Panel recommends gene-altering leukaemia treatment to US drug agency

Panel recommends gene-altering leukaemia treatment to FDA

Novartis developed the drug

Thursday, July 13, 2017

A panel has recommended the US Food and Drug Administration approve a cancer treatment which alters a patient’s own cells to fight the disease.

Scientists are calling the treatment “a living drug” as it harnesses the immune system to beat cancer, according to The New York Times.

It is thought to be likely the FDA will approve the gene therapy and if so, it will be the first such therapy to go on the market.

Pharmaceutical company Novartis worked on the cancer drug, which is for B-cell acute lymphoblastic leukaemia, but it is also working on other treatments for another type of the disease as well as an aggressive brain tumour and multiple myeloma.

The recommendation is that the therapy should be available to those whose cancer has resisted treatment, or people who have relapsed from ages 3 to 25.

The technique involves removing some of a patient's cells and sending them frozen to a Novartis plant where they are thawed and processed. The cells are then sent back to the patient's treatment centre.

This treatment has given patients, who were facing death as other treatments failed, long remission periods and possibly even cured them.

Novartis conducted a study of 63 patients and treated them between April 2015 and August 2016. A total of 82.5% of the patients went into remission, which is a high rate for the disease. However 11 involved in the study passed away.

The youngest patient to be given the altered cells was 12-year-old Emily Whitehead, who started the treatment aged six.

Although she suffered severe side effects, including crashing blood pressure, and lung congestion she is now free of cancer.

Her father said: “We believe that when this treatment is approved it will save thousands of children’s lives around the world."

Doctor Gwen Nichols, the chief medical officer of the Leukemia and Lymphoma Society, says the nest step is to discover “what we can combine it with and is there a way to use it in the future to treat patients with less disease, so that the immune system is in better shape and really able to fight.”

Spokeswoman for Novartis, Julie Masow, has said the company will file for approval of the treatment in the European Union later this year.