Gene therapy for leukaemia set for FDA go-ahead

A drug for leukaemia that genetically alters patients' own cells to fight cancer, has cleared a critical hurdle in gaining commercial approval.

CTL019, or tisagenlecleucel, was unanimously recommended by the Oncologic Drugs Advisory Committee (ODAC), which advises the US regulatory body the Food and Drug Administration (FDA). If CTL019 does go on to be approved – as is likely now – it will be the first gene therapy to gain FDA approval.

'It is encouraging to see the FDA panel's recommendation and continued momentum behind this innovative therapy, which has potential to help young patients with relapsed/refractory B-cell ALL,' said Professor Carl June, team leader at the University of Pennsylvania.

The new drug belongs to the so-called CAR-T cell immunotherapies, and is designed to treat the most common childhood cancer in the USA, B-cell acute lymphoblastic leukaemia (B-cell ALL). The treatment requires harvesting T-cells, a type of white blood cell – from each individual patient, and then genetically reprogramming them to attack B-cells which turn malignant in leukaemia. The genetically-modified T-cells are then reinfused back into the patient.

Professor June called the altered T-cells 'serial killers' in the New York Times - as one can fight up to 100,000 tumour cells. CTL019 was developed by researchers at the University of Pennsylvania and then licensed to Novartis to treat children and young adults.

The ODAC's recommendation was based on evidence from Novartis, including a study in which 83 percent of 63 patients treated with CTL019 achieved remission.

The panel also heard from Tom Whitehead, father of Emily Whitehead, who was the first child to be given the gene therapy in 2012. Despite experiencing severe short-term side effects, she was cured and the cancer has not returned.

'We believe that when this treatment is approved it will save thousands of children's lives around the world,' said Mr Whitehead. 'I hope that someday all of you on the advisory committee can tell your families for generations that you were part of the process that ended the use of toxic treatments like chemotherapy and radiation standard treatment, and turned blood cancers into a treatable disease that even after relapse most people survive.'

But the panel raised major concerns over potential side-effects including short-term acute reactions, and longer-term issues such as possible life-threatening inflammatory conditions. For this reasons, patients receiving the CTL019 therapy will be entered in a registry and monitored for 15 years.