A propos de nous
Notre mission
L’Alliance mondiale ECD se consacre à la sensibilisation, au soutien, à l’éducation et à la recherche dans les domaines suivants Erdheim-Chester la maladie. L’organisation travaille avec la communauté mondiale pour accomplir ce qui suit :
Sensibilisez les gens à ECD. Nous nous efforçons de faciliter le diagnostic précoce de la maladie et de stimuler l’intérêt et les efforts pour améliorer les connaissances sur la maladie et les traitements.
Apporter un soutien aux personnes touchées par la maladie. Nous continuerons à rechercher tous les moyens d’alléger autant que possible le fardeau des personnes touchées par la maladie ECD. L’information, l’orientation et le contact individuel avec d’autres personnes touchées par la maladie ne sont que quelques-uns des soutiens offerts par l’intermédiaire de ECDGA.
Défendre et soutenir les programmes de recherche. L’objectif de ces programmes de recherche est d’en apprendre davantage sur (a) la maladie, (b) les traitements efficaces de la maladie, (c) les remèdes possibles, et/ou (d) les moyens de prévenir complètement la maladie. L’alliance comprendra, entre autres, une collaboration avec le personnel médical, le personnel de recherche et d’autres organismes de santé pour aider à définir les domaines dans lesquels la recherche est nécessaire, pour financer la recherche et pour aider à promouvoir les avantages de la recherche sur le site ECD auprès d’autres organismes de santé. L’alliance servira également d’intermédiaire pour transférer les informations nécessaires de ses membres à la communauté des chercheurs, conformément à des lignes directrices documentées.
Kathy Brewer, fondatrice et présidente
Partager du matériel éducatif et faciliter l’échange d’informations entre les parties intéressées concernant ECD. Il s’agit notamment de fournir des informations actuelles, fiables et factuelles sur le site ECD aux patients, à leurs proches, au personnel médical, au personnel de recherche, aux médias et à d’autres organisations. Il s’agira également de promouvoir, de parrainer et d’organiser des ateliers, des symposiums et d’autres réunions afin de promouvoir les objectifs de l’alliance.
Une décennie d’impact
Pour la plupart des gens, l’expérience de la maladie commence par une recherche frustrante et effrayante de réponses. Erdheim-Chester maladie commence par une recherche frustrante et effrayante de réponses.
Ce fut certainement le cas pour la fondatrice de ECD Global Alliance, Kathy Brewer, et son défunt mari, Gary. Au moment de sa mort, Gary était malade depuis 20 ans et très malade depuis trois ans. Il présentait des symptômes de maladie rénale et avait d’ailleurs subi une greffe de rein. Mais ce n’est qu’à l’autopsie que Kathy a appris que la maladie de Gary était la véritable cause de son décès. Erdheim-Chester la véritable cause de son décès.
C’est le défi que pose une maladie très rare. Lorsqu’une maladie ne touche qu’environ 1 000 personnes dans le monde, peu de médecins ont la familiarité nécessaire pour la diagnostiquer, en particulier lorsqu’elle peut se manifester par des symptômes allant de douleurs osseuses et d’une perte de poids à des problèmes de vision et d’équilibre. (Parmi les nombreux systèmes de l’organisme, ECD peut affecter l’hypophyse et les glandes surrénales, les poumons, la membrane entourant le cœur, les reins et le cerveau).
Cependant, l’histoire de ECD est remarquablement différente aujourd’hui de ce qu’elle était en 2008, et la raison en est l’Alliance mondiale ECD. Lancée par des soignants qui se sont trouvés grâce à l’internet, l’Alliance est passée de six familles à près de 800, ce qui a radicalement changé les perspectives pour les patients du site ECD.
Aujourd’hui, la plupart des patients du site ECD peuvent contrôler la progression de la maladie grâce à l’un des nombreux traitements actuellement utilisés. Nombreux sont ceux qui sont passés d’une espérance de vie typique de 3 à 5 ans à une gestion des symptômes qui leur permet de continuer à vivre.
Notre espoir pour l'avenir
Découvrez le plan stratégique quinquennal actuellement mis en œuvre par votre groupe de pression.
L’Alliance a mis en place un réseau de centres de soins dans des établissements médicaux de premier plan au niveau international, chacun disposant d’une équipe de spécialistes préparés à diagnostiquer et à traiter ECD.
« Avant ECDGA, il n’y avait pas de lieu central pour traiter la maladie ou obtenir des informations, pas d’organisation. Vous rencontriez un médecin qui vous proposait de vous soigner et vous découvriez qu’il n’avait jamais traité un seul patient de ECD. » – aidant d’un patient ECD
Elle a créé un réseau de soutien pour les familles et a réuni les membres de la communauté internationale ECD pour une série de conférences annuelles – une occasion pour les patients de se rencontrer et de parler avec des médecins spécialisés dans ECD.
Elle a lancé un registre de patients pour constituer des archives de données sur ECD et s’est associée aux NIH pour recruter des patients dans le cadre d’une étude de l’histoire naturelle, essentielle pour comprendre la progression de la maladie.
Et, fait le plus marquant, elle a contribué à rendre possible les travaux de thérapie génique qui ont conduit à l’approbation par la FDA d’un médicament efficace dans le traitement de ECD.
Plus de réalisations
| 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. |