Cure JM Foundation Research Grant Recipients
Hanna Kim MD- Fellow, Division of Pediatric Rheumatology Children's National Medical Center and National Institutes of Health
Novel Biomarkers associated with Disease Activity in JDM
In order to expand biomarker analysis to broad proteomic analysis, she is replacing cytokine analysis from her previous grant. The new SomaLogic platform will replace the previously proposed platform called Luminex and is much more likely to successfully identify a new biomarker in JDM without requiring more blood samples.
Claire Deakin MD Post-Doctoral Fellow at University College, London
Genetic Risk Factors in Juvenile Dermatomyositis
This grant which will enable inclusion of North American samples to enhance the statistical power of her study. The project will study genetic risk factors for JDM using samples from a large number of North American and UK patients with JDM. By studying large numbers of patients, this project may also be able to study genetic risk factors for developing specific features, such as the formation of calcium deposits. It will also help us find out whether the age patients are when they get disease influences the role of genetic differences in JDM or its subgroups. The biological effect of these genetic differences will also be studied in order to help us understand the cause of disease better. This research may lead to more information about the causes of JDM and why certain patients develop certain features.
James Jarvis MD Professor of Pediatrics from the University of Buffalo, NY
Plasma Exosomes in Juvenile Dermatomyositis
JDM is a disease with extensive inflammation in blood vessels. Exosomes are found in considerable numbers in the circulation, and are a means through which cells communicate with one another Dr. Jarvis is trying to understand how blood vessels become injured in JDM, as he thinks it's how the muscle injury starts. He will culture exosomes from children with JDM with blood vessel cells and determine how the small RNA molecules disturb the regulation of genes in the blood vessel cells. By comparing what is seen with JDM exosomes to the exosomes of healthy children, we will have new ways to understand how blood vessels are injured in JDM.
Chack-Yung Yu, D.Phil., Professor of Pediatrics; Professor of Molecular Virology, Immunology and Medical Genetics, Center for Molecular and Human Genetics, The Research Institute at Nationwide Children's Hospital and The Ohio State University, Columbus, OH
Complement C4 in Disease Risk and Pathogenesis of Juvenile Dermatomyositis
This study will analyze whether variations of C4A genes and changes of C4 proteins can be used to reveal the severity of JDM and how the illness may develop. The research team will analyze hundreds of DNA samples from JDM patients and healthy subjects. They will use cutting-edge techniques to perform experiments -some of the techniques were originally designed by this research team, who are pioneers in the research field of immune genetics. They hope, through their research, to be able to develop better and more specific ways to diagnose and treat the disease, and hopefully to find a cure in the future.
Hanna Kim, MD, Fellow, Division of Pediatric Rheumatology Children's National Medical Center and National Institutes of Health
Novel Biomarkers Associated with Disease Activity in JDM
This study will compare and contrast serum markers and gene expression patterns in JDM with those of other closely related disorders (CANDLE and SAVI) with the hopes of better understanding the cause(s) of JDM and possible treatments. The study also intends to identify dysregulated pathways in JDM using RNA-Sequencing to improve biomarkers related to disease activity. By developing a better biomarker, the research team hopes to better predict who will respond to a particular therapy, indicate when to stop and start therapy and develop more JDM-specific therapies in the future.
Dr. Megan Curran, Attending Physician, Division of Rheumatology, Ann & Robert H. Lurie Children's Hospital of Chicago; Assistant Professor, Northwestern University Feinberg School of Medicine; Program Director, Northwestern/McGaw Pediatric Rheumatology Fellowship Program
Physician Education Program
Cure JM Foundation funded a multi-stage project to educate physicians about JM across North America. The goal is to help physicians diagnose JM faster so that patients can begin treatment without delay. The first part of this project was a survey of 400 JM families to better understand the patients’ symptoms and path to diagnosis. The second part of this project was an E-newsletter sent to 10,000 pediatricians to help educate them about the signs and symptoms of JM so they can make an accurate and timely diagnosis. The pediatricians receive CME credit (Continuing Medical Education) for reading it and taking a short test afterwards. CME credits are required in most states for physicians to keep their medical licenses. The next stage of this project will be developing and delivering materials to pediatricians with images of the primary signs/symptoms of JM.
Ann Reed, MD
Predictive Model of Disease Outcomes using Computational Biology Modeling in Children with Inflammatory Muscle Disease
Research study at the Mayo Clinic to determine associations between disease outcomes and various features of JDM, which may lead to the prediction of which patients would benefit from particular treatment choices.
Susan Kim, MD
Lymphocyte Repertoire in Juvenile Dermatomyositis
Research study at Boston Children's Hospital using "next generation sequencing" to study detailed T & B cell differences in JDM. This should lead to a better understanding of changes in the immune system, which may help to advance the understanding of JDM and improve future outcomes.
Dawn Wahezi, MD
Premature Atherosclerosis in Juvenile Dermatomyositis
Research study at Children's Hospital at Montefiore that aims to identify which risk factors may be the most significant indicators of early heart disease in children with JDM.
Anne Stevens, MD, PhD. Seattle Children's Research Institute
Simultaneous Genomics and Microbiotica Phenotyping in Juvenile Dermatomyositis (JDM)
Long Term Aim: To identify specific oral and fecal microbial communities in JDM that may be targeted in future therapeutic trials. The influences of susceptibility genotypes for known immune function genes and dietary elements known to alter the microbiome will be characterized to provide the rationale for future diagnostic and therapeutic trials.