The Food and Drug Administration (FDA) is on the verge of approving the first-ever gene therapy treatment for use in the United States.
If it goes through as expected, the therapy will be used to treat children and young adults with advanced acute lymphoblastic leukemia (ALL).
The FDA’s Oncologic Drugs Advisory Committee earlier this month recommended that the agency approve Novartis’ experimental chimeric antigen receptor (CAR-T) therapy, CTL019 (tisagenlecleucel).
It’s an individualized therapy in which a person’s T cells are removed from their blood and reengineered to fight cancer. Then they’re infused back into the patient.
That’s why it’s called a “living drug.”
In a recent clinical trial, 83 percent of patients experienced complete remission or complete remission with incomplete blood count recovery within three months. The FDA is expected to make a decision in September. The agency usually follows its committee’s recommendations.
Dr. Santosh Kesari is a neuro-oncologist and chair of the Department of Translational Neuro-oncology and Neurotherapeutics at the John Wayne Cancer Institute at Providence Saint John’s Health Center in California.
Kesari told Healthline that if approved, the therapy will be a revolutionary breakthrough in cancer treatment.
“It will be the first application of this type — combining gene therapy and immune therapy to modify a patient’s own cells to go attack cancer cells,” he said.
Kesari explained that this application could work in other cancers where there’s a specific target.
He pointed to a City of Hope case study involving a 50-year-old man with recurrent multifocal glioblastoma, a type of brain cancer.
The treatment was part of a phase I clinical trial to test the safety of CAR-T therapy when delivered directly to brain tumors. A successful response was sustained for more than seven months, longer than would usually be expected.
“So, there is potential for application in solid tumors if we identify the right marker, making sure we manage side effects,” said Kesari.
n an email interview with Healthline, Dr. Swati Sikaria, medical oncologist from Torrance Memorial Medical Center in California, explained that the goal is to choose a target as unique to the cancer as possible while avoiding damage to the noncancerous cells of the body.
“This initial success paves the way for creating CAR-T cells with targets for other malignancies,” she explained.
“Whether the success in ALL can be replicated in other types of cancers, I’m cautiously optimistic. We’ll have to see what ongoing and future clinical trials show. The most notable progress has been in multiple myeloma as well as glioblastoma, the type of tumor Senator [John] McCain was recently diagnosed with,” said Sikaria.