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Fig. 1 A Congressional staffer examines a differentiated induced pluripotent stem cell (iPSC) as part of the demonstration conducted by Robert Colbert, MD, PhD.
Courtesy of NIAMS/NIH

AAOS Now

Published 12/1/2014
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Simit H. Pandya

AAOS Hosts Congressional Tour of NIAMS Labs

Staffers get behind-the-scenes look at National Institute of Arthritis and Musculoskeletal and Skin Diseases

While their bosses were home campaigning for reelection in October, several congressional staffers attended the 2014 Congressional Tour Day: Inside the NIAMS Labs, an event hosted by the American Academy of Orthopaedic Surgeons (AAOS) and other organizations belonging to the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) Coalition.

This biennial event seeks to educate Congressional staffers about the burden of diseases that affect the bones, joints, muscles, and skin and the critical need for NIAMS research to identify the causes of, and treatments for, these conditions.

According to data from the U.S. Bone and Joint Initiative, the annual direct and indirect costs for bone and joint health are $950 billion, which represents 7.4 percent of the country’s gross domestic product. Furthermore, the burden of musculoskeletal conditions is expected to escalate in the next few decades due to an aging population. Yet, less than 2 percent of the National Institutes of Health’s (NIH) annual budget is dedicated to funding NIAMS.

Setting priorities
Stephen I. Katz, MD, PhD, NIAMS director, educated participants about the Institute’s three-pronged mission and funding stream, the types of research activities and programs currently under way, and the importance of continued support for these types of research activities. Specifically, he cited NIAMS-funded research conducted by AAOS Past President Stuart L. Weinstein, MD, on the effects of bracing in adolescents with idiopathic scoliosis as an example of the types of research studies that are funded through the NIH’s extramural program.

Additionally, Dr. Katz laid out several key factors that NIAMS takes into consideration when setting its research priorities, including the following:

  • studies that may present the greatest scientific opportunities
  • the percentage of the population affected by a disease
  • the effect of the disease on patients’ quality of life
  • research areas that need scientific pursuit

Because a 2012 industry-funded analysis found that results in approximately one-half of basic science studies focused on cancer were not reproducible, one congressional staffer challenged Dr. Katz on the reproducibility of NIH-funded studies. In response, Dr. Katz pointed to NIAMS’ vigorous peer-review process as a means to promote a high reproducibility of research studies.

John O’Shea, MD, NIAMS’ scientific director, provided an overview of the Intramural Research Program. He listed the types of research activities funded through the intramural research program, including studies on orthopaedic outcomes and autoinflammatory diseases, such as spondyloarthritis and juvenile arthritis. He also outlined several programs funded by the NIAMS Scholars Program, including the Rheumatology Fellowship, the Scholars in Translational Research Program, and a program aiming to assist clinical investigators.

Dr. O’Shea also gave an overview of several clinical trials that examine the use of Janus kinase (JAK)-3 inhibitors to treat rheumatoid arthritis (RA). JAK inhibitors are a new class of immunomodulatory drugs that function by inhibiting the effect of one or more enzymes that act as signaling pathways and can trigger the symptoms of inflammatory and other diseases.

A panel discussion led by NIAMS staff on the admissions process at the NIH’s Clinical Research Center was also on the agenda. The Center is the world’s largest biomedical research hospital. Shirley Aviles, a lupus patient from Fredericksburg, Va., shared her experience receiving treatment at the clinical center. She was grateful for the ability to receive treatment at the NIH at no cost to her.

Finally, the group was given an opportunity to visit and learn more about several NIAMS research projects and laboratories.

Making the invisible visible
In the molecular immunogenetics lab, participants were given an opportunity to use advanced microscopes to look at green fluorescent proteins (GFP) on a single cell under ultraviolet light. By studying GFPs on single cells, researchers have been able to study the location and movement of specific proteins. This information will enable scientists to one day illustrate the relationships between specific proteins and diseases.

Using stem cells to understand disease
Robert Colbert, MD, PhD, discussed the use of induced pluripotent stem cells (iPSC) in studies on the pathogenesis of ankylosing spondylitis, a chronic and painful inflammatory disease affecting the spine. These cells enable researchers to generate and study the differing responses of cell lines developed from normal and affected patients. Congressional staffers were able to view a differentiated iPSC that “beats” in culture like a cardiac cell (Fig. 1).

The NET effect
The delegation also learned about neutrophil extracellular traps (NETs), which are networks of extracellular fibers composed of DNA from a type of white blood cell (neutrophils) that bind pathogens. They were able to view these NETs through a fluorescence microscope and heard about how this normal biologic response to invading pathogens may contribute to cardiovascular disease and organ damage in autoimmune diseases like RA and lupus.

Gait lab demonstration
The group visited the Clinical Center’s Clinical Movement Analysis Laboratory, which uses technology employed by athletic trainers and movie special effects artists to quantitatively analyze the biomechanic forces generated when walking.

Supporting orthopaedic advocacy
This Congressional Tour Day, formerly known as NIAMS Awareness Day, supplements the AAOS’ annual Research Capitol Hill (RCH) days event. During RCH days, AAOS fellows and their patients meet with members of Congress to educate them on the need for continued investments in NIH-funded biomedical research. The next AAOS RCH days will take place March 4–6, 2015, in Washington, D.C. For more information, visit
www.aaos.org/researchdays

The NIAMS Coalition is an independent consortium of more than 80 voluntary and professional associations; members include the American Academy of Physical Medicine and Rehabilitation, the Lupus Foundation of America, the Arthritis Foundation, and the AAOS. Congressional staff from key committees, including the House Energy & Commerce Committee, Appropriations Committee, Rules Committee, and Oversight & Government Reform Committee, attended the event.

Simit H. Pandya is the government relations specialist in the AAOS office of government relations. He can be reached at pandya@aaos.org