A compilation of news from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS). Published three times a year. Just scan these “shorttakes” for information on what’s happening at the NIAMS, or access the complete articles for viewing or use in your own newsletter or other publication.

From the Director . . .

Highlights from the NIAMS Intramural Research Program

The NIAMS Intramural Research Program (IRP) has an established tradition of excellence, with a strong focus on long-term, high-risk research into the genetics and pathophysiology of human disease, and the development of innovative therapies for a number of serious disorders for which satisfactory treatments previously did not exist. The IRP receives approximately 10 percent of the Institute’s budget, and I would like to share an update on this important program.

For much of 2014, the intramural research programs across the NIH have been taking a look inward to develop a vision for intramural research for the next decade and beyond. At the request of NIH Director, Francis S. Collins, M.D., Ph.D., NIAMS and other Institutes consulted with experts to elaborate on what makes the IRP unique, and how those traits can be leveraged and enhanced for the future. In addition, an Advisory Committee to the NIH Director (ACD) Working Group was assembled to examine and assess the IRP to identify areas of opportunity, enhance the uniqueness of the IRP, evaluate the sustainability of current approaches, and guide the future vision of the IRP. The Working Group reported on its findings at the December 11, 2014, meeting, making recommendations regarding research, workforce, training, and infrastructure and facilities. The NIH and the NIAMS are considering these recommendations as we strive to continuously improve our program.

As we consider the path intramural research will take in the future, NIAMS investigators continue to generate outstanding basic, translational, and clinical research discoveries that have a significant impact. Basic research conducted in NIAMS laboratories is furthering our understanding of how the genome works to control gene expression, and how errors in those processes can lead to disease. In addition, translational research is enhancing understanding of cardiovascular disease risk in lupus and other autoimmune diseases, potentially leading to novel interventions. The NIAMS clinical program continues to excel in identifying the genetic basis of rare pediatric autoinflammatory diseases. Some of the characteristics of these conditions appear in other immune diseases, and the work could lead to novel insights and new treatments that reach far beyond a single rare disease.

In addition to enhancing the NIAMS’ research mission, the IRP also is a key component of training the next generation of researchers, and hosts several innovative programs to meet this goal. Our Summer Research Program provides outstanding opportunities for high school, undergraduate, graduate, and medical students contemplating a career in biomedical research or academic medicine to gain valuable experience. In addition, NIAMS has developed a robust series of research and training programs for clinician-researchers. The combination of our Rheumatology Fellowship, Scholars in Translational Science, and Assistant Clinical Investigator programs are developing a robust cohort of highly skilled investigators who will conduct clinical research throughout the nation for years to come.

Finally, I would like to highlight two recent scientific honors that the NIAMS IRP Scientific Director John O’Shea, M.D., received. In April, Dr. O’Shea was named the 2014 recipient of the Ross Prize in Molecular Medicine. Conferred by the Feinstein Institute for Medical Research and the journal Molecular Medicine, the Ross Prize is bestowed upon an active investigator who has “produced innovative, paradigm-shifting research that is worthy of significant and broad attention in the field of molecular medicine.” In addition, this fall, Dr. O’Shea was elected a member of the prestigious Institute of Medicine. Election to the IOM is considered one of the highest honors in the fields of health and medicine and recognizes individuals who have demonstrated outstanding professional achievement and commitment to service. We are very proud of Dr. O’Shea’s accomplishments.

As you can see, the NIAMS IRP plays a vital role in the mission of the Institute, and in the scientific community. You can keep up to date on our IRP through the NIAMS website and the IRP Facebook page.

Stephen I. Katz, M.D., Ph.D.
Director
National Institute of Arthritis and
Musculoskeletal and Skin Diseases

Research Watch and Announcements . . .

Researchers have identified a pattern of biological molecules in the blood of people with systemic lupus erythematosus (SLE) that signals the onset of a disease flare.

Correcting an imbalance between two key molecules in bone-producing cells shows promise for improving bone health in neurofibromatosis, a rare genetic disease that affects skeletal integrity.

Studies shed light on how psoriasis arises and how the body works to repair the damage.

Scratching an itch brings short-lived relief but often makes the itching worse in the long-term. Researchers found that serotonin intensifies itch in mice.

Recommended standards for clinical low-back pain research hold promise for more consistently designed research and, in the long term, better treatment solutions to support those living with chronic low-back pain.

Genetic sequencing is expected to greatly improve the rate of diagnoses for rare conditions in the future. In studies funded in part by the NIAMS, research teams analyzed the results of whole-exome sequencing in a clinical setting.

Injecting an inflammation-blocking medication into a joint area immediately following an injury prevents the onset of post-traumatic osteoarthritis (PTOA) in mice and may lead to new treatments for patients.

A study funded by the NIH comparing treatment with widely used blood pressure medications atenolol or losartan in patients with Marfan syndrome who had an enlarged aortic root found no significant difference in the rate of aortic-root dilation between the two treatment groups over three years.

Researchers have discovered a form of the thyroid hormone receptor that accounts for some of thyroid hormone’s effects, including its role in bone development and maintenance.

Prolonged ultraviolet (UV) light exposure results in an increase of a substance known as ?-endorphin, a chemical produced by the body that reduces sensitivity to pain and is associated with addiction.

Certain types of hair keratin also help form dental enamel, the tough outer covering of teeth.

NIH has funded additional rare disease research through the Rare Diseases Clinical Research Network. Among the funded consortia are the Brittle Bone Disorders Consortium and the Vasculitis Consortium.

The NIH awarded grants to 11 research groups across the United States to establish the Accelerating Medicines Partnership in Rheumatoid Arthritis and Lupus (AMP RA/Lupus) Network.

Scientists investigated the role of Stat3 in muscle regeneration and the potential for Stat3 inhibition to improve muscle repair and muscle cell proliferation.

Researchers uncovered a gene linked to a potentially life-threatening autoinflammatory disorder called macrophage activation syndrome (MAS).

Two types of antibody molecules — rheumatoid factor (RF) and anti-citrullinated protein antibody (ACPA)—act in concert to stimulate inflammation in people with rheumatoid arthritis.

An image of bone formation submitted by a cell biologist at the University of Massachusetts Medical School in Worcester, was named one of the winners of the Federation of American Societies for Experimental Biology’s 2014 BioArt competition.

NIAMS announced the release of its Fiscal Year 2015-2019 Long-Range Plan.

Grants and Contracts . . .

For information on NIH Funding Opportunities related to the NIAMS, please subscribe to the monthly NIAMS Update or visit the Funding Opportunities List on the NIAMS website and the NIH Guide for Grants and Contracts.

Highlights From the Hill, DHHS and NIH . . .

New Congressional Leadership:

The 114th Congress opened on January 6 with a Republican majority in both the House and the Senate for the first time in eight years. The leadership of the following House and Senate Committees of particular interest to the NIH are:

Senate:

• Senate Appropriations Subcommittee on Labor, HHS, Education and Related Agencies
  • Roy Blunt (R-MO), Chairman
  • Patty Murray (D-WA), Ranking Minority Member
• Senate Committee on Health, Education, Labor, and Pensions (HELP)
  • Lamar Alexander (R-TN), Chairman
  • Patty Murray (D-WA), Ranking Minority Member

House:

• House Appropriations Subcommittee on Labor, HHS, Education and Related Agencies
  • Tom Cole (R-OK), Chairman
  • Rosa DeLauro (D-CT), Ranking Minority Member
• House Energy and Commerce Subcommittee on Health
  • Joe Pitts (R-PA), Chairman
  • Gene Greene (D-TX), Ranking Minority Member

Future Directions of Lupus Research

The NIAMS is leading an effort to evaluate progress on The Future Directions of Lupus Research (lupus plan) and to develop a coordinated action plan for future NIH lupus research at the request of the Congressional Lupus Caucus. To help inform this process, NIAMS issued a Request for Information to obtain input from the public on significant accomplishments since the release of the 2007 lupus plan, important scientific opportunities in lupus research, and suggestions for updating the Broad Goals and Priorities elaborated in the 2007 plan.

Congressional Briefing: Lupus Foundation of America and Rheumatology Research Foundation Host Event on the Accelerating Medicines Partnership

On September 9, at the request of the Lupus Foundation of America (LFA) and the Rheumatology Research Foundation (RRF), NIAMS Deputy Director Robert H. Carter, M.D., participated in a congressional briefing titled, “Advancing the Understanding of Lupus through the Accelerating Medicines Partnership.” Representative Jim Moran (D-VA), Co-Chair of the Congressional Lupus Caucus, provided brief opening remarks, and Sue Manzi, M.D., M.P.H., Medical-Scientific Advisory Council, LFA, moderated the briefing. Martin Hodge, Ph.D., Senior Director for Immunoscience, Pfizer Worldwide Research and Development, and David Karp, M.D., President, RRF, also participated. Congressional and NIAMS staff attended the event, as well as researchers, patients, and advocates from the lupus community.

Congressional Tour Day: Inside the NIAMS Labs

On October 15, the NIAMS welcomed Congressional staff to the NIH Clinical Research Center (CRC) to learn more about the NIH and the NIAMS, and to tour several labs in the NIAMS Intramural Research Program (IRP). This biennial event is sponsored by the NIAMS Coalition to raise awareness about NIAMS research. Five Congressional staffers took part in the visit, representing appropriations and authorizing committees, as well as Member offices. Several members of the NIAMS Coalition leadership also attended. The attendees engaged with NIAMS leadership, key researchers from the NIAMS IRP, a patient advocate who has been treated for lupus at the NIH CRC, and viewed several lab demonstrations designed to showcase the state-of-the-art capabilities of the IRP.

New Law: Wellstone MD-CARE Amendments Act of 2014

On September 26, 2014, the President signed the Paul D. Wellstone Muscular Dystrophy Community Assistance, Research and Education (MD CARE) Amendments Act (H.R. 594), sponsored by Rep. Michael Burgess (R-TX). Among other provisions, the bill amends the Public Health Service Act to revise the muscular dystrophy research program at NIH. Some key changes include: specifying more forms of MD within the law, increasing the size and changing the membership of the Muscular Dystrophy Coordinating Committee (MDCC), requiring the MDCC to meet at least twice per calendar year, and adding or clarifying topics to be covered in the Action Plan for the Muscular Dystrophies developed by the MDCC. The bill became Public Law 113-166.

New Law: Sunscreen Innovation Act

On November 26, 2014, the President signed the Sunscreen Innovation Act (S. 2141), sponsored by Sen. Jack Reed (D-RI). The bill established a process for the review and approval of over-the-counter sunscreen active ingredients, and is primarily directed at the Food and Drug Administration regulatory process. The bill became Public Law 113-195.

For More Information

For other related legislative highlights, please refer to the NIH Office of Legislative and Policy Analysis website.

Budget Update

FY 2014

In FY 2014, the NIAMS funded 288 new and competing continuation applications for a success rate of 18.1 percent—a figure higher than last year’s rate of 15.9 percent. The overall NIH success rate is 18.1 percent. The NIAMS FY 2014 Funding Plan can be reviewed on the NIAMS website.

FY 2015

On December 16, 2014, the President signed into law the Consolidated and Further Continuing Appropriations Act, 2015, providing funding through September 30, 2015, as P.L. 113-235. The funding level for the NIAMS is $521.7 million, which is an increase of $1.6 million from the FY 2014 budget. The operational funding plan for NIAMS for FY 2015 is available on the NIAMS website.

FY 2016

On February 2, 2015, President Obama released his FY 2016 budget request to Congress. The amount requested for NIH is $31.3 billion, which is an increase of approximately $1 billion over the FY 2015 budget. The amount for NIAMS is $533.2 million, which represents an increase of approximately $12 million above FY 2015. The NIAMS FY 2016 Congressional Justification (CJ) is available on the NIAMS website. In addition to budget tables, the CJ document includes narrative sections about the Institute’s programs and how funds will be allocated.

NIAMS Faces . . .

The NIAMS Advisory Council welcomes five new ad hoc Advisory Council members.

• Joan E. Bechtold, Ph.D., is the Gustilo Professor of Orthopaedic Research and Graduate Professor in Orthopaedic Surgery and Mechanical and Biomedical Engineering at the University of Minnesota. Dr. Bechtold’s research focuses on bone healing in the context of challenges such as infection, trauma, smoking, and joint replacement. She is particularly interested in developing new bone/implant interfaces to improve outcomes for patients who have revision joint replacement surgeries. She has been an NIH grantee since 1995, and has served on various NIH Study Sections. Dr. Bechtold is actively involved in professional organizations such as the Orthopaedic Research Society, where she currently holds the position of Immediate Past President.
• V. Michael Holers, M.D., is the Scoville Professor of Rheumatology at the University of Colorado School of Medicine, where he also is head of the Division of Rheumatology. His research interests include B lymphocyte activation and development, the role of complement in inflammation and autoimmunity, analysis of pre-clinical autoimmunity in rheumatoid arthritis, and etiopathogenesis of rheumatoid arthritis and other related conditions. Dr. Holers is a member of various scientific and professional organizations, including the Arthritis Foundation and the Lupus Research Institute. He has authored or co-authored over 200 publications.
• Sundeep Khosla, M.D., is the Dr. Francis Chucker and Nathan Landow Research Professor, a Mayo Foundation Distinguished Investigator, and the Dean for Clinical and Translational Science at the Mayo Clinic College of Medicine in Rochester, Minnesota. As principal investigator on a number of NIH grants, Dr. Khosla’s research projects include investigating the mechanisms of bone loss in women and in men, sex steroid action on bone, and the biology of osteoprogenitor and stem cells. He is a former president of the American Society for Bone and Mineral Research, and currently serves as the chair of the Governance Committee of the National Bone Health Alliance and as the Editor-in-Chief of the journal Bone.
• Amy S. Paller, M.D., is the Walter J. Hamlin Professor and Chair of the Department of Dermatology at Northwestern University’s Feinberg School of Medicine. She is also the director of cutaneous clinical trials research at the Ann and Robert H. Lurie Children’s Hospital of Chicago. Dr. Paller’s research interests include the role of sphingolipids in skin and the use of nucleic-acid nanoconjugates as topical gene therapies for certain skin conditions. As a pediatric dermatologist, she specializes in clinical research related to genetic and immune-mediated inflammatory conditions in children. She has led more than 70 clinical trials, and currently is studying eczema, psoriasis, localized scleroderma, and epidermolysis bullosa. She is a former president of the Society for Investigative Dermatology.
• Richard F. Seiden, J.D., is a partner with Foley and Lardner, LLP, in Los Angeles and has been a member of the California Bar since 1973. His experience includes serving as outside general counsel to major nonprofit hospital systems, and assisting clients in the development of integrated healthcare delivery systems in a managed care environment. Mr. Seiden joins our Council as a public representative. He is a former chair of the board of trustees of the National Psoriasis Foundation.

The NIAMS welcomes one newcomer to the Institute, and we bid farewell to those who are leaving us.

The NIAMS welcomes D. Lee Alekel, Ph.D., to the Division of Musculoskeletal Diseases in the NIAMS Extramural Program (EP). Dr. Alekel joins the NIAMS from the newly renamed National Center for Complementary and Integrative Health where she served for almost four years as a program director managing a broad portfolio of grants including Women’s Health and Aging. Prior to joining the NIH, Dr. Alekel was a professor of nutrition at Iowa State University.

William J Sharrock, Ph.D., program director of the Integrated Physiology and Genetics of Bone Program in the Division of Musculoskeletal Diseases, EP, is retiring after 23 years at the NIH. Throughout his career at the NIH, Dr. Sharrock has focused on the cellular and molecular mechanisms of bone development and metabolism—phenomena that are relevant to osteoporosis as well as the rare skeletal diseases, many of which are genetic in origin.

Charles N. Rafferty, Ph.D., former chief of the EP’s Scientific Review Branch, has retired from federal service. Dr. Rafferty joined the NIAMS in 2009, and immediately became involved in review activities related to the American Recovery and Reinvestment Act (ARRA). Dr. Rafferty’s efforts were instrumental in establishing the NIAMS chartered study section for review of clinical trials.

Stephen J. Stahl, Ph.D., senior investigator in the Protein Expression Laboratory of the NIAMS Intramural Research Program (IRP), has retired after 18 years at NIH. The NIAMS Protein Expression Laboratory supports intramural scientists across NIH as well as extramural researchers in studying the structure and function of Human Immunodeficiency Virus (HIV) proteins.

After 30 years at the NIH, Mildred E. Wilson, R.N., has retired from the NIAMS IRP Clinical Research Program, where she focused on natural history studies investigating the mechanisms of rheumatic, autoimmune, and other rare diseases.

After five years at the NIH, Sharon O. Hines has taken a position with the Food and Drug Administration as a program analyst. Ms. Hines served as the Rheumatology Fellowship Program Coordinator in the NIAMS IRP.

NIH Faces . . .

Don Lindberg, M.D., director of the National Library of Medicine for more than 30 years, will retire at the end of March 2015.

Kathy Russell has retired as the CEO of the Children’s Inn. On January 7, she was honored at a special event where the community also welcomed the new CEO, Jennie Lucca.

Kudos. . .

NIAMS Director Stephen I. Katz, M.D., Ph.D., was awarded the Austrian Society of Dermatology and Venereology Gold Medal, the highest honor given by this society, for “excellent scientific or practical contributions in the field of dermatology or venereology.” The award honors “a person who has given outstanding contributions to the development of dermatology/venereology and who is seen by the Society as a signpost for the younger generation.”

NIAMS Scientific Director John J. O’Shea, M.D., has been elected to membership in the Institute of Medicine (IOM) of the National Academy of Sciences. Current, active IOM members elect new members annually from candidates nominated for professional achievement and commitment to issues that affect the public’s health. The announcement was made in October at the IOM’s 44th annual meeting in Washington, D.C.

Joan A. McGowan, Ph.D., director of the NIAMS Extramural Program’s Division of Musculoskeletal Diseases, was honored by the University of Connecticut School of Medicine’s Group on Women in Medicine and Science (GWIMS). Dr. McGowan was recognized for “excellent service in academic inspiration and guidance” and “exemplary service and dedication in supporting biomedical research.” The award was presented at the GWIMS annual meeting.

Alasdair C. Steven, Ph. D., chief of the NIAMS IRP Laboratory of Structural Biology Research, received the Eduard Kellenberger Medal from the International Federation of Societies for Microscopy. The award recognizes achievement in microscopy applications in the life sciences. Dr. Steven accepted the medal in September at the 2014 International Microscopy Congress in Prague, Czech Republic.

Mariana J. Kaplan, M.D., chief of the NIAMS IRP Systemic Autoimmunity Branch, was recently selected as a member of the Henry Kunkel Society. This society is dedicated to fostering patient-oriented research, particularly in the field of immunology, as exemplified by Dr. Kunkel. Dr. Kaplan will be recognized in April at the annual scientific meeting of the society in Annecy, France.

NIAMS Communications and Outreach Update. . .

Multicultural Outreach

For the third consecutive year, the NIAMS has developed a set of multicultural health planners titled, “A Year of Health: A Guide to a Healthy 2015 for You and Your Family.” These planners, tailored for four multicultural communities (African Americans; American Indians/Alaska Natives/Native Hawaiians; Asian Americans/Pacific Islanders; Hispanics/Latinos), provide research-based health tips and information about staying healthy and managing conditions of the bones, joints, muscles, and skin. The health planners continue to be in great demand in underserved ethnic communities.

The 2014 bilingual (Spanish/English) health planner received a Gold Award (the highest honor) in the Health Promotion category from the Health Information Resource Center (HIRC), a national clearinghouse for consumer health professionals who work in health education fields.

Downloadable versions of the 2015 health planners are available on the NIAMS National Multicultural Outreach Initiative website.

Public Liaison

NIAMS Coalition Activities

The Institute continues to work with the NIAMS Coalition to share the latest research advances and related developments, and to foster dialogue on the future path and directions of NIAMS-funded research. The NIAMS Coalition, a group of nearly 90 professional and voluntary organizations, raises awareness about research into the basic understanding, causes, incidence, treatment and prevention of diseases of the bones, joints, muscles, skin, and connective tissues.

The Institute is currently working with Coalition leadership to plan the biennial NIAMS Coalition Outreach and Education Day, which will take place in October 2015. Throughout the year, we will continue to interact regularly with Coalition members via teleconferences, opportunities to meet with Institute leadership, and presentations at professional and voluntary meetings.

Social Media

The NIAMS social media following continues to grow steadily on all platforms. Nearly 10,000 people are following the NIAMS on social media.

On September 30, NIAMS hosted a bilingual (Spanish/English) Twitter chat on lupus, in recognition of Hispanic Heritage Month. Tweets were posted in both languages, and questions were answered in the language in which they were received. Subject matter experts for the event included Mariana J. Kaplan, M.D., chief of the Systemic Autoimmunity Branch, IRP, NIAMS, Susana A. Serrate-Sztein, M.D., director, Division of Skin and Rheumatic Diseases, EP, NIAMS, and Irene D. Blanco, M.D., assistant professor of rheumatology at the Albert Einstein College of Medicine of Yeshiva University. An archive of the chat (#NIAMSLupusChat) can be found on the Storify website at https://storify.com/NIAMS/niamslupuschat.

Media Highlights

NIAMS Director Stephen I. Katz, M.D., Ph.D., highlighted current activities and plans at the NIAMS and the NIH in an article in the January issue of The Rheumatologist, titled “National Institute of Arthritis and Musculoskeletal and Skin Diseases Report for 2015.”

Michael J. Ombrello, M.D., head of the Translational Genetics and Genomics Unit, Office of the Clinical Director, IRP, spoke to MedPage Today about autoinflammatory diseases and systemic juvenile idiopathic arthritis. The story, “Work in Progress: Causes for Systemic JIA,” was published in September.

In November, Michael M. Ward, M.D., acting chief of the Clinical Trials and Outcomes Branch, IRP, was interviewed by Rheumatology News for an article on treatment approaches for axial spondyloarthropathy.

Joan A. McGowan, Ph.D., director of the NIAMS Division of Musculoskeletal Diseases, EP, was featured in the January issue of NIH News in Health for the cover story, “Osteoporosis in Aging: Protect Your Bones with Exercise.”

James P. Witter, M.D., Ph.D., F.A.C.R., director of the NIAMS Rheumatic Diseases Clinical Program, EP, appeared in the PharmaVOICE article, “Using Consumer Data in R&D.” Dr. Witter discussed the use of the NIH Patient-Reported Outcomes Measurement Information System (PROMIS) in clinical research.

Upcoming Events

Look for the NIAMS exhibit at the following events between now and the June 2015 Shorttakes issue:

• American Academy of Dermatology, San Francisco, CA, March 20-24
• Joint Conference of the American Society on Aging and the National Council on the Aging, Chicago, IL, March 23-27
• American Academy of Orthopaedic Surgeons Annual Mee