Cure JM Foundation Newsletter

May 2015

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Cure JM Awards Two New Research Grants

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

Thanks to your outstanding support, Cure JM is pleased to announce that we are funding two new research grants to begin work immediately.

These two research studies hope to identify genetic markers that cause JDM (Juvenile Dermatomyositis, a form of Juvenile Myositis), so that we can develop better and more specific ways to diagnose and treat JDM.

These two studies are:

Complement C4 in Disease Risk and Pathogenesis of Juvenile Dermatomyositis

Investigator: 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

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
Investigator: Hanna Kim, MD, Fellow, Division of Pediatric Rheumatology Children's National Medical Center and National Institutes of Health

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.

To evaluate and award these grants, Cure JM collaborated with a scientific peer-review committee from CARRA, the Childhood Arthritis and Rheumatology Research Alliance. CARRA is a North American organization with over 400 members involved in pediatric rheumatology research.

Twice per year, with the help of this peer-review process, Cure JM identifies and funds the most promising and scientifically sound Juvenile Myositis research projects available - with the ultimate goal of finding better treatments and a cure.

Cure JM's next round of grants will be awarded in September. Physicians and researchers from across North America are invited to submit their proposals by July 31st.

In addition to these two new grants, Cure JM is also funding research at the Mayo Clinic, Boston Children's Hospital, Children's Hospital at Montefiore, Seattle Children's Hospital, Stanford University, the George Washington University Myositis Center, and the Cure JM Program of Excellence in JM Research at Stanley Manne Children's Research Institute, affiliated with Ann & Robert H. Lurie Children's Hospital of Chicago.

Thank you for making this research possible. Click to learn more about Cure JM-funded research.

21st Century Cures Act Unanimously APPROVED: 51-0

Last week, the United States House of Representatives' Energy and Commerce Committee unanimously passed H.R. 6, known as the 21st Century Cures Legislation.

Last week, the United States House of Representatives' Energy and Commerce Committee unanimously passed H.R. 6, known as the 21st Century Cures Legislation.

This is a crucial step in advancing measures that enhance drug discovery and development of treatments and cures for patients.

"The nonpartisan legislation will help to modernize and personalize health care, encourage greater innovation, support research, and streamline the system to deliver better, faster cures to more patients," according to the committee press release.

Some benefits for rare disease organizations like Cure JM include:

pharmaceutical companies will receive incentives to repurpose existing medications for rare diseases
the "Rare Pediatric Disease Priority Review Voucher Program," which provides major incentives to pharmaceutical companies to develop novel rare disease treatments, will be extended for 4 years
breakthrough drugs will be expedited through FDA approval
drug development will include patient involvement
the NIH will receive staffing for natural history registries (especially for rare diseases); the staff will collect, maintain and analyze data
the NIH will receive increased funding
data from NIH studies will be shared with all NIH researchers

"Cure JM Foundation, along with many other rare disease organizations, endorsed this legislation with letters of support," says Cure JM co-founder Shari Hume. "We are grateful to Chairman Fred Upton (R-MI) and the members of the House Energy and Commerce Committee for approving this momentous legislation for the rare disease community. The 21st Century Cures Act would enable Cure JM to speed our quest for a cure and improve the lives of patients and their families."

Cure JM will provide more updates as this bill continues through the legislative process.

If you would like to get involved or learn more, email info@curejm.org.

The Cure JM National Conference is in St. Petersburg, Florida, January 15th-17th

Join over 80 families at the Cure JM National Conference, January 15-17, 2016 in St. Petersburg, FL

Join over 80 other Cure JM families for an unforgettable weekend at the 9th Cure JM National Conference.

The conference will be held in St. Petersburg, Florida, January 15-17, 2016.

The St. Petersburg Conference is shaping up to be Cure JM's biggest and most exciting conference yet!

Some highlights will be:

Intimate break-out sessions about coping with real life issues facing JM patients, families and caregivers
An Educational Forum and Q&A with prominent Juvenile Myositis researchers
Special sessions for dads, moms and grandparents who care for children with JM
A Teens and Tweens cyber lounge and workshop on transitioning from pediatric to adult care
A Kids' Fun Camp where patients and siblings will enjoy activities, have fun and form lasting friendships with kids just like them
The St. Pete Beach Classic, where Cure JM runners, walkers and volunteers will help raise funds for JM research. This is a high point of the weekend!

Learn more here or contact us at info@curejm.org or (780) 487-1079.

Cure JM Attends CARRA’s Annual Scientific Meeting

Cure JM President Mitali Dave attended CARRA's annual scientific meeting last month in Austin, TX

As part of Cure JM's ongoing partnership with CARRA, Cure JM President Mitali Dave represented Cure JM at CARRA's annual scientific meeting last month in Austin, TX.

CARRA, the Childhood Arthritis and Rheumatology Research Alliance, is a North American organization with over 400 members involved in pediatric rheumatology research.

At the conference, Mitali participated in a parent panel that discussed the benefits of involving patients and their families in the research process so that their needs and concerns are addressed. Parental involvement is important because while physicians have deep medical knowledge and training, for rare diseases like JM, most physicians only see a few affected children, while today's parents are often connected with a strong community of affected families. Even for more common diseases, physicians only see infrequent snapshots of a child in a clinical setting, whereas parents see the whole child 24/7.

Mitali also participated in various small work group sessions where she represented the patient's voice.

Some working groups were developing studies to compare the effectiveness of different treatments. Physicians do not know definitively which treatments will work best for which patients, and they are unable to give patients and their families the data that would help them make an educated choice among a range of treatment options. These work groups hope to provide that critical data.

The JM populations that they hope to test treatment plan options for are:

1) Recently diagnosed children where skin is the predominant problem

2) More long-term patients whose skin issues do not go away after standard treatment

3) More long-term patients with a difficult disease course who have failed first-line therapies and need a biologic

4) More long-term patients with calcinosis

Two other CARRA JM working group sessions centered on developing better quality of life measures and surveying adults with JM to better understand adult outcomes.

Cure JM welcomes this more collaborative approach of patients and researchers working together.

Q & A with Rheumatologists & JM Researchers

Dr. Huber

Dr. Adam M. Huber, whose current research interests include improving how children with Juvenile Myositis are evaluated and treated and understanding outcomes of children with Juvenile Myositis.

Rheumatologists and JM researchers are answering your questions. Questions can be submitted to info@curejm.org.

Question: How should families handle the transition from pediatric to adult care? Are there adult rheumatologists who know a lot about Juvenile Myositis?

Answer: Provided by Dr. Adam M. Huber, Pediatric Rheumatologist and Professor of Pediatrics at the IWK Health Centre and Dalhousie University in Halifax, Nova Scotia, Canada.

It is very important to distinguish between "transition" and "transfer".

"Transition" is an ongoing process that should go on for much of your time with your pediatric rheumatologist. It is all about the process of patients taking an increasing amount of responsibility for their illness: understanding it, knowing medications, taking medications, looking after themselves, being able to talk to the doctor, identifying when there is a problem and knowing what to do. At the same time, parents are giving up some of that responsibility (which is sometimes very difficult).

"Transfer" is the one-time event when a pediatric patient is formally transferred to the adult system.

The pediatric rheumatologist and clinic should be engaged in the transition process over many visits, ideally over years. Things often start (baby steps) around 12 years old but it can earlier. For example, I ask my patients what medications they are on no matter how old they are. When this works, it means that patients and families are well prepared and ready for transfer when the time comes.

The right time to transfer to adult care depends on a number of factors. Sometimes it is related to an age (often defined by the health care system) like 16 or 18. Sometimes it is related to graduations from high school. It may also be influenced by the presence of other illnesses, disease severity or availability of adult care.

Children with Juvenile Myositis would normally be transferred to an adult rheumatologist. Your pediatric rheumatologist will know who is available in your community (or the community that you are going to for school or work). Just like in the pediatric community, some adult rheumatologists know a great deal about myositis, and others know less. Exploring this with your pediatric rheumatologist is very important. There might even be circumstances where you might travel to a specialized clinic, although this would probably not be typical.

What is most important is that you have these conversations with your rheumatologist.

JM Research Studies Need Patients

I Need a Cure

Volunteers are urgently needed for Juvenile Myositis research studies

Volunteers are urgently needed for Juvenile Myositis research studies.

With the rarity of Juvenile Myositis, your participation is crucial for keeping research moving forward.

Pediatric identical twins are urgently needed. Sets of twins are needed where one twin has Juvenile Dermatomyositis (JDM) and the other twin does not. For further information please call (312) 227-6277. Study conducted at the Cure JM Program of Excellence in JM Research at Stanley Manne Children's Research Institute, affiliated with Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL.

Join the Cure JM Patient Registry. This information is collected online and will be used by researchers as part of a demographic survey to determine if there are clusters of children affected by Juvenile Myositis (JM) in the same geographic area. Click here to join.

Pilot Calcinosis Study conducted at George Washington University, Washington, D.C. This is a study for treating calcinosis (calcium deposits in the skin) in individuals with Adult or Juvenile Dermatomyositis who have not responded to other treatments. Participants must be between the ages of 18-65 years, have stable disease which is mildly active or in remission, and have failed at least one therapy for superficial calcinosis. Please call (800) 411-1222.

Twin/Sibling Study conducted by the National Institute of Environmental Health Sciences (NIEHS) in Bethesda, MD. You may enroll at your local doctor's office, Bethesda, MD or in Research Triangle Park, NC. This is a study for families with siblings or twins in which one sibling has developed an autoimmune disease and the other has not. Now enrolling patients within 5 years of diagnosis. Siblings need to be of the same gender, within 5 years of age and without an autoimmune disease. There is no charge for study-related evaluations. Please call (800) 411-1222.

Microbiome Study conducted at Seattle Children's Hospital, Seattle, WA. Currently enrolling children with JDM who are less than 14 years old. Study participants must enroll either before any treatment has started or be off of immunosuppression for 3+ months and beginning to flare. Please call (206) 987-2057.

MYORISK Study conducted at the National Institutes of Health (NIH) in Bethesda, MD. You may be enrolled at your local doctor's office or a number of locations around the United States. Currently enrolling patients who were diagnosed in the last two years. Doctors at the NIH are conducting pioneering research into the environmental risk factors that could lead to myositis. Please call (800) 411-1222.

Study of Premature Atherosclerosis in Juvenile Dermatomyositis conducted at the Children's Hospital at Montefiore, Bronx, NY. Currently enrolling patients age 2-21 with a diagnosis of JDM. To be eligible patients cannot be currently using medications that alter lipid metabolism or endothelial function (including lipid lowering agents), be a smoker or have another chronic illness (other than JDM).Please call (718) 696-2405.

Click here for a complete list of research studies needing volunteers.

Upcoming Events

Dinner, Auction and Roaring 20's party benefiting Cure JM
Friday, May 29
Hillsboro, OR

Chuck E. Cheese party benefiting Cure JM
Friday, May 29
Mentor, OH

Graceyfest 2015: Music, Food and Fun
Sunday, May 31
Mt. Washington, KY

Basket Raffle and Silent Auction
Sunday, May 31
Rutland, VA

Dmitri's Mission to Find a Cure
Saturday, June 6
Mineville, NY

Race for a Cure at Bronco Raceway
Saturday, June 20
Cleveland, TX

2nd Annual Cowboy Brad Concert
Saturday, June 27
Estes Park, CO

11th Annual Strawberry Festival
Sunday, June 28
Crown Point, NY

3rd Annual Texas Family "Mini-Conference"
Saturday, July 18
Martindale, TX

Evening Under the Stars with Don Middlebrook and the Pearl Divers
Saturday, August 1
Warsaw, IN