Over ons
Onze missie
De ECD Global Alliance zet zich in voor bewustwording, ondersteuning, onderwijs en onderzoek met betrekking tot Erdheim-Chester ziekte. De organisatie werkt samen met de wereldwijde gemeenschap om het volgende te bereiken:
Bewustmaking van ECD. We streven ernaar een vroegtijdige diagnose van de ziekte te vergemakkelijken en de bezorgdheid en inspanningen te stimuleren om de kennis over de ziekte en behandelingen te vergroten.
Ondersteuning bieden aan mensen die door de ziekte zijn getroffen. We zullen alle wegen blijven bewandelen om de last voor de getroffenen van ECD zoveel mogelijk te verlichten. Het verstrekken van informatie, doorverwijzingen en één-op-één contact met anderen die door de ziekte zijn getroffen, zijn slechts enkele van de ondersteuningen die via ECDGA worden aangeboden.
Pleiten voor en ondersteunen van onderzoeksprogramma’s. Het doel van dergelijke onderzoeksprogramma’s is om meer te weten te komen over (a) de ziekte, (b) effectieve behandelingen voor de ziekte, (c) mogelijke genezing en/of (d) manieren om de ziekte helemaal te voorkomen. Dit omvat, maar is niet noodzakelijk beperkt tot, samenwerking met medisch personeel, onderzoekspersoneel en andere gezondheidsorganisaties om gebieden te helpen definiëren waar onderzoek nodig is, om onderzoek te financieren en om de voordelen van ECD onderzoek te helpen promoten bij andere gezondheidsinstanties. De alliantie zal ook dienen als doorgeefluik voor de overdracht van benodigde informatie van zijn leden naar de onderzoeksgemeenschap volgens gedocumenteerde richtlijnen.
Kathy Brewer, oprichter en voorzitter
Didactisch materiaal delen en de uitwisseling van informatie tussen belanghebbende partijen vergemakkelijken met betrekking tot ECD. Dit omvat het verstrekken van actuele, betrouwbare en feitelijke informatie met betrekking tot ECD aan patiënten, naasten, medisch personeel, onderzoekspersoneel, media en andere organisaties. Het omvat ook het promoten, sponsoren en houden van workshops, symposia en andere bijeenkomsten om de doelen van de alliantie te promoten.
Een decennium van impact
Voor de meeste mensen begint een ervaring met Erdheim-Chester ziekte beginnen met een frustrerende, beangstigende zoektocht naar antwoorden.
Dit gold zeker voor ECD Global Alliance oprichtster Kathy Brewer en haar overleden echtgenoot Gary. Op het moment van zijn dood was Gary al 20 jaar ziek en drie jaar erg ziek. Hij had symptomen van een nierziekte en onderging zelfs een niertransplantatie. Maar pas bij zijn autopsie kwam Kathy erachter wat de ware oorzaak was. Erdheim-Chester wat de ware oorzaak was.
Dit is de uitdaging bij een zeer zeldzame ziekte. Wanneer een aandoening wereldwijd slechts 1.000 mensen treft, zijn er maar weinig artsen die de diagnose kunnen stellen, vooral wanneer de symptomen variëren van botpijn en gewichtsverlies tot problemen met het gezichtsvermogen en het evenwicht. (Naast vele andere lichaamssystemen kan ECD de hypofyse en bijnieren, de longen, het membraan rond het hart, de nieren en de hersenen aantasten).
Het verhaal van ECD is vandaag de dag echter opmerkelijk anders dan in 2008, en de reden daarvoor is de ECD Global Alliance. Gestart door patiëntenverzorgers die elkaar via het internet hebben gevonden, is de Alliance gegroeid van zes families naar bijna 800 en heeft het de vooruitzichten voor ECD patiënten drastisch veranderd.
Tegenwoordig kunnen de meeste ECD patiënten de progressie van de ziekte onder controle houden met een van de verschillende behandelingen die nu worden gebruikt. Velen worden niet langer geconfronteerd met een normale levensverwachting van 3-5 jaar, maar kunnen de symptomen onder controle houden terwijl ze verder gaan met hun leven.
Onze hoop voor de toekomst
Lees over het 5-jaren strategieplan dat momenteel wordt geïmplementeerd door jouw belangenorganisatie.
De Alliantie heeft een netwerk van Care Centers opgezet in vooraanstaande medische instellingen over de hele wereld, elk met een team van specialisten die voorbereid zijn op het diagnosticeren en behandelen van ECD.
“Vóór ECDGA was er geen centrale plek om de ziekte te behandelen of informatie te krijgen, geen organisatie. Je ontmoette een arts die aanbood je te behandelen en ontdekte dat hij nog nooit een ECD patiënt had behandeld.” – verzorger van een ECD patiënt
Het creëerde een ondersteuningsnetwerk van families en bracht leden van de internationale ECD gemeenschap samen voor een reeks jaarlijkse conferenties – een gelegenheid voor patiënten om elkaar te ontmoeten en te praten met artsen die gespecialiseerd zijn in ECD.
Het lanceerde een patiëntenregister om een archief van gegevens op te bouwen op ECD en werkte samen met de NIH om patiënten te werven voor een studie naar de natuurlijke geschiedenis – de sleutel tot het verkrijgen van inzicht in de progressie van de ziekte.
En, het meest opvallend, het hielp het werk in gentherapie mogelijk te maken dat leidde tot goedkeuring door de FDA van een medicijn dat effectief is bij de behandeling van ECD.
Meer prestaties
| Year | Accomplishment | Details |
|---|---|---|
| 2021 | Webinars, Chats, and Strategy continue | The organization continues to support patients, families, and physicians with meaningful connections as much as possible during the COVID pandemic. All while implementing our 5-Year Strategy of growth and improvement. |
| Virtual Medical Symposium | With a pandemic that no one expected or hoped for, the annual events were canceled for 2020. This year we pulled together our top physicians and researchers to create an online conference, continuing the collaboration and growth in the medical field without delay. | |
| 2020 | Community Webinars | Educational webinars hosted by our expert medical members replaced the in-person events normally held annually for the ECD community. This involved partnerships with other histiocytosis advocacy groups. These partnerships have strengthened and streamlined this project, benefiting all involved. |
| High-Level Strategy Planning | The ECD Global Alliance is a small but extremely effective organization leading the fight against the disease. Over the past decade, it has helped achieve major advances in treatment and offered invaluable support to patients and caregivers. As a result, the Alliance has helped change the meaning of an ECD diagnosis. Going forward, with expanded donor investment, the Alliance has plans to accomplish even more. | |
| 2019 | Endocrinology Conference ECD Exhibit | The purpose of the ECD Global Alliance exhibition is to generate awareness of Erdheim-Chester Disease, communicate the recent research landscape and new treatment opportunities for patients, and facilitate the growth of the ECD Global Alliance medical professional network among interested physicians and scientists. In addition, connect with other exhibitors offering services that can help the ECDGA further awareness and education of ECD. |
| Second Consensus Article Published | As a result of the ECD Medical Symposia in previous years, the second-ever comprehensive guidelines were published by leading ECD experts. This important document was a collaborative collection of data gleaned from extensive research of the disease and the treatment of patients worldwide. | |
| Chat Program | The infamous COVID pandemic pushed the ECDGA to find creative ways to engage the community. With online meetings becoming normal, the Alliance began hosting chats for various audiences. This includes patients, friends, family, caregivers, physicians, and regional and international language meetings. | |
| 2018 | Hematology Exhibit | Hematologists from around the world eagerly engage with ECDGA exhibitors to learn more about this rare disease. |
| Grand Round Medical Presentations | ECD-knowledgeable physicians provided education on ECD at the University of Miami and Houston Methodist. | |
| 6th Annual Event held in Orlando, FL at Orlando Health | A total of 109 members from eight countries attended to learn about the drug trial process, how to manage multiple medications, how to care for yourself with nutrition and exercise, and about care management. | |
| 7th Annual Events held in Milan, Italy | The purpose of the medical symposium is to unite international medical professionals in discussion and education of studies, experiences, and knowledge regarding Erdheim-Chester Disease. In this space, medical professionals are encouraged to present findings and knowledge to audience members. During the patient meeting, patients and family members are encouraged to ask questions, reach out and learn about the ultra-rare disease that affects their lives. This meeting was co-hosted with Dr. Lorenzo Dagna. | |
| $50,000 Grant Awarded to Dr. Nicole Coufal at UCSD (Announced in 2020) | In 2019, a $50,000 young investigator award was granted to Nicole Coufal with the University of California, San Diego. “Pathophysiology of Neurodegenerative Erdheim-Chester Disease and Langerhans Cell Histiocytosis” | |
| 2017 | 1st FDA-Approved Drug: Vemurafenib (Zelboraf) | The Memorial Sloan Kettering Basket Trial led to favorable results for the Erdheim-Chester patients enrolled, eventually resulting in approval. The ECDGA assisted in the recruitment process to make this possible. |
| Care Centers Added to Network | Locations in Israel, China, Nashville, Canada, and Australia were welcomed to the network. | |
| Grand Round Medical Presentations | ECD-knowledgeable physicians were sponsored by the ECDGA to give educational talks at universities in Houston, TX, New Orleans, and Shreveport, LA. | |
| Pathology Conference Exhibit | The ECDGA medical community volunteered to create materials to educate attendees at an annual pathology conference. The ECDGA team hosted an exhibit booth, where they introduced and discussed ECD at length with pathologists around the world. | |
| 5th Annual Events in New York | Patients, family members, nurses, research teams, and numerous physicians attended the 2017 Patient & Family Gathering and Medical Symposium held in New York. Attendees were brought up to date on the latest research, treatments, and care management techniques, including the psychological effects of living with ECD. This meeting was co-hosted with Dr. Eli L. Diamond. | |
| $50,000 Grant Awarded to Dr. Benjamin Durham at MSK (Announced in 2018) | The ECD Global Alliance awards Erdheim-Chester disease research grant to MSK to define the disease-initiating cell population in ECD and compare genetic alterations. | |
| 2016 | 4th Annual ECD Patient & Family Gathering | The first European event, held in Paris, France, included 63 attendees from 17 different countries who heard from 16 physicians about ECD organ involvement, the use of scans to monitor the disease and its treatments, techniques to manage pain, and the latest research findings including treatment options. This meeting was co-hosted with Prof. Julien Haroche. |
| 4th ECD Medical Symposium | The first European ECD Medical Symposium was held in Paris, France at Hôpital Pitié-Salpêtrière, where over 50 physicians gathered to collaborate and share data on ECD. This meeting was co-hosted with Prof. Julien Haroche. | |
| 10 Languages Added to Website | Volunteers from around the globe dedicated their time and talents to translating important content in ten languages for the ECDGA website. | |
| 2015 | Development of ECD Care Center Network | Care centers with ECD-knowledgeable medical teams began to form a network to support patients with care and consultation around the world. To date, the ECDGA collaborates with 35 ECD Care Centers serving patients. |
| New Website | An updated website was developed to engage members on all devices. | |
| $143,000 for Three Research Grants Awarded | ||
| 3rd Annual Patient & Family Gathering | Patients, families, and physicians united in Houston, TX for a memorable two-day gathering. | |
| 3rd Annual ECD Medical Symposium | The symposium was hosted at MD Anderson Cancer Center, with Dr. Filip Janku. | |
| 2014 | 2nd ECD Patient & Family Gathering | Patients and family members gather in Bethesda, MD for another two-day meeting including ECD presentations delivered by the experts. |
| 2nd International Medical Symposium | Physicians and researchers presented their work and discussed the state of Erdheim-Chester disease care for patients around the world. | |
| $316,000 Grant to Dr. Eli L. Diamond and Pr. Matthew Collin (Announced in 2015) | Drs. Eli L. Diamond and Matthew Collin were awarded for the development of the ECD Patient Registry. This allowed patients and physicians the opportunity to collaborate and lead research more effectively so that treatment and care can continue to improve and evolve. | |
| 2013 | 1st ECD Medical Symposium | In addition to the patient meeting, the first international meeting was held this year, inviting doctors to present ECD research, case studies, and treatments. This meeting was also the catalyst for collaboration in developing the significant 2014 Blood Journal ECD Consensus document. Collaborative annual meetings have continued to take place around the world each year since. |
| ECD Documentary Film | A patient documentary film was developed and produced to support outreach and education of Erdheim-Chester disease. | |
| 3rd ECD Research Grant Awarded | $50,000 was awarded to Drs. Abdel-Wahab, Diamond, and Hyman at Memorial Sloan-Kettering Cancer Center (MSK). “Somatic Genetic Alterations in the Pathogenesis and Therapy of Histiocytic Disorders” | |
| 2012 | 2nd ECD Research Grant Awarded | $50,000 was awarded to Drs. Haroche and Arnaud ECD research. “Understanding dendritic cell lineages in Erdheim-Chester disease: towards a non-invasive diagnosis” |
| 1st ECD Medical Meeting | A meeting was held in Houston, TX with investigators from various institutions in the US. | |
| 1st ECD Patient & Family Gathering and Medical Symposium | ECD patients and their families came together for the first time this year and the annual event has been a successful way to bring families together ever since. | |
| 2011 | First Grant Awarded to ECD Research | For the first time, $50,000 was awarded to an ECD researcher, and each year following. This progressive mission has set out to enhance research discoveries in Erdheim-Chester disease with the leading researchers and institutions in the field. A total of $750,000 has been granted toward this initiative to date. |
| ECD Natural History Study | The National Institute of Health (NIH) was integral in ECD research and pursuing new treatments that help educate the medical community about ECD. NIH clinical director, William Gahl, M.D., Ph.D., and principal investigator, Juvianee Estrada-Veras, M.D. conducted a natural history study on the disease. The study began in 2011 and this led to a treatment trial in 2014. Multiple medical journals have been published with the data collected from this thorough study of ~60 patients. | |
| 2010 | 501(c)3 Tax Exemption Granted | ECDGA was granted federal tax exemption following the formation of the Board of Directors and Medical Advisory Board. |
| 2008 | ECD Caregivers Meet | In April 2008, a few ECD caregivers, Carol, Kathy, and RuthAnn found each other via the internet. |
| First Chat Session Held | With a spirit of “If you build it, they will come,” an online chat session was scheduled for all those affected by ECD. We started with a group representing only six patients. | |
| ECD Website Published | A website was created with the hope that it would provide much-needed information to families in search of answers for this rare illness. |