Introduction
This site provides a compilation of www resources and scientific literature on primary sclerosing cholangitis (PSC), a chronic cholestatic liver disease characterized by periductal inflammation of both intrahepatic and extrahepatic bile ducts. Progressive loss of bile ducts impairs bile flow, ultimately resulting in cirrhosis. Males are affected by PSC more often than females (2:1), and the disease is often accompanied by inflammatory bowel disease; mostly ulcerative colitis and sometimes Crohn's disease. The exact causes of PSC are unknown, but it is thought that the disease might be triggered by as yet unidentified environmental factor(s) in genetically susceptible individuals.
The genetic basis of PSC is likely to be complex; certain of the genes determining susceptibility are thought to reside in the major histocompatibility complex - a genetic region of central importance in a number of autoimmune diseases. Because PSC is often associated with inflammatory bowel disease, the complex genetic basis of ulcerative colitis and Crohn's disease may be relevant to understanding how PSC is initiated and sustained.
The National Institute of Diabetes and Digestive and Kidney Diseases, the Office of Rare Diseases, and the Morgan Foundation jointly sponsored a Primary Sclerosing Cholangitis Conference in Bethesda, MD, September 19-20, 2005. The aims of the meeting were to stimulate clinical and basic research interest in PSC and identify gaps in knowledge and challenges for medical research in this disease:
The European Association for the Study of the Liver (EASL) held a Monothematic Conference on “Primary Sclerosing Cholangitis” in Oslo, Norway June 21-23, 2009. Central questions that were addressed at this conference included:
- Epidemiology and natural history of PSC
- Pathogenesis of PSC
- Overlap syndrome and autoimmunity in PSC
- Malignancies in PSC: pathogenesis and treatment
- Transplantation in PSC including recurrence
The Morgan Foundation has established a research initiative entitled STOPSC: 'Studies of Primary Sclerosing Cholangitis'. STOPSC is a collaborative effort between centers in the United States and Canada whose primary objective is to collect and analyze information required to understand the etiology, pathogenesis, and treatment of primary sclerosing cholangitis (PSC). Researchers who are interested in learning more about STOPSC should contact the STOPSC Data Coordinating Center (see About STOPSC).
This site is associated with PSC Partners Seeking a Cure (www.pscpartners.org), a 501(c)3 nonprofit foundation that endeavors to find a cure for Primary Sclerosing Cholangitis. Please consider a donation to help us conquer this devastating disease. For more information about how to make a tax-deductible donation, please visit PSC Partners Seeking a Cure (www.pscpartners.org).
The three-fold purpose of the PSC Partners Seeking a Cure foundation is to:
(i) raise funds with which to research the causes and cures of PSC,
(ii) provide education and support to PSC patients and their families, and
(iii) promote PSC and organ donation awareness.
 The 7th PSC Partners Seeking a Cure conference will be held April 29 - May 1, 2011 at the Sheraton Grand in Sacramento, California in conjunction with Dr. Chris Bowlus and his colleagues at the University of California Davis. Please join us in our next fundraiser ... Itching for a Cure: Road to Sacramento.
If you are a PSC researcher seeking grant support, please consult the PSC Partners Seeking a Cure: Request for Proposals. If you are a PSC patient or caregiver, please consider joining our mailing list:
PSC Partners Seeking a Cure has recently donated $40,000 to support the STOPSC research initiative of the Morgan Foundation; see press release from: Memphis Business News, Jan 8, 2007.
PSC Partners Seeking a Cure awards a $3,000 prize annually at the annual meeting of the American Association for the Study of Liver Diseases (AASLD). The recipient of the 2007 PSC Partners Seeking a Cure AASLD Award was Dr. Thomas H. Karlsen (Medical Department and Institute of Immunology, Rikshospitalet-Radiumhospitalet Medical Center, Oslo, Norway), as described in the AASLD 2007 Annual Report. The recipients of the 2008 PSC Partners Seeking a Cure AASLD Awards were Dr. I. Tornai (2nd Department of Medicine, University of Debrecen, Debrecen, Hungary), and Dr. P. G. Blanco (Beth Israel Deaconess Medical Center, Boston, MA, USA), as described in the AASLD 2008 Annual Report. The 2009 award winner was Dr. V.S. Teaberry (Duke University, Durham, NC), as described in the AASLD 2009 Annual Report. The 2010 award winner was Dr. John Eaton (Mayo Clinic, Rochester, MN).
In its 2009 grants competitions, PSC Partners Seeking a Cure made 8 awards to the following investigators and projects:
- Konstantinos N. Lazaridis, M.D., Assistant Professor of Medicine, Center for Basic Research in Digestive Disease, Mayo Clinic College of Medicine 200 First Street SW, Rochester, MN 55905. Examining the Disease Impact of Genetic Variation in Logical Candidate Genes for PSC: a PROGRESS Study. $40,000 over 2 years.
- Pietro Invernizzi, MD, Ph.D, Assistant Professor of Medicine, Division of Rheumatology, Allergy, and Clinical Immunology, University of California, Davis GBSF suite 6515, Davis, CA 95616. Cholangiocarcinoma-associated serum microRNAs in primary sclerosing cholangitis: Identification and prognostic potential. $40,000 over 2 years.
- Cyriel Y. Ponsioen, MD, PhD, Department of Gastroenterology & Hepatology, Academic Medical Center, C2-112, P.O. Box 22700, 1100 DE Amsterdam, The Netherlands. Aberrant homing of lymphocytes to the liver in patients with primary sclerosing cholangitis; the missing link between colon and liver. $40,000 over 2 years.
- Christopher Bowlus, M.D., Associate Professor, Division of Gastroenterology, UC Davis Medical Center, 4150 V Street, PSSB 3500, Sacramento, CA 95817. alpha4Beta7-Integrin Ligand Development for the Treatment of PSC. $40,000 over 2 years. This project will be entirely funded by a generous donation from Abe and Rachel Gomel.
- Gregory T. Everson, M.D., Transplant Center and Hepatology, Clinic Mail stop B, 154 Anschutz Outpatient Pavilion, 1635 N. Ursula Street, Room 7085, Aurora, CO 80045. Non-invasive Assessment of Disease Progression in Primary Sclerosing Cholangitis. $40,000 over 2 years.
- Richard M. Green, M.D., Associate Professor of Medicine, Division of Hepatology, Northwestern University Feinberg School of Medicine. The Role of Abcb11 and Fibroblast Growth Factor 15/19 in the Pathogenesis of Primary Sclerosing Cholangitis. $40,000 over 2 years. The first year of this project will be funded by a generous donation from Ros and David Parry.
- Keith D. Lindor, M.D., Mayo Clinic, 200 First Street, SW, Rochester, MN 55905. A Pilot study of Vancomycin or Metronidazole in patients with Primary Sclerosing Cholangitis. $25,706 over 2 years. Clinical Trial NCT01085760.
- Gideon Hirschfield, MA MB BChir MRCP PhD, Assistant Professor of Medicine, University of Toronto, Liver Centre, Toronto Western Hospital 399 Bathurst St, 6B Fell, Rm 162, Toronto, ON, M5T 2S8, Canada. Quality of life in patients with primary sclerosing cholangitis. $10,000 over 2 years. This project will be funded in large part by a generous donation from Hoops 4 Healing.
In its 2010 grants competition, PSC Partners Seeking a Cure made 5 awards to the following investigators and projects:
- Mario Strazzabosco, M.D., Ph.D, Department of Internal Medicine Section of Digestive Diseases, Yale University, 333 Cedar Street-1080 LMP, P.O. Box 208019, New Haven, CT 06520-8019, and Michael Trauner, M.D., Ph.D, Department of Internal Medicine III, Divison of Gastroenterology and Hepatology, Medical University of Vienna, Waehringer Guertel 18-20, A-1090 Vienna, Austria. Pathogenesis of PSC: Role of TGR5 in the regulation of the innate immune response in the biliary epithelium. Amount awarded = $40,000 over 2 years. The first year of this project will be funded by a generous donation from Craig and Ali Wiele.
- Carlo Selmi, MD, PhD, Assistant Professor of Internal Medicine, University of Milan, Physician Scientist, IRCCS Istituto Clinico Humanitas, Rozzano, Milan, Assistant Professor of Medicine, University of California, Davis, Division of Rheumatology, Allergy, and Clinical Immunology, GBSF suite 6515, 451 E Healthy Sciences Drive, Davis, CA 95616. Epigenetics associated with primary sclerosing cholangitis in monozygotic twins discordant for the disease. Amount awarded = $40,000 over 2 years.
- Judy Cho, M.D., Associate Professor of Medicine and Genetics, Section of Digestive Diseases, Director, Inflammatory Bowel Disease Center, Yale University, Internal Medicine, 333 Cedar Street, Room LMP-1072, P.O. Box 208056, New Haven, CT 06520-8056. Identification of genetic profiles unique to PSC-IBD. Amount awarded = $40,000 over 2 years. This project will be entirely funded by our anonymous donor who generously donated $100,000 in our Itching for a Cure: Road to Connecticut fundraiser of 2009/2010 to help support PSC research.
- Shi-Ying Cai, Ph.D., Research Scientist, Section of Digestive Diseases, Department of Internal Medicine, Yale Liver Center, Yale University School of Medicine, 1080 LMP, 333 Cedar Street, New Haven, CT 06520, and James L. Boyer, M.D., Ensign Professor of Medicine, Section of Digestive Diseases, Department of Internal Medicine, Yale Liver Center, Yale University School of Medicine, 1080 LMP, 333 Cedar Street, New Haven, CT 06520. Combination treatment with ursodeoxycholic acid and all-trans retinoic acid for primary sclerosing cholangitis (PSC). $37,000 over 2 years. This project will be entirely funded by our anonymous donor who generously donated $100,000 in our Itching for a Cure: Road to Connecticut fundraiser of 2009/2010 to help support PSC research.
- Sridhar Mani, M.D., Professor, Medicine, Oncology and Genetics, Miriam Mandel Faculty Scholar in Cancer Research, Albert Einstein College of Medicine, 1300 Morris Park Ave, Chanin 302D-1, Bronx, NY 10461. Establishing the role and molecular mechanisms for pregnane X receptor in progressive sclerosing cholangitis. $40,000 over 2 years.
Congratulations to Drs. Lazaridis, Invernizzi, Ponsioen, Bowlus, Everson, Green, Lindor, Hirschfield, Strazzabosco, Trauner, Selmi, Cho, Cai, Boyer and Mani! For further details of these awards, please see:
Research Awards. In total, PSC Partners Seeking a Cure has invested $527,706 in PSC research.
For those newly diagnosed with PSC, and/or their caregivers, we highly recommend joining a PSC support group:
Please consider becoming an organ donor, see: Coalition on Donation - Donate Life
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