The special feature. The CAR T cells were modified using an innovative virus-free technology called the Sleeping Beauty Transposon System. This was brought to clinical maturity in Leipzig under GMP conditions and enables safer and more efficient production than conventional methods. The therapy targets the protein ROR1, which is present on the cell surface in many types of cancer but plays hardly any role in healthy tissue. The first patient treated suffers from a rare adrenal carcinoma. The main aim of the phase I trial is to determine safety, tolerability and the correct dose. At the same time, the doctors and researchers are hoping for initial indications that the therapy is working, for example through a reduction in tumour growth or measurable immune responses.
Up to 46 patients are to be treated in the LION 1 study, including at the NCT sites in Berlin and Cologne. The Fraunhofer IZI is not only supplying the cell preparations, but has also played a key role in the preclinical development of the ROR1 CAR T therapy. The LION 1 study is therefore also a prime example of how excellent research from Leipzig finds its way directly to the patient's bedside.
News from "Uniklinikum Würzburg" on 25 June 2025