ATMPs are very promising treatments for the severely ill. However, the path leading to their registration and clinical use is long and fraught with difficulties. Through the intense collaboration of its members, EUHA (and EUCCAT) is leading the translation of academia-developed ATMPs into clinical applications. Last week, EUHA colleagues met in Berlin to discuss the progress of the EUHA-driven cross-border clinical trials that aim to address hurdles and create new sustainable models for ATMP development in Europe.
Cell and gene therapies are at the very centre of healthcare innovation and are the fastest-growing area of therapeutics that can induce a paradigm shift in the treatment of diseases including cancer, autoimmune disorders, neurodegenerative disorders, and many rare diseases. The path leading to registration and clinical use of these therapies is long and often fraught with difficulties that compromise the safe, efficient and equitable access to ATMPs (Advanced Therapy Medicinal Products) and threaten the sustainability of such products within EU healthcare systems.
For this reason, in 2019, EUHA started EUCCAT, the European Centre for Gene and Cellular Therapies, to facilitate the clinical use of academia-developed ATMPs and leverage the potential of basic research conducted in Europe. Importantly, the group has initiated a new model for doing decentralised clinical studies and ATMPs manufacturing within EUHA hospitals.
The group, which brings together basic research groups, drug manufacturing facilities, clinical trial capacity, and implementation expertise, has recently met at Charité – Universitätsmedizin Berlin to discuss the progress of the ongoing EUHA-driven clinical trials and the new scope of the group.
New sustainable models for the development of ATMP Trials
EUCCAT is experimenting with new models for ATMPs development through the collaboration between EUHA members based on the principles of cost-sharing, fair reward, and ethical pricing. In 2022, the group started a cross-border decentralised study to treat patients with melanoma and head or neck cancer with their gene-engineered cells.
In Berlin, the group assessed the status and progress of such EUHA-driven trials and discussed the next steps. Experts at Erasmus MC reported how ATMPs have successfully been manufactured in their hospital and patients have been treated there.
To advance cellular therapies and make these therapies better accessible to patients, we need to join forces and test academic trials across borders to identify and address transnational challenges. Reno Debets (Erasmus MC)
To advance cellular therapies and make these therapies better accessible to patients, we need to join forces and test academic trials across borders to identify and address transnational challenges.
As a multi-step trial, patients at AP-HP and Charité – Universitätsmedizin Berlin will also be treated with the ATMPs produced at Erasmus MC. Thus, the trial acts as a lead trial within EUCCAT to address regulatory and logistic hurdles and make therapy more competitive, affordable and accessible in Europe.
EUCCAT expands its scope to non-cancer diseases
New models for clinical development of ATMPs based on collaboration between specialised university hospital centres apply to many diseases. While EUCCAT initially concentrated on cancer, the group had a fruitful discussion and finally opted to broaden its focus to encompass various other medical conditions, with the overarching goal of establishing a sustainable supply of ATMPs across Europe.
ATMP may become game-changers not only for cancer treatment but also for many other diseases. That is why we decided to broaden the scop of EUCCAT. Johan Van Eldere (European University Hospital Alliance)
ATMP may become game-changers not only for cancer treatment but also for many other diseases. That is why we decided to broaden the scop of EUCCAT.
JOIN4ATMP: Advocating broad access to new cell and gene therapies
The group also met to discuss the initiation of JOIN4ATMP, a project recently approved for funding by the European Commission. This EUHA-driven project is the result of the fruitful collaboration of EUHA members within the EUCCAT working group and brings together the specific expertise of all EUHA members and other partners to accelerate the development – and thus, broad-based availability – of innovative ATMPs in Europe.
ATMPs can be better tailored to individual patients than conventional drugs and are particularly suitable for the treatment of rare diseases and diseases that could previously not be treated or could only be treated inadequately. Despite the large number of ATMPs currently in development, only a few products have been approved for the European market due to regulatory and logistic hurdles that prevent the approval of new treatments.
Through JOIN4ATMP, we will make suggestions on how current medical, regulatory and economic obstacles can be overcome and how different requirements can be integrated into individual countries. Annette Künkele (Charité – Universitätsmedizin Berlin)
Through JOIN4ATMP, we will make suggestions on how current medical, regulatory and economic obstacles can be overcome and how different requirements can be integrated into individual countries.
For this reason, this EUHA-driven project, coordinated by Charité – Universitätsmedizin, comes in to help accelerate the development and affordable access to ATMPs in Europe. The consortium operates at the Europe-wide level to bring expertise from university medicine, together with the biotech sector in the preclinical development, production, clinical trials and regulatory approval of ATMPs.
EUHA members and university hospitals play a pivotal role within the consortium and will actively contribute to the sustainability of ATMPs at the European level. Their invaluable expertise, particularly in the development of ATMPs within academic settings and their subsequent translation to clinical applications, enhances the overall strength and impact of the consortium.
The official kick-off the JOIN4ATMP project will take place early 2024.
Now more than ever, it is important to find innovative and sustainable solutions for healthcare systems as they face increasing demands, escalating costs, shortages of personnel, and potential emergencies of the uncertain future. For this reason, it is crucial to build resilient and sustainable healthcare systems that can navigate the uncertainty of the future.
In the field of ATMPs, EUCCAT contributes to this by driving the sustainable development of innovative therapies, emphasising the need to rethink current models, and proposing new strategies based on cross-border collaboration. For this reason, EUCCAT is the perfect example of how university hospitals, through EUHA, can explore joint European efforts to find solutions.