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 most important white blood cells of the immune system.
CAR-T cells are created from the patients’ own T cells that are genetically engineered to produce an artificial T-cell receptor for use in immunotherapy, enabling them to target a specific target protein to fight cancer.
Kymriah (Tisagenlecleucel) became the first CAR-T cell therapy to receive regulatory approval in ALL when it was approved by the FDA in August 2017. In 2018 it received the second FDA approval for the treatment of Diffuse Large B-cell Lymphoma (DLBCL) and EC approval in both ALL and DLBCL indications.
DLBCL is the most common subtype of non-Hodgkin lymphoma (NHL), is an aggressive disease characterized by frontline therapy failure rates of more than 30%. Adults with R/R DLBCL experience limited eligibility for and poor outcomes to standard of care therapies.
Kymriah is a one-time treatment for paediatric and young adult patients up to 25 years of age with B-cell precursor acute ALL that is refractory, in relapse post-transplant or in second or later relapse and in adult patients with relapsed or refractory DLBCL after two or more lines of systemic therapies.
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 can provide deep and durable remission in difficult-to-treat patients with R/R B-cell haematologic malignancies, with a well-characterized, manageable safety profile, while improving patient quality of life.
- Kymriah Summary of Product Characteristics. Novartis Pharma AG; 2018
- The Science of CAR-T Cell Therapy, Novartis.com website
- What is CAR-T cell therapy? Novartis.com website
- CAR-T Cell Therapy: Pioneering Cancer Therapy, Novartis.com website
- Kymriah, European Medicines Agency website
- KYMRIAH (tisagenlecleucel), FDA website