Dupuytren Foundation 2016 Mid-Year Report
2016 has already exceeded expectations for the Dupuytren Foundation. Here are some of the highlights.
Progress on International Dupuytren Data Bank Research
The International Dupuytren Data Bank (IDDB) launched in November 2015. The goal of the IDDB is a cure for Dupuytren disease. This research has been made possible through your support.
A blood test is the missing piece of the Dupuytren puzzle needed to develop and test medical treatments. Right now, the only tools we have to measure Dupuytren/Ledderhose disease are the ruler, protractor, and camera. These are like a car odometer: they show what has already happened, how far the disease has gone. That’s not helpful if the goal is to slow or stop Dupuytren disease. To do that, we need to measure how fast the disease is going right now – a Dupuytren/Ledderhose speedometer. A blood test could show what is happening right now, including effects of medical treatment, diet, physical activity, or other lifestyle changes – before any changes in lumps or angles. A blood test could give the power to test treatments for Dupuytren disease more quickly and accurately than has ever been possible.
The first goal of the IDDB is to develop a Dupuytren blood test. We’re starting with a list of potential blood tests based on what we know from prior research. The list includes tests of genetic activity, the immune system, inflammation, nutritional factors, and others.
Legal and Privacy Oversight
The IDDB is a collaboration between the Dupuytren Foundation and the Arthritis Research Center (ARC). The IDDB is an extension of the existing ARC research platform. Privacy policies, investigational board review oversight and database structures already in place for Arthritis Research Foundation research apply also to the IDDB. A Clinical Research Organization (CRO) is needed for any large-scale medical research, and the IDDB is contracting with Palm Beach CRO to provide this additional oversight and logistic support.
Thanks to your support, the IDDB currently has over 1400 enrollees from 25 countries. The majority are from the United States, Canada, the United Kingdom and Australia. Some of the demographics are different than prior research. This is because the IDDB is designed to include people missed by older approaches, such as those with early disease, or those who haven’t seen a specific doctor. Compared to traditional studies, a higher percentage of IDDB enrollees are women – almost half. The average age is 59, but spans from 17 to 88. On average, enrollees have lived with Dupuytren disease for 10 years.
Because Dupuytren disease behaves differently in each person, more enrollees are needed before these demographics will have statistical strength. That’s why it’s so important for the Dupuytren community to spread the word about this study and to encourage others with Dupuytren disease to enroll. How important? Almost three-quarters of enrollees found out about the IDDB online; less than one out of ten heard about the study from their own doctor. The online shortcut link is easy: DupStudy.com. The first follow-up survey is about to go online to dig even deeper into the nature of Dupuytren disease.
Plans are also underway for the next half of this study: finding a Dupuytren blood test – a biomarker. The most promising tests are not done by standard laboratories. Our starting list includes fibrocytes, T cells, RBC Magnesium levels, WT1, CCN2, TGFβ1, TGFβ2, TNFα, TNFR1, TNFR2, CITP, PIIINP, IGF-II, IGFBP-6, interferon gamma, interleukins, antibodies to WT1, MicroRNA, DNA genotyping, and DNA methylation.
Some of these blood tests are very expensive – too expensive to run all the blood tests on everyone in the study all at once. This part of the study will start small and then grow in competitive cycles. In each cycle, groups of people will be selected based on disease severity to collect blood samples and identify the best blood tests to repeat in the next cycle. Following cycles will do the same with more people and fewer tests until the best tests are confirmed. This strategy is designed to reduce overall costs. This strategy of repeated testing is also the fastest way to confirm the accuracy and reliability of a test available to all researchers to develop a cure for Dupuytren disease and related conditions.
The starting point for this project is a pilot study of 100 participants chosen from online survey enrollees. Blood test results of 50 people with severe Dupuytren disease will be compared with 50 demographically matched people without Dupuytren disease. After analysis of these results, the next research phase will involve more participants, a refined range of tests, and an additional group of participants with mild Dupuytren disease to enhance data quality. This pilot study will pave the way for greater funding opportunities and further research to develop a cure for Dupuytren disease and related conditions.
Test, analyze, revise, retest – until biomarkers are identified, as outlined here:
We’ve reached the survey enrollment benchmark to begin this next phase. We’re still working on the logistics of processing each of the blood tests for this first round. We anticipate each round of this project to take twelve to eighteen months. Depending on the number of cycles, funding requirements, and luck, the entire process of identifying should take three to six years – very short for a solution two centuries overdue. This is all very exciting: a new era for Dupuytren research with the promise of new ways to evaluate Dupuytren disease and to speed up the process of developing preventative treatments.
If you haven’t enrolled, now is the time. Visit http://DupStudy.com
Progress on Dupuytren Foundation Research Committee
Dupuytren biology is a complicated mix – genetics, immunology, inflammation, wound healing, biomechanics, gender, and the biology of aging. Biologists in these fields are superspecialists, each working on specific areas. Understanding Dupuytren biology requires bridging the boundaries of these separate fields. Our 2016 Research Committee members bring a wide range of knowledge and experience from five countries to find a cure for Dupuytren disease. It’s an amazing team: https://dupuytrens.org/research-committee/
Progress on Dupuytren Foundation Workforce and Workplace
In early 2016, we relocated our office to a permanent location in Palm Beach County, Florida. I am pleased to announce two new employees added to the team at this new location to assist with our rapid growth: Dupuytrens.org/development-staff/. Chief Development Manager Patricia Rowen and Manager of Outreach John Mac have come in and hit the ground running. Both bring a wealth of non-profit experience and have already made a huge difference in the direction and current development of the organization. We’ve completed a complete overhaul of the foundation email and contact list database. The Dupuytren Foundation has been awarded Guidestar gold level nonprofit transparency rating: http://www.guidestar.org/profile/26-3886958.
I’m also very pleased to announce the addition of John Blades and Stuart Landow to our Board of directors https://dupuytrens.org/homepage/board-of-trustees/. They bring with them a new level of nonprofit, business and legal experience to the Dupuytren Foundation. I’m particularly grateful for the growing involvement and volunteer work of Dupuytren patient advocates including Leslie Wieslander Rosenthal, Eric Forman, Anne Sjoukje Schurer, Dr. Denise Nagel, Ken Bernstein, Dr. Wolfgang Wach, and far more than I can list, working hard behind the scenes to support the quest for a cure. This growing group of advocates play crucial roles in outreach and improvement of the IDDB participant experience.
Progress on Research Support
Our Fundraising goal for the year is $200K to be able to begin IDDB blood collection. This can be achieved through your continued support, in addition to grant support from family and corporate foundations. Support this research with a charitable gift at https://dupuytrens.org/donate/. Results from this initial pilot study will provide the basis for larger grants and government funding for the next phase of research.
And Just For You…
I understand email overload – that’s why the number of newsletters is kept to a manageable level. However, there is much going on behind the scenes and much more available on the Dupuytren Foundation website. Browse through the list of blog posts at https://www.dupuytrens.org/dupuytren-blog-index/. Browse Dupuytren.tv, our YouTube channel. Browse the drop-down lists on the top of Dupuytrens.org. It’s all there just for you. I invite you to contact the Foundation to share your concerns, ask questions, or just check in. You deserve this – your help has made this possible!
Charles Eaton MD