Novartis, however, is poised to be the first one to get approved.
It is thought to be likely the FDA will approve the gene therapy and if so, it will be the first such therapy to go on the market.
A year ago, the family was preparing for a bone-marrow transplant when they learned about the cell treatment, which Connor then underwent at Duke University. Diefenbach, who was not involved in researching the drug and has no ties to its manufacturer, Novartis, described its results as "astounding".
Data from the U.S. multicenter trial and single site trial assessing the safety and efficacy of CTL019 among pediatric and young adult patients with r/r B-cell ALL also backed the recommendation and the biologics license application (BLA). ALL is the most common childhood cancer in the US. But if the cancer comes back, the prognosis can be dire. There are about 3,100 new cases of ALL each year, but roughly 70 percent can be pushed into remission by standard therapy.
"This is the beginning of something big", she added.
Statistics show that patients with ALL who fail chemotherapy typically only have a 16 to 30 percent chance of survival. In a briefing document to the ODAC, FDA staffers cautioned that a major consideration for manufacturing CTL019 was the need for "a well-controlled manufacturing process that can consistently produce high-quality vehicle T cells that are safe, pure, and potent".
In a clinical trial, 79 percent of patients given the Novartis therapy were alive a year later. The majority were also relapse-free in the same time frame.
"The word is out there that this is different from traditional chemotherapy", Dr. Nichols said. That is because it was the first global pediatric CAR-T cell therapy registration trial.
Dr. Carl June, a lead researcher at the University of Pennsylvania who helped create the new treatment, called the re-engineered T-cells "serial killers". Cancer cells arise from normal cells, so the immune system doesn't always recognize that anything is wrong.
But there are already concerns that supply chain costs will be transferred to the final products, potentially slapping a $300,000-plus list price tag onto the gene therapies. Brody said it could take decades to conclusively say this does not happen. "As an investor I've never seen anything like it". "It's not an opinion".
This last-ditch treatment, CAR-T therapy, retrains a patient's immune system to home in on tumor cells and kill them.
"You can put someone else's red blood cells into you", he said.
Only a few thousand people are affected by the B-cell acute lymphoblastic leukemia that proves stubborn in the face of conventional treatments for about 15 percent of cases, a lot of them in children and young adults. Baldrick's Pediatric Cancer Dream Team.
"This is truly a turning point in the management of this disease", said Maris.
"While CAR-T is a promising new type of immunotherapy, it is not commercially available and we have yet to complete our evaluation", said T.J. Crawford, a spokesman for Aetna Inc. Novartis' therapy is not identical to the CAR-T cells used in those trials, which were administered in adults, but the deaths cast a pall over the entire field.
The FDA previously approved Amgen's T-VEC, which injects a modified herpesvirus into melanoma cells, causing them to rupture.
CTL019 offers a potential lifeline, with clinical trial evidence showing remission rates of around 83%, but the FDA's Oncologic Drugs Advisory Committee (ODAC) was more concerned with the safety of the therapy. Cripe was a member of the FDA advisory panel that voted Wednesday to support the drug's approval. The agency is expected to rule on the drug by the end of September.
The FDA didn't respond to a request for comment.