Gene therapy found to save lives in U.S. study

An experimental gene therapy that turns a patient’s blood cells into cancer killers worked in a major study, with more than one-third of very sick lymphoma patients showing no sign of disease six months after a single treatment, its maker says.

Eighty-two per cent of patients had their cancer shrink at least by half at some point in the study. Its sponsor, Californiabased Kite Pharma, is racing Novartis AG to become the first to win approval of the treatment, called CAR-T cell therapy, in the U.S. It could become the U.S.’s first approved gene therapy.

One hopeful sign is that the number of patients in complete remission at six months — 36 per cent — is barely changed from partial results released after three months, suggesting this one-time treatment might give lasting benefits for those who do respond well.

“This seems extraordinary, extremely encouraging,” said independent expert Dr. Roy Herbst, cancer medicines chief at the Yale Cancer Center in New Haven, Connecticut.

The worry has been how long Kite’s treatment would last and its side-effects, which, he said, seem manageable in the study. Followup beyond six months is still needed to see if the benefit wanes, Herbst said, but he added: “This certainly is something I would want to have available.”

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 deemed due to the treatment.

It was developed at the U.S. government’s National Cancer Institute, then licensed to Kite. The U.S. Leukemia & Lymphoma Society helped sponsor the study.

Results were released by the company and have not been published or reviewed by other experts. Full results will be presented at the American Association for Cancer Research conference in April. The company plans to seek approval from the U.S. Food and Drug Administration by the end of the month and in Europe later this year.

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 one of three types of non-Hodgkin lymphoma, a blood cancer, and had failed all other treatments. Median survival for such patients has been about six months.

Kite study patients seem to be living longer, but median survival isn’t yet known. With nearly nine months of followup, more than half are still alive.

Six months after treatment, 41 per cent still had a partial response (cancer shrunk at least in half) and 36 per cent were in complete remission (no sign of disease).

“The numbers are fantastic,” said Dr. Fred Locke, a blood cancer expert at Moffitt Cancer Center in Tampa, Florida, who co-led the study and has been a paid adviser to Kite.

“These are heavily treated patients who have no other options.”

One of his patients, 43-yearold Dimas Padilla of Orlando, was driving when he got a call saying his cancer was worsening, chemotherapy was no longer working and there was no match to enable a second try at a stemcell transplant.

“I actually needed to park. I was thinking how am I going to tell this to my mother, my wife, my children,” he said. But after CAR-T therapy last August, he saw his tumours “shrink like ice cubes” and is now in complete remission.

“They were able to save my life,” Padilla said.