Sun Sentinel Palm Beach Edition
Study: Gene therapy ‘extraordinary’ at fighting blood cancer
An experimental gene therapy that turns a patient’s own blood cells into cancer killers worked in a major study, with more than one-third of sick lymphoma patients showing no sign of disease six months after a single treatment, its maker said Tuesday.
In all, 82 percent of patients had their cancer shrink at least by half at some point in the study.
Its sponsor, Kite Pharma, is racing Novartis AG to win approval of the treatment, called CAR-T cell therapy, in what could become the nation’s first approved gene therapy.
The number of patients in complete remission at six months, 36 percent, is barely changed from partial results released after three months, suggesting the onetime treatment might give lasting benefits for those who do respond well.
“This seems extraordinary, extremely encouraging,” said Dr. Roy Herbst, cancer medicines chief at the Yale Cancer Center.
But the therapy is not without risk. Three of the 101 patients in the study died of causes unrelated to worsening of their cancer, and two of those deaths were due to the treatment.
It was developed at the National Cancer Institute and licensed to Kite. The company plans to seek approval from the U.S. Food and Drug Administration by the end of March.
The treatment involves filtering a patient’s blood to remove key immune system soldiers called T-cells, altering them in the lab to contain a gene that targets cancer, and giving them back intravenously.
Doctors call it a “living drug” — permanently altered cells that multiply in the body into an army to fight the disease.
Patients in the study had non-Hodgkin lymphoma, a blood cancer, and had failed all other treatments. Median survival for such patients has been about six months.