The Spanish Agency of Medicines and Medical Products (AEMPS) has approved the CARxALL clinical trial led by the company OneChain Immunotherapeutics (OCI). 

It will be the world’s first trial with CAR-T technology for patients with a subtype of T-cell leukaemia.

The CARxALL trial will evaluate, for the first time, the CAR-T CD1a (OC-1) therapy.

This treatment specifically targets a subtype of T-cell leukaemia, cortical T-cell acute lymphoblastic leukaemia (coT-ALL), which accounts for 30-40% of T-ALLs and has a poor prognosis in patients who do not respond to currently available treatments.

The clinical production of the CD1a CAR-T therapy for the trial has been led by Dr Maria Castellà, a doctor in the Immunology Service at the Biomedical Diagnostic Centre of Hospital Clínic Barcelona, directed by Dr Manel Juan.

The trial will be conducted at Hospital Clínic and Hospital Sant Joan de Déu in Barcelona, under the direction of Dr Núria Martínez and Dr Susana Rives, principal investigators for the study.

It is expected to include both paediatric and adult patients without any therapeutic alternatives.

Although the patients will be treated in Barcelona, patients from Spain and across Europe may join the trial.

The group led by Dr Menéndez, founder of OCI and director of the Josep Carreras Institute’s stem cell biology, developmental leukaemia and immunotherapy group, was the first in the world to develop and validate a CD1a-specific CAR-T for coT-ALL.

The study, led by Dr Diego Sánchez and published in the journals Blood and the Journal for ImmunoTherapy of Cancer, has been conducted with animal models using cell lines derived from patients with coT-ALL.

Preliminary results show that these CAR-T cells persist in vivo in the long term and retain their anti-leukaemia activity.

The CARxALL clinical trial represents another step towards developing adoptive cell immunotherapy, such as CAR–T cell therapy, a treatment that consists of extracting a patient’s T cells (cells that protect the body from infection), modifying them in the laboratory and returning them to the patient.

This modification enables the cells to attack and eliminate the receptors located in the membranes of tumour cells.

With this technique, the patient’s own modified cells attack the cancer cells in a directed way, without damaging healthy cells.

This is a successful case of technology transfer and public-private collaboration involving recovery of public investment in research.

Article: Fratricide-resistant CD1a-specific CAR T cells for the treatment of cortical T-cell acute lymphoblastic leukaemia

Source: Josep Carreras Leukaemia Research Institute