‘Astounding’ gene therapy cancer drug heading for FDA approval

A new gene therapy drug, the first in its class, was recommended for approval to the US Food and Drug Administration by an advisory committee on Wednesday. If approved by the FDA, the agency would consider it the first gene therapy to hit the market.

The drug may provide a second chance to some leukemia patients whose first-line drugs have failed.

A panel of experts voted to endorse the immunotherapy drug, known as tisagenlecleucel, which treats a type of leukemia that is more common among children. Ten committee members voted in favor, and one left early without voting. None voted against.

The drug enables patients’ own immune cells to recognize and kill the source of the cancer: a different immune cell gone awry.

“Which one wins is really the question of life or death,” said Dr. Catherine Diefenbach, clinical director of lymphoma at the NYU Perlmutter Cancer Center. Diefenbach, who was not involved in researching the drug and has no ties to its manufacturer, Novartis, described its results as “astounding.”

The research presented to the committee studied the drug as a treatment for the relapse of a blood cancer known as B-cell acute lymphoblastic leukemia, or ALL. This is the most common type of cancer among children, according to the National Cancer Institute.

Nearly 5,000 people were diagnosed with acute lymphoblastic leukemia in 2014, the most recent year on record, according to the US Centers for Disease Control and Prevention. Although more than half of those with this diagnosis were children and teens, they represented only 14% of those who died that year.

The vast majority of people with ALL recover through other treatments, including chemo, radiation and stem-cell transplantation. But if the cancer comes back, the prognosis can be dire.

“The patients who are left behind when chemotherapy doesn’t work are left in really tough shape,” Dr. Stephan Grupp, director of the Cancer Immunotherapy Program at Children’s Hospital of Pennsylvania, said…

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