Physician’s Brief clinical and research highlights for our colleagues in rheumatology Ye a r- E n d 2014
The U-M Scleroderma Program Collaborating to solve a painful mystery Patients battling the debilitating and painful progression of scleroderma face a painful journey. The exact cause of the disease is not yet clearly understood, and there are few good treatment options — let alone a cure — for it. Yet although we have a long way to go to defeat scleroderma, survival rates continue to improve as research advances lead to better treatments for the specific internal organ complications it causes. Many of those breakthroughs are being made in the labs and clinics of the University of Michigan. Since the U-M Scleroderma Program launched in 2004, we have remained focused on our dual mission of developing more effective therapies for scleroderma and its complications while investigating the causes and mechanisms of this complex disease. That requires a multidisciplinary approach to everything we do, involving U-M faculty from disciplines including
the Division of Rheumatology, the Interstitial Lung Disease Program in the Division of Pulmonary and Critical Care Medicine, the Pulmonary Hypertension Program of the Division of Cardiology, Pediatric Rheumatology, the Department of Dermatology, the Division of Hand Surgery, the Department of Occupational Therapy and the Cancer Center. Clinical care is provided primarily to patients throughout Michigan and the Midwest, but the program also serves as an international referral center.
One “switch” closer to a cure In the spring of 2014, a unique collaboration between investigators at U-M and Michigan State University published a groundbreaking study detailing both their identification of a signaling pathway that turns off
scleroderma and a class of chemical compounds that can flip the switch. “The majority of drug treatments that exist today for fibrosis basically look at reducing just the inflammation,” says Dinesh Khanna, M.D., M.S., director of the U-M Scleroderma Program. “There are other drugs that block one or two of the signaling pathways that cause the disease, but scleroderma has many of these pathways.” These new compounds target the genetic switch that controls the formation of myofibroblasts – cells that produce too much collagen, which leads to thickening of skin and damage to other organs. The work originated in the lab of former U-M pharmacology professor Richard Neubig, M.D., Ph.D., now chair of the Department of Pharmacology and Toxicology at the MSU College of Osteopathic Medicine. “The genetic switch that Continued on page 2
New U-M Comprehe Musculoskeletal Cen
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World class care for bone
Dinesh Khanna, M.D., M.S.
David Fox, M.D.
controls the fibrosis process for scleroderma is one that we’ve been studying in my lab for about 10 years,” says Neubig. “But we only recently discovered new compounds that have shown initial success in blocking that switch. We have begun testing those compounds in fibroblasts from scleroderma patients, and are very enthusiastic about their potential to treat and possibly to reverse scleroderma based on the results we’ve seen so far.” Numerous research steps must be completed before the discovery can be translated for use in human patients, but if successful, the approach may be have the potential to slow or stop the progression of other diseases besides scleroderma, including idiopathic pulmonary fibrosis and Crohn’s disease.
David Fox, M.D., chair of the U-M Division of Rheumatology, agrees that the initial efforts are promising, but cautions that it could take several years before continued success in the laboratory might translate into clinical trials. “We need to expand the cell culture studies, greatly expand the research in animal models, including looking at the ability of these compounds to target fibrosis in the lungs — the most deadly aspect of the disease — and also refine the delivery method to ensure it will be well-tolerated,” Fox says. “A few years may sound like a long time,” he adds, “but when you’re talking about something that has the potential to dramatically improve the quality of life for hundreds of thousands of patients, it’s important to get the science right.”
The University of Michigan has filed for patent protection for the discovery and is currently looking for a licensing partner to help bring it to market.
In response to a growing regional and national demand, a U-M program launched in May, 2014 offers premium patient resources for musculoskeletal care. The U-M Comprehensive Musculoskeletal Center (CMC) brings together over 250 physicians from 10 specialties under one umbrella, helping position U-M as a regional and national leader in the field. Care teams can now provide services at 50 different clinics located throughout Ann Arbor and Southeast Michigan with specialties ranging from rehabilitation after a sports injury to managing pain from arthritis. The new U-M brand has a major presence at the U-M Health System’s newest facility, the Northville Health Center, which opened in July. The CMC at Northville, as well as 21 other U-M facilities across nine communities in Southeastern Michigan, provides care related to arthritis, spine disease, pain management, sports medicine and fractures. The new center anticipates the growing need for musculoskeletal care, especially with an aging population that often faces such problems as arthritis and fractures from osteoporosis.
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ne, joint and spine health The center will strengthen connections among experts across the health system and bring diagnostic and therapeutic services together, allowing for a wider range of treatment options than in a traditional physician’s office. Services range from advanced therapies to minimally invasive surgery, with everything from ultrasounds to MRIs managed within the center’s facilities. This multidisciplinary coordination will also help patients get timely and accurate diagnosis and treatment. The Musculoskeletal Center’s arthritis clinic, for example, offers the expertise of rheumatologists, physical medicine and rehabilitation physicians and orthopaedic surgeons who work together to diagnose a patient and determine treatment options and whether surgery is recommended. For the patient, that may mean one appointment instead of being referred from one office to another.
To consult with one of our musculoskeletal experts, contact M-LINE at 800-962-3555.
U-M an Autoimmunity Center of Excellence for Basic and Clinical Research Only institute to be named ACE in both disciplines In spring 2014, U-M received Autoimmunity Center of Excellence (ACE) awards for clinical and basic research – the only institution to be awarded both. The ACE program is designed to encourage and enable collaborative basic and clinical research across scientific disciplines to identify effective treatments for autoimmune diseases. It is a cooperative effort of the National Institute of Allergy and Infectious Diseases (NIAID), the Office of Research on Women’s Health (ORWH) and the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), all components of the National Institutes of Health (NIH). Michigan is one of only 10 institutions in the U.S. to be named an Autoimmunity Center of Excellence. In total, four programs have been awarded ACEs for
clinical research and seven for basic research, with Michigan the only program to receive the distinction for both disciplines. Together, the centers will conduct clinical trials and basic research on new immune-based therapies for autoimmune diseases, enhancing interactions between scientists and clinicians in order to accelerate the translation of research findings into medical applications. David Fox, M.D. and Dinesh Khanna, M.D., M.S. direct the U-M Clinical ACE, and Bruce Richardson, M.D., Ph.D. and Amr Sawalha, M.D. direct the U-M Basic ACE.
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