For more information contact: Slideshare@marcusevans.com. Forming Powerful Partnerships to Drill Down into the Areas of Expertise of Each Stakeholder and Unravel Disease Mechanisms Eric Low Cascais, 16 March 2015

1. www.myeloma.org.uk
EricLow
Cascais,16March2015
FormingPowerfulPartnershipstoDrillDown
intotheAreasofExpertiseofEachStakeholder
andUnravelDiseaseMechanisms

2. 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

3. www.myeloma.org.uk
Anew 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.’’

4. www.myeloma.org.uk
Research Continuum
Reverse translational model – from patients to
discovery and back again

5. 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

6. 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

7. www.myeloma.org.uk
Why this matters
‘’Despite recent advances in treatment and
care, myeloma remains an incurable,
debilitating cancer.’’

8. 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

9. 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

10. 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

11. 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

12. www.myeloma.org.uk
Email: eric@myeloma.org.uk
Web: www.myeloma.org.uk
Twitter: @MyelomaUK | @EricLowMUK