Review and update meetings Glasgow and Liverpool 22 February 2014
Duchenne Research Breakthrough Fund review and update
Jul 28, 2015
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Review and update meetingsGlasgow and Liverpool
22 February 2014
Today’s agenda
• The Fund - overview
• Research update
• Regulatory challenges
• Story from local ambassador
• Questions
The Fund
Phase 1 completed - £800,000 invested Phase 2 - £1.4million target by 2016/17
4 research projects completed - £446k
3 continuing research projects - £230k
Clinical trial readiness & infrastructure - £72k
Parliamentary campaigning - £30k
7 new research projects - £554k
3 continuing research projects - £126k
Parliamentary campaigning - £160k
Clinical trial readiness & infrastructure £380k
New clinical research fellow - £180k
Overall investment into Duchenne muscular dystrophy - £2.2million
The Duchenne Forum
Paving the road to treatments
Dr Neil BennettResearch Communications Officer
Dr Alison StevensonSenior Grants Manager
Email: [email protected]: www.muscular-dystrophy.org
• The research department
• Clinical trial update
• Why and how we fund research
• Update on Duchenne research projects
• The future
Overview
• Research grant program• Applications undergo a rigorous peer-review process• Only the best science is funded
• Facilitate collaboration/communication between scientists• Organising conferences and workshops• Funding support for the European Neuromuscular Centre
• Research Communications Service• Target Research• Weekly web-based news service• Information on clinical trials
Our Activities
Exon-skipping clinical trials• Two initiatives currently trialling exon-skipping technology
• Prosensa/GSK and Sarepta Therapeutics targeting exon 51
• Eteplirsen trial - slower decline in walking ability• The distance boys can walk is stable after 120 weeks• Very small phase 2b study - further trials required
• Drisapersen phase 3 trial failed to show effectiveness• Large phase 3 trial of 180 boys• Companies now analysing the data in more detail
• Prosensa testing molecular patches to exons 44, 45, 53
Other clinical trials
• Ataluren for Duchenne muscular dystrophy • Phase 3 trial under way• Could help cells overcome nonsense mutations
• SMT-C1100 • Phase 1b trial started• Has the potential to increase production of utrophin
• Other trials include• HT-100• Stem cells
Why fund research?• Not all potential treatments in clinical trials will work
• We don’t know which therapeutic approach will work best
• Initial treatments may need to be improved
• Different approaches may be needed in different boys
• Exploring a broad range of therapeutic approaches will maximise the chance of finding an effective treatment
• The charity was founded in 1959 by a clinician, a scientist, and the parent of a boy with Duchenne
• A strong focus on funding research to find treatments and eventually cures
• In the last 10 years the charity has invested:• £4.3M into Duchenne research• £2.7M into UK muscle centres• £0.5M into databases
• Spearheaded a number of promising, potential treatments
Duchenne Research: a focus within the charity
How do we choose research projects
Research Grant Application
Scientific ApplicationLay Application
International Peer ReviewLay Research Panel
Medical Research Committee
Board of Trustees Meeting
Peer Review
• What is it Peer Review?• It is used to assess the quality of scientific ideas • Independent scrutiny by qualified experts (peers)
• What does it tell you?• Scientifically valid, significant and original• Timely and achievable• Not unnecessarily duplicating other work• Using appropriate methodologies• Carried out by researchers with the right skills and facilities• Value for money• Fits with our research strategy
Quality control for science
Strategic topics
1. Developing potential therapeutic approaches
2. Promoting clinical trial readiness
3. Clinical trials and pilot studies
4. Understanding the cause of neuromuscular diseases
5. Building clinical and scientific capacity
6. Improving quality of life
7. Foster sharing of knowledge and networking within the scientific community in the UK and internationally
8. Building partnerships
Gene identificationTherapydelivery
Therapeutic concepts
Testing drugs inanimal models
Improved diagnostics
Better disease characterisation
Well characterised animal models
Understanding muscle function
Understanding what goes wrong
Basic Science PreclinicalStudies
The research we fund
Clinical trialsinfrastructure
Regulatoryaffairs
Gene identificationTherapydelivery
Therapeutic concepts
Testing drugs inanimal models
Improved diagnostics
Better disease characterisation
Well characterised animal models
Understanding muscle function
Understanding what goes wrong
Basic Science PreclinicalStudies
The research we fund
Clinical trialsinfrastructure
Regulatoryaffairs
Professor Nic Wells at the Royal Veterinary College is investigating ways to make blood
vessels more leaky to improve the delivery of molecular patches to the muscles
Professor Matthew Wood at the University of Oxford is looking at using short protein fragments, called peptides, to improve the delivery of the molecular patches to the heart
Ongoing Duchenne research projects
Professor George Dickson is using exon skipping to boost muscle growth by blocking the activity of a protein called myostatin.
New research projects
• The 2013 grant round focused on research for Duchenne muscular dystrophy
• Seven projects were awarded; developing various potential therapeutic approaches
• With the contribution of other Duchenne charities through the Duchenne Forum our commitment for the next 4 years will be £850,000
New research projects
Prof Kay Davies (Oxford University)• £128,000 for 2 years• Developing small molecules to target
Duchenne muscular dystrophy
Dr Jennifer Pell (University of Cambridge)• £121,000 for 2 years• Investigating inflammation in Duchenne
muscular dystrophy
• Dr Angela Russell (Oxford University)• £110,000 for 4 year PhD studentship• Understanding how small molecules can
increase levels of utrophin
New research projects
Prof George Dickson (RHUL)• £80,000 for 3 year PhD studentship• Genome surgery for Duchenne muscular dystrophy
• Prof George Dickson (RHUL)• £180,000 for 3 years• Development of a triple-transplicing system
for Duchenne muscular dystrophy
Prof Jenny Morgan (UCL)• £111,000 for 4 year PhD studentship• Altering the muscle environment to
influence stem cell behaviour
New research projects
Prof Matthew Wood (Oxford University)• £110,000 for 4 year PhD studentship• Searching for biomarkers of Duchenne
muscular dystrophy
Translational research• The transfer of scientific discoveries into clinical applications
Bench – to – Bedside
• Several challenges due to the rarity of the conditions• How to find the patients• Assessing the benefit of a potential treatment
• The promotion of public-private partnership is important particularly with regards to the cost
The translational triangle
New Technology
Patients Outcome measures
Supporting clinical (trial) infrastructure• Clinical Training and Research Fellowships
• Clinicians who undertake a research project• Current call for applications for a Fellow who will
focus on Duchenne muscular dystrophy
• Clinical Trial Co-ordinators • Based at the muscle centres in London and Newcastle• Help the centres with the administrative burden of
organising clinical trials
• Neuromuscular database (Northstar)• Collects natural history data from boys with Duchenne• Currently being expanded to collect data from boys no
longer able to walk
• More and more clinical trials starting to take place
BUT…
• Clinical trials are expensive and don’t always work
HOWEVER…
• There is a growing confidence in the scientific community that therapies will start to appear on the market in the next few years
Into the future…
Speeding up access to drugs for rare diseases
Nic BungayDirector of Care, Campaigns & Information
Rebecca JohnstonNeuromuscular Outreach Officer
Introduction and Context
• Potential treatments on the horizon
• Regulatory hurdles
• Approval process
• Funding arrangements
• Parliamentary activity
Who do we need to influence?
• European Medicines Agency and relevant Committees
• Medicines and Healthcare products Regulatory Agency (MHRA)
• National Institute for Health and Clinical Excellence (NICE)
• NHS England
• Health Ministers
Parliamentary Inquiry – background
All Party Parliamentary Group for Muscular Dystrophy inquiry in 2013:
“To determine the future of access to high cost drugs for orphan diseases in the NHS; the regulatory and reimbursement environment for orphan drugs; and the future of funding arrangements for research into treatments for children and adults
with rare diseases”
Parliamentary Inquiry – evidence
• Researchers and pharmaceutical companies
• Patient and family views
• Regulatory, approval and funding perspective
• Department of Health
• Charitable organisations
Parliamentary Inquiry – recommendations
• Government should establish a ring-fenced fund for rare disease drugs
• NICE should assess treatments for rare conditions differently from less rare conditions
• the Medicines and Healthcare Products Regulatory Agency (MHRA), NICE and NHS England should speed up access to life-changing drugs after the final stages of clinical trials
• NHS England should ensure specialist centres are equipped with an appropriate range of health professionals to deliver treatments
Parliamentary Inquiry – report and follow-up
• Report presented to Health Minister Norman Lamb in September 2013 at MDC parliamentary reception
• Subsequent roundtable and one-to-one meetings with MPs and Peers, MHRA, NICE and NHS England
• Parliamentary pressure – debates and questions in the House of Commons and House of Lords
THANK YOU
ANY QUESTIONS?
Fundraising What can you do to help?
What can I do?
• Place collection tins in your local area
• Make a regular donation by Direct Debit
• Take part in our events programme
• Help out at an organised collection
• Organise your own fundraising event
• Set up a Family Fund
Family Funds
THANK YOU
ANY QUESTIONS?
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