Approval in pediatric ALL now seems all but assured; the FDA rarely bucks the advice of its advisory committees.
In clinical trials, 83% of patients who received CTL019 treatment were free of cancer after three months. "I think it will be important in the commercial phase to assess both failure rates and turnaround times for CAR-T production", said Krishna Komanduri, director of the Sylvester Comprehensive Cancer Center Adult Stem Cell Transplant Program at the University of Miami, who has treated patients with other experimental CAR-Ts, but not the one from Novartis.
Austin Schuetz, one of a handful of CAR-T patients to achieve multi-year remission of his cancer, requires biweekly infusions of immunoglobulin to replace B-cells wiped out by the engineered cancer-killing T-cells. "We believe when this treatment is approved, it will save thousands of children's lives around the world".
The Emily Whitehead Foundation, which raises funds for immunotherapy research, tweeted pictures on Wednesday of Emily before and after her treatment, along with a quote from her father's comments to the panel, saying the treatment killed Emily's cancer in "just 23 days".
Fatalities due to cerebral oedema have occurred with other CAR-T therapies however - notably Juno Therapeutics' JCAR015 which was discontinued in March and another candidate from Kite Pharma - and as a result the safety of CTL019 had been expected to come under close scrutiny.
The FDA is expected to make a decision on approving Novartis' treatment within the next few months.
The committee's approval for CAR-T therapy applies to children and young adults, ages 3 to 25, with a form of leukemia called relapsed or refractory B-cell acute lymphoblastic leukemia (ALL). That happens to more than 600 patients in the USA each year.
The agency is expected to decide by November 29 whether to approve Kite's CAR-T, axi-cel, for treatment of adults with advanced and aggressive lymphoma. This is the most common type of cancer among children, according to the National Cancer Institute.
In drug trials, the immunotherapy results worked far better than chemotherapy and newer types of cancer drugs.
The main side effect of CTL-019 is cytokine release syndrome, a blistering immune reaction to the drug and the detritus of dying cancer cells. For now, however, patient advocates are cheering what may well be the first step on the road to a revolutionary new kind of personalized cancer medicine. In 2015, the company acquired a cell manufacturing facility in Morris Plains, New Jersey, and past year it said it plans to develop centers in Europe and Japan. When the modified cells are returned to the patient, they seek out and destroy the cancer. Doctors call it a "living drug" - permanently altered cells that continue to multiply in the body to fight the disease.
Whitehead's treatment process took about 16 weeks, which the FDA advisors said can be too long for some desperately ill patients. As such, the treatment is tailored for each patient.
Spokeswoman for Novartis, Julie Masow, has said the company will file for approval of the treatment in the European Union later this year.
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".
The drug company Novartis presented its case for the treatment to be approved by the FDA for use on patients.