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You can play a big part in fighting ECD.
If you are a patient or direct caregiver, please participate in this survey by October 30, 2021.
When the ECD Global Alliance (ECDGA) was formed research was needed to help improve the diagnosis and treatment of Erdheim-Chester Disease (ECD). Little was known about the disease and research was difficult because patient access was limited, and funding was almost non-existent. Only a little over a year after the formation of the ECDGA, the organization began to fund ECD-focused research.
The ECDGA has historically funded nine (9) research projects worth over $700,000 proposed by the medical community. This has worked well to push ECD research forward. In the past decade, much has been learned about ECD and there have been substantial breakthroughs in the treatment of ECD, including an FDA-approved treatment. Although not FDA-approved, other targeted treatments have been found as well. With better treatments, patients are now living more normal lives.
To date, the research performed has focused on (1) basic science, (2) transitional research, and (3) clinical research.
1) Basic Science Research allows scientists to do lab investigations to increase knowledge and understanding of ECD. The ECD-funded research to better understand the various mutations seen in ECD patients was funded in part as a basic science research project. The findings eventually allowed more targeted treatments to be discovered for many histiocytic patients.
2) Translational Research takes knowledge learned in basic research to determine how that knowledge might be “translated” into clinical care. For instance, when different ECD mutations were found, the translational research projects were able to discover the possible treatments that could be used to target those mutations.
3) Clinical Research looks at increasing the understanding of what works best for patients regarding diagnosis, treatment, and care management. Diagnostic research is important to help patients arrive at a diagnosis early in disease progression when less irreversible damage may have occurred to organs. Treatment and care management research allows an increased understanding of what treatments and care bring about the best outcomes for patients.
Some of the ECD clinical research performed to date has focused not only on better diagnostic and treatment options in general but also on better understanding how ECD affects different organs, most notably the brain and neurological system.
The ECDGA also funds, in part, an ECD Patient Registry which is a database to collect patients’ medical histories in an organized format. This allows information about patients who are seen at many different institutions to be analyzed together in a more holistic way. By analyzing the ECD population, scientists can begin to see patterns and clues that will help understand ECD and hopefully find the most effective treatments in a given circumstance. The ECDGA has also partnered with the National Institute of Health for a Natural History Study of ECD. The collected data continues to be used to further research.
To ensure that further gains in the knowledge of ECD are in line with the issues most relevant to patients and their families, the ECDGA Board of Directors is now considering funding research projects based on patient/caregiver priorities, in addition to research proposed by the medical community needs.
To understand the priorities and needs of patients and their loved ones, a survey has been developed to gather input from the community. We invite all ECD patients and their family members to complete and submit this survey before October 30, 2021.
The information collected from the survey will be analyzed and discussed with the ECD medical community to arrive at possible research projects that could bring about knowledge that would be most meaningful to the community.
If you have not already, we ask you to complete your survey by selecting the button below.
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