Gene-changing leukemia therapy nears approval

Feds open door to okaying cancer-fighting treatment that genetically alters patient's cells

An FDA panel has given a unanimous vote of confidence to a gene-altering leukemia therapy.

According to a story by Denise Grady in The New York Times today, the Food and Drug Administration panel has unanimously recommended, following successful clinical trials, that "the agency approve the first-ever treatment that genetically alters a patient's own cells to fight [lethal] leukemia, transforming them into what scientists call 'a living drug' that powerfully bolsters the immune system to shut down the disease."

The agency is likely to accept the recommendation, Grady's story indicates, an action that would make Novartis' experimental treatment the first gene therapy to reach the market — ending in September a decades-long competition by researchers and drug companies.

"To use the treatment," the article continues, "a separate treatment must be created for each patient" — at enormous cost: between $300,000 and $600,000 for the requisite single infusion, according to analysts.

The CAR T-cell technique has multiple steps: Cells removed from the patient must be frozen, thawed and processed at a plant run by the manufacturer, frozen again and shipped back to the treatment center.

Severe side effects — including "fever, crashing blood pressure, lung congestion" — can potentially be life-threatening, but scores of patients have experienced long remissions and, in some cases, possible cures, the Times story maintained.

On the other hand, "re-engineering cells for treatment sometimes take four months," it noted, leaving some patients "so sick that they died before their cells came back."

Dr. Carl H. June

Dr. Carl H. June, an immunology professor and leader of the University of Pennsylvania research team that developed the treatment and licensed it to Novartis, has labeled the altered cells "serial killers."

One cell purportedly can destroy up to 100,000 cancer cells.

The panel's recommendation specifically dealt with leukemia "that has resisted treatment, or relapsed, in children and young adults ages 3 to 25" — for patients who typically had a bleak prognosis.

The clinical trials, according to a story by Laurie McKinley in The Washington Post yesterday, took place "in almost a dozen countries," with results of 83 percent of patients going into remission.

That story also indicated that "researchers are exploring CAR T-cell therapy's use for multiple myeloma and chronic lymphocytic leukemia [and] are also tackling a far more difficult challenge — using the therapy for solid tumors in the lungs or brain."

Meanwhile, Dr. Stephen Schuster, a Penn oncologist and leader of a lymphoma study, is quoted by the Post as saying, "We're saving patients who three or four years ago we were at our wit's end trying to keep alive."

Details on a variety of cancer therapies can be found in "Rollercoaster: How a man can survive his partner's breast cancer," a VitalityPress book I, Woody Weingarten, aimed at male caregivers.