Gene therapy successfully used as a cancer treatment

Layla Richards, a one-year old girl, has had her leukaemia successfully treated with genetically engineered immune cells. Before her first birthday, Layla’s parents were told that none of the treatments she had been given had worked as her acute lymphoblastic leukaemia (ALL) was particularly aggressive. However, her family and doctors at Great Ormond Street hospital were determined not to give up on her and agreed to give Layla an experimental new treatment that had previously only been trialled in mice with leukaemia.

Previously researchers at UCL had shown that using T-cells that were genetically engineered to target specific antigens on the surface of tumour cells could be highly useful in the treatment of certain kinds of cancer. Layla was given 1ml of these white blood cells and her parents were told to look for a rash that would be indicative of the treatment working. Within a few weeks, the rash appeared and Layla was cleared of leukaemia. She is still free of the cancer months later.

Currently, only 25% of children with ALL survive. It is thought that this therapy could be suitable for up to 10 children each year who develop ALL. The first clinical trials of this are scheduled to occur in 2016, funded by the French biotechnology firm Cellectis. The implications of Layla’s story also reach further; researchers want to modify these T-cells further to test their use as a therapy for other types of cancer and inherited diseases such as sickle-cell anaemia.