APPENDIX 11
Memorandum submitted by the Leukaemia
Research Fund
ABOUT THE
LEUKAEMIA RESEARCH
FUND
1.1 The Leukaemia Research Fund was established
in 1960 and is now the UK's third largest national cancer charity
with a current annual research spend of around £14.5
million. The present five year commitment to approved research
awards is just under £37 million.
1.2 The Fund's research portfolio encompasses
all the haematological malignancies ie the leukaemias, lymphomas,
multiple myeloma, aplastic anaemia, and related less common disorders.
There are 18,000 new cases of these malignancies in the UK each
year.
1.3 The Fund supports laboratory based research
which has been rigorously evaluated by external peer review and
by the Fund's Medical & Scientific Advisory Panel. The Fund's
peer review procedures are cited as an example of best practice
by the Association of Medical Research Charities. The Fund currently
supports 30 long-term programme grants, 10 senior fellowships
and lectureships, 120 project grants and 30 junior fellowships
and studentships. Details of the Fund's research is published
annually as a Directory of Research. A copy is enclosed as Appendix
I[11].
This is also available at www.lrf.org.uk. The Directory is a resource
for the UK research community, for haematologists and medical
oncologists and for Research Councils and companion research charities.
1.4 The Fund has established a Clinical
Trials Advisory Panel for the treatment of leukaemia and related
malignancies. There is a need for phase II trials as a necessary
precursor to national phase III randomised trials which historically
have been supported by the MRC. There is concern in the UK haematology
community that financial constraints are threatening the continued
funding of national phase III randomised leukaemia trials by the
MRC. The task of the LRF Clinical Trials Advisory Panel is to
deliver innovative phase II trials which will unequivocally translate
into informative national phase III randomised trials that will
be acceptable for support by the MRC. Annex I contains (i) a briefing
note from Professor Alan Burnett, Chairman, Adult Leukaemia Working
Party and Dr Brenda Gibson, Chairman, Childhood Leukaemia Working
Party concerning MRC leukaemia trials, (ii) the terms of reference
of the Clinical Trials Advisory Panel and (iii) a diagram to show
the pivotal roles of the MRC and LRF in partnership with the NHS
and pharmaceutical companies for the development of new treatments
through clinical and laboratory-based research.
1.5 LRF grantholders are leading academic
staff in Universities, Medical Schools, teaching hospitals and
research institutes who are expert in haematology, medical oncology,
epidemiology, immunology, cell biology and molecular biology.
Funding is directed to campuses that offer the right multi-disciplinary
research and clinical infrastructure appropriate for the particular
topic under investigation. The LRF national research portfolio
is thus a "virtual" institute which capitalises on the
individual strengths of particular centres of research and clinical
excellence. This ensures that the whole research enterprise of
the LRF is greater than the sum of its individual projects.
1.6 The Leukaemia Research Fund has four
main goals:
— to discover the causes of leukaemia
and related cancers
— to understand how genetic lesions
in blood cells cause them to become cancerous
— to continually refine diagnostic
methods for the rapid and accurate assessment of disease
— to devise new strategies for the
treatment of these cancers.
1.7 The funding strategy of the Leukaemia
Research Fund is very simple. It is to support the right project,
in the right place, at the right time, with the right people.
To achieve this the Fund is both proactive and reactive in building
its research portfolio. On average about 175 applications are
received each year (reactive mode funding) for consideration for
short term funding of project grants, training fellowships and
studentships. Each application is rigorously reviewed by UK and
international experts for its scientific quality, relevance to
the Fund and its priority for funding. The Fund currently draws
on the expertise of 1,000 referees both from within the UK and
overseas for this purpose. About 30 per cent of the applications
are recommended for support.
1.8 The transfer of knowledge from laboratory
based research into clinical benefit to patients is a key task
for the Leukaemia Research Fund. To facilitate this the Fund co-ordinates
and plans a national research strategy through its Specialist
Programme grants (proactive mode funding). These are for teams
of investigators to work long term on programmes of translational
research at the interface between the laboratory and the clinic.
The appraisal and review of Specialist Programmes is by a two-day
site visit of a committee of international experts to the host
laboratory. There are 25 Specialist Programmes at present. Examples
are programmes to investigate cord blood stem cell transplantation,
immunotherapy, bone marrow failure, immuno- and molecular diagnosis
of disease and the molecular pharmacology of contemporary chemotherapy.
Criteria for programme grant support are identified by (i) consolidation
of consistent project grant support into a structured programme,
(ii) strategic calls for applications in areas of new interest
eg immunotherapy and (iii) consortium funding with other funding
agencies, Universities and teaching hospitals.
1.9 The Leukaemia Research Fund places a
high priority on the training and career development of outstanding
young clinicians and scientists. The Fund offers elective bursaries,
studentships and fellowships for training in research and the
award of a higher degree. The purpose is two fold (i) to recruit
and retain future leaders in UK haematological research and (ii)
to provide the NHS with high quality haematologists and medical
oncologists for the benefit of patients with leukaemia and related
malignancies.
1.10 In partnership with a host University
the Fund will provide five years pump priming support for the
establishment of new academic posts. This has been particularly
successful in establishing new clinical senior lectureships in
haematology in Glasgow, Leeds, Manchester, Nottingham, Southampton
(two) and London (three). The host University has assumed responsibility
for these posts at the end of the five year period.
1.11 The Fund provides five year senior
research fellowships which include up to two years in an overseas
laboratory for outstanding young post doctoral scientists and
clinicians to progress to independent investigator status. The
Fund also offers three travelling fellowships for two year appointments
in the world's premier research institutes.
1.12 The Leukaemia Research Fund also recognises
the importance of contact and communication with international
colleagues for the dissemination and acquisition of new research
findings. The Fund organises an annual Research Symposium in London
and also supports Symposia as part of the annual meetings of the
British Society of Haematology and the European Haematology Association
(see Appendix V). The Fund encourages grantholders to attend and
contribute to these meetings and, additionally, the annual American
Society of Haematology meeting. Travel bursaries are provided
for this purpose.
CO-ORDINATION
WITH OTHER
ORGANISATIONS
2.1 The mission of the Leukaemia Research
Fund is very clear; namely the routine cure of the haematological
malignancies. It is therefore straightforward to clearly delineate
the Fund's research portfolio and to co-ordinate this with other
organisations. The LRF works particularly close with the Medical
Research Council (MRC), the Cancer Research Campaign (CRC) and
the Kay Kendall Leukaemia Fund (KKLF).
2.2 The Fund provides complimentary funding
to existing core programmes in MRC and CRC research institutes.
This amounts to over £6 million in the past five years.
2.3 It is the Fund's task to deliver international
quality phase II trials (see [1.4] above) which can be taken into
national phase III trials by the MRC. The Fund also provides programme
and project grant support for laboratory based studies which are
integral to national MRC sponsored phase III leukaemia trials.
This amounts to over £3.5 million in the past five years.
2.4 The Fund anticipates closer co-operation
with The Wellcome Trust in the future as the Trust will now consider
parallel funding of fundamental studies alongside applied cancer
research. The Fund will encourage holders of Specialist Programme
grants in particular to seek complementary funding from the Wellcome
Trust to add value to the LRF portfolio.
2.5 The Fund is a full member of the recently
constituted Cancer Research Funders Forum. The first chairman
of the Forum is Professor George Radda, Chief Executive, MRC.
The Fund welcomes this initiative to consider issues of mutual
interest to agencies funding cancer research. The fund expects
the Forum to establish concensus in areas that would benefit from
an integrated and strategic approach by the member agencies.
2.6 The Fund is a full member of the United
Kingdom Co-ordinating Committee on Cancer Research (UKCCCR). The
UKCCCR takes a lead role in facilitating applications to the major
cancer funding agencies for national and international trials
and associated research projects. This function has been strengthened
in the past two years with the establishment of an extensive national
clinical cancer network to co-ordinate trials and research on
14 separate malignancies. This reconfiguring of the UKCCCR role
will be of considerable benefit to the principal cancer research
agencies to ensure continuity and uniformity in trial activity
in the UK. It also provides a focus for multi-agency support of
national studies such as the UK Childhood Cancer Study. This exceptionally
successful study has been funded by a consortium of cancer charities
and the Electricity Association. This study has taken seven years
to collect the data and could not have been done without affiliation
to a UKCCCR Management Committee under the chairmanship of Sir
Richard Doll. The Leukaemia Research Fund applauds the aims and
objectives of the UKCCCR and will contribute fully to its purpose
as a focus for the co-ordination of UK cancer research.
CO-ORDINATION
WITH INDUSTRY
3.1 The Fund invites an expert scientist
from the industry to serve for three years as a member of the
Medical and Scientific Advisory Panel. Presently the member is
from Glaxo Smith Kline Beecham.
3.2 A LRF Business Manager is part of the
team at Cancer Research Ventures (CRV) which is the technology
transfer company responsible for the protection and exploitation
of intellectual property (IP) generated from the Fund's research
portfolio. CRV has been established with support from a UK Department
of Trade and Industry Biotechnology Platform Exploitation award.
The Leukaemia Research Fund is one of the partners in this award.
CRV now also receives funding from the Vanguard Medica Group and
from the European Commission to further CRV's European business.
The LRF Business Manager advises on all technology transfer issues,
monitors the research portfolio and, in partnership with the host
University, ensures that appropriate patents are filed and commercialised
fully with agreed revenue shares. When LRF projects are managed
directly CRV will co-ordinate and finance the patent process,
develop a commercialisation exploitation strategy, consider various
commercialisation options, put together the legal framework and
carry out post signing management of the project. CRV select the
commercialisation route that is most appropriate to the specific
technology. This may include:
— Direct Licensing to biotech/pharmaceutical
companies;
— Option/Licensing arrangements;
— Research Collaboration with research
partners able to bring funding and/or complementary skills to
advance the programme. Such "sponsored collaborations"
are generally linked to downstream option and/or licence arrangements.
— Formation of Start-up Companies or
Joint Ventures with existing companies.
SUMMARY
1. Strengths
National cancer charity with stringent peer
review procedures.
Nationally co-ordinated research portfolio in
centres of excellence.
Delivery of substantive long-term funding to
Universities/Medical Schools throughout UK.
Integration of laboratory research with clinical
studies eg national leukaemia trials.
Excellent European and international profile.
2. Weaknesses
Minimal value of LRF funding in Research Assessment
Exercise.
Lack of core HEFCE resources in LRF supported
laboratories.
3. Opportunities
Partnership funding for major initiatives in
UK centres of excellence for cancer research and treatment.
Contribute to career development of the next
generation of scientific and clinical investigators.
Delivery of top quality phase II trials for
new treatments.
Improved co-ordination through Cancer Research
Funders Forum and UKCCCR.
4. Threats
Poor and inconsistent implementation of the
Concordat for service support and excess treatment costs.[12]
Continuing disenfranchisement from core support
for academic cancer research.[13]
Lack of uniformity and procedures for protection
of intellectual property and its commercial exploitation.[14]
Loss of outstanding young scientists and clinicians
from cancer research due to career uncertainty, financial pecuniary
and lack of adequate research infrastructure.[15]
11 Not printed. Back
12
Clinical trials are an absolute requirement for the advancement
of best medical practice. The best available treatment can only
be superseded by one which is compared against it and shown to
be superior in a properly conducted randomised trial of sufficient
statistical power. Since the mid-1960s MRC leukaemia trials have
become internationally acclaimed for their contribution to seminal
advances in treatments for leukaemia. The outstanding success
of leukaemia trials has been due to maintaining continuity from
one trial to the next. Continuity is vital to the maintenance
of a trial infrastructure encompassing all the major teaching
hospitals and district general hospitals throughout the UK. It
is also vital to the maintenance of internationally competitive
research as an integral part of a clinical trial. The trials are
run as a partnership between the MRC, the national health service
(NHS) and the pharmaceutical companies who provide the drugs under
test. In recent years this has been formalised as a Concordat
between the MRC and the NHS which essentially allows trials to
be conducted with NHS patients even though there may be additional
support and treatment costs to the health service for this "research"
activity. Trials sponsored by cancer charities are recognised
under a partnership arrangement with the NHS as detailed in Annex
B of Health Services Guidelines (97)32 Statement of Partnership
on Non-Commercial R & D in the NHS in England. Interpretation
and implementation of the Concordat is extremely variable
and is proving prejudicial to trial activity. Back
13
Core research resources eg technical and administrative support,
central facilities etc are provided (i) by HEFCE as a result of
the RAE and (ii) by the MRC as an integral part of research grants.
Cancer research funded by the national charities does not qualify
for the allocation of core resources by these routes. Any allocation
is essentially dependent on local benevolance and not subject
to any nationally agreed policy. The lack of core resources is
prejudicial to the pursuit of internationally competitive research
and its application to patient benefit. Back
14
Biomedical research is part of the "knowledge economy".
The exploitation of intellectual property (IP) generated from
cancer research is not well organised at present. Practices and
procedures vary from University to University. The separate interests
of funding agencies, technology transfer offices and potential
commercial partners are difficult to reconcile. There is a lack
of informed expertise to transfer cancer related IP to the market
place. There are few incentives to encourage closer commercial/academic
collaboration. Policy on start-up companies and business plans
is muddled and often naïve. Guidance is required to ensure
a uniform approach by Universities, funding agencies and companies
to this important source of wealth generation. Back
15
The recruitment and retention of clinical researchers of international
standing is a concern across the whole of academic medicine. The
reasons for this are a complex amalgam of changes to training
schemes, conflicting demands between the service and academic
sector, salary restrictions, withdrawal of posts, lack of success
in successive RAEs etc. This debilitation of the clinical research
base compromises the effectiveness of the cancer charities to
strategically invest in long-term internationally competitive
programmes that will deliver new treatments. Back
| 
