By: Laurie McGinley
Food and Drug Administration advisers on Wednesday enthusiastically endorsed a first-of-its-kind cancer treatment that uses patients’ revved-up immune cells to fight the disease, concluding that the therapy’s benefits for desperately ill children far outweigh its potentially dangerous side effects.
The unanimous recommendation from the Oncologic Drugs Advisory Committee means the treatment could be approved by the FDA by the end of September, forging a new path in the immunotherapy frontier.
Timothy Cripe, a panel member who is an oncologist with Nationwide Children’s Hospital in Columbus, Ohio, called the treatment the “most exciting thing I’ve seen in my lifetime.”
Novartis, the drugmaker behind the CAR T-cell therapy, is seeking approval to use it for children and young adults whose leukemia doesn’t respond to traditional treatments — a group that numbers 600 or so patients a year in this country. But the approach also is being tested for a range of diseases from non-Hodgkin lymphoma and multiple myeloma to solid tumors.
If cleared by the FDA, it would be the first gene therapy approved in the United States. But unlike traditional gene therapy, the new treatment doesn’t replace disease-causing genes with healthy ones. Instead, it uses technology to reprogram immune cells called T cells to target and attack malignancies.
When a patient is treated under the Novartis process, T cells are extracted from a patient’s blood, frozen and sent to the company’s plant in Morris Plains, N.J. There, the cells are genetically modified to attack the cancer, expanded in number, refrozen and shipped back to the patient for infusion.
Once inside the body, the cells multiply exponentially and go hunting for the CD19 protein, which appears on a kind of white blood cell that can give rise to diseases, such as leukemia and lymphoma. The turnaround time for manufacturing the therapy, called “vein-to-vein” time, will be an estimated 22 days, Novartis officials told the committee Wednesday.
From the start of Wednesday’s meeting, committee members made clear that they were not concerned about the treatment’s efficacy, which has been well established — 83 percent of patients went into remission in the pivotal Novartis trial. Rather, the panel homed in on how to best to handle possible shot-term toxicities, as well as long-term safety risks and manufacturing quality.
Most patients in the Novartis study experienced something called cytokine release syndrome, which causes fever and flulike symptoms that can range from mild to extremely severe, said Stephan Grupp, an oncologist at the Children’s Hospital of Philadelphia who led the Novartis trial. Some patients in that study also had neurological problems, including seizures and delirium. But there were no cases of fatal brain swelling, as occurred in another company’s trial, Grupp said.
To try to ensure safety, Novartis is limiting the therapy’s availability to 30 to 35 medical centers where personnel have had extensive training with the treatment. The company also plans to post Novartis employees at hospitals using the therapy and to follow patients for up to 15 years.
During the committee meeting, hundreds of people packed the hearing room at FDA headquarters in Silver Spring, Md., including prominent scientists, such as Carl June of the University of Pennsylvania, who developed the treatment. Though the FDA isn’t required to follow the guidance of its advisory committees, it usually does.
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