Chimeric antigen receptor(CAR) T-cell therapy is effective in relapsed/refractory large B-cell lymphoma(LBCL), but evidence in elderly patients(≥70y) is limited. We compared outcomes of tisagenlecleucel(tisa-cel) and axicabtagene ciloleucel(axi-cel) and assessed prognostic factors. We analyzed 1,191 patients(aged ≥70y) reported to the EBMT(tisa-cel n=402; axi-cel n=789). Median follow-up was 22.7 months. Outcomes included cytokine release syndrome(CRS), immune effector cell-associated neurotoxicity syndrome(ICANS), overall survival(OS), progression-free survival(PFS), relapse incidence(RI), and non-relapse mortality(NRM). Multivariable models adjusted for age, sex, ECOG, disease status, prior transplantation, and year of infusion. Median age at infusion was 74y; 65.6% were aged 70-75y. Progressive disease at infusion was 59% vs 53% for tisa-cel and axi-cel(p=0.04). Tisa-cel patients were older(74.0 vs 73.7y, p=0.019). At day-30, CRS(any grade) occurred in 62% vs 77%(p.
Vanderson Rocha, J. Mooyaart, R. Ram et al.· Haematologica· 0 citations
Gene- and cell-based therapies (GCTs) represent a disruptive and transformative class of biomedical innovations. They address diseases by adding, removing, repairing, or replacing genes and/or by endowing distinct living cells with additional biological functions. Through this plethora of options, numerous conditions—including genetic disorders, cancers, and degenerative diseases—have become potential targets for a curative therapy. Thus, GCTs are considered the “Future of Medicine” as they (i) offer a potential cure, particularly for rare and severe disorders previously considered untreatable, (ii) expand the treatment options for common diseases, and (iii) possess the possibility to complement currently applied conventional treatment options. Recognizing both the scientific promise and translational challenges of GCTs, Germany has launched a coordinated national initiative—the National Strategy for Gene- and Cell-Based Therapies. The Strategy was commissioned by the German Federal Ministry of Research, Technology and Space (BMFTR, formerly the German Federal Ministry of Education and Research [BMBF]) and developed through a multi-stakeholder process. The latter involved more than 150 experts from academia, industry, health care sector, professional associations, and patient organizations, who were nominated by the community and assembled into eight working groups to identify current roadblocks and propose possible solutions. Summarized in the Strategy Paper, which was submitted to the BMFTR and published on June 12, 2024, a comprehensive roadmap was developed in this bottom-up process to accelerate the development and clinical implementation of GCTs in Germany. Although it initially had a national focus, the resulting framework is increasingly contributing to the international GCT landscape through growing exchange with GCT initiatives launched in other European member states and with the European Society of Gene and Cell Therapy (ESGCT). In brief, the initiative is focusing on translation starting from research through all steps to clinical application and beyond. This includes workforce development, regulatory frameworks, manufacturing capacity, patient access, and communication with the general public. Numerous targeted measures have been developed by the participating experts in the working groups and are currently being implemented in this broad, collaborative, and bottom-up multi-stakeholder approach. They encompass, for example, the establishment of a website as central information platform, including the GCT-Atlas, a web-based networking and information tool for stakeholders and actors in the GCT field, tailored communication and outreach formats, a Regulatory Support Unit providing independent regulatory guidance for publicly funded early-stage, nonclinical product development, different funding and entrepreneurship programs offering researchers and clinicians financial, educational, and mentoring support, as well as the establishment of translational infrastructure and exchange formats with investors to specifically foster the necessary scale-up and commercialization. Overall, the main goal of the German National Strategy for GCT is to ensure patient access to advanced therapies while strengthening Germany’s position as an international hub for biomedical innovation. To accomplish this, existing resources need to be coordinated, streamlined, and prioritized to increase efficiency and support the long-term sustainability of the system. These objectives are closely aligned with current emerging European initiatives, including the EU Biotech Act and the Horizon Europe work program 2026, which aim to further optimize the framework conditions for this strategically important field and enhance future European competitiveness.
Christian Gallus, F. Ayuk, P. Beckhove et al.· Human Gene Therapy· 0 citations
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