ATMPs are cutting-edge treatments that could drastically change patient life quality and expectancy in the coming years.
We interviewed Johan Van Eldere, Emeritus Professor of Microbiology at the Catholic University of Leuven and Hasselt University and Secretary-General of the European University Hospital Alliance (EUHA) on this topic.
EUHA brings together eleven leading University Hospitals in Europe. We took the opportunity to interview Prof. Van Eldere during the EUHA Members’ Assembly that took place from November 11 to 14 2024, hosted by IRCCS San Raffaele Hospital and Vita-Salute San Raffaele University, marking the conclusion of their six-month joint presidency of EUHA.
ATMP is the acronym for Advanced Therapy Medicinal Products – for most people it would be much clearer if we just said that these are cell - and gene – therapy products. When we’re talking about ATMPs we are mostly referring to cells from a patient that we modify genetically and then administer again to the patient and that will, for example, attack tumours or cancers, or restore a function that the patient has lost or correct a genetic disorder.
ATMPs are fantastic drugs: they can in some cases cure illnesses that could until recently only be managed at best, thus dramatically changing life quality and expectancy of the patients. To get ATMPs on the market and available for patients, you obviously need to follow a certain procedure with EMA (European Medicine Agency) as for any other drug. However, the current EMA procedure is designed for chemical drugs, and is not adapted for, nor suited to ATMPs.
To make chemical drugs you take a few chemical constituents, you put them together in a sometimes very complex chemical reaction and if you use the same basic ingredients and the same chemical conditions you consistently obtain the same products. Furthermore, this process can be scaled up to produce great quantities. The problem with ATMPs is that we start from cells from a patient, which are therefore different every time because every patient is different, and we genetically transform them by using, for instance, viral vectors. This makes it much more difficult to predict what the final product will be and whether it will have the same effect on patients. For this reason, to obtain a market authorisation (the permission to use them in patients) for lifesaving ATMPs based on procedures designed for chemical drugs is currently very difficult.
Prof. Johan Van Eldere
Another difficulty is that in particular universities or academic institutions or small and medium enterprises do not have sufficient personnel, time and money to go through this complex procedure with EMA, to get these products on the market.
In addition, for universities or academic institutions or small and medium enterprises it is much more difficult to get approval by EMA, since they often make products targeted towards rare diseases. When you have few patients affected by a rare disease, it is much more challenging to follow EMA procedures based on the principle of “randomised controlled trials”: hospitals simply don’t have enough patients to form a group that you treat and a group that you do not treat. It therefore becomes problematic to evaluate drugs for rare diseases with the methodology applied to evaluate chemical drugs.
With this in mind, EUHA has initiated EUCCAT, the European Center for gene & Cellular Therapies, a trans-institutional and trans-national group that brings together basic research groups, medicinal production facilities, clinical trial capacity and translational know-how. EUCCAT is actively pursuing its objectives through the JOIN4ATMP project.
Join4ATMP is a EUHA project funded by Horizon Europe (the EU program for research and innovation for the period 2021-2027). Within the project Join4ATMP, we are trying to develop adapted pathways to market authorisations, to facilitate academic institutions and small and medium enterprises in bringing these new drugs to the market. That would not only be good for academia, but also for the patients! We know that industry is not interested in developing these drugs for many rare diseases, because commercially these products are not considered interesting. The whole concept of an ATMP is less suitable for industry because industry likes to make things in large volumes that can be sold to many patients. On the opposite side, the world of academia can make a great contribution to the development of the ATMPs with great advantage for patients and their families.
To make that possible, we need to adapt the rules for market authorisations.
At the IRCCS Ospedale San Raffaele and Vita-Salute San Raffaele University, many ATMPs are currently under development and study. For this reason, I believe it was especially important to mark the conclusion of the EUHA presidency by OSR-UniSR with an in-depth reflection on ATMPs.”
One of the key moments of the Milan EUHA Autumn Meeting was the Symposium entitled “European Academia at the Forefront of Advanced Therapies Medicinal Products Development”, which explored collaborative EU-wide solutions to align ATMPs with regulatory standards, underscoring the urgency of accessibility and affordability for patients across Europe. Several UniSR researchers participated in the Symposium: among them Chiara Bonini, full professor of Blood Diseases, and Fabio Ciceri, full professor of Blood Diseases, who gave their point of view during the session titled “From ATMPs to other innovative approaches in medicine: an academic perspective”.
Alessandro Aiuti, full professor of Paediatrics, chaired the Symposium session “How to translate academic ATMP development into the regulatory context”. Professor Aiuti recalled that San Raffaele is at the absolute forefront in developing and studying ATMPs, therefore actively engaging with both the opportunities and challenges inherent to these groundbreaking treatments:
“Advanced therapies are now a therapeutic reality, and we have been among the pioneers in the field, developing one of the first gene therapies to reach the European market, that for ADA-SCID immunodeficiency, approved in 2016, and that for Metachromatic Leukodystrophy, approved in 2020 in Europe and in 2024 in the United States. Alongside significant therapeutic successes, we have continued to work on clarifying the underlying mechanisms of diseases and refining gene transfer strategies, thanks to the support of the Telethon Foundation and important international partners.
However, today the challenge is no longer only scientific: the sustainability of these therapies, especially when aimed at ultra-rare diseases, is extremely at risk if current models of development, approval, and reimbursement do not change. For this reason, synergy among all stakeholders involved is important, and the Alliance of University Hospitals, of which San Raffaele Hospital is a part, will play a crucial role in upcoming challenges”.