Groundbreaking Gene Therapy Treatment Cures Terminal Cancer Patients

This is huge. Because 1/3rd of the cancer patients — for whom no other treatment worked — were completely cured.


Call it a miracle or a revolution; a groundbreaking gene therapy treatment has cured terminal cancer patients in a clinical trial.

Called CAR-T cell therapy, the treatment radically filters a patient’s blood to remove key immune system cells or T-cells (immune system cells that can be genetically engineered in a lab to identify cancer cells), cuts the brakes, and boosts the patient’s own immune cells so that they are able to attack the cancerous cells more effectively.

Dr. Jeff Wiezorek, senior vice president of clinical development at Kite Pharma, remarked: “These results with axicabtagene ciloleucel are exceptional and suggest that more than a third of patients with refractory aggressive NHL could potentially be cured after a single infusion of axicabtagene ciloleucel.”

In the first six months of its clinical trial sponsored by California-based Kite Pharma (founded less than a decade ago by an Israeli-American oncologist), more than one-third of very sick lymphoma patients confirmed no sign of disease six months after a single CAR-T cell therapy treatment.

Dr. Frederick L. Locke, a blood cancer expert at Moffitt Cancer Center in Tampa who co-led the study, noted: “The numbers are fantastic. These are heavily treated patients who have no other options. The ZUMA-1 study results suggest that axicabtagene ciloleucel could become a new standard of care for patients with refractory aggressive lymphoma.”

CAR-T cell therapy
These scans show a 62-year-old man with non-Hodgkin lymphoma, at left in December 2015, and three months after treatment with Kite Pharma’s experimental CAR-T cell therapy at MD Anderson Cancer Center in Houston.

The 101 patients who participated in the trial had one of three types of non-Hodgkin lymphoma, a type of blood cancer, had failed all other treatments, and had been given only a few months to live.

However, following the first round of CAR-T cell therapy, which took place nine months after the trial began, half the patients are not only alive, but 36% are in complete remission at six months; while 82% of patients have their cancer shrink by at least half.

Dr. Roy Herbst, cancer medicines chief at the Yale Cancer Center, commented: “This seems extraordinary… extremely encouraging.  The worry is how long Kite’s treatment would last and its side effects, which seem manageable in the trials. Follow-up beyond six months is still needed to see if the benefit wanes, but this certainly is something I would want to have available.”

While Kite Pharma is racing Novartis AG to win approval of America’s first approved gene therapy treatment, Marilynn Marchione, chief medical writer at The Associated Press, observed the CAR-T cell therapy is not without risk.

“Of the study participants, 13% developed a dangerous condition where the immune system overreacts in fighting the cancer, but that rate is lower than in some other tests of CAR-T therapy. The rate fell during the study as doctors got better at detecting and treating it sooner.”

Acknowledging the risks, Dr. Locke noted: “Roughly a third of patients developed anemia or other blood-count-related problems, which were easily treated. And 28% had neurological problems such as sleepiness, confusion, tremor or difficulty speaking, but these typically lasted just a few days.”

Full results of the trial will be presented at the American Association for Cancer Research conference in April. Even as Kite Pharma plans to seek approval from European regulators later this year, the National Cancer Institute’s Dr. Steven Rosenberg, pointed out:

“It’s a safe treatment, certainly a lot safer than having progressive lymphoma, and comparable to combination chemotherapy in terms of side effects.”

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