It is time we got more personal with advanced therapies- How do we create the right ecosystem for more effective ATMP development in Europe?

Main Article Content

E. Oldoni A. Ussi A. L. Andreu D. Morrow

Abstract

Over the past few years, Advanced Therapy Medicinal Products (ATMPs), especially cell and gene therapies, have brought about a remarkable transformation in the field of therapeutics. ATMPs have the potential to be tailored to individual patients based on their distinct molecular characteristics, making them a crucial aspect of personalized medicine (PM) strategies. Unlocking the full potential of ATMPs is crucial for them to become the treatments of the future. Despite their immense promise, their success is hindered by significant complexity, as evidenced by various systemic bottlenecks in the realms of science, clinical implementation, and regulation. Presently, ATMPs face challenges such as a limited understanding and predictability of in vivo cell fate specific to each patient, regulatory issues caused by rapid technological advancements, inadequate standardization in data acquisition, limited reproducibility during preclinical development, and insufficient knowledge exchange among key stakeholders. Addressing these aspects is essential to fully harness the benefits of ATMPs in healthcare. EATRIS, the European Research Infrastructure for Translational Medicine, is actively enhancing its capabilities in the field of PM through a series of key initiatives. These efforts aim to support also ATMP development and are focused on delivering novel and innovative scientific tools for the scientific community. The final aim is to create the right ecosystem for more effective ATMP development in Europe, by better serving academia and industry in the translation of ATMPs for patient benefit.

Keywords: advanced therapies, ecosystem for more effective ATMP, effective ATMP development in Europe

Article Details

How to Cite
OLDONI, E. et al. It is time we got more personal with advanced therapies- How do we create the right ecosystem for more effective ATMP development in Europe?. Medical Research Archives, [S.l.], v. 11, n. 9, sep. 2023. ISSN 2375-1924. Available at: <https://esmed.org/MRA/mra/article/view/4322>. Date accessed: 15 may 2024. doi: https://doi.org/10.18103/mra.v11i9.4322.
Section
Research Articles

References

1. van Dongen GA, et al. EATRIS, a European initiative to boost translational biomedical research. Am J Nucl Med Mol Imaging. 2013; 3: 166-174. www.ajnmmi.us /ISSN:2160-8407 /ajnmmi1301007

2. Gilliland CT, et al. Putting translational science on to a global stage. Nat. Rev. Drug Discov. 2016; 15:217–8. doi:10.1038/nrd. 2016.33

3. Ussi AE, et al. In Search of System-Wide Productivity Gains - The Role of Global Collaborations in Preclinical Translation. Clin. Transl. Sci. 2017; 10, 423–425. doi:10.1111 /cts.12498

4. Morrow D, et al. EATRIS: Providing the right tools, at the right time, for vaccine development in a pandemic. Vaccine Insights. 2022. 1(3), 131–137. doi: 10.18609/vac.2022. 022

5. Oldoni E, et al. European research infrastructure join forces to provide innovative cancer research services across Europe- how can they support the cell and gene therapy develop? Cell & Gene Therapy Insights. 2023; 9(7), 1003–1008. doi: 10.18609/cgti.2023.127

6. Oldoni E, et al. Tackling the translational challenges of multi-omics research in the realm of European personalised medicine: A workshop report. Front. Mol. Biosci. 2022; eCollection 2022. doi.org/10.3389/fmolb. 2022.974799

7. Fosse V, et al. Recommendations for robust and reproducible preclinical research in personalised medicine. BMC Med. 2023; 21(1): 14. doi: 10.1186/s12916-022-02719-0.

8. Marshall D, Sharpe M, Ward S. Cell & gene therapies, and the evolving role of personalized medicine. Cell Gene Therapy Insights. 2016; 2(2), 277-286. doi:10.18609/ cgti.2016.034

9. Koshkina O , Lajoinie G, Bombelli F. Multicore Liquid Perfluorocarbon-Loaded Multimodal Nanoparticles for Stable Ultrasound and 19F MRI Applied to In Vivo Cell Tracking. Adv Funct Mater. 2019; 29(19): 1806485. doi: 10.1002/adfm.201806485

10. Srinivas M, Mann C, Andreu AL, Ussi A, Morrow D. Broadly applicable imaging platforms are necessary for optimizing cell therapies in solid tumors. Cell & Gene Therapy Insights. 2019; 5(7), 629-638. doi:10. 18609/cgti.2019.071

11. Helfer BM, et al. Options for imaging cellular therapeutics in vivo: a multi-stakeholder perspective. Cytotherapy. 2021; 23(9): 757–773. doi: 10.1016/j.jcyt.2021.02. 005

12. Chapelin F, et al. Fluorine-19 MRI for detection and quantification of immune cell therapy for cancer. J Immunother Cancer. 2018; 6. 105. doi: 10.1186/s40425-018-0416-9

13. Ahrens ET, et al.Tracking immune cells in vivo using magnetic resonance imaging. Nat Rev Immunol. 2013;13:755–63. doi: 10.1038/ nri3531

14. Frangioni JV, et al. In vivo tracking of stem cells for clinical trials in cardiovascular disease. Circulation. 2004; 110:3378–83. doi: 10.1161/01.CIR.0000149840.46523.FC

15. Ashmore-Harris C, et al. Non-invasive reporter gene imaging of cell therapies, including T cells and stem cells. Mol Ther. 2020; 28:1392–416. doi: 10.1016/j.ymthe. 2020.03.016

16. Morris EC, et al. Cytokine release syndrome and associated neurotoxicity in cancer immunotherapy. Nat Rev Immunol. 2022; 22:85–96. doi.org/10.1038/ s41577-021-00547-6

17. Balagopal S, et al. Emerging approaches for preventing cytokine release syndrome in CAR-T cell therapy. J. Mater. Chem. B. 2022; 10:7491-751. doi: 10.1039/D2TB00592A

18. Cosenza M, Sacchi S, and Pozzi S. Cytokine Release Syndrome Associated with T-Cell-Based Therapies for Hematological Malignancies: Pathophysiology, Clinical Presentation, and Treatment. Int J Mol Sci. 2021; 22(14):7652. doi: 10.3390/ijms 22147652

19. Hort S, Herbst L, Backel N. Toward Rapid, Widely Available Autologous CAR-T Cell Therapy – Artificial Intelligence and Automation Enabling the Smart Manufacturing Hospital. Front Med (Lausanne). 2022; 9: 913287.doi: 10.3389/fmed.2022.913287

20. European Parliament. EU AI Act: first regulation on artificial intelligence. https://www.europarl.europa.eu/news/en/headlines/society/20230601STO93804/eu-ai-act-first-regulation-on-artificial-intelligence (Press Release) 08-06-2023

21. Iglesias-Lopez C, Agustí A, Vallano A. Current landscape of clinical development and approval of advanced therapies. Mol Ther Methods Clin Dev. 2021; 10(23): 606–618. doi: 10.1016/j.omtm.2021.11.003

22. DARE-NL- To accelerate clinical testing of novel oncological ATMPs to ensure timely and sustainable access to potentially curative treatment options for cancer patients. https://www.dare-nl.nl/

23. Saxocell https://www.saxocell.de/en/news-press-media/