “a portfolio of research that has made a real difference in the health of the nation and the health of the world.” Dr. Collins describes the NIAMS as consistently making strides in treatment development and understanding the immunology and genetics of its mission areas, from funding the Study of Osteoporotic Fractures in the mid-1980s to supporting research that recently led to the first FDA-indicated treatment for lupus in more than 50 years. Explaining one of the reasons he is excited about plans for the NIH’s proposed National Center for Advancing Translational Sciences, Dr. Collins says, “We have the chance to focus even more intentionally now on taking the deluge of basic science discoveries pouring out of laboratories across all of the diseases that are in the NIAMS’ portfolio and making sure that we apply those as quickly as possible in the translational arena.”

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9:00 - 9:30 a.m.
Institute Champion The Honorable John Edward Porter
Chairman, Research!America

Taking the audience back to the time before “iPads, iPhones, and Twitter,” the Honorable John Edward Porter reminds the audience of how the NIAMS has changed the research landscape, saying, “Twenty-five years ago, there was no Pediatric Rheumatology Clinic to diagnose and treat children with arthritis and other rheumatic diseases. Twenty-five years ago, the discovery of proteins that regulate bone growth and repair—findings which paved the way for advances in bone-grafting and cartilage regeneration—had yet to occur. Today, the millions of people living with arthritis, lupus, inherited skin disorders, and other diseases are grateful for these and other findings.” Mr. Porter also discusses the current budget climate from the perspective of someone who chaired the U.S. House of Representatives subcommittee that oversees the NIH budget for many years, emphasizing the need for both political parties to work together to resolve the nation’s budget crisis. He notes that it was a Democrat and a Republican working together, “Congressmen Henry Waxman of California and the late Ed Madigan of Illinois, who formally established the NIAMS 25 years ago.” Mr. Porter encourages bench scientists and clinicians to create opportunities to share their stories of medical research and breakthroughs with the public and Congress.

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9:30 - 10:45 a.m.
Scientific Session and Patient Perspective Panel 1 Joan A. McGowan, Ph.D.
National Institute of Arthritis and Musculoskeletal and Skin Diseases

Dr. Joan McGowan, Director of the Division of Musculoskeletal Diseases, NIAMS, introduces and moderates Scientific Session and Patient Perspective Panel 1.

Richard Moxley, M.D.
Department of Neurology
University of Rochester

Dr. Richard Moxley discusses how NIAMS and NIH funding has moved the field of muscular dystrophy research forward via a critical disease registry, a network of research centers, and individual study grants. He discusses the strides being made, particularly in the field of myotonic dystrophy—the most common of the muscular dystrophies—“because of the breakthroughs that we think we are now poised to pursue,” including the discovery that toxic ribonucleic acid (RNA) causes the gene defect in myotonic dystrophy and the exciting advances that have been made in treating the disease in animal models.

Clifford Rosen, M.D.
Center for Clinical and Translational Research
Maine Medical Center Research Institute

Dr. Clifford Rosen, Director of Clinical and Translational Research and senior scientist at the Maine Medical Center Research Institute, gives a “tour” of the recent history of osteoporosis research and the NIH’s and the NIAMS’ role in sponsoring much of it. Among the examples Dr. Rosen highlights are genetic studies that have improved our understanding of a major cell-signaling pathway in osteoporosis, the wealth of new basic and translational research spawned by that understanding, and the importance of data that continue to come from the NIH-funded Study of Osteoporotic Fractures, now in its twenty-first year.

Priscilla Ciccariello
National Marfan Foundation

Ms. Priscilla Ciccariello, who lost her husband, eldest son and a grandson to complications of Marfan syndrome, a connective tissue disorder, describes how new surgeries and other treatment advances have given a more hopeful future to her surviving two sons and the many others living with Marfan syndrome.

Panel 1 participants take questions from the audience.

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11:15 - 12:30 p.m.
Scientific Session and Patient Perspective Panel 2 Robert H. Carter, M.D.
Deputy Director, National Institute of Arthritis and Musculoskeletal and Skin Diseases

Dr. Robert Carter, Deputy Director of NIAMS, introduces and moderates Scientific Session and Patient Perspective Panel 2.

Daniel Kastner, M.D., Ph.D.
National Human Genome Research Institute

Dr. Daniel Kastner recalls “the wonderful opportunities” that the NIH afforded a young scientist and shares some behind-the-scenes stories of his research discoveries. About finding the gene for Familial Mediterranean Fever while working in the NIAMS Intramural Research Program, Dr. Kastner says, “This was back at that wonderful time, the dawn of the Human Genome Project, when with positional cloning—that method in which if you could recruit a group of families, if you knew it was a single gene disease—you could discover the gene, even if you didn’t know anything about it. Seeing the dramatic inflammatory phenotype...I thought: That’s for me!...The adventure was on.”

John O’Shea, M.D.
National Institute of Arthritis and Musculoskeletal and Skin Diseases

Dr. John O’Shea describes the NIH as a place where “people come from around the world and give their heart and soul to tough problems.” He recounts his own tough problem, “a bedside to bench to bedside story,” which began in 1994, when his intramural research team at the NIAMS discovered a protein, Jak3, the absence of which causes severe immunodeficiency in humans. Dr. O’Shea’s team cloned the gene that encodes Jak3, about which he says, “Back when we were doing this, it was actually really hard to clone a gene...now you can just sit at home, drinking a cup of coffee, and go on the Internet, and you can find out all this stuff. But it took us months, and months and months.” The slow-going work paid off: Dr. O’Shea and his colleagues have collaborated with Pfizer to develop tofacitinab, a compound that helps block transplant rejections and might help treat certain autoimmune diseases.

George Beach
Beach Creative Communications

Mr. George Beach shares how a new biologic treatment gave his artistic career a second act after 25 years of being unable to paint due to joint damage and pain caused by rheumatoid arthritis. When his arthritis treatment began to “kick in,” in 2001, Mr. Beach says his “brushes came alive again.” Later that same year, the World Trade Center tragedy and America’s response to it inspired Mr. Beach to put his rediscovered skills to work in his award-winning painting, "Indivisible", featured in this video.

Panel 2 participants take questions from the audience.

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1:30 - 2:45 p.m.
Scientific Session and Patient Perspective Panel 3 Susana A. Serrate-Sztein, M.D.
National Institute of Arthritis and Musculoskeletal and Skin Diseases

Dr. Susana Serrate-Sztein, Director of the Division of Skin and Rheumatic Diseases, NIAMS, introduces and moderates Scientific Session and Patient Perspective Panel 3.

John Stanley, M.D.
Department of Dermatology
University of Pennsylvania

Dr. John Stanley, a professor in the department of dermatology at the University of Pennsylvania, argues that although historically there has been “a long lag between research and improvement in patient care,” those lag times are getting shorter, thanks, in large part, to public investment in the research enterprise. Dr. Stanley uses pemphigus as his example, describing how it took 100 years to achieve an understanding of the histology of the disease, leading to the first major advance for patients. But he says more recent advances—from discovering prednisone as a pemphigus treatment to improvements in diagnosis—have benefitted patients more rapidly. Other examples of research with tangible benefits include discovering antibodies associated with pemphigus, coming to understand pemphigus as an autoimmune disease, and the development of animal models of pemphigus.

Jane Salmon, M.D.
Department of Medicine and Rheumatology
Mary Kirkland Center for Lupus Research
Hospital for Special Surgery

Dr. Jane Salmon thanks the NIAMS for taking a risk in funding her team’s PROMISSE study of lupus patients, despite what she calls its “unconventional” hypothesis. She describes the early NIAMS-funded mouse studies that led to PROMISSE’s development and shares some of PROMISSE’s results to date, including findings that suggest that women with lupus who have antiphospholipid antibodies are at increased risk for preeclampsia, a disease of pregnancy that endangers the lives of both fetuses and mothers. Dr. Salmon also notes data that show, importantly, that women with lupus who do not have antiphospholipid antibodies are likely to have a healthy pregnancy if their lupus is stable at conception.

Amye Leong, M.B.A.
Healthy Motivation

Ms. Amye Leong shares her story of being diagnosed with rheumatoid arthritis at age 18—and later with Sjögren’s syndrome and osteoporosis—and her journey to become a patient advocate, speaker and author who defies disability. About her role as the international spokesperson for the Bone and Joint Decade, Ms. Leong says, “One of the things I have been a strong advocate for is the engagement of patients at the research level. And that sounds really bizarre: ‘We’re going to study you; we don’t want to talk to you about your cells!’ But, I would propose to you that when you engage us in the development of research design, we will open your eyes in ways that you will find very beneficial.”

Panel 3 participants take questions from the audience.

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2:45 - 4:15 p.m.
Scientific Session and Patient Perspective Panel 4 Robert H. Carter, M.D.
Deputy Director, National Institute of Arthritis and Musculoskeletal and Skin Diseases

Dr. Robert Carter, Deputy Director of NIAMS, introduces and moderates Scientific Session and Patient Perspective Panel 4.

Emily Smith
Arthritis Foundation

At the age of two, Emily Smith was diagnosed with juvenile arthritis, and she describes the decade that followed as a “roller coaster” in which her arthritis waxed and waned, and her parents and doctors experimented with different dosages of the standard drugs available. Shortly before her eleventh birthday, however, Ms. Smith started taking one of the then-new biologic treatments, and everything changed, dramatically. After an early childhood spent going “days, and sometimes months, nearly immobile from pain and inflammation,” in high school she was pole vaulting on the track team. She says, “I have been lucky enough to see kids with RA [rheumatoid arthritis] completely avoid the misery that I went through. I am able to live my life to the fullest.”

Helen Lu, Ph.D.
Department of Biomedical Engineering
Columbia University

Dr. Helen Lu describes the challenge facing the tissue engineer today with an analogy: “How do you connect a rope to a wall without any fixation pins or screws?” Surgeons today use mechanical fixation devices, she explains, but for tissue engineers like herself, Dr. Lu says the hope is that in the near future, they can better mimic how ligaments and tendons are naturally connected to the bone. She says her long-term goal, and the goal of others in her field, is to eventually be capable of engineering total joint systems. Dr. Lu also talks about the value of having researchers interact with clinicians and the role of mentorship in science, saying, “We stand on the shoulders of giants.” She thanks her mentors, including Dr. Cato Laurencin, and her students, as well as her collaborators at the NIAMS Intramural Research Program and at private institutions.

Cato Laurencin, M.D., Ph.D.
School of Medicine
University of Connecticut

Dr. Cato Laurencin says that to meet the grand challenges in medicine, we need three things: “bold ideas; bold, smart people; and organizations that believe in us and in funding ideas that are bold.” Describing tissue engineering as one of the bold ideas of the 1980s, he says his group is now trying to think in terms of “regenerative engineering,” which he describes as “the integration of tissue engineering with advances in material science, stem cells sciences and developmental biology.” Dr. Laurencin credits the NIAMS with providing ongoing financial support for such engineering, with widening the field, and for having the courage to believe in bold ideas.

Maria Morasso, Ph.D.
National Institute of Arthritis and Musculoskeletal and Skin Diseases

Dr. Maria Morasso describes her research in the NIAMS on mouse models of ectodermal dysplasias—a group of skin disorders caused by genetic mutations—to illustrate more generally how basic research can lead to clinical applications. She emphasizes that the end goal in basic research is always the better understanding of a process or pathology with the intention of using that knowledge to improve quality of life. Dr. Morasso also emphasizes that science is not done alone, saying that researchers need mentors and they need to work in teams.

Panel 4 participants take questions from the audience.

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4:15 4:30 p.m.
NIAMS Coalition Annie Kennedy
NIAMS Coalition Co-Chair
Muscular Dystrophy Association

Tiffany Schmidt, J.D., M.B.A.
NIAMS Coalition Co-Chair
American College of Rheumatology

Ms. Annie Kennedy and Ms. Tiffany Schmidt discuss the role of the NIAMS Coalition, a group of more than 70 professional and voluntary organizations concerned with the research of the NIAMS. Describing herself as the “ghost of Coalitions past and present,” Ms. Kennedy explains how the Coalition has worked to create opportunities for legislators and policymakers to interact with patients and scientists so they can better understand the value of the NIAMS and its role in lessening the burden on the nation of the diseases within its mission. Ms. Kennedy describes the NIAMS’ commitment to its stakeholders as “unparalleled,” and says, “I’m proud to report that the Coalition is more robust today than ever before.” As the “ghost of Coalitions future,” Ms. Schmidt says, “One of the things we will try to do is bring patients, researchers and physicians to Capitol Hill, so that we can go from office to office,” educating representatives and senators about NIAMS’ mission areas. Encouraging members in the audience to join the Coalition in sharing their own stories with legislators, she says, “I was thinking today how much members of Congress would have learned if they had been sitting here today.”

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4:30 - 4:45 p.m.
Closing Remarks Stephen Katz, M.D., Ph.D.
Director, National Institute of Arthritis and Musculoskeletal and Skin Diseases

Dr. Stephen Katz thanks the organizations and presenters who made the NIAMS 25th Anniversary symposium possible, and thanks the audience for attending. He encourages the audience to visit the NIAMS poster session in an adjacent room, saying the posters provide a good sample of “the outstanding quality of science that we have here.” Concluding the symposium portion of the day’s celebration, Dr. Katz says, “This year, and this day in particular, has highlighted the very real progress and promise of our efforts.”

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