2017 Accomplishments: Spread The Word!
One of the most meaningful ways that our community members can contribute to the mission of PSC Partners Seeking a Cure is to spread the word about the work that we do! We are always looking for ways to reach PSC patients and caregivers who haven’t heard about our organization, so that they can have access to the education, support, and resources that are available. PSCers and their caregivers have an unbelievably wide reach through their existing networks, including hepatologists, gastroenterologists, family, and friends. Our goal is to ensure that no PSC patient or caregiver feels isolated and alone. Please help us reach this goal by spreading the word about what we do. It is often said that the PSC community is like a family – help us find patients and caregivers who don’t know that this family exists!
Please keep reading for a review of the exciting news that we have to share.
As the holiday season begins, we want to express our gratitude for the support of our community. In 2017, we continued to expand our efforts and further our mission of providing education and support to PSC patients, families and caregivers, and to raise funds to research causes, treatments and cures for primary sclerosing cholangitis. As the year ends, please consider making a tax-deductible donation to PSC Partners to help us continue to pursue this critical mission. Thanks to the support of members of our community, we are able to provide important information and encouragement to PSC patients and caregivers and fund the most promising PSC-related research performed by thought leaders in top academic institutions worldwide.
Thank you to everyone who has so generously given to PSC Partners through the years. Your continued support and dedication are truly appreciated. We couldn’t be where we are without you!
Together in the Fight,
PSC Partners Seeking a Cure
Canadian residents can receive tax receipts by donating to our affiliate, PSC Partners Seeking a Cure Canada, by clicking here.
Education and SupportPSC Partners Seeking a Cure is the only non-profit organization in North America to provide education and support for primary sclerosing cholangitis patients and their caregivers and to fund research into better treatments and a cure for PSC. Our most significant accomplishments this year were:
- PSC Partners continued to reach patients and caregivers, providing them education and support.
- Over 300 patients and caregivers attended the 2017 annual conference organized in collaboration with the Cleveland Clinic Transplant Center. Preparations are ongoing for the 2018 conference to be held on June 22-24 in Sacramento in conjunction with the University of California Davis.
- Our reach on social media expanded to over 5,000 Facebook members in public and closed groups, 1,112 Twitter followers and over 4,150 active contacts on our mailing list.
- PSC Partners partially funded an innovative 3-D animation video on PSC for patients to understand their disease and for physicians to explain PSC to their patients. The project was created by Dr. Cyriel Ponsioen and his team in the Netherlands and is available for viewing. You can find out more on this PSC Partners grant here, or you can go straight to the video by clicking here.
- Together with PSC Support in the UK, we launched a successful educational initiative designed to differentiate between PSC and PBC, two diseases of the bile ducts, and to diminish the risk of confusion between the two diseases. This work was possible thanks to a grant provided by Intercept Pharmaceuticals.
- We updated our brochures to reflect current information about PSC.
- PSC Partners began its video interview series entitled “Behind the Scenes,” created by a volunteer member who has been interviewing PSC experts on educational topics such as fecal transplant, clinical trials, and the PSC Forum. The videos can be viewed here.
- PSC Partners worked with a dedicated group of volunteers to help kick off a local support group program for PSC patients and caregivers.
- In November, volunteers presented a request to the Social Security Administration that PSC be included on the list of conditions approved for Compassionate Allowances.
- We expanded our Mentorship Program designed to pair newly diagnosed PSC patients and their caregivers with PSC patients and caregivers of similar ages and need.
Since 2009, PSC Partners has awarded over $2.6 million to support promising international research grants in adult and pediatric research. In 2017, PSC Partners focused yet again on filling gaps in PSC research, and funded seven studies that have the potential of leading to practicable tools and treatments:
- Dr. Mark Deneau of University of Utah received a PSC Partners grant to bring together pediatric data from over thirty medical centers and to enable robust pediatric studies.
- Dr. David Ellinghaus of Kiel University in Germany is studying the genetic changes in the gut-liver axis that predispose patients to PSC-IBD and aims to subsequently identify concrete gene targets to treat PSC.
- Dr. Richard Green of Northwestern University in Chicago is studying the impact of reduction of a specific protective protein observed in the liver in PSC and investigating the effects of this decreased protein on disease progression and on liver and bile duct cancers. He is searching for ways of increasing this protein to treat PSC.
- Dr. David Katzmann of the Mayo Clinic in Rochester adds a novel perspective to understanding PSC by studying the increased bile duct cell-to-cell communication in PSC. Identifying this cell-to-cell activity would bring a new, yet-untested path to PSC treatment.
- Dr. Kostas Lazaridis of the Mayo Clinic in Rochester and Dr. Tom Karlsen of Oslo University in Norway, running the two largest PSC databases in existence, are joining forces to study how to genetically predict PSC outcomes, specifically, the development of liver transplant, bile duct cancer and colon cancer. If these PSC outcomes can be predicted, then the risks to the patients would be lessened.
- Dr. Massimo Pinzani of the University College London in the UK is working on diagnosing and classifying PSC patients through a blood test measuring certain impaired proteins in PSC patients. This test would serve as biomarker for PSC, which does not exist yet and is highly sought after as a means to identify success of clinical trials.
- And lastly, the Canadian PSC Partners affiliate funded a grant for a study by Dr. Yury Popov of Harvard University in Boston who has identified an epithelial tissue which is nonexistent in normal bile ducts and the liver and that is present in bile duct cancer and in PSC. Since drugs inhibiting these tissues are already being developed for other diseases, they may be effective for the treatment of bile duct cancer and PSC.
The PSC Partners Scientific/Medical Advisory Committee (SMAC) selected this wide range of studies to target PSC at multiple levels and using different paths – genetic, cellular, protein, blood – for the purposes of diagnosis, prediction, prevention, and treatment of PSC. Patients and their families can move mountains. We are all in this together!
For full descriptions of all grants previously funded by PSC Partners, please click here.
Our International PSC Partners Patient Registry, established in collaboration with the NIH, is the only PSC patient-driven de-identified database. The registry attracts researchers and pharmaceutical companies that conduct clinical trials and are interested in developing PSC treatments. Our registry achievements this year include:
- We reached a milestone of 1, 100 patients in the registry.
- Ricky Safer and Rachel Gomel presented registry data at the 2017 PSC Forum Meeting in DC and at the Mayo Endowment meeting in Rochester, MN.
- Our registry data was the topic of three posters authored by PSC Partners, Dr. Greg Everson, Dr. Keith Lindor, and Dr. Christopher Bowlus. They were presented at the AASLD Liver Meeting in DC, at the European Association for the Study of the Liver (EASL) in Barcelona, and at the Digestive Disease Week (DDW).
- Two studies written with the collaboration of PSC Partners are based on data from the PSC Partners Patient Registry.
- “Development and validation of a primary sclerosing cholangitis specific patient-reported outcome (PSC PRO) instrument,” Hepatology (official journal of the AASLD; upcoming publication)
- “Characteristics and patient reported outcomes of primary sclerosing cholangitis from a patient-driven online registry,” Clinical Gastroenterology and Hepatology (The study is a comprehensive analysis of our registry data so far). Under revision
International and National Collaborations and Advocacy
Through its consistent engagement with the PSC community and support of cutting-edge PSC research, PSC Partners has become a valuable resource for parties interested in hearing the voice of PSC patients and caregivers. This year our collaborations include:
- Serving on the steering committee of the PSC Forum, part of the Forum for Collaborative Research / The Liver Forum and representing the voice of PSC patients (Ricky Safer)
- Serving on the External Advisory Committee of The Mayo Clinic Carlos Endowment to find a cure for PSC (Ricky Safer)
- Presented at CALD (Center for Autoimmune Liver Disease), an exciting collaboration for the study of pediatric PSC and AIH at Cincinnati Children’s Hospital Medical Center (Ricky Safer)
- Staffed booths at AASLD (American Association for the Study of Liver Disease meeting) and NASPGHAN (National Association for Pediatric Gastroenterology, Hepatology and Nutrition meeting); attended the EASL meeting in Amsterdam and the National Organization for Rare Disorders (NORD) Summit, which addresses the unique needs of rare disease patients and organizations.