Periodic Reporting for period 2 - MUSIC (Multisystem Cell Therapy for Improvement of Urinary Continence)
Reporting period: 2018-07-01 to 2019-12-31
The main aim of the MUSIC consortium is to translate basic knowledge on cellular therapies and RM into the clinic by undertaking a ""first-in-man"" multisystem study using autologous muscle precursor cells (MPCs) in a combination with neuromuscular electromagnetic stimulation (NMES) in 40 female patients. Throughout the project’s lifetime, we will specifically address safety and efficacy of the proposed novel treatment, as well as reproducibility of the therapeutic effect. Further objectives are the optimization of the advanced-therapy medicinal product (ATMP) towards a manufacturing procedure that is totally free of animal-derived components (xeno-free) and that is facilitated by using a bioreactor. Furthermore, the introduction of a novel injection technique will allow for a more efficient and precise implantation of the final product.
The MUSIC consortium features a unique infrastructure, combining expertise in the fields of RM, urology, cellular biology and biomaterials throughout Europe (CH, NL, UK, A, D). We have an exclusive opportunity to determine the validity of this MPC cellular treatment in combination with NMES and to further develop its practicability and clinical efficacy. Our ultimate goal is to significantly improve the patients’ quality of life and to exploit future commercial opportunities by spreading the know-how to various smaller RM centers and companies within Europe and beyond, thus, making personalized medicine using autologous cells a realistic SUI treatment option.
Due to a fruitful collaboration and successful transfer of know-how within the consortium, we managed to kick-start the development of further objectives of the MUSIC project. The advances in the set-up of a totally xeno-free cell culturing procedure are promising. Fine-tuning for the production settings is currently ongoing. Moreover, the transfer of our current culturing system into a bioreactor-automated setting was successful, allowing a highly efficient cell expansion. Furthermore, with combined efforts, we are developing two injection techniques/tools in parallel, the prototypes of which are already available for pre-clinical testing, in order to optimise the settings for precise and efficient implantation of the final product prior to introduction into a clinical setting.
The new procedure to treat SUI will result in a long- lasting relief for patients with minimal risk of complications. An important goal for the consortium is to develop a procedure that is safe, affordable and minimally invasive for the patients. To optimize the safety of the new cell-based product, we aim to have a production process that is completely free of animal-derived components. Two of our consortium partners are developing components that are either synthetic or human-derived. In addition, to ensure that the new procedure remains affordable, we are also working towards a more efficient way to grow the cells. Automation of the cell culture process will reduce production cost and thereby the overall costs for the therapy, making it reasonably priced for the general patient population. Finally, to minimize patient discomfort, we are optimizing the application method to make it minimally invasive. The developments can be used in other areas as well, so that the impact of the results of this project go beyond the application in SUI. As a result, the MUSIC project will provide a superior treatment for thousands of women (and later on men) with SUI, while the developed technologies will benefit the wider field of cell-based therapies.