Go-ahead for the optimization of new immunotherapies
Fighting autoimmune diseases and cancer with human immune cells more efficiently: medicine is moving a step closer to this vision with new processes developed by ISAR Bioscience GmbH. The Bavarian research company’s concepts for the rapid, reliable and at the same time economical production of therapeutic immune cells have now been positively evaluated by the German regulatory authority. The Paul Ehrlich Institute (PEI) considers the processes to be promising - and thus gives the go-ahead for the further development at ISAR Bioscience.
So-called C(A)ART cells are the key elements of the new therapies. The abbreviation stands for Chimeric (Auto-) Antigen Receptor Cells and thus for a promising therapeutic approach that is intended to strengthen the fighting ability of the body’s own immune cells (T cells) against serious diseases such as cancer, but also in autoimmune diseases.
The preparation of the highly personalized therapy is complex and takes several weeks. First, T cells are obtained from a patient’s white blood cells. In the case of cancer, these so-called autologous cells are then genetically modified in the laboratory so that they can recognize and destroy tumor cells in the body. Finally, the patients receive “their” specifically optimized cells back by infusion. In Germany, this very expensive therapy has already been approved for certain leukemias and lymphomas.
A new technology platform at ISAR Bioscience promises a much faster and more cost-effective procedure. In this process, stem cells are modified by genome editing in order to reduce a possible pathological autoimmune reaction against the body’s own tissue. Subsequently, the modified stem cells are used to permanently generate therapeutic “battle-ready” chimeric T cells. The new platform allows for the mass production of standardized batches of cells that can be used over and over again in the long term. This, it is expected, will significantly reduce treatment costs compared to current clinically approved autologous CART cell therapies.
A second innovation is expected to allow T cells to be used in autoimmune diseases. CAART cells are expected to make this possible. The second A in this abbreviation stands for auto-antigen: These are the body’s proteins that are attacked by its own immune system. CAART cells are intended to suppress this attack. The first disease the researchers at ISAR Bioscience want to tackle is hyperthyroidism triggered by antibodies. However, other autoimmune diseases, such as rheumatic diseases, may also be amenable to such therapy.
The Bavarian research company specializes in the production of customized stem cells and aims to bring medical research rapidly to application. The encouraging signal for the new technology platform for the use of human pluripotent stem cells (hiPSC) for the generation of therapeutic immune cells is the result of a “Scientific Advice” meeting recently held with the Paul Ehrlich Institute.
The positive evaluation is associated with advice from the PEI on the further development of the platform. This involves, for example, the generation of a Master Cell Bank (MCB) in the laboratories of ISAR Bioscience at the company’s site in Planegg near Munich. The planned MCB will enable continuous cell production in accordance with “Good Manufacturing Practice” (GMP). Specifications for the release of the MCB have already been jointly defined by ISAR Bioscience and PEI. Further preclinical studies are in preparation. Thereafter, toxicological tests are planned and, after their completion, clinical tests in humans.
Background information: https://www.pei.de/DE/home/home-node.html
