APPROACH
Project Management and Coordination
UCL will be in charge of the scientific, contractual and financial management of the project. This stage of the project will ensure proper quality and timeliness of project progress and results, address risk planning and management as well as considering ethical criteria and gender issues.
Cell-microcarrier manufacturing process
Led by IST, this will establish a robust, consistent and scalable GMP compliant procedure for attachment of human SMDC to TIPS microcarriers. This standardised procedure will be verified by UCL before being transferred to NHSBT, where the process will be validated in readiness for manufacture of the cell-microcarrier combination.
Manufacture of clinically ready TIPS microcarriers
The microcarriers will be manufactured at pilot scale. UCL will oversee confirmation that the microcarriers meet specifications. Stability studies will provide data on the long-term stability of the implantable cell-microcarrier combination. Critical evaluation of the manufacturing process at this stage will determine the feasibility of scaling-up the process for commercial quantities.
Clinical trial plan and regulatory approval
Regulatory and Ethical approvals will be sought in the set-up phase of the trial. Each country will have all relevant approvals in place prior to recruitment and will be overseen by UCL. The clinical trial will test whether ASMDC-microcarrier implantation leads to a minimum clinically relevant reduction in frequency of total FI episodes. Patients, carers and the public will be involved in the delivery of the study and the dissemination of results.
Non-clinical safety studies required for regulatory approval
The expected biological behaviour of the cellular component in the finished product will be demonstrated together with the local tolerance, persistence and toxicological properties of the cell-microcarrier combination in order to verify its safety.
Verification of product stability
A robust and consistent process for preparing the finished product in a state suitable for storage and transportation will be designed and validated.
Dissemination and Stakeholder Engagement
Bowel Research UK and TT will work together with all AMELIE beneficiaries to engage with business and industrial beneficiaries, healthcare professionals, patients and the public, charities and the third sector, and academics.
Knowledge Management and Exploitation
This package will seek to understand the merit and basis of a business case for the application of the implantable cell-microcarrier technology in treating FI and explore potential other markets where the technology could also be effectively used.
Lesley Booth
MBE, Director of Research and PPIE at Bowel Research UK
“The reality of living with faecal incontinence can be devastating. It is definitely life-limiting. And the fact that it’s a condition which people don’t discuss freely just adds to the difficulties of leading a normal life. Finding a therapy which works would not just be a major medical breakthrough, it would be life-changing for millions.”
Richard Day
Professor of Regenerative Medicine Technology at University College London
“We are tremendously excited by the prospect of the AMELIE project and the potential benefits that may arise from the new therapeutic approach being investigated.
We look forward to working with the exceptional consortium of academics, clinicians, industry and charity partners from across Europe to develop our pioneering regenerative medicine approach for treating this debilitating condition. This will be a radical and innovative approach never before attempted at such a scale.”