Newsletter_04-2024_EN

EU-funded JOIN4ATMP project aims
to overcome regulatory obstacles
They offer hope for people who do not respond to conventional treatments or
for whom there is no effective therapy yet available: advanced therapy medicinal
products (ATMPs). The first of these novel medications, developed using genetic
and cell technologies, were officially approved just five years ago. Some of them
have been great successes, while others have made a mere blip on the market.
JOIN4ATMP, a Europe-wide project, is now being launched under the coordination
of Charité – Universitätsmedizin Berlin, with support from the Berlin Institute
of Health at Charité (BIH). The project aims to identify the obstacles standing
in the way of these new treatments and determine what is needed to ensure
that people in Europe can have fast, safe, and equitable access to them.
netically modify them in a lab so that, once
reintroduced into the body, they can detect
and destroy cancer cells. ATMPs can be tailored
to individual patients more effectively
than traditional medications, which makes
them especially suitable for treating rare diseases
and cancers that currently have no or
inadequate treatment options.
Although there are many ATMPs currently
in development, very few have been
approved for the European market to date.
The problem is that the regulatory rules for
approval of conventional medicinal products
– which require clinical trials involving
large numbers of patients, for example
– do not translate to these complex gene and
cell therapies.
Reshaping the landscape
GMP-compliant manufacturing of gene and cell therapies in a special lab at Charité
© Charité | Arne Sattler
Gene and cell therapies are among the most
important innovations in the healthcare
sector. And they reflect advances in science
and technology. They have the potential to
radically reshape the treatment of cancer,
autoimmune diseases, neurodegenerative
disorders, and many rare genetic conditions.
But the path to approval and clinical use
of these products is long and often fraught
with difficulty.
That was the reason the European University
Hospital Alliance (EUHA) founded
the European Center for Cell and Gene
Cancer Therapies (EUCCAT) four years ago.
The center’s aim is to facilitate the clinical
use of ATMPs developed at higher education
institutions and further consolidate the
basic research conducted in Europe. The
newly launched JOIN4ATMP project originated
with the virtual institute. All members
of the EUHA, together with the existing
EU-funded RESTORE and T2EVOLVE
networks, biotech companies, and patient
advocacy organization EURORDIS – Rare
Diseases Europe, will work together to identify
obstacles and propose solutions geared
toward real-world practice – so that these
innovative treatments are made affordable
and accessible to all patients.
“Living” medications
ATMPs are based on genes, tissue, or cells,
so they often contain living components. For
example, it is possible to take white blood
cells from a patient with leukemia and ge-
This is where JOIN4ATMP, which is slated
to receive about three million euros in funding
from the European Commission over
a three-year period, comes in. “Our goal is
to devise concrete recommendations for
how patients in Europe can gain access to
innovative gene and cell therapies faster,”
says Prof. Annette Künkele-Langer of the
Department of Pediatric Oncology and Hematology
at Charité, which is leading the
consortium. “To that end, we are bringing
knowledge and experience in preclinical
development, production, clinical testing,
market approval, and reimbursement of
ATMPs together Europe-wide and analyzing
the obstacles and how they can be
overcome at the medical, regulatory, and
economic levels.” The experts will present
their conclusions in the form of guidelines,
recommendations, and white papers, thereby
advancing the European strategy for
novel therapies. They will form the basis for
new approval processes tailored to ATMPs
and create the overall conditions needed
for standardized, decentralized manufacturing
of gene and cell therapies even as the
application of rigorous good manufacturing
practices (GMPs) is expanded at the European
level.
Charité – Universitätsmedizin Berlin
D 10117 Berlin
www.reinraum.de | www.cleanroom-online.com NEWSLETTER | Edition EN 04-2024
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