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, the first available in the United States, could be approved by the FDA by the end of September, forging a new path in the immunotherapy frontier.
Dr. 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.”
“This is a major advance,” said panel member Dr. Malcolm A. Smith of the National Cancer Institute. He said the treatment is “ushering in a new era.”
Novartis, the drugmaker behind what is called 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 — which generally follows the advice of its experts — it would be the first gene therapy approved in the United States.
The 10-0 vote by the experts came after lengthy discussion and impassioned pleas from the fathers of two young patients whose lives were saved by the therapy.
“Our daughter was going to die and now she leads a normal life,” said Tom Whitehead of Philipsburg, Pennsylvania. His daughter Emily, now 12, was the first child to receive the experimental therapy, five years ago. “We believe when this treatment is approved, it will save thousands of children’s lives around the world.”
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, New Jersey.
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 will be an estimated 22 days, Novartis officials told the committee Wednesday.
In a key test, results of the therapy were far better than chemotherapy and even newer types of cancer drugs. Of the 52 patients whose results were analyzed, 83 percent had complete remission, meaning their cancer vanished. Most patients suffered serious side effects but nearly all recovered.
The panel on Wednesday homed in on how best to handle possible short-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.
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.
One of the big issues in CAR-T cell therapy — the cost — wasn’t discussed because that is beyond the FDA’s purview.
Other biotech and pharmaceutical companies are developing types of gene therapy to treat solid cancers and rare gene-linked diseases.
A few products have been approved elsewhere, including in Europe — GlaxoSmithKline’s Strimvelis for a deadly condition called severe combined immunodeficiency and launched with a $670,000 price tag.
This report includes material from the Associated Press.