Fighting haemophilia A, a bleeding disorder, with the body's own cells: That is the goal of a new international research consortium led by scientists from Würzburg. The EU funds the project with around €5.5 million.
People suffering from haemophilia A have a genetic deficiency in clotting factor VIII which causes increased bleeding. In extreme cases, unstoppable bleeding may occur that can be life-threatening, in particular if the head or brain region is affected. Joint bleeds occur frequently that can cause permanent damage and even destruction of joints when left untreated.
Although effective treatment of the symptoms is available, there is no cure at present. Patients have to get lifelong infusions several times a week to compensate for the missing clotting factor. The costs of treatment for adults vary between €200,000 and 800,000 per year (source: http://www.g-ba.de).
Genetically modified cells produce the clotting factor
An international research consortium is therefore seeking to develop a novel curative therapy for the disease. Their idea: The patients' own cells are genetically modified outside the body to produce the missing clotting factor using precursor cells of endothelial cells flowing in the bloodstream. Subsequently, these cells are transplanted back into the patient's body in a kind of "cell pouch".
Made of plastic, the pouches measure about 6 x 8 centimetres. They are implanted under the abdominal wall and after they have grown into the tissue, the genetically modified cells are filled into the pouches through a kind of valve. Because the pouches are linked to the blood circulation, the cells are capable of continuously producing the clotting factor and releasing it into the bloodstream for a long period of time. This should mitigate the disease's impact noticeably, increase the patients' quality of life and reduce the cost of therapy.
The research consortium
Under the name HemAcure, the new research consortium unites companies and scientific institutions from Germany, Italy, the UK and Canada. The project will be funded with around €5.5 million under the European Union's Horizon 2020 programme for the next three years. It is headed by Dr. Joris Braspenning, a member of the Department of Tissue Engineering and Regenerative Medicine at the University Hospital Würzburg.
"HemAcure brings together experts from science and industry in a powerful coalition," Joris Braspenning explains. Their knowledge and skills will allow cell-based medical products that comply with European regulations to be developed more quickly and efficiently. He believes that the project will not only benefit patients suffering from severe forms of haemophilia A, but also promote advanced therapies of the future.
Heike Walles, Head of the Department of Tissue Engineering and Regenerative Medicine, is also convinced of the research project's value as, in her words, "it ideally complements the department's value chain and the Translational Center for Regenerative Therapies in Würzburg.
The parties involved
Besides the Department of Tissue Engineering and Regenerative Medicine of the Würzburg University Hospital, the following institutions are involved in HemAcure: GABO:mi, a Munich-based enterprise that specialises in the management of EU-funded collaborative research projects, will be in charge of project management. The quality management will be monitored by IMS - Integrierte Management Systeme in Heppenheim, Germany. The company acts as a point of contact for international projects in the pharmaceutical and medical engineering sector.
The Würzburg University Hospital will be responsible for isolating the cells. Moreover, the entire process will be set up in line with GMP (Good Manufacturing Practice) guidelines for the production of pharmaceutical products.
The Università del Piemonte Orientale (Italy) will perform the gene correction of the patient cells. Scientists from Loughborough University (UK) will focus on the manufacturing process and safety testing. Sernova, a Canadian company, will supply the "cell pouches" for implanting the therapeutic cells.
The Horizon 2020 programme
Horizon 2020 is the biggest EU research and innovation programme ever with nearly €80 billion of funding available over seven years (2014 to 2020). It promises more breakthroughs, discoveries and world firsts by taking great ideas from the lab to the market, for example in the field of personalised medicine providing novel therapies such as gene or cell therapy.
Dr. Joris Braspenning, Project Manager HemAcure & Business Development Manager
Phone: +49 931 31-88598, firstname.lastname@example.org
Gunnar Bartsch | idw - Informationsdienst Wissenschaft
Tracking down pest control strategies
31.01.2018 | Technische Universität Dresden
Polymers and Fuels from Renewable Resources
29.01.2018 | DECHEMA Gesellschaft für Chemische Technik und Biotechnologie e.V.
A newly developed laser technology has enabled physicists in the Laboratory for Attosecond Physics (jointly run by LMU Munich and the Max Planck Institute of Quantum Optics) to generate attosecond bursts of high-energy photons of unprecedented intensity. This has made it possible to observe the interaction of multiple photons in a single such pulse with electrons in the inner orbital shell of an atom.
In order to observe the ultrafast electron motion in the inner shells of atoms with short light pulses, the pulses must not only be ultrashort, but very...
A group of researchers led by Andrea Cavalleri at the Max Planck Institute for Structure and Dynamics of Matter (MPSD) in Hamburg has demonstrated a new method enabling precise measurements of the interatomic forces that hold crystalline solids together. The paper Probing the Interatomic Potential of Solids by Strong-Field Nonlinear Phononics, published online in Nature, explains how a terahertz-frequency laser pulse can drive very large deformations of the crystal.
By measuring the highly unusual atomic trajectories under extreme electromagnetic transients, the MPSD group could reconstruct how rigid the atomic bonds are...
Quantum computers may one day solve algorithmic problems which even the biggest supercomputers today can’t manage. But how do you test a quantum computer to...
For the first time, a team of researchers at the Max-Planck Institute (MPI) for Polymer Research in Mainz, Germany, has succeeded in making an integrated circuit (IC) from just a monolayer of a semiconducting polymer via a bottom-up, self-assembly approach.
In the self-assembly process, the semiconducting polymer arranges itself into an ordered monolayer in a transistor. The transistors are binary switches used...
Breakthrough provides a new concept of the design of molecular motors, sensors and electricity generators at nanoscale
Researchers from the Institute of Organic Chemistry and Biochemistry of the CAS (IOCB Prague), Institute of Physics of the CAS (IP CAS) and Palacký University...
15.02.2018 | Event News
13.02.2018 | Event News
12.02.2018 | Event News
23.02.2018 | Physics and Astronomy
23.02.2018 | Health and Medicine
23.02.2018 | Physics and Astronomy