www.myeloma.org.uk
Eric Low
Cascais, 16 March 2015
Forming Powerful Partnerships to Drill Down
into the Areas of Expertise of Each Stakeholder
and Unravel Disease Mechanisms
www.myeloma.org.uk
Content
• A new approach - the evolving role of patient-driven research
organisations
• The case for partnership working
• Myeloma UK Research Continuum
• Disease mechanisms in myeloma
• The Myeloma UK – Structural Genomics Research Consortium
partnership
• Summary
www.myeloma.org.uk
A new approach
‘’The role of research charities and patient
organisations has evolved from a primary
emphasis on grant funding to a driving force that
is advancing scientific development and leading
cutting edge patient-centred research.’’
www.myeloma.org.uk
Research Continuum
Reverse translational model – from patients to
discovery and back again
www.myeloma.org.uk
Research Continuum
• Predominantly a directed investment rather than response mode model
underpinned by sustainable, strategic investment, driven by milestones and
focused on results
• Innovative, cutting edge research underpinning Myeloma UK strategic themes
and objectives
• NIHR/NCRI partners, AMRC members
• Translational Research Network: focused on genetics, diagnostics, drug
discovery and development
• Early Phase Clinical Trial Network
• Health Services Research
• Research policy – adoption and diffusion
www.myeloma.org.uk
Working in partnership
• Evidently, one of the major issues seen in research in the past
has been silo working and thinking
• This has led to disparate, unconnected research plans and the
inefficient use of scare resources, expertise and funding
• This approach has been especially damaging in rarer diseases
where the totality of resources is arguably much more limited
• As a way to overcome this in myeloma, Myeloma UK are strong
proponents of working in collaboration and partnership where it
makes sense to do so and to establish, facilitate and/or be part of
consortia and networks
www.myeloma.org.uk
Why this matters
‘’Despite recent advances in treatment and
care, myeloma remains an incurable,
debilitating cancer.’’
www.myeloma.org.uk
Working in partnership
• Integration of researchers from multiple sectors (academia, government,
industry, non-profit, clinical care), particularly those researchers from the
same sector that normally “compete” with each other
• Agreement on a mission that addresses a shared need with a strategic and
milestone-driven plan to achieve outputs and outcomes that, in turn, can be
broadly used by each stakeholder
• A governance structure that provides each stakeholder with an opportunity to
provide input to the partnerships strategic objectives and operations
• An integrated research plan that leverages the research resources and
knowledge from each stakeholder
• Funded by both philanthropic and commercial investment models
www.myeloma.org.uk
Disease Mechanisms
Uncontrolled increase in plasma cells within the bone marrow:
• Fail to die • Keep growing • Grow in the wrong place • Damaged genetic code (DNA) • Make only one kind of antibody
(clonal paraprotein) • Relapsing/remitting
Damage to the body:
• Kidneys • Bone destruction • Bone marrow failure
www.myeloma.org.uk
Myeloma UK – SGC Partnership
OPTIMAL- Identifying novel therapeutic approaches to myeloma
• Target identification validation
• Target specific tool compounds
• Collaboration with SGC
• Collaboration with pharma
• Pre-clinical drug development
• Drug testing in patient cells
• Identification of pharmacodynamic markers
www.myeloma.org.uk
Summary
• Patient-driven organisations are often the catalyst to
bucking the trend
• Myeloma is a very challenging problem
• Working strategically and collaboratively aligned to a
common goal is critical
• Patient benefit is the single most important driver
• A relentless commitment is needed to be successful
www.myeloma.org.uk
Email: [email protected]
Web: www.myeloma.org.uk
Twitter: @MyelomaUK | @EricLowMUK
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