The ECD Global Alliance has been funding Erdheim-Chester Disease research since it was first given a 501(c)3 status in 2010 (retroactive back to 2009). The organization has awarded six $50,000 research grants to study Erdheim-Chester Disease, as well as a $316,000 grant to establish an ECD Patient Registry.
2015 ECDGA Grants Awarded
Three grants in 2015 were awarded to learn more about ECD.
1. Filip Janku, MD, PhD and Abdel-Wahab, MD were awarded a $50,000 grant for their project, “Understanding and targeting novel molecular alterations in Erdheim-Chester Disease without the BRAFV600E mutation.” The purpose of this study is to discover treatable mutations found in BRAF-negative ECD patients and test in the lab which treatments will most likely work best for these patients.
2. Eli Diamond, MD and Vaios Hatzoglou, MD were awarded a $43,000 grant for their project, “A Clinical, Structural, and Functional Neuroimaging Study of Cognition in Erdheim-Chester Disease.” The purpose of this study is to understand the neurological/cognitive effects of ECD.
3. Marina Ferrarini, MD and Lorenzo Dagna, MD were awarded $50,000 for their project, “Tailoring Treatment for Erdheim-Chester Disease”. The Ferrarini and Dagna study focuses on understanding how malignant cells in ECD patients accumulate and interact with neighboring normal cells to fuel the disease, causing lesions and tumors to form. With this understanding, the discovery of a treatment strategy that can interrupt the processes, stopping the disease from progressing, may be possible.
2014 ECDGA Grant Awarded to Eli Diamond, MD
The 2014 grant was awarded to principal investigators Eli Diamond, MD from Memorial Sloan-Kettering Cancer Center in New York, and Matthew Collin from Newcastle University in the UK, for the “Design and Creation of a Global Registry Database for Erdheim-Chester Disease”. The patient registry will be a collection of standardized information about the ECD group of patients to evaluate specified outcomes defined by this particular disease. The data will serve as a scientific, clinical purpose. This is expected to be a two (2) year effort with a budget of $316,000 being granted from the ECD Global Alliance.
2013 ECDGA Grant Awarded to Omar Abdel-Wahab, MD
In September 2013, the ECD Global Alliance announced a $50,000 2013 ECD grant was awarded to Omar Abdel-Wahab, MD, David Hyman, MD, and Eli Diamond, MD from Memorial Sloan-Kettering Cancer Center in New York (MSKCC), New York for the study described as, “Somatic Genetic Alterations in the Pathogensis and Therapy of Histicytic Disorders”. As described in the MSKCC press release, the aim of this study is to:
1. Identify recurrent somatic genetic events in addition to BRAFV600E mutations in patients with ECD.
2. Identify cell of origin in ECD.
3. Identify the optimal targeted therapy in histiocytic disorder patients with mutations activating kinase signaling.
This project was completed in February 2015. The team at MSKCC supported a phase II study of vemurafenib in ECD and LCH patients bearing the BRAFV600E mutation, performed a study in collaboration with Filip Janku, MD, PhD at MD Anderson Cancer Center to understand how blood and urine could be used to track the BRAFV600E mutation in ECD patients undergoing therapy, collaborated with Drs. Julien Haroche and Jean-Francois Emile in France to perform a large genomic study of ECD patients, and continues to perform and even larger genomics study in ECD. Multiple journal articles were published in part as a result of this project.
2012 ECDGA Grant Awarded to Julien Haroche, MD, Phd
Proposals for the 2nd $50,000 research grant awarded by the ECD-GA were due December 31, 2011. In April 2012, the organization announced the grant was awarded to Laurent Arnaud, MD, PhD and Julen Haroche, MD, PhD from SHôpital Pitié-Salpêtrière in Paris, France for the study described as, “Understanding dendritic cell lineages in Erdheim-Chester disease: towards a non-invasive diagnosis“. The aim of this study is to understand the primary mechanisms involved in the pathogenic differentiation of histiocytes in ECD, as well as to use these data to derive a screening & diagnostic test for ECD that would rely on blood sampling without the need for a biopsy. The study is taking longer than expected and final results are expected in late summer 2015.
2011 ECDGA Grant Awarded to Lorenzo Dagna, MD. Final report: “Hypoxia and inflammation in Erdheim-Chester disease microenvironment: insights on the pathogenesis and implications for therapy final report”.
In April 2010, only one month after receiving a 501(c)(3) designation, the organization announced it would be awarding a $50,000 ECD research grant and put out a Request for Proposals (RFP) to the international medical research community. In November 2010, the first grant recipient was chosen.
The ECD Global Alliance 2010 grant was awarded to Lorenzo Dagna, MD at San Raffaele Scientific Institute in Milano, Italy for the study described as, “Hypoxia and inflammation in Erdheim-Chester disease microenvironment: insights on the pathogenesis and implications for therapy proposal”. The aim of this study was to better define the microenvironment inside ECD lesions and to evaluate the possibility of using specific drugs, already available for human usage, as a means to find new therapeutic options for the disease. The study ran from January 2011 until June 2012. The results of this study can be found in the Journal of Clinical Oncology, Vol. 310, 2012, in an article entitled, “Tumor Necrosis Factor a As a Master Regulator of Inflammation in Erdheim-Chester Disease: Rationale for the Treatment of Patients With Infliximab”.
Last updated: February 12, 2016