Gene therapy for cancer nears FDA approval

Emily Whitehead, 12, seen here with her parents, Tom and Kari Whitehead, has been cancer-free since she underwent the experimental therapy known as CTL019 at age six.
Emily Whitehead, 12, seen here with her parents, Tom and Kari Whitehead, has been cancer-free since she underwent the experimental therapy known as CTL019 at age six.PHOTO: NYTIMES

Panel backs treatment targeted at a type of leukaemia and in patients aged 3 to 25

NEW YORK • A Food and Drug Administration (FDA) panel has opened a new era in medicine by unanimously recommending that the agency approve the first treatment that genetically alters a patient's own cells to fight cancer.

The cells will be transformed 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. Others are expected. Researchers and drug companies have been engaged in intense competition for decades to reach this milestone.

Novartis is now poised to be the first. Its treatment is for a type of leukaemia, and it is working on similar types of treatments in hundreds of patients for another form of the disease, as well as multiple myeloma and an aggressive brain tumour.

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The treatment known as CTL019 was developed by lead researcher Carl June at the University of Pennsylvania and licensed to Novartis as a treatment for paediatric acute lymphoblastic leukaemia. It works by taking a patient's own white blood cells, called T-cells, then altering them to allow them to recognise and kill cancer cells. A single one can destroy up to 100,000 cancer cells.

The panel on Wednesday recommended approving the treatment for B-cell acute lymphoblastic leukaemia that has resisted treatment, or relapsed, in children and young adults aged three to 25.

It is best known for saving the life of a young girl named Emily Whitehead, who is now 12 and has been cancer-free since she underwent the experimental therapy at age six.

The treatment... works by taking a patient's own white blood cells, called T-cells, then altering them to allow them to recognise and kill cancer cells. A single one can destroy up to 100,000 cancer cells.

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. Eleven others died.

Side effects, however, can be severe, and include spiking fever and crashing blood pressure.

As the treatment destroys not only leukaemic B-cells but also healthy ones, which help fight germs, patients need treatment to protect them from infection. Thus, every few months they receive infusions of immune globulins.

"Although this therapy is technologically somewhat complicated and is associated with certain serious side effects, it indeed has been proven to be amazingly effective," said Dr Kanti Rai, chief of the chronic lymphocytic leukaemia research and treatment programme at Northwell Health Cancer Institute in New York.

Analysts predict that these individualised treatments could cost more than US$300,000 (S$413,000), but a spokesman for Novartis, Ms Julie Masow, declined to specify a price.

As to whether the treatment will be available in other countries, Ms Masow said: "Should CTL019 receive approval in the US, 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."

She added that the company would file for approvals in the European Union later this year.

NYTIMES, AGENCE FRANCE-PRESSE

A version of this article appeared in the print edition of The Straits Times on July 14, 2017, with the headline 'Gene therapy for cancer nears FDA approval'. Print Edition | Subscribe