EBMT 2021 | The future of CAR-T and cellular therapy

Yeah, CAR-T therapies, we already heard a lot during the last EU CAR T-cell meeting, but we will hear more in many sessions here at EBMT. Basically, three key messages. First one is that for already established indications, mostly B-acute lymphoblastic leukemia, and B-non-Hodgkin’s lymphoma, refractory/relapsed forms of the disease. Real-world results are very reassuring when compared to the clinical results that were produced in the registration trials and the response rate, the overall response rate, and the safety profile of tisa-cel and axi-cel in real-world conditions are very much the same as they were in the registration studies, despite the fact that there are now many more centers that are in a position to administer those treatments to an increasingly diverse patient population. So that’s message one.

Message two is that we expect a significant increase in CAR T-cell activity as two new CAR T-cells that target not CD19 but BCMA, will soon be approved and reach the market. And so, we will see a population of patients with advanced multiple myeloma benefit of treatment with CAR T-cells targeting BCMA. So, we expect some sharp increase in CAR T-cell activity at European centers by the end of year 2021. And the results of the registration trials will be updated again at the EBMT annual meeting.

The third point is that we will see a blooming field with technological developments. We will see CAR T-cells targeting new tumor antigens with potential applications in diseases, ulcers, and lymphoid malignancies. We will see other types of immune effector cells than T-cells, namely natural killer cells that can be genetically engineered to express a chimeric antigen receptor, potentially bringing an improved safety profile while retaining a very significant clinical efficacy. And we will see new genetic constructs that could potentially improve the activity or the safety profile of CAR T-cells, in particular when patients develop significant side effects such as a cytokine release syndrome.

So again, very exciting developments to be presented at the EBMT annual meetings. And beyond CAR T-cells and immune effector cells, there are also other types of cellular therapies and gene therapies that come to the market. So, we expect some update, for example, on gene therapies for globin disorders, in view of the recently released information on safety issues and efficacy issues for us is a new category of drugs that still provide very exciting perspectives to treat these patients.