The Saudi Food and Drug Authority (SFDA) announced approval for the first phase 19 clinical study of a novel, locally developed gene therapy using engineered carcinoma-like T cells (CAR T-cells) to treat adults with CDXNUMX-positive acute lymphoblastic leukemia (ALL) who have failed to respond to treatment or have relapsed.
Food and Drug
This achievement is the first of its kind in the Kingdom, in terms of developing and manufacturing the treatment within a national research environment, using a closed transport system in an advanced manufacturing unit at King Faisal Specialist Hospital and Research Center in Riyadh. This reflects the integration between regulatory and scientific bodies in the Kingdom to advance the development of advanced gene therapies.
What is the target disease?
Acute lymphocytic leukemia (ALL) is one of the most serious types of blood and bone marrow cancers. It is characterized by its rapid spread, as it affects white lymphocytes, leading to the production of large quantities of immature cells, weakening the body's ability to fight infection, transport oxygen, and stop bleeding.
Mechanism of new gene therapy
The treatment under study relies on CAR T-cell technology, whereby a patient's T cells are modified outside the body to reprogram them to target cancer cells bearing the CD19 protein, and then reinjected into the patient intravenously. The current study aims to evaluate the safety of the treatment in patients in the first phase, aged 18 to 60.
The treatment is being developed by a national team of Saudi scientists and researchers in collaboration with Lentgen/Militoni, using precise manufacturing techniques that ensure the safety and quality of the biologic product.
A step towards healthcare leadership
The authority confirmed that this approval is part of its strategy to support innovation in advanced biotherapies and provide a flexible and transparent regulatory environment to accelerate the availability of promising treatments, particularly for incurable diseases.
She also explained that this step is in line with the objectives of the Health Sector Transformation Program, one of the programs of Saudi Vision 2030, which seeks to transform the Kingdom into a leading regional center for health research, development, and innovation.
for more information
The list of registered clinical studies can be viewed on the Authority’s website via the following link:
https://www.sfda.gov.sa/ar/drug_clinical_trials_list
