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Cure JM Foundation 2016 Annual Conference Recap

Conference Report from Cure JM Executive Director James Minow

JM Dad at Workshop

Jim Minow, Cure JM Executive Director, speaking at the Cure JM National Conference

Dear Cure JM Families,

I was honored to attend my first Cure JM Family Conference and Fundraising Event in St. Pete Beach, FL, and am pleased to share this report with all of our JM families, researchers, and community of funders and supporters.

For me, having been on the job for just one month, the Conference was an enlightening, rewarding, and humbling experience. Enlightening because it presented an opportunity for me to learn much about the research and to meet many of our amazingly dedicated research leaders. Rewarding because I met amazing families and children who are laser-focused on a future free of JM. Humbling because we have so much work ahead of us with tasks to expand the breadth and relevance of our research while dramatically increasing the level of funding Cure JM must invest toward new treatments and cures.

Prior to the Conference's opening day on January 15, the Cure JM board held an important two-day planning session to develop a roadmap for the future, and I'm pleased to share some of the significant outcomes.

First, the board thought it would be useful to underscore the Cure JM organizational focus and resolve through a review and, if necessary, an update of our Mission Statement. With co-founder Shari Hume’s sharp editorial pen, the board settled on a simple and pure statement that expresses our very reason for existence. The Cure JM mission is:

To find a cure for Juvenile Myositis and improve the lives of families affected by JM

Simple. Straightforward. Visionary. Everything a mission statement should be.

Second, the board focused most of its remaining time on research and fundraising strategies focused on local communities with national support which I will also share here.

Research Investment Strategy

On the research front, the board developed, with suggestions from the Medical Advisory Committee, a research investment strategy that will focus and concentrate future investments in four research areas, each with its own set of outcome objectives. The four areas are:

  1. Basic research. Investing to better understand the underlying mechanisms of JM and the cellular, genetic, and environmental causes required to guide therapeutic research
  2. Genetics.  Investing in longer-term solutions to correct the underlying genetic causes of autoimmune dysfunction in JM and discover methodology for gene therapy treatments
  3. Translational.  Speeding potential therapies to children—the "bench to bedside" process in cellular, biologic, drug, and gene therapies that are available to patients and provide effective therapeutic interventions.
  4. Drug Development.  Investing in the discovery of new drugs or to repurpose existing drugs to provide more and better treatment options

In addition, the board expressed a commitment for Cure JM to monitor and, when possible, support career development activities to encourage younger researchers and clinicians to pursue careers in the area of juvenile pediatric rheumatology and autoimmune disorders (JM specific).

Kim, Sue and and Kristen Carpenter

Cure JM's Medical Advisory Committee members answer questions at the Cure JM National Conference. Pictured from left to right are Dr. Lauren Pachman, Dr. Lisa Rider, Dr. Susan Kim and Dr. Adam Huber

Enhancing the Role of the Medical Advisory Committee

The board further expressed a commitment to enhance the role of the Medical Advisory Committee in developing a research implementation strategy and resource allocation strategy for Cure JM, with the goal of advancing new therapies as quickly as possible. In reaching this decision, the board determined that advancing the Cure JM mission will be strengthened and accelerated by a highly engaged and outcome- oriented Medical Advisory Committee working in close collaboration with board leadership and the Executive Director.

Research outcomes that lead to treatments and cures is at the heart of the Cure JM mission, and fulfilling that mission requires the active leadership of the scientific and medical community. We've been fortunate to already have that leadership in place, and look forward to a more formal and integrated role for the Medical Advisory Committee as we move ahead.

Research Program Highlights

Abatacept Clinical Trial

A new clinical trial is underway to test the efficacy of an existing drug, Abatacept (trade name of Orencia), in children with moderately active juvenile dermatomyositis who have not responded to prednisone and at least 1 other medication. Patients need to be at least 8 years of age and weigh at least 66 pounds as part of the study qualifications. The trial is being led by the George Washington University Myositis Center, one of two “Cure JM Centers of Excellence.”

Abatacept is a drug compound approved by the FDA for adult and juvenile rheumatoid arthritis. It works by preventing T Cells from becoming fully activated, thereby interrupting the autoimmune responses in the body. It's manufacturer, Bristol-Myers Squibb is also conducting clinical trials to test for efficacy in lupus and other autoimmune diseases.

The GW Myositis Center has just begun to enroll patients.

Understanding and Modulating the role of Natural Killer Cells

Another exciting research project is focused on understanding how natural killer cells (NK cells) proliferate in response to viral infection in children and then self-regulate to return to normal levels as the virus subsides. In children with autoimmune disorders, functional defects in natural killer cells may well exist, and ineffective NK cell responses to viral infections may contribute to the pathogenesis of juvenile myositis and other musculoskeletal disorders.

A collaborative venture between Dr. Lauren Pachman and Dr. Anthony French seeks to identify functional and specific NK cell defects in patients with autoimmune disorders (such as JM) that could lead to earlier and more effective therapeutic interventions to either modulate NK cell responses or stimulate more effective responses. Dr. Pachman leads the Cure JM Center of Excellence at the Ann and Robert H. Lurie Children's Hospital in Chicago, and Dr. French is an Associate Professor of Pediatrics at Washington University Hospital in St. Louis.

Tests developed at Dr. French's lab will be useful in screening a wider group of patients with autoimmune or autoinflammatory disorders. Detecting NK cell deficits in children and adolescents with JM has the potential to provide insights into the role of the innate immune response, and in particular NK cells, in the pathophysiology of pediatric musculoskeletal disorders.

Identification of Risk Factors in JM

A significant outcome of the recently completed MYOGEN study was the identification of risk factors associated with a class of genes associated with dermatomyositis and polymyositis—the HLA genes- that influence immune response. Most recently, the risk factor has been further isolated to certain sectors of the gene (known as alleles).

A different set of genes, also within the HLA region, may be a risk factor for calcinosis- this is an area of active research at present.

Researchers at Stanford University will soon conduct sequencing studies of the entire HLA region to further detail the HLA allele risk factors. This is important because researchers must locate and understand the genetic "misspellings" in alleles to better understand the pathways involved in the disease process.

These three projects are but a representative sample of the progress being made within the overall research portfolio supported by Cure JM. There are too many to report in this document alone, but look for additional updates on other projects on the Cure JM website soon.

The board also heard reports on advancement in patient care at Cure JM's Centers of Excellence at George Washington University Hospital and Chicago Children's Hospital. My thanks to all of the clinicians and researchers who attended the Family Conference and who shared their work with those in attendance. They include Drs. Rodolfo Curiel, Megan Curran, Adam Huber, Hannah Kim, Susan Kim, Takayuki Kishi, Lauren Pachman, Sukesh Sukumaran, Ann Reed, Lisa Rider, and Chack-Yung Yu.

New Compound Trial from Idera Pharmaceuticals

We also welcomed representatives from Idera Pharmaceuticals, a new and important partner with Cure JM as we increasingly move into an era of clinical trials. Idera is currently enrolling adult dermatomyositis patients in a Phase II clinical trial to assess the efficacy of its compound, IMO-8400, in treating the disease. The compound acts to inhibit the role of "Toll-like receptors" that are thought to play a key role in autoimmune diseases such as adult and juvenile dermatomyositis.

While the trial is currently enrolling adult patients, Idera is also assessing the possibility of testing efficacy and safety in juvenile dermatomyositis patients in the future.

The Cure JM Medical Conference

I would also like to thank board member and Vice President for Research Amy Gleason for her tireless work in developing the Cure JM Medical Conference in partnership with Johns Hopkins Medicine and All Children's Hospital in St. Petersburg. The conference, held on January 15, brought together dozens of researchers and clinicians from North America to share ideas, presentations, and research outcomes. Several Cure JM funded clinicians including Drs. Lauren Pachman, Ann Reed, Lisa Rider, Charles Spencer, and Chack-Yung Yu presented at the Conference.

Presentations from the conference will be summarized and shared on the Cure JM website in the near future.

Expanding Fundraising

In its planning session, the board also addressed Cure JM's need to add to its committed and passionate volunteer ranks and determined that the organization would benefit from a better defined support infrastructure for fundraising and outreach. The board approved a plan that provides for three organizing components.

First, the board authorized the creation of Cure JM chapters around the country. Chapters will have three primary responsibilities—to organize families and others in a Chapter region to raise funds to support the research mission; to provide information and support to chapter families and particularly to families with a newly diagnosed child; and, to reach out to the local pediatric medical and rheumatology community with information about juvenile myositis, to assure that all JM kids are diagnosed early and have access to the best possible care.

Second, the board is expanding the role of the Cure JM National Leadership Council (formerly the Advisory Council) to serve as high level leaders, facilitators, and "momentum builders" to move the mission of Cure JM. Leadership Council chair Nikki Hahn met with other Council members present to outline a vision for the future of the Council.

Nikki proposed a total membership of about 30 individuals spread across six committees: Development (fundraising and major gifts); Chapters and Events; National Walk; Communications and Social Media, and Medical and Research. Descriptions of the roles of each committee have been developed, and Nikki is in the process of recruiting committee chairs.

Third, the board itself recognizes the need to expand and begin the process of identifying the next generation of potential Cure JM leaders. Moreover, the board expects that as the scope and number of research projects increase, the research budget will necessarily increase as well on an order of several magnitudes (translational research and clinical trials are far more expensive than is basic research.) The board acknowledged its own responsibility to secure new revenues in working with the Leadership Council and Cure JM's newly formed chapters.

In closing, I'd like to thank the many volunteers who worked diligently to make the 2016 Family Conference such an unqualified success. I am especially grateful to Chairman Rhonda McKeever for her overall board and Family Conference leadership.

My thanks, too, go to Shannon Malloy for her excellent staff leadership to the Conference, and to our families for all of your year-end fundraising on CrowdRise and through the St. Pete Beach half marathon. And finally, a bow to our Conference sponsors--Alderfer, Bergen & Co., Borrow Lenses, Dun & Bradstreet, FedEx, Idera, JMC Services, Inc., Zimmer Biomet; and Wissmetac.

We simply could not have pulled off such a great conference without our sponsors and families. Should any of you have questions or wish to discuss ways that you can help further move the Cure JM mission, please feel free to contact me at james.minow@curejm.org. I look forward to hearing from you!

And please save the date for the next Cure JM National Conferene and fundraising event in Austin, TX February 17-19, 2017, more information coming this spring.  

With appreciation,

Jim Minow

Executive Director
Cure JM Foundation