The first half of 2024 has been very active in the global EHE research community. Central to the EHE Group’s mission is advancing research – it is why our foundations were set up, and much of that research is quite literally powered by the patient community, as high-lighted in Hugh Leonard’s presentation during the EHE 360 Patient Conference.
Denise Robinson wanted to share her observations following a number of events and interactions during the first half of the year. Denise explained:
“In recent months, I have had the opportunity to meet with expert clinicians from around the world and attend meetings with the US and EU regulatory bodies, FDA, and EMA. These interactions have led me to an unequivocal position – to find effective treatments, we need more data. By participating in research through the EHE Global Patient Registry, EHE Biobanks, and other projects, the patient community can come together to advance the science and treatment of EHE.
The EHE patient community is powerful – patients and their advocates are fierce in their quest for information, answers, and better treatments. As advocates for all EHE patients, The EHE foundations must work together to harness knowledge, bring forward lived experiences, and use data to make a case to drug developers concerning what we know about treatments used today.”
Denise continued:
“Drug developers and academic researchers continually ask, “How many people are living with EHE?” There is no good answer – we simply do not know. So this is a call-to-action. One of our most important objectives is to bring forward a research-ready patient community to help and be involved in finding treatments and a cure for EHE. Regulatory agencies and researchers require structured, ethically-collected data to make informed decisions about what drugs should be studied for EHE treatment and then to understand how those treatments help patients affected by the disease so that they can be approved for use. This was why the EHE Global Patient Registry was set up; to gather such data.
Over 220 patients have now joined the registry from 25 countries. This is amazing progress, and we know there are more EHE patients around the world who can share their experiences to make a difference in the future of this disease.
Put simply, joining the EHE Global Patient Registry is the easiest, most impactful way every patient can contribute to EHE science and further our understanding of the disease, by joining the patients who’ve already contributed their EHE experiences to help expand our understanding of this complex sarcoma. The paper we produced about patient experiences with sirolimus, published in February of this year, is an excellent example of how actual patient data can highlight key aspects of EHE and its treatment, and support our demands to regulators.”
As stated above, it is not just the EHE Global Patient Registry that can add so much to EHE research programmes. EHE Biobanks are another hugely important part of the EHE Group’s overall research initiatives, providing tissue, blood and fluid samples to support research and lead to new and better treatments.
Another example is The EHE Foundation’s work with EORTC to develop a Quality of Life assessment for EHE that could be used in the future to measure quality of life when testing a new drug. The EHE Group are also assisting in the development of a pain survey that will be conducted later this year to allow pain to be assessed as part of clinical studies.
All of these different initiatives are key if we are to make progress and find a cure for EHE. But the reality is that none will be effective without significant patient support and engagement. So please engage and help to bring forward the day when an EHE diagnosis need not be feared.