Periodic Reporting for period 4 - SCIENCE (Stem Cell therapy in IschEmic Non-treatable Cardiac diseasE (SCIENCE))
Okres sprawozdawczy: 2019-07-01 do 2020-12-31
Ischemic heart disease (IHD) caused by coronary artery disease is the most common cause of death with more than 17 million deaths worldwide each year and a major cause of hospital admissions in industrialized countries. It is an increasing economic health problem due to increasing morbidity in an ageing population. Classical therapies have reduced mortality of IHD significantly but left an increasing number of patients with chronic IHD and/or heart failure without further treatment options.
There is an unmet need for novel, effective treatments for chronic IHD and heart failure to improve patient’s survival and quality of life and reduce health care costs. Stem cell therapy is emerging as a viable therapeutic option in this patient group as well as in several debilitating diseases for which no cure is currently available.
The overall aim of the SCIENCE project was
• to conduct a multi-center double-blind placebo-controlled trial with stem cells from healthy donors to improve myocardial function in patients with ischemic heart disease and heart failure.
• establishing a manufacture facility for centralized production of the stem cell product in Copenhagen, Denmark.
• establishing financial health care data to demonstrate the impact of implementing stem cell treatment
• evaluating advanced existing and new imaging techniques of the function of the heart in order to establish future more precisely guided delivery of stem cells
There is an unmet need for novel, effective treatments for chronic IHD and heart failure to improve patient’s survival and quality of life and reduce health care costs. Stem cell therapy is emerging as a viable therapeutic option in this patient group as well as in several debilitating diseases for which no cure is currently available.
The overall aim of the SCIENCE project was
• to conduct a multi-center double-blind placebo-controlled trial with stem cells from healthy donors to improve myocardial function in patients with ischemic heart disease and heart failure.
• establishing a manufacture facility for centralized production of the stem cell product in Copenhagen, Denmark.
• establishing financial health care data to demonstrate the impact of implementing stem cell treatment
• evaluating advanced existing and new imaging techniques of the function of the heart in order to establish future more precisely guided delivery of stem cells
Within the 72 months grant period the consortium has established a centralized production of mesenchymal stromal cells from adipose tissue from healthy donors for the clinical SCIENCE trial in Copenhagen, Denmark.
The approval and initiation of the clinical project was a little delayed due to new regulation from The European Medicinal Agency for some of the component used in the stem cell production, which was released late 2015 to be implemented in EU 2017. The new regulation was released after our submission of the project to the competent authorities for an approval. We therefore had to establish a completely new production of stem cell for the clinical trial based on the upcoming new regulation and initiate a new approval process. Then the new production plan was approved by the competent authorities in the involved countries and the new production of the stem cell product was initiated.
The national Competent Authority approval process was delayed in some countries. The last approval was in Poland August 2017.
The first patient was treated January 2017. A total of 133 patients have been treated and followed for at least 12 months without any complications or adverse events caused by the stem cell product in the six involved clinical departments in Slovenia, Germany, Austria, Poland, The Netherlands and Denmark.
The trial and each site have been monitored by QMED Consulting A/S, which is a certified Contract Research Organisation (CRO).
All clinical trial data have been collected in a central database. The data have been analysed for safety and efficacy of the stem cell therapy in patients with heart failure Q2 2022.
The imaging data of the heart function have been collected from all sites and they echocardiographic investigations have been analysed by two independent groups to ensure maximum data quality and validity. The analyses were finalized Q2 2022.
The SCIENCE trial demonstrated that it was safe to treat patients with ischemic heart failure with direct injection of stem cells into the heart muscle.
The efficacy and 3-years long-term safety data have been submitted for publication June 2022.
A cost-effectiveness analysis was developed to estimate the cost of the intervention and relate this to the improvement in health resulting from the ASC treatment intervention.
The data of the patients from the SCIENCE trial made available for this cost-effectiveness analysis found was used in the developed cost-effective analysis.
The results of the analyze will be submitted for publication 2022.
The approval and initiation of the clinical project was a little delayed due to new regulation from The European Medicinal Agency for some of the component used in the stem cell production, which was released late 2015 to be implemented in EU 2017. The new regulation was released after our submission of the project to the competent authorities for an approval. We therefore had to establish a completely new production of stem cell for the clinical trial based on the upcoming new regulation and initiate a new approval process. Then the new production plan was approved by the competent authorities in the involved countries and the new production of the stem cell product was initiated.
The national Competent Authority approval process was delayed in some countries. The last approval was in Poland August 2017.
The first patient was treated January 2017. A total of 133 patients have been treated and followed for at least 12 months without any complications or adverse events caused by the stem cell product in the six involved clinical departments in Slovenia, Germany, Austria, Poland, The Netherlands and Denmark.
The trial and each site have been monitored by QMED Consulting A/S, which is a certified Contract Research Organisation (CRO).
All clinical trial data have been collected in a central database. The data have been analysed for safety and efficacy of the stem cell therapy in patients with heart failure Q2 2022.
The imaging data of the heart function have been collected from all sites and they echocardiographic investigations have been analysed by two independent groups to ensure maximum data quality and validity. The analyses were finalized Q2 2022.
The SCIENCE trial demonstrated that it was safe to treat patients with ischemic heart failure with direct injection of stem cells into the heart muscle.
The efficacy and 3-years long-term safety data have been submitted for publication June 2022.
A cost-effectiveness analysis was developed to estimate the cost of the intervention and relate this to the improvement in health resulting from the ASC treatment intervention.
The data of the patients from the SCIENCE trial made available for this cost-effectiveness analysis found was used in the developed cost-effective analysis.
The results of the analyze will be submitted for publication 2022.
The SCIENCE has demonstrated that it is possible to establish an industrialized centralized production of an allogeneic adipose-derived stem cell product stored in nitrogen in small vials ready to be used as an off-the-shelf product without any delay. Moreover, the study has demonstrated that the drug can be distributed without any problems to end-users of the drug in any country in Europe.
The treatment with the stem cell product has proven safety.
The clinical efficacy and 3-years safety data for the treatment of patients with ischemic heart failure with the allogeneic adipose tissue derived mesenchymal stromal cell product will have a great impact for the future planning of cell therapy in patients with heart failure and in patients with other diseases with a need for new treatment options.
Moreover, it is rather unique, that the SCIENCE trial also has collected health economic data for a European clinical trial. These data will have a great value in the next phase of implementing stem cell clinically.
The treatment with the stem cell product has proven safety.
The clinical efficacy and 3-years safety data for the treatment of patients with ischemic heart failure with the allogeneic adipose tissue derived mesenchymal stromal cell product will have a great impact for the future planning of cell therapy in patients with heart failure and in patients with other diseases with a need for new treatment options.
Moreover, it is rather unique, that the SCIENCE trial also has collected health economic data for a European clinical trial. These data will have a great value in the next phase of implementing stem cell clinically.