Coronary artery disease is responsible for 40% of all deaths in the European Union, and is the leading cause of death in both women and men. Total costs in connection with coronary artery disease are approaching a staggering EUR 196 bn. or almost DKK 1.5 trill. annually in the European Union alone. Hopefully, in the next few years these statistics will be changed by the stem-cell treatment of heart failure, developed in the Capital Region of Denmark using the investment from Innovation Fund Denmark.
"Even though we have come far with current treatment methods that have considerably reduced death rates as well as inconvenience and discomfort for patients, an increasing number of patients are still having to cope with severe discomfort from chronic coronary artery disease for which treatment options have been exhausted. There is a large, unsatisfied need for new and effective treatments like this that can increase survival rates, reduce patient discomfort, increase quality of life and employment as well as reduce costs for the health service," said Professor Jens Kastrup, Consultant and Head of the Cardiology Stem Cell Center at Rigshospitalet.
Cooperation with businesses
At Rigshospitalet, for many years Jens Kastrup and his colleagues have been treating patients suffering from coronary artery disease with patients' own stem cells from their bone marrow and fatty tissue from the stomach, and the treatment results have been good. However, stem cells only exist in very small quantities in tissue, and therefore they must be cultured in small bottles for six to eight weeks before they can be used in treatment. This is a slow process which has called for innovative thinking.
"In order to promote more widespread use of stem-cell treatment, using the latest technology to culture stem cells in bioreactors, we have now established stem-cell production from healthy donors. The finished cell product is stored frozen until the patient is ready to undergo treatment. The cell product can be defrosted in only five minutes. This opens for completely new possibilities," said Jens Kastrup.
The grant from Innovation Fund Denmark will enable Rigshospitalet to complete two Phase II trials and initiate extensive preparation for the Phase III trial which is to finally document the effect of stem-cell treatment and pave the way for approving it for general patient treatment.
"Strong cooperation with partners from the business community will bring production of Danish stem cells to the forefront and to the market, and ultimately improve treatment and quality of life for patients suffering from chronic heart disease," said Jens Kastrup.
"Developing and culturing the stem-cell line is a difficult balance in itself, particularly when legislation and guidelines for stem-cell production are constantly changing. We also want to take the next step and disseminate our knowhow in patient treatment, and therefore we need to be willing and ready to change, convert production and we need a pioneering spirit. Sometimes it feels just like we're driving a train at full steam ahead while the tracks are being laid in front of us. Sometimes we have to stop and relay the tracks before we can continue," said Jens Kastrup and concluded:
"The good results we have achieved in the Phase I and II trials strengthen my confidence that, with this new grant, we will be ready to take the last step to bring stem-cell production from the laboratory to the patient, and we will do this together with our partners."
The vision of the Cardiology Stem Cell Center is to establish stem-cell treatment as a general option for patients suffering from severe coronary artery disease and failure of the heart's pumping function. The grant from Innovation Fund Denmark is to be used to develop stem-cell treatment and to gather the documentation required by the European Medicines Agency for a new drug to be approved for general patient treatment. Moreover, clinical treatment studies will be conducted of patients undergoing treatment with the new stem-cell product. If these results turn out to be as good as results using patients' own stem cells, an application will submitted for authorisation to market the stem-cell product from healthy donors for general treatment of patients suffering from heart disease.
Facts: The three clinical trial phases
Before a new treatment can be taken into use, it must go through a number of clinical trial phases.
Phase I: Researchers test the new treatment method on a small group of people, typically between 20 and 80. The effects of the treatment on the body are tested, e.g. whether the treatment has any side-effects. Phase I may also include testing the treatment on animals. Phase I usually lasts between one and 1½ years.
Phase II: Researchers expose a larger group of patients to the treatment, typically between 20 and 300. Phase II studies long-term effects of the treatment and whether the treatment has different effects on different ethnic groups. For instance, whether Asian, Caucasian and African people react similarly to the treatment. Phase II lasts two-three years.
Phase III: The new treatment method is given to 2,000-4,000 patients. This phase places stricter requirements on who can receive treatment, e.g. so that the treatment is not influenced by a patient suffering from other diseases. In this phase, authorities should be able to get an idea of what the treatment will cost and whether it will be worth implementing. Phase III lasts three-four years.
Investment by Innovation Fund Denmark: DKK 25 mill.
Total budget: DKK 37 mill.
Duration: five years
Official title: Cardiology Stem Cell Center
About the partners
The Cardiology Stem Cell Center, Rigshospitalet, have conducted research for many years in establishing stem-cell treatment as a new option for heart patients.
Bioneer A/S is an expert in developing biomedicine and biotechnology.
Cook Regentec develops products for stem-cell treatment and will establish a business in Bjæverskov, south of Copenhagen, to produce a blood platelet product to culture stem cells.
Terumo BCT has developed the first approved automatic bioreactor to culture stem cells to treat of patients.