Novartis Leukemia Treatment Yields High Remission Rate

A new leukemia immune treatment being developed by Novartis AG led to the disappearance of cancer in 90 percent of patients, a study showed.

The therapy, a process that involved taking a patient’s T cells and reprogramming them to hunt cancer cells, resulted in complete remission of 27 of 30 patients with acute lymphoblastic leukemia, a deadly pediatric cancer, according to the study published today by the New England Journal of Medicine.

The early-stage trial of CTL019 engineered cells conducted by researchers at the University of Pennsylvania indicates the potential of the complicated treatment, which analysts have estimated may generate $10 billion a year if its uses expand beyond leukemia. The therapy can provoke serious side affects, however, and eight of the 30 patients required intensive care for severe cytokine-release syndrome.

“We are excited by these results, which indicated how effective CTL019 may be in fighting ALL, a leading cause of childhood cancer deaths,” said lead investigator Stephan Grupp, a professor at the university.

Immune therapies, which harness the body’s own defense system to fight cancer and other diseases, have taken the pharmaceutical industry by storm, with analysts estimating $35 billion in annual sales. Unlike products on the market such as Bristol-Myers Squibb Co.’s Yervoy, CTL019 therapy involves removing a patient’s blood to modify their blood cells.

Breakthrough Therapy

The published study updated trial data first reported in December at the American Society of Hematology’s annual meeting. A further update will be released at this year’s meeting.

The therapy is now in the second of three stages of human trials for acute lymphoblastic leukemia, with studies for other forms of cancer planned for next year. Novartis, based in Basel, Switzerland, received a breakthrough therapy designation from the U.S. Food and Drug Administration in July. The company will seek FDA approval in 2016.

Patients in the trial -- 25 children and five adults -- received infusions between April 2012 and February of this year. Of the 27 patients who were successfully treated, 19 remained in remission and 15 of them received no further therapy. Seven patients died after a relapse of the disease.

In 2013, more than 43,000 adults and 5,000 children and teens in the U.S. were diagnosed with leukemia, with about 6,000 having acute lymphoblastic leukemia.

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