Projects / Programmes
Repetitive Intramyocardial CD34+ Cell Therapy in Dilated Cardiomyopathy (REMEDIUM)
| Code |
Science |
Field |
Subfield |
| 3.06.00 |
Medical sciences |
Cardiovascular system |
|
| Code |
Science |
Field |
| B530 |
Biomedical sciences |
Cardiovascular system |
| Code |
Science |
Field |
| 3.02 |
Medical and Health Sciences |
Clinical medicine |
Heart failure, Dilated cardiomyopathy, Stem cell therapy
Researchers (12)
Organisations (2)
Abstract
Background. Stem cell therapy appears to represent a safe and effective treatment for patients with heart failure due to dilated cardiomyopathy.
Study Aims. The aim of this study is to investigate whether repetitive stem cell application would be associated with superior clinical benefits when compared to single stem cell application in patients with dilated cardiomyopathy.
Methods. The REMEDIUM trial will consist of a prospective randomized study design. We plan to enroll 80 patients with dilated cardiomyopathy. All patients will undergo stem cell mobilization; thereafter CD34+ cells will be collected via apheresis. Patients will be randomly allocated in 1:1 ratio in repetitive cell administration (Group A) and single administration (Group B). Group A will receive cell therapy at baseline, and again after 6 months; Group B will only receive one dose of cells at baseline. Before cell administration we will perform detailed electroanatomical mapping of the left ventricle, which will enable us to perform regional and global multimodality assessment of viability, contractility, and electrophysiological properties of the myocardium. The total duration of the follow up will be 1 year. At the time of enrollment, and at 6-month intervals thereafter, we will perform detailed clinical evaluation, echocardiography, high-resolution electrocardiography, 6-minute walk test, and measure plasma levels of NT-proBNP. Magnetic resonance imaging (MRI) will be performed in all eligible patients at baseline, 6 months, and 1 year.
Expected results. When compared to single cell administration we expect repetitive cell therapy to be associated with superior effects on myocardial structure and function, laboratory parameters, electro-anatomical parameters, patient exercise capacity and outcome.
Significance for science
The expected impact of REMEDIUM trial will provide important clinical results, which will serve to further define the role of stem cell therapy in the treatment of heart failure and DCM. By combining the clinical and translation aspects, the aim of this trial is to directly implement the existing pre-clinical data into a clinical setting. Futhermore, the experience with already conducted first long-term clinical trial of CD34+ cell therapy in DCM offers a unique advantage over other similar research efforts worldwide, and may serve to significantly improve the international competitiveness of Slovenian regenerative medicine. By combining all of the above, the development of personalized CD34+ cell product that could be stored and used as needed, the REMEDIUM trial could also serve as an important fundament for effective dissemination and exploitation of stem cell therapies.
Significance for the country
The research aspect of REMEDIUM trial will provide important clinical data that could be used to further expand the role of stem cell therapy in patients with DCM. The translational part of the trial will be implemented in close collaboration with the clinical part, and will aim to develop patient-specific CD34+ cell product.
Therefore, the trial also offers a unique business opportunity for both European and global markets through a novel concept of personalized stem cell banking. The aim of this model is to start the initiative to develop a network of stem cell banking facilities, which would serve to store CD34+ cells for patients with different stages of heart failure. If the concept is proven successful, the storage capacity could be further expanded to store CD34+ cells of healthy individuals. This would allow for a unique preventive concept in regenerative medicine: cryopreserved personalized CD34+ cell product of healthy individuals could be stored and used in case of development of heart failure. This would not only allow for treatment of heart failure in earlier stages of the disease, but would also represent a boost for strengthening and growth of companies focusing on stem cell banking and cryopreservation.
Most important scientific results
Interim report,
final report
Most important socioeconomically and culturally relevant results
Final report
