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Research
October 2009--Research Grants Extended to Two Centers
Your fundraising efforts have allowed us to extend two grants paying for research into better treatments and an eventual cure for JM. We recently gave the Cure JM Program of Excellence in JM Research at Children’s Memorial Research Center in Chicago a new grant to help cover another year of work while the JM research and treatment center at The George Washington University in Washington, D.C., received a grant for an additional year.
Research Update: Cure JM Program of Excellence in JM Research at Children’s Memorial Research Center in Chicago
The following is a research update from Dr. Lauren M. Pachman, MD at the Cure JM Program of Excellence in JM Research.
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October 21, 2009
Rhonda McKeever
Chairman
Cure JM Foundation
3223 Lake Ave, Unit 15C #393
Wilmette, IL 60091
Dear Rhonda:
Please accept my sincere thanks for Cure JM’s transformational support of the juvenile myositis research program at Children’s Memorial. I am humbled by the generosity of Cure JM and most grateful for your support.
Cure JM’s philanthropic investment helps us integrate cutting-edge research,
top-level scientists, and advanced technologies to accelerate our understanding
of juvenile myositis. With a talented team in the clinic and laboratory, we have great potential to better understand the cause of juvenile myositis, and move toward a future where no child has to struggle with this disease.
Thank you again for your belief in our work. It was wonderful to see you, and
other Cure JM leaders, at the Young Adults with JM: Transitioning Your Care workshop.
Sincerely,
Lauren M. Pachman, M.D.
Professor of Pediatrics
Division of Pediatric Rheumatology
Northwestern University, Feinberg School of Medicine
Co-Director, Center of Excellence in Clinical Immunology
Head, Cure JM Program of Excellence in Juvenile Myositis Research |
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(Click Arrows Below to Flip the Page)
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Research Update: George Washington University Myositis Center
The following is a research update from Dr. James D. Katz MD, FACP, FACR at the GW Myositis Center:
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The GW Myositis Center
Division of Rheumatology
The George Washington University
2150 Pennsylvania Avenue, NW Ste 3-416
Washington, DC 20037
http://www.gwdocs.com/1200000177214.html |
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The division of rheumatology of the George Washington University hosts a myositis clinic for both adults and children (ages 7 and above). The GW Myositis Center has been created with the help of a generous grant from CureJM, a nonprofit organization dedicated to finding a cure for juvenile-onset myositis. The center provides clinical consultation to individuals with established or suspected idiopathic inflammatory
muscle disorders; patients should be under the care of, and referred by, a primary physician for their myositis.
The Center also has a research mission. Cure JM’s generous support has enabled inclusion of a full time researcher, Dr. Gulnara Mamyrova, who has special interest and training in juvenile myositis, and a part time pediatric rheumatologist, Dr. Olcay Jones., chief of pediatric rheumatology at Walter Reed Army Medical Center.
In addition, research collaboration has been established with the Environmental Autoimmunity Group, NIEHS, from the National Institutes of Health that bolsters all aspects of the Center's mandate. Dr. Rider is a pediatric rheumatologist from Environmental Autoimmunity Group at NIEHS/NIH, is a consulting attending physician of the Center. Dr. Rider is a world-known expert in juvenile myositis with more than 16 years experience in the care and research of patients with juvenile myositis, who has spearheaded several of the largest national and international collaborative myositis studies performed. Results of these studies have been summarized in more than 100 publications and appeared in national and international peer reviewed journals worldwide.
The center also has a close affiliation with several specialists:
- Dr. Alison Ehrlich, Department of Dermatology (expert in systemic rheumatic diseases and issues of photosensitivity)
- Dr. Robert Jones, Department of Pathology (expert in muscle biopsy review)
- Dr. Kathleen Brindle, Department of Radiology (expert in musculoskeletal radiology, including MRI)
- Dr. Patience White, Department of Rheumatology, advisor to the clinic on issues of transition and career/vocational counseling/planning research
Trainees in rheumatology (fellows) are actively engaged in the clinic resulting in advanced understanding of these rare diseases as preparation for future clinical careers in rheumatology.
For more information about the GW Myositis Center, or to refer a patient, please contact Dr. Mamyrova at phone (202) 741-3069; fax: (202) 741-2490; email: gmamyrova@mfa.gwu.edu
Research Projects for the GWU Myositis Center:
Our first project “Health Care Transitioning in Pediatric Myositis Patients”
This is an IRB-approved survey for parents and children with myositis designed to help understand how involved children with myositis feel in the process of transitioning their care to an adult medicine provider.
The following links are for Parents to log on: http://www.surveymonkey.com/s.aspx?sm=y_2fU_2fJqgB57zKdwdA_2fAH2pA_3d_3d
and for Children to log on:
http://www.surveymonkey.com/s.aspx?sm=XZJKss8Rr3Y0dvILqMgjpA_3d_3d
We will post more new projects soon…. |
The GW Myositis Center Team
Olcay Jones, MD (pediatric rheumatologist);
Gulnara Mamyrova, MD, PhD (researcher);
James D. Katz, MD (rheumatologist - director
of clinic); Lisa Rider, MD (pediatric rheumatologist
from the NIH); Olena Guzhva,
MD (fellow trainee in rheumatology); Geeta
Nayyar, MD (fellow trainee in rheumatology
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Environmental Autoimmunity Group, National Institute of Environmental Health Sciences, National Institutes of Health
ONGOING PROJECT 1:
Discovering new genetic risk factors for dermatomyositis
Cure JM has supported a pilot study of whole genome association scanning in order to help in the discovery of new genes and potential pathways involved in the development of dermatomyositis in children and adults. This project has been led by Dr. Frederick Miller, Dr. Peter Gregersen, Director of the Robert S. Boas Center for Human Genomics at the Feinstein Institute for Medical Research and a leader in gene discovery in autoimmune diseases, and MYOGEN, a collaborative group of many of the major myositis researchers from the United States and Europe. Cure JM’s funds have enabled the typing of the DNA from almost 200 juvenile dermatomyositis patients of 1100 samples typed to date. Preliminary results suggest a number of possible genetic loci spanning a number of different chromosomal regions, including several previously unidentified genetic risk factors for myositis. These results are in need of replication in order to confirm and be certain of the findings. Currently MYOGEN is attempting to locate DNA samples from 1100 additional juvenile and adult dermatomyositis patients (several hundred DNA samples from JDM patients have now been identified), and raise the funds needed for Dr. Gregersen to type these samples. This replication phase should provide more definitive knowledge of the major genetic risk factors for juvenile and adult dermatomyositis and possibly lead to understanding new pathways to disease and novel therapies in the future.
ONGOING PROJECT 2: Understanding outcomes of juvenile myositis
Cure JM has also funded a project related to examining long term outcomes of juvenile myositis and the effects of different treatments for juvenile myositis on long term outcomes. For this project, Cure JM has extended funding to Dr. Peter Lachenbruch, Professor of Public Health and Statistics at Oregon State University and the immediate past president of the American Statistical Association, who has more than 15 years experience researching juvenile and adult myositis. The work, to date, has resulted in the finalization of the validation of the Myositis Damage Index, a tool that assesses longstanding changes from previously active disease, a description of the frequency and types of damage present in JDM patients who are more than 5 years from diagnosis, and predictors of damage in patients with myositis. Dr. Lachenbruch’s work has also resulted in studies of risk factors for the development of calcinosis, and the effects of initial treatments on long term damage. Several publications have already resulted from this work, which remains in progress:
Published papers:
- Rider LG, Lachenbruch PA, Monroe JB, Ravelli A, Cabalar I, Feldman BM, Villalba ML, Myones BL, Pachman LM, Rennebohm RM, Reed AM, Miller FW, for the International Myositis Assessment and Clinical Studies Group (IMACS). Damage Extent and Predictors in Adult and Juvenile Dermatomyositis and Polymyositis Using the Myositis Damage Index. Arthritis and Rheumatism, 2009;60(11):3425-3435.
- Lachenbruch PA, Miller FW, Rider LG. On Determining the Effects of Therapy on Disease Damage in Non-randomized Studies with Multiple Treatments: A study of Juvenile Myositis.” Comm. In Statist. - Theory and Methods. 2009, 38: 3268-3281.
Published abstracts:
- L Rider, P Lachenbruch, L James-Newton, I Cabalar, B Feldman, A Ravelli, B Myones, R Rennebohm, L Pachman, M Villalba, E Adams, C Lindsley, C Wallace, L Zemel, A Reed, F Miller, for the IMACS Group Predictors of Calcinosis in Juvenile and Adult Myositis. Arthritis and Rheumatism. 2008; 58 (suppl): S230.
- L. Rider, P. Lachenbruch, L. James-Newton, I. Cabalar, B.M. Feldman, A. Ravelli, B. Myones, R. Rennebohm, L. Pachman, M. Villalba, E. Adams, C. Lindsley, C. Wallace, S. Ballinger, L. Zemel, A. Reed, F. Miller, IMACS Group Effects of Treatment on Disease Damage in Juvenile and Adult Myositis . Arthritris and Rheumatism, 2008; 58S, S229.
Earlier Grants
In October 2003 the Cure JM Foundation began its mission to do whatever is necessary to help find a cure for juvenile myositis. Within
a year, through the fundraising efforts of the JM community and many generous contributions, we were able to grant two important research fellowships.
In June, 2004, Cure JM awarded a fellowship to Dr. Gulnara Mamyrova, M.D., Ph.D, to complete the Childhood Myositis Heterogeneity Study and projects related to analysis of data from this registry study.
This study was conducted under the guidance of Dr. Lisa Rider, M.D.
by the Environmental Autoimmunity Group at NIEHS, National
Institutes of Health. Deputy Chief Dr. Rider and Chief Dr. Frederick Miller, M.D., Ph.D. were the principal investigators of this study. Dr. Mamyrova’s fellowship funding began on November 25, 2004 and and continued until November 21, 2007. The total amount of this grant was $147,660.
Dr. Mamyrova’s research focused on identifying new immunogenetic risk factors for JM, studying specific complications of illness, such as lipodystrophy, and working on establishing the database and long term outcomes for several hundred patients enrolled in the Childhood Myositis Heterogeneity Study registry. Dr. Mamyrova has presented her work from this study at a number of national and regional meetings, including the American College of Rheumatology, the NIH Research Festival, and DC Rheumatism Society. She received recognition for her work on this study from the DC Rheumatism Society as a Poster Award Winner at their Fellows Forum.
Published papers:
- Mamyrova G, O’Hanlon TP, Monroe JB, Mercatante Carrick D, Malley JD, Adams S, Reed AM, Shamim EA, James-Newton L, Miller FW, Rider LG, for the Childhood Myositis Heterogeneity Collaborative Study Group. Immunogenetic risk and protective factors for juvenile dermatomyositis in Caucasians. Arthritis and Rheumatism. 2006; 54: 3979 – 3987.
- Mamyrova G, Kleiner DE, James-Newton L, Shaham B, Miller FW, Rider LG. Late-Onset Gastrointestinal Pain in Juvenile Dermatomyositis as a Manifestation of Ischemic Ulceration from Chronic Endarteropathy. Arthritis and Rheumatism (Arthritis Care and Research). 2007; 57: 881 - 884.
- Bingham A, Mamyrova G, Rother KI, Oral E, Cochran E, Premkumar A, Kleiner D, James-Newton L, Targoff IN, Pandey JP, Mercatante Carrick D, Sebring N, O’Hanlon TP, Ruiz Hidalgo M, Turner M, Gordon LB, Laborda J, Bauer SR, Blackshear PJ, Imundo L, Miller FW, Rider LG, for the Childhood Myositis Heterogeneity Study Group. Predictors of Acquired Lipodystrophy in Juvenile-Onset Dermatomyositis and a Gradient of Severity. Medicine.2008; 87: 70- 86.
- Mamyrova G., O’Hanlon TP, Sillers L, Malley K, James-Newton L, Parks CG, Cooper GS, Pandey JP, Miller FW, Rider LG, for the Childhood Myositis Heterogeneity Collaborative Study Group. Cytokine Gene Polymorphisms as Risk and Severity Factors for Juvenile Dermatomyositis. Arthritis and Rheumatism. 2008; 58: 3941 - 3950.
Published abstracts:
- L. Vegosen, C. Weinberg, T. O'Hanlon, I. Targoff, B. Koneru, G. Mamyrova, F. Miller, L. Rider, and the Childhood Myositis Heterogeneity Study Group. Seasonal Birth Patterns in Subgroups of Myositis Suggest a Role for Perinatal Environmental Factors in Etiology. Arthritis Rheum. 2005; 52 (suppl): S228.
- G. Mamyrova, L. Sillers, T. O'Hanlon, J. Pandey, C. Parks, J. Malley, L. James-Newton, C. Wallace, D. Sherry, L. Imundo, C. Lindsley, K. Madson, M. Perez, G. S. Cooper, F. Miller, L. Rider, and the Childhood Myositis Heterogeneity Study Group. Interleukin-1 and Tumor Necrosis Factor Alpha Polymorphisms are Risk and Protective Factors for Juvenile Dermatomyositis (JDM). Arthritis and Rheumatism. 2006; 54 (suppl): S831.
- G. Mamyrova, L. Sillers, T. O'Hanlon, J. Pandey, J. Malley, S. Adams, L. James-Newton, C. Wallace, D. Sherry, L. Imundo, C. Lindsley, K. Madson, M. Perez, F. Miller, L. Rider, and the Childhood Myositis Heterogeneity Study Group. Cytokine and HLA Alleles Modulate Disease Severity in Juvenile Dermatomyositis (JDM). Arthritis and Rheumatism. 2006; 54 (suppl): S692-693.
- A Bingham, G Mamyrova, E Cochran, E Oral, L James-Newton, A Pokrovnichka, L Imundo, F Miller, L Rider, A Premkumar. MRI Characterization of Regional Fat Loss in Patients with Lipodystrophy Complicating Dermatomyositis. Arthritis and Rheumatism. 2006; 54 (suppl): S169-170.
- G. Mamyrova, CA. Bingham, EA Oral, K. Rother, E. Cochran, N. Sebring, J. Pandey, L. James-Newton, T. O'Hanlon, I. Targoff, F. Miller, L. Rider. Clinical and Metabolic Characteristics of Acquired Lipodystrophy in Juvenile Dermatomyositis Arthritis and Rheumatism 2007; 55 (suppl): S145
In July, 2004, Cure JM Foundation also awarded a fellowship to Dr. Kelly Rouster-Stevens, M.D., Pharm. D., for the “Untreated Children with Juvenile Dermatomyositis: Impact of Serologic and Genetic Factors on Bone Mineral Density”. This study is being conducted at Children’s Memorial Research Center in Chicago, and is supervised by Dr. Lauren Pachman, M.D. This fellowship award began July 1, 2004 and will continue through June 30, 2006 for a total of $127,965. We would like to thank all those who helped make this possible and who are continuing to raise awareness and funds for further JDM research.
Research Subjects Needed
Twins/Siblings Study
The National Institutes of Health is conducting pioneering research into the genetic and environmental risk factors that may result in an autoimmune disease.
Specifically, they are seeking subjects who have been diagnosed within the last 4 years and who have a twin
or sibling of the same gender (within four years of age) without an autoimmune disease.
This is a great opportunity for JM sufferers to take an active role in the
fight against JM.
Click here for more information.
Other
Research Studies
Calcinosis in Juvenile Dermatomyositis: Pathogenesis and
Current Therapies
www.pedrheumonlinejournal.org/April/calinosis.html
Clinical Outcomes In JDM
(requires site registration, but registration is free)
www.medscape.com/viewarticle/444390
Clinical Trials
www.clinicaltrials.gov/ct/gui/action/SearchAction?term=Connective+Tissue+Disorders
Dermatomyositis
www.emedicine.com/derm/topic98.htm
www.thedoctorsdoctor.com/diseases/dermatomyositis.htm
Guidelines for safe use of IVIG
www.rheumatology.hss.edu/pat/specInfo/sideEffects/ivig_use.asp
Juvenile Dermatomyositis Presenting With Rash Alone -- Eisenstein et al. 100 (3): 391 -- Pediatrics
pediatrics.aappublications.org/cgi/content/full/100/3/391?maxtoshow=&HI
Juvenile dermatomyositis associated with lipodystrophy
www.ijdvl.com/article.asp?issn=0378-6323;year=2003;volume=69;issue=5;
spage=343;epage=344
Lipodystrophy in JDM
www.jrheum.com/abstracts/abstracts01/610.html
Association of TNFa-308A Allele
www.current-reports.com/article_frame.cfm?PubID=RR03-5-1-03&Type=Abstract&KeyWords=Allele&HitNum=91
Vitamin D Lowers Inflammation
www.mercola.com/2004/feb/28/vitamin_d.htm
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