Erdheim-Chester Disease ECD Global Alliance October 2010 What is Erdheim-Chester Disease? Non-Langerhans Cell Histiocytosis Causes Scar-Like Tissue to Surround the Organs & Arteries Too Many Histiocytes (cells which normally fight infections) Infiltrate Organs Bone, Brain, Kidney, Eyes, Lungs, Abdominal Tissue/Organs, Cardiovascular, Skin and More Rarely Others Symptoms Vary Between Patients Depending on Which Organs are Involved Without Successful Treatment: Organ Failure Why Have I Never Heard of ECD? First Described in 1930 by an Austrian (Dr. Erdheim) and an American (Dr. Chester) Pathologist Only About 350 Published Cases in the World Never Heard of by Most Doctors Diagnosis is Difficult and Time Consuming, Made Only by Chance in Some Cases Not Known if Cancer, Auto-Immune, Genetic, etc. What is the Status of ECD? Only a Handful of Doctors Have Treated Multiple Cases Anecdotal Treatments Available Immunotherapy, chemotherapy, radiation, surgery, hormone therapy, corticosteroids, anti-inflammatory drugs, antimetobolite, new cancer drug which specifically inhibits a particular enzyme Results are Variable Literature Says Most Succumb in 2 to 3 Years, However We See Some Patients Live for Decades International Disease Advocacy Group Formed – ECD Global Alliance – www.erdheim-chester.org First a Diagnosis is Needed “When you hear hoofbeats, think horses, not zebras” Specialists Often Only Know What it is Not Doctors have Not Seen or Studied Many of the Rare Diseases Often Patients Find the Validity of Their Symptoms Being Questioned Diagnosis Often Takes Years with Patients Shuffled from One Specialist to Another What Are the Patient Issues? Sickness Itself Getting a Diagnosis Finding Knowledgeable Doctors No Agreement on Best Treatments Health Care Costs / Insurance Coverage Expert Doctors Most Likely Out of Network Treatments not Scientifically Verified Transportation / Housing Costs Disability Difficult to Obtain Lack of Support Structure Lack of Information Relative to Progression Status of the ECD Global Alliance Carol, Kathy, RuthAnn Met by Phone Teleconference with ECD Patients and Dr. Kurzrock Apr. ‘08 Jan. ‘08 Jan. ‘09 Oct. ‘08 Sep. ‘08 First Online Chat Session Held Dr. Haroche’s HLA Study Communicated Oct. ‘08 Published www.erdheim-chester.org website Organizational Activity Dr. Vaglio’s Treatment Protocol Communicated Support Activity Medical Advisory Board Formed Mar. ‘10 Aug. ‘09 Apr. ‘09 First Newsletter Published Dec. ‘09 501(c)(3) Non-Profit Paperwork Submitted & Board of Directors Formed Bringing Information to Patients RFP for Research Project Communicated Apr. ‘10 501(c)(3) Tax Exempt Status Granted, Effective as of Aug. 28, 2009 Promoting Research ECD Global Alliance What is Needed? Raise Additional Research Funds Continue Dialogue with Research Physicians Create and Maintain Patient Registry Future ECD Symposium Support for Patients with Regard to Disability and Other Related Issues What Can We All Do? Be Supportive Be an Advocate Be an Organ Donor Be Generous ? ? ? ? ? Questions? ? ? ? ? ? ? ? ?