Engineered Cell Therapy for Cancer Gets Green Light from FDA
On Wednesday (13 July), the FDA panel advisory committee announced that it backed Novartis’ (NVS) new gene therapy treatment.
The therapy treats patients with B-cell acute lymphoblastic leukaemia or ALL, reports Seeking Alpha. It is a new treatment known as CAR-T or chimeric antigen receptor T-cell therapy.
In simpler terms, the patients T-cells are taken out of the body and re-engineered to target and kill cancerous cells only in the body. Once this has been completed they are placed back into the patient, and the T-cells get to work and begin combatting against the cancer. CART-cells are built with proteins where they seek out cancer protein cells in the body that they destroy.
The backing is a great success for the company, as this is the first CAR-T therapy to be recommended for FDA possible approval. The potential of this treatment is exciting; especially for children and young adults with B-cell ALL, as they would only need to be treated once and afterwards the therapy would work on its own accord. An issue hanging over the benefits is cost, some analysts expect it could cost up to $500,000.
However, at current despite receiving backing from the FDA, the body has until 3 October 2017 to decide if it wants to approve CTL019. The panel recommended 10-0 that the treatment should be approved by the FDA. It had ruled that the benefits of the patients receiving this treatment outweigh the overall risks.
The panel recommended CTL019 because of the positive clinical findings from a phase 2 trials, known as the ELIANA study. This is the first of its kind due to being the first global paediatric CAR-T cell therapy registration trial. Ultimately, the trial achieved statistical significance at the end of the study. Throughout the study was looking for an improvement in complete remission or complete remission with incomplete blood count recovery over time with treatment of CTL019.