2018: CAR-T cell therapy, Kymriah® second FDA approval and EC approval – unique to each patient

Nominated by: Novartis

Organisations in nomination: Novartis

Acute lymphoblastic leukaemia (ALL) is a type of cancer that affects white blood cells. It progresses quickly and aggressively and requires immediate treatment. Both adults and children can be affected and is the most common childhood cancer, occurring when a bone marrow cell develops errors in its DNA.

Correcting the DNA is a key target in the emerging field of immunotherapy, with research now yielding products that can finally be used in patients.

T cells are one of the important white blood cells of the immune system

CAR-T cells are created from T cells, an important type of white blood cells within our immune system.  The T cells are genetically engineered to produce an artificial T-cell receptor for use in immunotherapy, enabling them to target a specific target protein.

Kymriah became the first CAR-T cell therapy to receive regulatory approval when it was approved by the FDA in August 2017. In 2018 it received the second FDA approval and EC approval. Kymriah is a one-time treatment for patients up to 25 years of age with B-cell precursor acute ALL that is refractory, or in second or later relapse.

Kymriah makes use of a patient’s own T cells to fight cancer, using the 4-1BB costimulatory domain in its chimeric antigen receptor to enhance cellular expansion and persistence.

CAR-T is different from typical small molecule or biologic therapies because it is manufactured for each individual patient using their own cells. It is a one-time treatment after which patients may not need another therapy.

CAR-T cell therapy has been shown to be more effective than other treatments for haematologic malignancies, providing improvements in health outcomes for approved indications.

Kymriah® was later also approved by the FDA for non-Hodgkin lymphoma (NHL), and today is the only CAR-T therapy FDA-approved for two distinct indications.



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