STEMBANCC

Stem cells for biological assays of novel drugs and predictive toxicology
Banking on human iPS cells

FACTS & FIGURES

Start Date
End Date
Call
IMI1 - Call 4
Grant agreement number
115439

Contributions
IMI Funding
26 000 000
EFPIA in kind
20 761 386
Other
8 841 407
Total Cost55 602 793

Summary

The aim of the STEMBANCC project is to generate and characterise 1 500 high quality human induced pluripotent stem (iPS) cell lines that can be used by researchers to study a range of diseases, including diabetes and dementia, and test for drug efficacy and safety. The cell lines will help to improve and speed up the drug development process, and ensure that patients benefit from more effective and safer drugs.

Currently, many drugs fail rather late in the drug development process because the tests used in the earlier stages of drug development simply do not reflect what happens in real life when the drug is administered in patients. 

This is partly because these early tests rely heavily on animal cells, and when human cells are used, they have often been extensively modified to survive in culture and so no longer behave naturally. 

Those working in drug research and development therefore urgently need a good supply of cells that more accurately mimic what happens in the human body.

The power of pluripotency

Most adult cells can only divide to produce other cells of the same type – for example, skin cells can only make other skin cells, and blood cells can only make other blood cells. Only embryonic stem cells are ‘pluripotent’, i.e. able to give rise to all the different kinds of cell that make up the human body. However, in recent years researchers have developed a way of reprogramming ordinary adult cells to create so-called induced pluripotent stem (iPS) cells. Like embryonic stem cells, iPS cells are able to generate any kind of cell; as such, they offer researchers a good supply of different kinds of human cell that can be used in research and drug development. 

The research resulting in the creation of the first iPS cells was a major scientific breakthrough that won scientists John Gurdon and Shinya Yamanaka the 2012 Nobel Prize in Physiology or Medicine.

A unique resource

STEMBANCC’s goal is to generate 1 500 iPS cell lines from 500 people, characterise them in terms of their genetic, protein, and metabolic profiles, and make them available to researchers. All cell lines will also undergo a rigorous quality check. 

The raw materials for the project will be largely skin and blood samples taken from patients with certain diseases, people who display adverse reactions to drugs, and healthy individuals. The collection of these samples will be carried out with the individuals’ informed consent and in line with strict ethical standards. 

There will be a strong focus on peripheral nervous system disorders (especially pain); central nervous system disorders (e.g. dementias); neurodysfunctional diseases (e.g. migraine, autism, schizophrenia, and bipolar disorder); and diabetes. The project will also investigate the use of human iPS cells for toxicology testing; here the team will use the iPS cells to generate liver, heart, nerve and kidney cells. 

Ultimately STEMBANCC will be a source of well-characterised, patient-derived iPS cells that will help researchers study diseases, develop new treatments, and test the efficacy and safety of new drugs.

Achievements & News

StemBANCC and EBiSC contribute to the understanding of a rare pain disease
One of the biggest challenges in drug development is finding ways to translate results from early laboratory tests to human patients. Pluripotent stem cells derived from human patients might hold the key to this translational puzzle, and a new IMI study confirms this potential.### In a unique collaboration, both IMI’s EBiSCand StemBANCC project teams contributed to a study led by one of their industrial project partners, Pfizer. The study set out to improve the understanding of a rare pain disease called inherited erythromelalgia (IEM). IEM is a chronic condition with no adequate medical treatment, in which patients suffer from extreme pain that is made worse by heat. The disease is caused by a mutation in a gene leading to overactivity of a sodium channel in patients’ sensory neurons. Pfizer scientists examined if a new experimental drug could block this sodium channel and reduce the pain experienced by IEM patients. When tested on five patients, results indicated that the drug reduced pain upon exposure to heat in most sufferers. However, the experiment didn’t end there. The same patients allowed scientists to take their blood cells and transform them into pluripotent stem cells, which were deposited in the EBiSC project stem cell bank. Next, the StemBANCC scientists turned some of those stem cells into sensory neurons and studied their characteristics in a lab. Prior to treatment, the neurons showed hyper-excitability and an unusual response to heat: the more severe the disease was in a patient, the more the neurons became hyper-excited. When the same neurons were treated with the experimental drug, the hyper-excitability diminished, mimicking the drug effect that occurred in patients. 'This is an exciting study,' said Andrea Weston, Pfizer’s scientific lead for StemBANCC and EBiSC projects. 'Rarely is it possible for drug developers to be able to study the drug responses of the actual, relevant cells from individual patients who are involved in clinical trials of the same experimental drug. These results illustrate that stem cell technology has the potential to transform the way in which drugs are made, enabling scientists to make them much more precise and tailored to individual patients. This is also a good example of how technology can bridge the translational gap between preclinical models and clinical evaluation. The study was facilitated by the collaborative nature of IMI’s projects, and wouldn’t have been possible without the open exchange of knowledge and ideas between different project partners.’ (February 2017)

STEMBANCC recruitment on track
IMI’s stem cell project StemBANCC is advancing well with the recruitment of patients and control individuals (subjects). So far, around 50 subjects have been recruited to disease cohorts such as Alzheimer’s disease, neuropathy, ###diabetes, Parkinson’s disease, migraine and bipolar disorder by several project participants. The sites recruiting for the autism and schizophrenia cohorts expect to have their first patients in soon.
StemBANCC aims to generate and characterise 1 500 high quality human induced pluripotent stem (iPS) cell lines from 500 people that can be used by researchers to study a range of diseases and test for drug efficacy and safety. The raw materials for the project are largely skin samples taken from patients with certain diseases, people who display adverse reactions to drugs, and healthy individuals. The collection of these samples is carried out with the individuals’ informed consent and in line with strict ethical standards.
The recruitment of subjects began following the full ethics approval by the UK’s main research ethics committee in 2013. The University of Oxford, which is also the managing entity of the StemBANCC project, is responsible for recruiting more than half of the required target of 500 patient and healthy volunteers.
   -   Learn more about the StemBANCC project
(February 2014)

Participants Show participants on map

EFPIA companies
  • AbbVie Deutschland GmbH & Co. KG, Wiesbaden, Germany
  • AstraZeneca AB, Södertälje, Sweden
  • Boehringer Ingelheim International GmbH, Ingelheim, Germany
  • Eli Lilly and Company Ltd, Basingstoke, United Kingdom
  • F. Hoffmann-La Roche Ltd, Basel, Switzerland
  • Janssen Pharmaceutica NV, Beerse, Belgium
  • Merck KGaA, Darmstadt, Germany
  • Novo Nordisk A/S, Bagsvaerd, Denmark
  • Orion Corporation, Espoo, Finland
  • Pfizer Limited, Sandwich, Kent , United Kingdom
  • Sanofi-Aventis Research and Development, Chilly Mazarin, France
Universities, research organisations, public bodies, non-profit groups
  • Charité – Universitätsmedizin Berlin, Berlin, Germany
  • Helmholtz Zentrum Muenchen, Deutsches Forschungszentrum fuer Gesundheit und Umwelt (GmbH), Neuherberg, Germany
  • Institut National De L'Environnement Industriel Et Des Risques, Verneuil En Halatte, France
  • Institut National De La Sante Et De La Recherche Medicale, Paris, France
  • King's College London, London, United Kingdom
  • Linkopings Universitet, Linkoping, Sweden
  • Medizinische Hochschule Hannover, Hannover, Germany
  • Medizinische Universität Innsbruck, Innsbruck, Austria
  • Naturwissenschaftliches und Medizinisches Institut an der Universität Tübingen, Reutlingen, Germany
  • Region Hovedstaden, Hilleroed, Denmark
  • Tel Aviv University, Tel Aviv, Israel
  • The Hebrew University of Jerusalem, Jerusalem, Israel
  • Universite De Technologie De Compiegne, Compiegne, France
  • University College London, London, United Kingdom
  • University of Birmingham, Birmingham, United Kingdom
  • University of Cambridge, Cambridge, United Kingdom
  • University of Edinburgh, Edinburgh, United Kingdom
  • University of Luebeck, Luebeck, Germany
  • University of Oxford, Oxford, United Kingdom
  • Université de Genève, Genève 4, Switzerland
  • Université de Lausanne, Lausanne, Switzerland
  • Univesity of Newcastle upon Tyne, Newcastle upon Tyne, United Kingdom
Small and medium-sized enterprises (SMEs)
  • Concentris Research Management GmbH, Fürstenfeldbruck, Germany
  • Newcells Biotech Limited, Newcastle upon Tyne, United Kingdom
  • Univercell-Biosolutions, Toulouse, France
Third parties
  • Universitätsklinikum Schleswig-Holstein, Lübeck, Germany

CONTACT

Project coordinator
Martin Graf
F. HOFFMANN-LA ROCHE LTD.
+41 61 688 92 46
martin.graf[at]roche.com
Managing entity
Zameel Cader
The Chancellor, Masters and Scholars of the University of Oxford
zameel.cader[at]ndcn.ox.ac.uk