Novartis Oncology CEO Bruno Strigini said: "The panel's unanimous recommendation in favor of CTL019 moves us closer to potentially delivering the first-ever commercially approved CAR-T cell therapy to patients in need". Approval in pediatric ALL now seems all but assured; the FDA rarely bucks the advice of its advisory committees.
One of the parents testifying was Tom Whitehead, the father of Emily, who was the first patient to receive the immunotherapy.
All four are developing chimeric antigen receptor T-cell therapies (CAR-T), which harness the body's own immune cells to recognize and attack malignant cells.
The approval of the new treatment has potential consequences that extend well beyond treatment of leukemia.
"We believe that when this treatment is approved it will save thousands of children's lives around the world", Whitehead told the meeting, his voice cracking as he looked at his daughter beside him.
Novartis officials said they would follow patients for at least 15 years and investigate any cases of secondary cancers.
It's not without risks - some children who received the treatment spiked fevers of as high as 107 degrees and not all of them were cured.
CTL019 is an autologous vehicle T-cell therapy. Beyond representing a change in the treatment of relapsed/refractory ALL, approval for Tisagenlecleucel would be a landmark decision - ushering in CAR-T as a new class of personalized cancer treatments.
"When fully commercialized, this [CTL019] therapy will no doubt save the lives of many children and young adults who have had no other effective therapy for relapsed and refractory B-cell acute lymphoblastic leukemia [ALL]", said Dr. John Maris, a pediatric oncologist at The Children's Hospital of Philadelphia.
More than 600 patients in the United States each year reach that critical stage. An approval decision for the Kite CAR-T is expected on November 29.
In a test conducted, gene therapy results exceeded chemotherapy, and even newer types of cancer medicines.
What are the side effects? The drug was tried on patients whose cancer had relapsed or didn't respond to standard treatments.
Scientists are calling the treatment "a living drug" as it harnesses the immune system to beat cancer, according to The New York Times. After the cells are removed by medical personnel, they are frozen, shipped to Novartis (the therapy's maker), processed, refrozen, and shipped back to the medical center. For one thing, the manufacturing process is extremely intensive since each individual batch of the drug needs to be tailored to each individual patient.
"The potential for CAR-T cells is enormous".
The FDA approval of Novartis' new therapy, he said, gives the industry "our first glimpse from a commercial and regulatory perspective about how the FDA is thinking about this space". Speaking during the public hearing, Megan Polanin, a senior fellow at the National Center for Health Research who specializes in evidence-based mental health interventions, urged the FDA to take it slow in approving the vehicle T therapy. The dramatic effect of the treatment, known for years as CTL-019, was never questioned at the meeting.
The vote also underscores ODAC's ingrained acceptance of moderate risk when it comes to treating patients dying of cancer.
Patients with ALL who fail chemotherapy typically have only a 16 percent to 30 percent chance of survival.
There are quite a few risks with the CTL019 treatment by Novartis.