We need to understand more about Bone Marrow Failure Syndromes (BMFS). Current therapies are not well tolerated and today's BMFS patients and their families face a narrow range of treatment options that are very disruptive to daily life.
It is simply not acceptable to not have options for young BMFS patients.
To achieve better outcomes, and to ultimately find a cure for BMFS, we work with our highly reputable Scientific Advisory Committee to continue to develop a National Research Strategy. Their aim is to fund the research projects and people who we believe will discover the important breakthroughs this area of medicine so urgently needs. Since inception in 2015, Maddie's Vision has raised over $7 million and as at January 2019, over $2.4 million has already been committed to funding a range of projects in partnership with medial research institutes, hospitals and universities across Australia. Each year, we will look for and continue to support the projects we believe will lead to breakthroughs.
In 2018, with assitance from State Government funding, we established the world's first Centre of Excellence in Bone Marrow Biology. The CRE is a 'brains trust' in Bone Marrow Biology, a virtual centre that provides a centralised contact point for existing researchers, future researchers, commercial entities and community groups. The establishment of a CRE in bone marrow biology provides Maddie Riewoldt's Vision the capacity to incorporate additional projects, complete the critical mass of researchers and complimentary projects, provide additional infrastructure and the coordination of BMFS research required to find a cure for this disease.
In May 2019, the CRE presented Australia's Inaugural National Symposium on Bone Marrow Failure Syndromes. The symposium attracted 180 delegates, comprising the most prominent, innovative and talented clinicians and researchers in Bone Marrow Failure Syndromes Australia wide, allied health professionals, patients and family members. Our exceptional international guest speakers, Dr Akiko Shimamura, Professor of Paediatrics Harvard Medical School, and Director BMF and MDS Program Dana Farber/Boston Children's Cancer and Blood Disorder Centre, and Dr Austin Kulasekararaj of King's College Hosptal NHS Foundation Trust, London, presented their groundbreaking work and expertise, and discussed opportunities to globally collaborate.
With cutting edge research projects underway we are now fundraising for further nationally and globally significant projects, including the establishment of a National Bone Marrow Failure Biobank. The purpose is to create a national biobank that will collect all tissues, blood and genetic material to store and analyse. This project will require a commitment of $1M over the first 3 years and we are well on our way with $350K already committed.
Thoughout everything we do, we are committed to:
- funding and supporting cutting edge medical research
- the development of collaborative, accessible information systems
- fixed term financial support for young researchers through Fellowship positions and Grant in Aid support
- national collaboration via diagnosis registry, establishment of a biobank to collect and store tissue samples
- governance and support of the Centre of Research excellence in Bone Marrow Biology.