A spin-off to develop therapies for leukaemia based on CAR-T, by Josep Carreras Institute

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 tumours, 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”.

Prof. Evarist Feliu, president of the Josep Carreras Leukaemia Research Institute’s Delegate Committee, says, “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 tumours”.

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”.

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ADDITIONAL INFORMATION

Thymic cortical T-cell acute lymphoblastic leukemia

More than 350 children are diagnosed with leukaemia every year in our country. Leukaemia accounts for around a third of childhood neoplasias.

Childhood leukaemia is an aggressive kind of blood cancer. The affected cells can be of the lymphoid or myeloid lineage. The most usual kind of leukaemia in children is of lymphoid origin, the most common kind being acute lymphoblastic leukaemia (ALL), which accounts for approximately 80% of childhood cases.

Acute lymphoblastic leukaemia in turn can originate in B or T lymphocytes. 80% of childhood acute lymphoblastic leukaemias are B type.

8 of every 10 children manage to overcome the disease. In the case of T-type ALL fewer than 100 cases are diagnosed every year in Spain and these are “subdivided” into four main kinds by means of immunophenotyping studies: Pro-T, Pre-T, thymic cortical and thymic mature.

Cortical T-cell acute lymphoblastic leukemia (coT-ALL) accounts for 40% of T-type acute leukaemias. Fewer than 30 children are diagnosed with this every year in our country. Usually they are adolescents between the ages of 11 and 17. A third of them do not respond to existing therapies and we can not offer them any further opportunities with the current arsenal of therapies.

About Dr. Pablo Menéndez

Dr. Menéndez was born in Avilés, Asturias, in 1974. He graduated in biochemistry from the University of Salamanca (1997) and as a Doctor of Medicine (Hematology) from the same university in 2002, under the supervision of Prof. Alberto Orfao and Prof. Jesús San Miguel. He carried out his postdoctoral training in stem cell biology at the Mick Bhatia laboratory in Canada from 2003 to 2005, and in childhood leukaemia at the laboratory of de Mel Greaves in London from 2005 to 2007. In 2007 he was appointed director of the Stem Cell Bank of Andalusia and directed his own laboratory at the Biomedical research Centre in Granada until July 2011, when he commenced work as a lead researcher at the Pfizer-University of Granada-Junta de Andalucía Centre for Genomics and Oncological Research (GENYO). In June 2013 he was appointed ICREA Research Professor and Research Director of the Josep Carreras Leukaemia Research Institute’s Hospital Clínic-UB Campus.

About the Josep Carreras Foundation

The Josep Carreras Leukaemia Foundation was established in 1988 with the intention of contributing to finding a definitive cure for this disease. Its efforts are concentrated on four basic areas: administering the Spanish Bone Marrow Donor Registry (REDMO), scientific research, carried out by the Josep Carreras Leukaemia Research Institute, patient guidance through an online patient consultation channel, and reception apartments for patients who need to undergo treatment and have to spend a long time far from home.

About the Josep Carreras Leukaemia Research Institute

The Josep Carreras Leukaemia Research Institute, a public centre pertaining to the Generalitat de Catalunya’s CERCA network, was established in 2010 with the aim of furthering biomedical research and personalised medicine in the field of leukaemia and other onco-hematological diseases. It is the first research centre in Europe exclusively focussed on leukaemia and malignant blood diseases, and one of the very few in the world. The Josep Carreras Institutes has three coordinated but independent scientific campuses: University of Barcelona Hospital Clínic Campus, The Catalan Institute of Oncology/Germans Trias i Pujol Campus, and the Sant Pau – Autonomous University of Barcelona (UAB) Campus.

About ICREA

ICREA, Catalan Institution for Research and Advanced Studies, is a foundation funded by the Catalan Government. ICREA was established in response to the need for new contracting formulas that would make it possible to compete on a level playing field with other research systems, with a view to contracting outstanding and highly-talented academic and scientific staff. ICREA is an open institution. It works shoulder to shoulder with Catalan universities and research centres to integrate ICREA researchers into the Catalan research system. ICREA offers researchers from around the world permanent posts to come and conduct research in Catalonia. Over the years it has become synonymous with academic excellence around the world. ICREA has researchers in all fields of knowledge who carry out their research work in 48 universities and research institutions in Catalonia.

About CDTI

The Centre for Industrial Technological Development (CDTI) is an organ of the General State Administration that supports knowledge-based innovation. It provided guidance and public funding by means of grants and partially reimbursed funds. The CDTI also internationally projects business R&D projects and innovation for companies and Spanish organisations. It manages Spanish participation in international R&D&I organisms, such as Horizonte2020 and Eureka, and in the Science and Space industries. Furthermore, through the Innvierte Economía Sostenible initiative, it supports and facilitates the capitalisation of technological companies.

About Invivo Ventures

Invivo Ventures FCR invests in life-science companies at an early stage and is administered by Invivo Capital Partners, an administrative company founded by Dr. Lluís Pareras and Albert Ferrer. Both administrators have wide experience in the sector and also administer the risk capital company, Healthequity SCR. Invivo Ventures FCR has a majority of private investors with the support of a number of institutional investors such as, the European Investment Fund (EIF), Fond-ICO Global, the Institut Català de Finances and the Institut Valencià de Finances.