F.D.A Panel Recommends Approval for Gene-altering leukemia treatment
A US Food and Drug Administration panel opened a new era in medicine, unanimously recommending that the agency approve the first-ever treatment that genetically alters a patient’s own cells to fight cancer.
The treatment transforms genes into what scientists call a living drug that powerfully bolsters the immune system to shut down the disease.
If the FDA accepts the recommendation, which is likely, the treatment will be the first gene therapy to reach the market. Novartis is poised to be the first with a treatment for a type of leukemia. It is working on similar types of treatments for another form of the disease, as well as multiple myeloma and an aggressive brain tumor.
A single dose of the resulting product has brought long remissions, and possibly cures, to scores of patients in studies who were facing death because every other treatment had failed.
The panel recommended approving the treatment for B-cell acute lymphoblastic leukemia that has resisted treatment, or relapsed, in children and young adults aged three to 25.
The main evidence that Novartis presented to the FDA came from a study of 63 patients who received the treatment from April 2015 to August 2016. Fifty-two of them, or 82.5 per cent, went into remission – a high rate for such a severe disease.
The panel raised concerns about potentially life-threatening side effects like those Emily experienced, and longterm worries about whether the infused cells could cause secondary cancers or other problems.
Treatment of disease
The treatment was developed by researchers at the University of Pennsylvania and licensed to Novartis.
Use will not be widespread at first, because the disease is not common. It affects only 5,000 people a year, about 60 per cent of them children and young adults. Most children are cured with standard treatments, but in 15 per cent of the cases – like Emily’s and Connor’s – the disease does not respond, or it relapses.
Analysts predict that these individualised treatments could cost more than US$300,000 (S$413,000), but a spokesman for Novartis declined to specify a price.
Because the treatment is complex and patients need expert care to manage the side effects, Novartis will initially limit its use to 30 or 35 medical centres where staff will be trained and approved to administer it, the company said.
As to whether the treatment, known as CTL019, will be available in other countries, a Novartis spokesman said by e-mail: “Should CTL019 receive approval in the United States, it will be the decision of the centres whether to receive international patients. We are working on bringing CTL019 to other countries around the world.”
The treatment requires removing millions of a patient’s T-cells – a type of white blood cell – and genetically engineering them to kill cancer cells. The technique employs a disabled form of HIV, the virus that causes Aids, to carry new genetic material into the T-cells to reprogramme them. The process turbocharges the T-cells to attack B-cells, a normal part of the immune system that turn malignant in leukaemia. The T-cells home in on a protein called CD-19 that is found on the surface of most B-cells.
The altered T-cells – called chimeric antigen receptor cells – are then dripped back into the patient’s veins, where they multiply and start fighting the cancer.