Report
Teaser, summary, work performed and final results
Periodic Reporting for period 1 - OPTOGENERAPY (Optogenetic Protein Therapy for Multiple Sclerosis)
Teaser
Optogenerapy - Optogenetic Protein Therapy for Multiple Sclerosis- aims to develop and demonstrate a new optogenetics implant for controlled beta interferon (IFN-ß) protein delivery for treating Multiple Sclerosis patients, in a 3 years project funded by the European...
Summary
Optogenerapy - Optogenetic Protein Therapy for Multiple Sclerosis- aims to develop and demonstrate a new optogenetics implant for controlled beta interferon (IFN-ß) protein delivery for treating Multiple Sclerosis patients, in a 3 years project funded by the European Commission within the Horizon 2020 Programme.
Optogenerapy represents an innovative and effective therapeutic delivery with an impact on slowing the disease progression and increasing the Multiple Sclerosis patients’ quality of life. The novelty lies in the continuous delivery of IFN-ß, overcoming current limitations of short drug half-life in vivo, adverse immune reactions, and pain and irritation at the site of local injection.
The main objective is to develop and validate a new bio-electronic cell based implant device to be implanted subcutaneously providing controlled drug release during at least 6 months. The cell confinement within a chamber sealed by a porous membrane allows the device to be easily implanted or removed. At the same time, this membrane acts to prevent immune rejection and offers long-term safety in drug release while overcoming the adverse effects of current cellular therapies.
Wireless powered optogenetics – light controlling the cellular response of genetically engineered cells – is used to control the production of IFN-ß.
It is a low-cost system enabling large scale manufacturing and reduction of time to market up to 30% compared to other cell therapies, combining:
• Polymeric biomaterials with strong optical, biocompatibility and barrier requirements, to build the cell chamber and to encapsulate the optoelectronics.
• Optoelectronics miniaturization, autonomy and optical performance.
• Optimal cellular engineering design, enhanced by computer modelling, for stability and performance of the synthetic optogenetic gene pathway over long-term implantation.
• Micro moulding enabling optoelectronics and membrane embedding for safety and minimal invasiveness.
Optogenerapy socio-economic impact:
Impact for the patients:
• Improved drug effectiveness: The novel solution for IFN-β continuous systemic release will improve quality of life of patients with Multiple Sclerosis due to improved drug effectiveness as eliminates the serum levels peaks, the flu-like effects after injection, and liver toxicity in the long term.
• Improved compliance with drug taking: The Optogenerapy solution will also improve patients’ treatment adherence as there is no need for daily to weekly self-injections. Patients would only need go to the doctor for follow-up, which helps preventing patients to stop their medications by therapy fatigue and delaying disease progression.
Impact for the Healthcare System:
• Reduce direct and indirect costs linked to Multiple Sclerosis: Optogenerapy therapy deployment will save the costs of non-adherence to the healthcare system and to the society, mainly related to labour market productivity losses.
Impact on Society
• Social awareness of efficient therapies improving patient compliance: Optogenerapy outputs will contribute to increase the social awareness about more efficient therapies with improved patient compliance thanks to the project’s highly innovative concept and the real interest and capability to persist during the exploitation strategies once the project ends.
• On the other hand, the project will help to generate awareness about Multiple Sclerosis and its prevention measures, treatment options and adherence benefits.
Work performed
The design and the development of the Optogenerapy implant including the selection of materials and components to comply with the mechanical, optoelectronics, wireless, biocompatibility, and minimal invasiveness requirements of the new device have been set. After the initial phase of the implant technological development the implant requirement specifications were transferred into the device manufacturing process.
Engineering process for integrating the pathway is defined and NIR-Photoactivable cell lines were constructed. A preliminary pathway model describing network behaviour has been developed and it will be refined with additional data. An Optogenerapy clinical trial design will be delivered at the end of the project.
Different iterations on the tooling and manufacturing process adjustments have led to the manufacturing of implant prototypes for validation purposes. Further refinements in terms of functionality will be carried out in the following months to improve performance. The most suitable sterilization methods have been selected and testing on some implant parts has started showing positive results.
Concerning quality of life, a systematic literature review regarding patient preferences in treatments has been completed and a scientific publication is under preparation. A cross-sectional survey to be used on MS patients was also designed, with the scope of the survey and the measures already defined. Data collection for the patient survey will start in late September 2018/ early October 2018.
A cost-effectiveness analysis is on-going, with a systematic review completed and the design of the decision model in progress. The physician interviews have commenced.
The first draft for regulatory submission has been completed and the consortium is on the right track in terms of the regulatory strategy/plan. The necessary standards and regulatory requirements affecting the different developments of the project (GMP cell manufacturing, instrumentation…) have been identified and will be closely monitored.
Final results
The main expected results include:
• Optogenerapy implant prototypes
• Flexible optoelectronics prototypes
• Therapeutic cell lines
• Computer modelling of the optogenetic pathway
• Optical design
• Micro-injection moulding process
• Implant sterilisation protocol
• Surgical instruments prototypes
• Standardisation studies
• Pre-clinical regulatory studies
• Disability and Quality of life impact assessment
Website & more info
More info: http://www.optogenerapy.eu.