April, 2021
	Your Fundraising at Work
	Mental and Emotional Health
	COVID-19 Vaccines Update
	Cure JM Town Hall: The Importance of Exercise In JM

YOUR FUNDRAISING AT WORK

Our support of NIH is a classic example of your fundraising dollars at work. We are able to leverage your dollars to focus some of the institute’s scientific prowess and firepower on improving treatments for our rare disease -- JM. Because of your fundraising, Cure JM’s funded programs at NIH have identified several new, potentially effective drugs for JM. Those drugs are now moving into trials and that means that better treatments are on the way for our kids.

Because any trials for JM must be approved by the FDA, we are hard at work to ensure that the challenges JM patients and families face and the need for better therapies are well understood by the FDA regulators. These regulators are charged with evaluating and approving the use of new drugs in the U.S. You've probably heard a lot about these FDA professionals as they were responsin ble for evaluating and extending the emergency approval for several COVID-19 vaccines.

Five Cure JM families recently appeared before the FDA to raise JM’s profile and testify for the critical need for better drugs. Many thanks to Nikki and Addie Hahn, Kendall and Vickie Tiffany, Sasha and Zamaya Sumling, Shari Hume, and Kristine, and Katherine Alderfer for bravely sharing their stories and making sure that the FDA knows about juvenile myositis and understands the urgent need for better treatments to treat JM.

Earlier this year, the Cure JM Board of Directors approved increases in grants to three Cure JM Centers of Excellence at Duke University and Children’s Hospital, George Washington University Hospital, and Seattle Children’s Hospital/University of Washington.

Why are these important?

As one example, Duke University is taking the lead in advancing a clinical trial for vamorolone. Vamorolone is a newer steroid that does not have most of the side effects of other steroids our children rely on, such as prednisone. Vamorolone is not yet approved for use in any disease, but a clinical trial for another pediatric autoimmune disease is nearing completion, and the drug’s eventual approval by the FDA could be a gamechanger for JM families. When approved, our goal is for the FDA to next approve a JM trial that, if successful, will lead to vamorolone’s use in JM children.

Similarly, our Center of Excellence at George Washington University has enrolled the final patient in a clinical trial for a drug known as abatacept. This drug is already approved for use in another pediatric autoimmune inflammatory disease—a form of juvenile arthritis. We hope that the results of this trial, to be completed this year, will provide safety and efficacy information that will lead to increased use and availability to use the drug in JM.

MENTAL AND EMOTIONAL HEALTH

Parents are acutely aware that for many JMers, mental and emotional health challenges go with the territory of living with the disease. Statistics from numerous surveys indicate that children with JM are suffering from depression and anxiety at two to three times a higher rate than those without JM. We also know that pediatric rheumatologists throughout the U.S. often lack the familiarity, training, and resources to effectively help our parents and children with mental health issues.

The Cure JM Board of Directors recognized the lack of training and resources for medical personnel as well as the lack of access to assessment and treatment pathways for JM kids. In response, through the Cure JM grant program, Cure JM will invest $100,000 over the next two years to enhance, develop and disseminate written and digital materials about mental health resources across the U.S. to our clinicians and families.

Families need support, too. We intend to develop support structures (Town Halls and possible peer support groups) for our families. We will work with our JM clinicians to help facilitate access to mental health assessments and, most importantly, treatment pathways in their practices.

We'll post additional information on mental health that may be of importance to you in the coming months through Cure JM publications and our social networks.

With appreciation,

Jim
James Minow
Cure JM Executive Director

COVID-19 VACCINES UPDATE

We have received many questions from our families on whether their child with JM should receive one of the COVID-19 vaccines recently granted emergency authorization from the FDA. Cure JM Foundation and our Medical Advisory Board have been monitoring the safety data of the vaccines and reviewing expert information and guidance relevant to our families.

In February 2021, the American College of Rheumatology (ACR) released guidance for rheumatology providers on the use of the COVID-19 vaccines for patients with rheumatic and musculoskeletal diseases (RMDs), which includes Juvenile Myositis.

As of now, there is no evidence that COVID-19 vaccines result in increased risk to patients with autoimmune diseases. Patients 16 and older should consider vaccinations at the earliest date of eligibility. ACR further recommends that patients consider delaying (or advancing) the use of certain medications from the date of the vaccination as an additional precaution. Please review the medication chart provided by ACR here.

Please note that this information is based on what we know now, so please check-in daily with reputable sources such as the CDC, your state and local public health agencies, and your healthcare provider. We will update you as soon as more relevant information becomes available.

CURE JM TOWN HALL:
THE IMPORTANCE OF EXERCISE IN JUVENILE MYOSITIS

Sunday, April 25th @ 3:00pm EST, 2:00pm CST, 1:00pm MST, Noon PST

This presentation will explore the importance of different types of exercise in the management of JM.

This Town Hall will be presented by Sue Maillard, a specialist physiotherapist at Great Ormond Street Hospital, London. She has specialized in Pediatric Rheumatology Physiotherapy for over 30 years and has a specialist interest in Juvenile Myositis. She has completed research in the safety of exercise in JDM and leads a physiotherapy team to provide an inpatient rehabilitation program for children, as well as reviewing and managing home treatment programs.