EHE is one of nearly 80 diseases collectively known as ultra-rare sarcomas which each have an incidence ≤1 case/million people per year. Ultra-rare sarcomas include bone and soft tissue tumours, that collectively constitute around 20% of all sarcoma cases. This means many ultra-rare sarcoma patients face challenges that all patients with ultra-rare cancers face, such as:
- Having minimal data on the natural history of their disease;
- An acute lack of research funding to comprehensively study ultra-rare diseases;
- Few and poorly defined treatment options; in EHE there are no disease-specific approved treatments
- Major hurdles in designing and performing large, randomised clinical trials within a reasonable timeframe and cost due to the extreme rarity of patient populations
- Difficulties in drug development and access due to low interest from commercial drug developers
To try and address these serious challenges, a broad group of representatives from the global sarcoma community under the umbrella of the Connective Tissue Oncology Society (CTOS), including clinicians, scientists, patient advocates and non-profit organisations are establishing a consortium to address these hurdles, through the development of a new project called the PUSH (Pushing Rare Sarcoma towards Hope) Platform. The central aim of the PUSH Platform is to maximise the knowledge that is gained from interacting with and treating every single patient affected by an ultra-rare sarcoma and to support developing new treatments to improve outcomes and quality of life.
EHE is one of these ultra-rare sarcomas, and so the EHE Group immediately supported the development programme. Hugh Leonard is a member of the PUSH Executive Committee, as well as sitting on various working groups that have been created to help take the project forward.
Ultimately it is hoped that a substantial number of institutes and academic centres across the globe will join the project, forming the PUSH Consortium, giving the PUSH Platform real momentum and an ability to change the landscape for ultra-rare sarcomas. This will include collating relevant prospective and retrospective data from substantial numbers of patients from around the world. PUSH will incorporate data from a huge range of different sources that will in turn be used, with regulatory involvement and input, to define and undertake appropriate new prospective studies and associated analyses to help deliver new treatments for patients in faster, lower-risk, well understood and defined processes. PUSH is designed to also include prospective clinical trials. The first study will aim to support the prospective assessment of sirolimus as a treatment for progressive EHE.