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: eric@myeloma.org.uk

Web: www.myeloma.org.uk

Twitter: @MyelomaUK | @EricLowMUK