Josep Carreras Institute creates spin-off to develop CAR-T based therapies for leukemia

The Josep Carreras Leukaemia Research Institute, a public centre pertaining to the Generalitat de Catalunya's (Government of Catalonia) CERCA network, has created the OneChain Immunotherapeutics (OCI) spin-off, the aim of which is to develop new immuno-oncological therapeutic tools with various preclinical candidates, based on CAR-T technology for different tumors, such as cortical T-cell acute lymphoblastic leukemia (coT-ALL), a rare subtype of leukaemia that mainly affects children, and which has a poor prognosis.

The spin-off is being funded initially through a round of seed capital, amounting to €3,050,000, provided by Invivo Ventures, the Ministry of Industry, through CDTI-Innvierte, and the Josep Carreras Foundation, and it clearly makes the case for bringing the research carried out by Dr. Pablo Menéndez's group closer to patients. Dr. Mendez is an ICREA research professor and the scientific director at the Josep Carreras Leukaemia Research Institute's Hospital Clínic-UB Campus.

Dr. Menéndez, founder of OCI and world renowned for his work in the field of childhood leukaemia, directs the Josep Carreras Institute's stem cell biology, leukaemia development and immunotherapy group. More than 180 of his studies have been published by the world's most prestigious scientific journals and, outstanding in his wide-ranging professional career is having been awarded the triennial ED Thomas Postdoctoral Fellowship grant, awarded by the Josep Carreras Foundation in 2006, as well as three grants awarded by the European Research Council (ERC), the most important scientific funding organisation in Europe.

Dr. Menéndez's group was the first in the world to develop and validate a CD1a specific CAR-T for coT-ALL. The study, published in the journal Blood, has so far been conducted with animal models using both cell lines and primary blasts derived from patients with coT-ALL. Preliminary results show that these CAR-T cells persist in vivo over the long term and retain their anti-leukaemia activity.

The creation of this spin-off marks a further step towards developing adoptive cell immunotherapy, such as CAR-T cell therapy, a treatment that consists of extracting a patient's T cells (the ones that defend the organism), modifying them in the laboratory, and returning them to the patient. The modification enables the cells to attack the receptors that are located in the membranes of tumour cells (called CD antigens) and eliminate them. With this technique it is the patient's own modified cells that that attack the cancer cells, and they do so in a directed way, without damaging other, healthy, cells. The funding achieved through this first round is essential for turning research results into products that reach patients because, before they can be used clinically, they must pass safety, efficacy and production process scale tests, and these are technically highly complex and very expensive.

Dr. Menéndez says, "OCI is the vehicle that will enable us to take all the work we have been doing over all these years and apply it to clinical use. At the academic level it is very complicated to develop these strategies and manage all the regulatory mechanisms associated with the development of a product. OCI will enable us to carry out all the necessary steps so that all our knowledge can be available to patients".

Antoni García Prat, The Josep Carreras Foundation's administrator, says "The Foundation is participating in the initial investment by Invivo Ventures and CDTI in support of translational research and also in support of the foundational team. It should be borne in mind that the projects are directed towards rare or childhood blood diseases, or others with few therapeutic alternatives, and that, should the Foundation benefit financially, the proceeds will be entirely invested in the fight against leukaemia, always to the benefit of patients".

At the present time immunotherapy constitutes a wide and hopeful field for cancer treatment. Its major clinical interest is currently focussed on immune system checkpoint inhibitors and adoptive therapy strategies with T cells, with more than 700 clinical trials taking place throughout the world for the treatment of malignant blood diseases and, to a lesser extent, solid tumors".

Prof. Evarist Feliu, President of the Josep Carreras Leukaemia Research Institute's Delegate Committee

Jorge Alemany, CEO of OneChain, adds, "OneChain is a perfect example of Public Private Partnership (PPP) for the benefit of patients. A representative of the voluntary sector (FJC) promotes a project thanks to the support of the public sector (ICREA and CDTI) and private effort (Invivo)".

Dr. Lluís Pareras and Albert Ferrer, founding partners of Invivo Ventures, say, "We are joining forces with the Josep Carreras Foundation and the Josep Carreras Institute to develop a CAR-T platform with potential for the clinical application of solutions for devastating blood diseases. This platform, led by Dr. Pablo Menéndez, includes the CAR-T CD1a programmes, as well as two other platforms, which we can not disclose at the moment, but which we hope will be able to significantly improve the lives of many cancer patients".