Cure JM May 2024 – Virtual Symposium

The Cure JM Clinical Care Network hosted our second quarterly virtual symposium of 2024, “Juvenile Dermatomyositis – Advances in Clinical Care, Basic Research, and Translational Studies.” World-leading researchers and clinicians presented sessions on a wide variety of topics, with the primary goal of enhancing the overall patient and provider experience through collaboration and best practice sharing.

Intended Audience:

  • Pediatric and adult rheumatologists, dermatologists, and immunologists
  • Residents and fellows
  • Professors of dermatomyositis, polymyositis, and related fields of study
  • HCPs who treat autoimmune conditions in adults or juveniles
  • Industry representatives

We are pleased to share each presentation and outcomes from each session below:

Welcome and opening remarks – Andrew Heaton, Ph.D., Chief Scientific Officer, Cure JM Foundation

Scientific Session (3.0 CME credits are pending through Duke University)

Victoria P. Werth, M.D., University of Pennsylvania, USA

About this session:
Victoria Werth, M.D., will discuss the identification and treatment of skin manifestations associated with dermatomyositis, including diagnostic criteria, various skin manifestations, and the latest treatment approaches and insights into the complexities of managing dermatomyositis, aiming to improve patient outcomes and quality of life.

About Dr. Victoria Werth:
Dr. Werth is a Professor of Dermatology and Medicine at the University of Pennsylvania School of Medicine and Chief of the Division of Dermatology at the Philadelphia Veterans Administration Hospital. Dr. Werth earned her medical degree from Johns Hopkins University School of Medicine in Baltimore, Maryland. Her professional achievements include being recognized for her groundbreaking research in autoimmune skin diseases, particularly in the areas of lupus and dermatomyositis. She has also published extensively in top-tier medical journals, presented her work at major scientific conferences, and received numerous awards and honors for her contributions to dermatology and autoimmune diseases.

Marc Jansen, M.D., Ph.D., University Medical Center Utrecht, Netherlands

About this session:
The Utrecht JDM Cohort represents a pioneering initiative to unravel the complex pathophysiology of JDM flares and develop predictive tools for clinical practice. Marc Jansen, M.D., Ph.D., presents groundbreaking research elucidating the role of interferons in JDM pathogenesis and introduces an innovative scoring system for flare prediction.
About Dr. Marc Jansen:

Dr. Jansen is a pediatrician and researcher at the University Medical Center Utrecht (UMC Utrecht) and the Wilhelmina Children’s Hospital. He obtained his Doctor of Medicine (MD) degree from the University of Utrecht and completed his pediatric residency at the UMC Utrecht. Jansen received his Ph.D. in Pediatrics from the University of Utrecht in 2005.

He is a leading expert in pediatric nephrology, with a focus on juvenile idiopathic arthritis-associated nephritis (JIA-AN). His research has been recognized with several awards and honors, including the “Best Paper” award at the European Society for Pediatric Nephrology meeting in 2011. Dr. Jansen has published numerous papers in peer-reviewed journals, including the Journal of the American Society of Nephrology, Pediatric Nephrology, and the Journal of Clinical Hypertension.

His research interests include pediatric nephrology, particularly juvenile idiopathic arthritis-associated nephritis (JIA-AN), the development of biomarkers and prediction models for disease activity and flares in children with JIA-AN, and the investigation of novel treatments and therapies for pediatric kidney diseases. One of his notable publications is “The Utrecht JDM Cohort: Flare Prediction and the Development of an Interferon-Based Score” (2022).

Polly Livermore, Ph.D., University College London, Great Ormond Street Children’s Hospital, UK 

About this session:
Polly Livermore, Ph.D., will share her work with parents of children and young people with myositis, where she has been learning how we could further improve their support and, in turn, benefit their child and wider family. She will share some of the results so far about designing, developing, and testing a technology intervention to help families.

About Dr. Polly Livemore:
Dr. Livermore is a Senior Pediatric Rheumatology Nurse at Great Ormond Street, currently serving as the NIHR GOSH Biomedical Research Centre (BRC) Clinical Academic Programme Lead. In 2020, she completed her NIHR-funded Clinical Doctoral Research Fellowship (CDRF) at Great Ormond Street, focusing on the psychosocial needs of children and young people with Juvenile Dermatomyositis (JDM). She subsequently became the first nurse in the UK to receive the prestigious NIHR Advanced Clinical Academic Fellow (ACAF) award in April 2023.

Her research initiative involves designing, developing, and testing a chatbot to support parents of children and young people with rheumatic conditions. Additionally, Polly holds various leadership roles, including Chair for Health Professionals at the Pediatric Rheumatology European Society, Associate Editor for Rheumatology Advances in Practice, and Communications Lead for the Clinical Academic Research Implementation Network (CARIN). She is also actively involved in The Centre for Adolescent Rheumatology at UCL, UCLH & GOSH and the Centre for Outcomes and Experience Research in Child Health, Illness, and Disability (ORCHID).

Matthew Sherman M.D., M.H.Sc., National Institutes of Health, USA

About this session:
Matthew Sherman, M.D., M.H.Sc., will explore different types of autoantibodies associated with juvenile myositis, their diagnostic and prognostic significance, and potential implications for treatment strategies. He will also delve into recent research findings, advancements in understanding the pathogenesis of juvenile myositis, and future directions in diagnosis and management.

About Dr. Matthew Sherman:

Dr. Sherman is a physician and researcher at the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) of the National Institutes of Health (NIH). He is a Fellow in the Clinical Fellowship Program at the NIAMS, specializing in the Muscle Disease Section.

He received his Doctor of Medicine (MD) degree from the University of Pennsylvania Perelman School of Medicine, completed his Residency in Internal Medicine at the University of California, San Francisco (UCSF), and Fellowship in Clinical Neurology at the University of California, Los Angeles (UCLA).

His research interests include neuromuscular diseases, including muscular dystrophy and peripheral neuropathy, muscle biology and physiology, clinical trials, and translational research in muscle disease.

Stacey E Tarvin, M.D., M.S., FAAP, FACR, University of Indiana School of Medicine, USA

About this session:
Hear from Dr. Stacey Tarvin about the use of JAK inhibitors in JDM and the potential efficacy of JAK inhibitors, such as tofacitinib and baricitinib, in improving muscle strength and skin symptoms in patients.

About Dr. Stacey Tarvin:
Dr. Tarvin is an Assistant Professor of Clinical Pediatrics at Riley Hospital for Children and Indiana University School of Medicine. She is the Vice Chair of the CARRA JDM Research Committee and Co-Lead of the CARRA JDM Therapeutics group, as well as a member of the PReS JDM working party.

She leads Clinical Research efforts in her division, including PI for pharma trials, registry studies, and investigator-initiated projects. Her focus is on therapeutics, consensus treatment plans, and telehealth in JDM.  She is the Chair of Cure JM’s Clinical Care Network.

Special Presentation by Invitation

Tania Gonzalez Rivera, M.D., FACR, VP Medical Affairs, Cabaletta Bio, USA

About this session: Tania Gonzalez Rivera, M.D., discusses the application of CAR-T cell therapy in treating myositis and delves into the mechanism of CAR-T therapy, its successes, challenges, and future prospects in revolutionizing autoimmune disease treatment.

About Dr. Tania Rivera:

Dr. Rivera is a renowned physician and executive leader in the biotechnology industry. She currently serves as the Vice President of Medical Affairs at Cabaletta Bio, a company dedicated to developing novel therapies for autoimmune diseases. Dr. Rivera completed her Doctor of Medicine and Residency at the University of Puerto Rico School of Medicine and her Rheumatology Fellowship at the University of Michigan Medical School.

Her research and clinical expertise include autoimmune diseases, particularly in the areas of rheumatology and dermatology. She has published numerous papers in peer-reviewed journals on topics such as psoriasis, lupus, and skin cancer and presented research findings at major scientific conferences, including the American Academy of Dermatology and the American College of Rheumatology.

The Potential of JAKs in Fighting JM

In our June 2024 “Ask the Doc” Town Hall, Dr. Julie Paik joins in a Q&A session to shed light on what JAK inhibitors are, how they work in JM, when parents might consider discussing JAKs as a treatment option, and the pros and cons of their use.

The Potential of CAR-T Therapy in Myositis.

The Very Real Potential of CAR-T Therapy in Myositis

We expected to hear promising news about the potential for CAR-T therapy to treat myositis at the Global Conference on Myositis (GCOM).

What we heard was more than promising—it was astounding.

What if I told you that there was a therapy that would “reset” the immune system—virtually eliminating the autoimmune response in myositis that causes inflammation, pain, muscle weakness, and other JM conditions of which we are all too familiar.

Pioneering JM Care – Integrating Research With a Multidisciplinary Approach to Care

Dr. Jessica Turnier of the University of Michigan / Mott’s Children’s Hospital is dedicated to creating new directions for JM research and wider perspectives on how it is treated. About the University of Michigan, she states, “When I came to Michigan, there were a lot of myositis patients, and I just developed a really strong connection with those patients.”

Join Cure JM

Membership is free and we’ll connect you with a network of support, encouragement, and resources.

Cure JM supports families, patients, and the juvenile myositis research community.

Interested in DIY fundraising but need help?

We’re here to help!

To have your fundraiser matched, add “DIY Match” in the memo of your online gift or check.