Download ORGAN TRANSPLANTATION - The Problem ONE

ONE STUDY_BROCHUREv3_Layout 1 10/12/12 11:15 Pagina 1
ONE FOCUS
ORGAN TRANSPLANTATION - The Problem
The short-term success rates of transplant surgery
have improved remarkably over the last half century,
making this procedure a life-saving option for many
patients with organ failure. Early outcomes in transplant
recipients are outstanding: patients normally recover
from surgery with a well-functioning replacement organ
and typically return to an active lifestyle in a short period.
However, transplant practice is complicated by the
immune reactions against allogeneic (non-self) tissues,
resulting in donor organ destruction within a matter of
weeks after transplantation. To impede this rigorous
immunological response, an armamentarium of
general immunosuppressive drugs has been developed.
The concession of this therapeutic practice, in terms of
safety and early recovery, is that the whole immune
system is impaired and a myriad of side-effects are
typical. Thus, patients after organ transplantation often
suffer the consequences of drug toxicity, the development
of chronic rejection, reduced resistance to infections
and a high rate of cancer occurrence. Besides the
morbidity associated with life-long immunosuppression,
there are high financial costs for the families and for
society to bear. Therefore, use of immunosuppressive
drugs needs to be minimised.
Integrating
Basic
Research
Ideas
for
Improved
Health
Care
Preventing immunological rejection of transplanted
organs without the need for long-term use of
pharmacological immunosuppression is a primary
objective in transplantation medicine. For this
reason new transplant research should concentrate
on early strategies that support long-term immunological
acceptance of transplants, allowing for at least a
reduction in the use of general immunosuppression.
Conditioning the immune response of solid organ
transplant recipients towards allograft acceptance
using cell-based therapies is now becoming technically
feasible and clinically promising.
The central focus of the ONE Study cooperative work
programme is to:
produce and manufacture distinct populations of
haematopoietic immunoregulatory cells
comparatively study the immunosuppressive
characteristics of these regulatory cell types
test these cell therapy products side-by-side in a
clinical trial in living donor renal transplant recipients
The health economics of cell therapy as a new medical
technology is another essential aspect of the ONE
Study work programme that will be fully evaluated.
True viability of the proposed new cellular treatments
will depend not only on their clinical benefit, but also
on an acceptable health-economics profile.
ONE OUTCOME
This very focused scientific research programme is
expected to identify the most promising regulatory cell
products for further clinical testing and commercial
exploitation in the field of organ transplantation.
More generally, the anticipated scientific advances will
also lead to a rationalisation of research efforts into
cell-based techniques for the reduction of immunosuppressive
drugs and will be potentially applicable for the
treatment of other immunological conditions.
ONE DESIGN
Regulatory
Affairs
Cell Product Technology
Lymphocytes
Macrophages
Dendritic Cells
Clinical & Laboratory
Assessments
Basic Research
& Development
Economic
Assessments
ONE Clinical Trial
Cell Therapy
Practicality - Safety - Effect
Proof of Concept
Which cell type is optimal?
Optimal production methods?
A real-world treatment option?
ONE ALLIANCE
The ONE Study consortium is presently made up of
sixteen partners, of which ten are academic institutions
and six are companies that support research. Six countries
are represented: France, Italy, Germany, Switzerland,
the United Kingdom, and the United States of America.
The coordinating centre in Regensburg has extensive
experience in the conduct of large, complex,
international, clinical trials in organ transplantation. All
academic partners are internationally recognised groups
with outstanding scientific backgrounds in the field of
organ transplantation. Each participating partner has
investigators with scientific expertise and the facilities
to manufacture haematopoietic regulatory cells, and
is locally affiliated with an experienced renal
transplant program to test the cell therapy approach
in our ONE Study clinical trial. The companies and SMEs
allied in this study are committed to the advancement
and commercialisation of cell therapy technologies.
ONE STUDY_BROCHUREv3_Layout 1 10/12/12 11:16 Pagina 2
ACADEMIC INSTITUTES:
University of Regensburg
University Hospital Regensburg, Germany
www.uniklinikum-regensburg.de
King's College London, UK
www.kcl.ac.uk
w
University of Oxford, UK
ww.ox.ac.uk
Charité, Germany
www.charite.de
Fondazione Centro San Raffaele, Italy
www.sanraffaele.org
Nantes University Hospital, France
www.chu-nantes.fr
University of Loughborough, UK
www.lboro.ac.uk
University of Wisconsin, USA
www.wisc.edu
University of California, San Francisco, USA
www.ucsf.edu
Massachusetts General Hospital, USA
www.massgeneral.org
COMPANIES:
Miltenyi Biotec GmbH, Germany
www.miltenyibiotec.com
Electro Scientific Industries, UK
www.esi.com
A Unified Approach to Evaluating
Cellular Immunotherapy
in Solid Organ Transplantation
Project Coordinator
Edward K. Geissler, Ph.D.
Professor of Experimental Surgery
University Hospital Regensburg
Department of Surgery
Franz-Josef-Strauss-Allee 11
93053 Regensburg
Germany
[email protected]
Beckman Coulter Life Science, USA
www.beckmancoulter.com
SMEs
clinIT AG, Germany
www.clinit.net
Pharmatching GmbH, Germany
www.pharmatching.com
MANAGEMENT
ALTA Srlu, Italy
www.altaweb.eu
WWW.ONESTUDY.ORG
EU contribution:
A Unified Approach to Evaluating
Cellular Immunotherapy
in Solid Organ Transplantation
Tr1
ONE
Treg
DC
Mreg
FOCUS
OUTCOME
DESIGN
ALLIANCE
10,836,201Euro
Total Costs:
14,833,854 Euro
EUROPEAN
COMMISSION
EUROPEAN
COMMISSION