With the approval of two cell-based gene therapy drugs, the so-called CAR-T cell therapeutics, the European Commission (EC) has opened the way for a new innovative therapy. The medicines Yescarta (Axicabtagene Ciloleucel) and Kymriah (Tisagenlecleucel) are used to treat patients with certain types of blood cancer. They provide an additional treatment option for a group of cancer patients who have not responded to several conventional treatments.
"The marketing authorisation marks another milestone in developing an entirely new immunological therapy approach for the treatment of major diseases. Over a few decades, gene therapy has evolved from a promising concept to a real treatment option for largely untreatable stages of certain types of cancer,"
says Professor Klaus Cichutek, president of the Paul-Ehrlich-Institut (PEI), "I am pleased that, in terms of regulation,experts from PEI could contribute to the development and evaluation, thus paving the way of this new treatment option to patients."
Kymriah and Yescarta belong to the group of Advanced Therapy Medicinal Products (ATMPs). They must undergo the centralised authorisation procedure before they can bemarketed to the EU . Specifically for this group of medicines, the Committee for Advanced Therapies (CAT) of the European Medicines Agency (EMA) has been established, which is responsible for the scientific assessment of quality, safety and efficacy. The PEI currently provides the chair of the CAT and another member and representative.
For the production of CAR-T cells, T cells belonging to the white blood cells (lymphocytes) are taken from the patient, equipped with the gene of a chimeric antigen receptor (CAR) outside the body with the aid of a viral gene transfer vector, then multiplied and administered intravenously. These genetically modified immune cells can selectively kill cancer cells in the body.
Kymriah has been approved for the treatment of a sub-type of leukemia (B-ALL, B-cell acute lymphocytic leukemia) in children and a subset of a lymph node tumor (diffuse large B-cell lymphoma). The Yescarta approval concerns the treatment of diffuse large cell and mediastinal B-cell lymphoma.
The most common and serious side effects with this type of therapy are a cytokine release syndrome with fever and hypotension, as well as transient central nervous system depression. These potentially life-threatening side effects can be controlled by medical measures. A CAR-T cell therapy will be performed in qualified therapy centers by trained healthcare professionals.
More information will be available shortly on the EMA website.
Updated: 31.08.2018
