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University College London Hospitals Biomedical Research Centre
Research with
impact
Biomedical research centre annual update
Welcome to this summary of a
really fabulous year for the BRC.
What is striking to me is that the
work the BRC supports has such a
powerful impact in so many areas
of clinical medicine with clear
evidence of increasingly rapid
‘pull through’ of home grown
discovery science to develop
future novel treatments and
diagnostics for patients.
This really is ‘research with impact’. Universities are also
talking excitedly about impact with good reason. Whilst for
the moment traditional academic metrics remain dominant,
the delivery of impact has assumed great importance
and this is likely to increase with greater emphasis on
building the infrastructure to facilitate impact, and stronger
interdependency with partners such as the NHS and
industries, big and small. This can only be good for clinical
research and we must seize the moment. I think we are
on the cusp of a renaissance in experimental medicine and
translational clinical research.
The vast majority of the infrastructure to facilitate high
impact health research in universities and the NHS has come
from the NIHR via the BRCs, CRFs etc. Without this, the pull
through of clinical science for patient benefit and impact
would be much less impressive and that would be such an
opportunity missed. Indeed, the impact of NIHR funding was
acknowledged by HEFCE in the recent REF as being critical to
many of the highest quality impact statements submitted by
universities, no doubt many of these from UCL.
The BRC and NIHR infrastructure funding are also key to the
development of highly specialised medicine and surgery in
our leading hospitals such as UCLH and this is going to grow
in importance as treatments become more personalised and
complex through genomics, innovative imaging and other
methods for deep phenotyping.
Our goal is to be at the forefront of this fast-paced change
and continue to build clinical research infrastructure and
equip our research teams to tackle the big unresolved
questions in medicine so that we can continue to deliver
‘research with impact’.
Professor Bryan Williams
Director of the NIHR University College London Hospitals
Biomedical Research Centre.
Research with impact: biomedical research centre annual update
We are a leading biomedical research centre
• Our BRC has the largest portfolio of experimental
medicine, particularly in interventional studies,
compared with the other biomedical research
centres in the UK.
• We have also initiated the largest number of
studies. As a result, our BRC had the highest
number of active clinical research studies (over
700) in 2013/14, almost 200 more than the next
most active BRC for clinical research studies and
one of the largest portfolios of phase l and ll
clinical trials.
Our BRC is a leader in leveraging external
funding
• The BRC leveraged £153m in external grant
funding and increased collaborative contracts
by 20% over the last year.
• Healthcare technology investment company
Syncona invested £30m into a new UCL ‘spin
out’ company called Autolus, to develop and
commercialise next-generation engineered
T-cell therapies for haematological and solid
tumours. These therapies are based on the
work of BRC-supported Dr Martin Pule, a
clinical haematologist at the UCL Cancer
Institute and consultant at UCLH.
• BRC-funded projects have been establishing
laboratory space at the Stevenage Bioscience
Catalyst, including Dr Andrew Williams’
and Professor Rachel Chambers’ project
‘Targeting CCL7 in acute lung injury’. The
Stevenage Bioscience Catalyst is the UK’s first
open innovation bioscience campus, located
next to GlaxoSmithKline and BRC funding has
meant that UCL industry-related projects can
take up lab space on the campus and access drug
development expertise and facilities.
• UCL Business, Imperial Innovations and the Cell
Therapy Catapult formed Catapult Therapy TCR
Ltd to develop a novel cell therapy approach
for acute myeloid leukaemia. The treatment
being tested is a novel T-cell therapy targeting
malignancies associated with overexpression
of the WT1 antigen, such as acute myeloid
leukaemia and myelodysplastic syndrome. The
Cell Therapy Catapult will invest up to £10m to
take the therapy into and through Phase II trials.
Developing research infrastructure
• With BRC support, next generation sequencers
have been approved for cancer, neuroscience and
infection areas.
• UCL has given the go ahead for building of a
BRC-funded GMP laboratory for clinical grade PET
tracers which will enable researchers to develop
novel tracers for use in UCLH’s PET/MRI facility.
• The BRC awarded £500K to support
infrastructure in imaging and bio-banking of
stroke patients, contributing to an understanding
of phenotype/genotype.
• Two research fellows were established by the
BRC as part of the new cardio-oncology research
programme being set up across UCL and UCLH.
• The Cardiometabolic Phenotyping Unit was
opened by HRH The Princess Royal and hosts
a growing portfolio of Phase I/II cardiovascular
physiology studies. The unit provides researchers
with much needed space specifically designed to
carry out early stage cardiovascular physiology
research.
• At our NIHR Clinical Research Facility (CRF)
recruitment to studies has increased by 25% in
the last year and there has been an increase in
early phase studies – 53% of open studies are
phase I or phase II and 82% of studies in set up
are phase I or II. Occupancy has been consistently
higher over the last year and attendances average
about 300 per month.
• The NIHR CRF has been a centre of world-leading
research and over the last few months has given
the first in human dosing in the world for three
studies. It is the only global site for a Phase I/
II cancer study and the only UK site for a trial of
a treatment for thrombotic thrombocytopenic
purpura and a proof of concept Phase II trial of
treatment for antiphospholipid syndrome.
Research with impact: biomedical research centre annual update
Drug breakthrough
news
• Preparatory work for a trial of a drug known as
CPHPC to treat Alzheimer’s disease is making
good progress, with the trial scheduled to start in
April 2016. The drug was developed by Professor
Sir Mark Pepys FRS, Director of the UCL Wolfson
Drug Discovery Unit. It depletes circulating serum
amyloid P component (SAP) and completely
removes SAP from the brain. Sir Mark has also
developed a drug that is the first treatment aimed
at directly removing amyloid deposits from the
tissues of people with the rare and usually fatal
disease, systemic amyloidosis. The first clinical
trial by GlaxoSmithKline began in 2013 and
continues to deliver encouraging results.
Data and genomics updates
• 2014 saw the blossoming of the UCL BioResource
project which aims to enlist 10,000 volunteers to
give DNA and consent to being contacted in the
future about suitable research trials:
• O
ver 2380 patients and healthy volunteers
have been recruited.
• S tudies currently recruiting include: Personality
Disorders – a computational psychiatry
approach; and Epilepsy Pharmacogenomics:
delivering biomarkers for clinical use.
• U
CL BioResource is taking part in its first
Stage 2 study, with volunteers answering an
online questionnaire on thinking styles.
• The BioAid project which is a cross-BRC
collaboration with Imperial and King’s has taken
off. The aim is to create a registry of 5,00010,000 adults presenting with infectious disease,
and link clinical phenotype and microbiological
data to host DNA, RNA and plasma biobanking.
A dedicated research nurse was appointed
in August 2014, and 355 patients have been
recruited at UCH to date.
• The BRC continued to be a part of the major
health informatics programme – the National
Institute for Health Research Health Informatics
Collaboration (HIC). Clinical data are now being
shared between the 5 BRCs who are collaborating
on NIHR HIC. UCLH has been leading on the
critical care theme and has received approval from
the Confidentiality Advisory Group for the use
of identifiable data without the need for explicit
consent, a reflection of the strong information
governance framework that has been put in
place for the project centred around the UCL Safe
Haven for data. The study has been designed
with significant public and patient input and
community engagement revealed overwhelming
public support.
The recent Research Excellence Framework has
highlighted how the BRC is underpinning UCL’s
highest impact research, through the provision of
essential clinical infrastructure:
• As a result of Professor Mark Emberton’s research
into self-management intervention for men with
lower urinary tract symptoms, most evidencebased practice guidelines now recommend selfmanagement as the initial therapy. The estimated
cost saving to the NHS is around £20m annually.
• The current standard treatment for relapsed and
resistant Hodgkin lymphoma has adopted high
dose therapy and autologous transplantation as
a result of work by BRC-supported researchers.
This follows research into the use of high dose
chemotherapy, studies of the factors predicting
successful stem cell harvests and a refining of
allogeneic transplantation.
• Research led by Professor David Linch has
changed the way patients with different forms
of non-Hodgkin lymphomas (NHL) are managed
worldwide. In aggressive NHL, the role of
autologous stem cell transplant was established in
patients who relapse after primary chemotherapy.
In indolent NHL, it was shown that watchful
waiting allows chemotherapy to be postponed
for an average of two and a half years with no
reduction in survival rates.
Research with impact: biomedical research centre annual update
Impact on global healthcare
The core research infrastructure that the BRC has
built up has had a fundamental impact on global
healthcare as revealed by the latest Research
Excellence Framework. Examples include:
• Researchers led by Professor Nicholas Wood were
an integral part of the group that first described
mutations in the gene LRRK2. This represented a
significant shift in understanding of Parkinson’s
disease (PD) that had been seen as a non-genetic
disease. The team’s discovery of the mutation
G2019S in this gene showed for the first time
that a relatively rare genetic variant could not only
cause familial PD, but could also play a significant
role in sporadic PD. The team developed a now
widely available genetic test.
• UCL research has led to a new treatment for
patients with severe juvenile idiopathic arthritis
(JIA) transforming their long-term outcome.
UCL led the first clinical study outside of Japan
to investigate escalating doses of the drug
tocilizumab and this led to the TENDER trial, a
multi-centre phase III study which demonstrated
unequivocally the efficacy of tocilizumab, which is
now the standard care of children with systemic
onset JIA.
• BRC support for researchers, high impact projects
and the Farr Institute has been instrumental in
the huge impact of UCL research on the quality
and outcomes of care of people with or at risk
of cardiovascular disease. Research by Professor
Harry Hemingway and his team has directly
informed national and international clinical
guidelines, including NICE guidelines, for treating
cardiovascular disease, including the management
of stable angina.
• World-leading collaborative research led by
BRC-supported Professor Michael Hanna has
helped identify new disease mechanisms for
genetic muscle channelopathies. The result
is the development of new tests supporting
faster diagnosis and better patient outcomes.
The UCL team was commissioned by NHS
Highly Specialised Services to provide the
UK’s only diagnostic and treatment centre for
channelopathies.
• A team led by Professor Tony Schapira at UCL
developed scales to assess non-motor symptoms
which may be some of the early indicators of
the onset of Parkinsons. The questionnaire they
developed is now used to identify people at risk
of developing Parkinsons and is recommended
by the Department of Health in the 18-week
commissioning pathway way for tremor.
• UCL investigators have been at the forefront of
assessing HIV drug resistance soon after the very
first HIV drug was licensed. Professor Deenan
Pillay was instrumental in setting up the UK HIV
Drug Resistance Database. This national-level
collaboration was extended firstly into a
Europe-wide database and then into surveillance
of drug resistance on a global scale. This work
has influenced the WHO surveillance strategy and
the rollout of anti-retroviral therapies around the
world.
• BRC-supported Professor Steve Humphries,
director of the UCL Genetics Institute, has
led on research into the genetics of familial
hypercholesterolaemia (FH) that has had
a significant impact on the identification
and treatment of patients with FH and the
development of new DNA tests and diagnostic
protocols now in wide use across the UK.
• The MRC Prion Unit developed a validated blood
test for vCJD. The UCL blood test is now in use
at the National Prion Clinic (NPC) – the national
referral centre for prion disease based at the
National Hospital for Neurology and Neurosurgery
– to allow diagnosis of vCJD. Approximately 115
patients have been tested so far through the
clinic. The blood test has been demonstrated to
detect infection in over 70% of patients with
vCJD with, to date, 100% specificity. The NPC’s
Prion disease cohort is funded by the BRC.
• Research at the UCL Institute of Neurology
has resulted in the licensing of natalizumab
(Tysabri), a potent treatment for highly active
relapsing remitting multiple sclerosis. By 2013,
over 115,000 patients had received natalizumab,
which has been shown to reduce relapse rate by
two-thirds and relapse-related disability by 50%.
• BRC funding for research teams at the UCL
Institute of Cardiovascular Science has resulted
in significant improvements in the identification
and treatment of patients with cardiomyopathies
(diseases of the heart muscle) and genes
identified by the research are now regularly tested
for across the UK and around the world.
Research with impact: biomedical research centre annual update
The BRC has funded significant scientific
breakthroughs
• BRC researchers discovered that genetic
mutations which cause a decrease in dopamine
production in the brain and lead to a form of
childhood-onset Dystonia also play a role in the
development of Parkinson’s disease – opening
up the way for more research and possible
therapeutic implications.
• BRC-supported researchers found high dose of
the drug simvastatin significantly reduces brain
shrinkage in people with secondary progressive
multiple sclerosis. The study provides an exciting
platform that needs to be taken into a late phase
III trial to see if the drug has a sustained effect on
disability over a three-year period.
• Researchers found switching off the mutant
protein that causes the fatal brain disorder
Huntington’s disease can reverse abnormalities in
living cells taken from patients with the disease.
Developing academic and clinical academic
careers
The Academic Careers Office (ACO) helps develop
academic and clinical academic careers in the BRC
and School of Life Medical and Sciences. Highlights of
the year include:
• MiniMD, a two-week intensive course run in
collaboration with London Cancer for non-clinical
scientists, was attended by researchers, heads
of laboratories and researchers from charities.
As well as MiniMD Cancer, a MiniMD in medical
imaging (neuro and vascular) will be running this
year and will form part of the Medical Imagining
CDT training portfolio.
• The BRC is collaborating with the Francis Crick
Institute and other London BRCs to fund new
clinical research fellows working in experimental
medicine.
• The BRC ran two Experimental Medicine
Conferences – one on cardiometabolic medicine
and another on experimental medicine in a
pandemic. Two conferences are currently being
planned on neurosciences and cancer.
• The BRC supported staff to take part in a modular
based leadership development programme
offered in partnership with UCLP staff college.
• The ACO and BRC in collaboration with the NIHR
Trainees Coordinating Centre launched a scheme
enabling PhD and MD trainees supported by the
NIHR to spend time in other parts of the NIHR
infrastructure.
Research with impact: biomedical research centre annual update
Engaging with patients and the public
of the preceding year’s scheme revealed that
79.3% of students are going to university, 58.6%
of these to a Russell group university and all of
those were studying for a STEM degree.
The BRC has worked to develop patient and public
involvement (PPI) in research
• With funding from Health Education England, the
BRC ran a programme of training workshops for
researchers based in UCLP partner organisations.
120 researchers took part and evaluation revealed
a dramatic increase in researchers’ confidence.
• The BRC provided regular PPI training sessions
– 60 researchers attended six workshops with
100% finding the session ‘excellent’ or ‘good’.
The BRC also ran three workshops for 26 lay
people, demystifying research processes – 66%
reported better understanding of research and
their role.
• Engagement work included a successful open day
at the Clinical Research Facility with schools and
public tours, and a tour of research stalls around
the different UCLH hospital sites.
• The BRC continued to feature news stories about
research it supports, publishing online an average
of 10 news stories a month.
• Press coverage of BRC research included:
• D
r Rachel Batterham’s weight loss surgery
recommendations featured in the national
press The Independent, The Guardian, Sky,
Radio 4, Daily Mail
• £
10m awarded for foetal surgery research in
Professor Sebastien Ourselin’s project featured
by CNN, The Telegraph and the Mirror.
• B
reakthrough by Dr Jeremy Chataway’s team
suggesting statins could help progressive MS
patients featured by the BBC, The Guardian
and The Telegraph.
• Our cardiometabolic programme held a series of
seminars for lay people on the latest in research.
• Research capability funding enabled the BRC to
employ a PPI coordinator for three months to
work with 12 researchers at grant application
stage. Help included finding lay people and
setting up and facilitating meetings.
• A grant from the Wellcome Trust enabled the BRC
to set up a PPI bursary fund. Nearly £20k has so
far been awarded.
The BRC took the lead in raising the profile of
research:
• The BRC hosted a massive research open day in
the atrium of UCLH, with 28 stalls, school tours
and interactive activities. The day was the first of
its kind at UCLH and was attended by the CEO,
chairman and directors.
• The BRC worked with industry partner Infonetica
to develop the UCLH Research Gateway, which
enables patients to search all recruiting trials.
Probably the first of its kind, the gateway was
featured in The Guardian online.
• The BRC provided a BRC summer school of
25 places on a lab placement scheme for
disadvantage gifted ‘A’ level students – 86.2% of
students live in the most deprived areas in London
and 76% were on free school meals. Evaluation
www.uclhospitals.brc.nihr.ac.uk
@UCLHresearch
www.uclh.nhs.uk
www.ucl.ac.uk
1st Floor, Maple House
149 Tottenham Court Road
London W1T 7NF
Telephone: 020 7679 6639
Photography:
David C Bishop, Medical Illustration Services
NHS Photo Library
February 2015
Design: Susan Rentoul Design