FDA approves first GM individualised cell therapy


Novartis has received FDA approval for its CAR-T cell therapy, marketed as Kymriah, for the treatment of a particular type of blood cancer, acute lymphoblastic leukaemia (“ALL”).

ALL (which is most common in childhood) is an acute form of leukaemia or cancer of the white blood cells. People suffering from ALL have an excess of cancerous cells which accumulate in the bone marrow thereby preventing the production of normal healthy cells. The result is that ALL sufferers may be anaemic and bruise or bleed more easily. ALL is the most common type of childhood cancer in the US.

Novartis’ new therapy  uses chimeric antigen receptor (CAR) cells, a type of T cell, which are extracted from the patient’s own blood and thereafter genetically modified. The modification essentially reprograms the T cells so that they are genetically coded and can locate and kill the cancerous cells when re-injected into the body. Once they have located their target they multiply. This form of individualised treatment is the first of its kind and the fact that it is personal to each patient makes it markedly different to more standard therapies such as chemotherapy, radiation or surgery.

The therapy is suitable for children, teenagers and young adults who have not responded to standard treatment and is therefore only likely to be available to a few hundred children and young people per year. Nevertheless, the initial results seen has led to Kymriah being hailed as a revolutionary treatment. To give an example, the first child treated with Kymriah at the Children’s Hospital of Philadelphia was described by doctors as being near to death but, following treatment has now been cancer free for 5 years. In addition, a clinical trial conducted by Novartis found that of the 63 patients treated with Kymriah, 83% went into remission.

Despite these successes, the treatment is not without side-effects and can cause cytokine release syndrome, a potentially life-threatening condition which results from the rapid propagation of the CAR-T cells in the body. In addition, this type of therapy, whilst looking promising for the treatment of blood cancers, has proved less effective in treating other cancers such as lung cancer or melanoma which are characterised by the presence of solid tumours.

However, there can be no denying that the approval of Kymriah marks the start of an exciting new chapter in the use of CAR-T cell technology as a means of treating cancer. To quote the CEO of Novartis Joe Jimenez,  “with the approval of Kymriah, we are once again delivering on our commitment to change the course of cancer care”. In this regard, readers may be interested to know that both the FDA and the EMA are currently considering whether to approve Kite Pharma’s genetically modified  CAR-T cell therapy axi-cel as a treatment for certain subtypes of non-Hodgkin’s lymphoma. A decision from the FDA is expected before the end of the year with approval from the EMA expected in 2018.