Gene therapy to fight blood cancer shows encouraging results in study
02 March 2017
An experimental gene therapy that turned a patient's own blood cells into cancer killers has worked in a major study, with over a third of very sick lymphoma patients showing no sign of the disease six months after a single treatment, according to the pioneers of the therapy.
The therapy was able to shrink cancer by at least half in 82 per cent of patients, at some point in the study.
California-based Kite Pharma, which is the therapy's sponsor is racing Novartis to win approval of the treatment, called CAR-T cell therapy, in the US. The therapy could become the nation's first approved gene therapy.
According to experts, the number of patients in complete remission at six months, 36 per cent had hardly changed from the partial results released after three months, which indicated lasting benefits for those who responded well to the one-time treatment.
"This seems extraordinary ... extremely encouraging," said one independent expert, Dr Roy Herbst, cancer medicines chief at the Yale Cancer Center, AP reported.
There had been some worry as to how long the treatment would last and its side effects, which he said seemed manageable in the study.
He added, follow-up beyond six months was still needed to see if the benefit waned, adding, "this certainly is something I would want to have available."
CAR T-cell drugs are made by genetically altering patients' own T-cells to add a component of antibodies that made more effective at indentifying and killing cancer cells.
CAR T cell therapies were in various states of development in laboratories of a number of drugmakers like Juno Therapeutics Inc, Novartis AG and bluebird bio Inc.
Kite said it planned to submit a marketing application for the therapy with European regulators this year. According to Jefferies analyst Biren Amin, he expected accelerated approval for drug from the US Food and Drug Administration by the end of 2017, Reuters reported.