From Haematologic to Autoimmune DiseaseThe CAR-T Therapy Second Revolution

Cell and gene therapies have revolutionised the field of medicine, offering new treatment options for patients with various diseases that, by type of disease or severity, were considered untreatable until a few years ago. Rapid technological advancements in genetic and biological engineering and improvements in the quality and standardisation of the manufacturing process are fostering a marked increase in clinical development of these advanced therapies, generally named cell and gene therapies CGT), and they are expected to receive more approvals in the near future.

The Alliance for Regenerative Medicine reported in its January 2024 meeting that, up to 17 regulatory approvals in the U.S. and Europe are anticipated for the coming year in cell & gene therapies, compared to eight approvals in 2023. This is despite the impact & changes in priorities caused by COVID-19. In the CGT field, chimeric antigen receptordirected (CAR-T) cell therapies have emerged as a groundbreaking approach for treating patients with cancers that previously had proved to be resistant to available conventional therapies. Specifically, CAR-T cell therapies targeted against antigens expressed on B cells (CD19 and BCMA) have been successful in treating blood-related cancers. Now we are seeing early promising results in expanding CAR-T application to autoimmune diseases, providing new hope for patients suffering from these debilitating conditions.

Evolution and Medications Approved to Date

CAR-T therapy has come a long way since its inception, with significant advancements in design, manufacturing and clinical applications. The first CAR-T therapy approved by the U.S. Food and Drug Administration (FDA) in 2017, Novartis’s Kymriah, targeted CD19-positive B cell malignancies, such as acute lymphoblastic leukemia and non-Hodgkin lymphoma. Since then, five more CAR-T therapies targeting CD19 and BCMA antigens (Tecartus, Abecma, Yescarta, Breyanzi and Carvykti) have been approved. To date, across all six FDA-approved CAR-T therapies, drugmakers have reported treating about 34,400 patients worldwide, and these therapies have demonstrated remarkable efficacy, with durable remissions in patients who have exhausted other treatment options. The success of CAR-T therapy in hematologic diseases has sparked interest in exploring its potential in treating other indications.