Quiénes somos
Nuestra misión
La Alianza Global ECD se dedica a la concienciación, el apoyo, la educación y la investigación relacionados con Erdheim-Chester la enfermedad. La organización trabaja con la comunidad mundial para lograr lo siguiente:
Sensibilizar sobre ECD. Nos esforzamos por facilitar el diagnóstico precoz de la enfermedad y estimular la preocupación y los esfuerzos por aumentar los conocimientos sobre la enfermedad y los tratamientos.
Proporcionar apoyo a los afectados por la enfermedad. Seguiremos buscando todas las vías para aliviar en lo posible la carga de los afectados por ECD. Proporcionar información, derivaciones y contacto personal con otros afectados por la enfermedad son sólo algunos de los apoyos que se ofrecen a través de ECDGA.
Defender y apoyar los programas de investigación. El objetivo de dichos programas de investigación es conocer mejor (a) la enfermedad, (b) los tratamientos eficaces para la enfermedad, (c) las posibles curas, y/o (d) las vías para prevenir la enfermedad por completo. Esto incluirá, entre otras cosas, la colaboración con el personal médico, el personal investigador y otras organizaciones sanitarias para ayudar a definir las áreas en las que se necesita investigación, financiar la investigación y ayudar a promover los beneficios de la investigación de ECD entre otros organismos sanitarios. La alianza también servirá de conducto para transferir la información necesaria de sus miembros a la comunidad investigadora según las directrices documentadas.
Kathy Brewer, Fundadora y Presidenta
Compartir material educativo y facilitar el intercambio de información entre las partes interesadas en relación con ECD. Esto incluye proporcionar información actual, fiable y objetiva relacionada con ECD a pacientes, seres queridos, personal médico, personal investigador, medios de comunicación y otras organizaciones. También incluirá la promoción, el patrocinio y la realización de talleres, simposios y otras reuniones para promover los objetivos de la alianza.
Una década de impacto
Para la mayoría de las personas, una experiencia con Erdheim-Chester enfermedad comienza con una búsqueda frustrante y aterradora de respuestas.
Esto es lo que les ocurrió a la fundadora de la Alianza Global ECD, Kathy Brewer, y a su difunto marido, Gary. En el momento de su muerte, Gary llevaba 20 años enfermo y tres muy enfermo. Tenía síntomas de enfermedad renal y, de hecho, se sometió a un trasplante de riñón. Pero no fue hasta su autopsia cuando Kathy supo Erdheim-Chester cuál era la verdadera causa.
Éste es el reto de una enfermedad muy rara. Cuando una enfermedad afecta sólo a unas 1.000 personas en todo el mundo, pocos médicos tienen la familiaridad necesaria para diagnosticarla, sobre todo cuando puede manifestarse con síntomas que van desde dolor óseo y pérdida de peso hasta problemas de visión y equilibrio. (Entre otros muchos sistemas corporales, ECD puede afectar a las glándulas pituitaria y suprarrenales, los pulmones, la membrana que rodea el corazón, los riñones y el cerebro).
Sin embargo, la historia de ECD es notablemente diferente hoy de lo que era en 2008, y la razón es la Alianza Global ECD. Iniciada por cuidadores de pacientes que se encontraron a través de Internet, la Alianza ha pasado de seis familias a casi 800, y ha cambiado radicalmente las perspectivas de los pacientes de ECD.
Hoy en día, la mayoría de los pacientes de ECD pueden controlar la progresión de la enfermedad con uno de los diversos tratamientos que se utilizan actualmente. Muchos han pasado de enfrentarse a una esperanza de vida típica de 3-5 años a controlar los síntomas mientras siguen con sus vidas.
Nuestra esperanza para el futuro
Lee sobre el Plan Estratégico Quinquenal que está aplicando actualmente tu grupo de defensa.
La Alianza estableció una red de Centros de Atención en instituciones médicas líderes a nivel internacional, cada uno de ellos con un equipo de especialistas preparados para diagnosticar y tratar ECD.
«Antes de ECDGA, no había ningún lugar central para tratar la enfermedad u obtener información, ninguna organización. Te encontrabas con un médico que se ofrecía a tratarte y descubrías que nunca había tratado a un solo paciente de ECD «. – cuidador de un paciente de ECD
Creó una red de apoyo de familias y reunió a miembros de la comunidad internacional de ECD en una serie de conferencias anuales, una oportunidad para que los pacientes se conocieran y hablaran con médicos especializados en ECD.
Puso en marcha un registro de pacientes para crear un archivo de datos sobre ECD y se asoció con los NIH para reclutar pacientes para un estudio de historia natural, clave para comprender la progresión de la enfermedad.
Y, lo que es más sorprendente, ayudó a hacer posible el trabajo en terapia génica que condujo a la aprobación por la FDA de un fármaco eficaz en el tratamiento de ECD.
Más logros
| 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. |