The Food and Drug Administration requires companies that make specialized cancer treatments known as CAR-T to add a boxed warning that the treatments themselves can cause cancers.
The agency noted that the benefits still outweigh the risks of the treatment, which involves removing a type of white blood cell — T cells — and then genetically engineering them to create proteins called chimeric antigen receptors (CARs). Infused back into the patient’s blood, the modified cells allow the T cells to attach to the cancer cells and kill them.
But the treatments, which mainly treat blood cancers including multiple myeloma, have already drawn warnings of dangerous immune reactions and neurological risks. And the new warning follows reports of about 25 cases of secondary cancers that federal health officials and others suspected were caused by CAR-T therapies, though more research may be needed to establish a clear link. The treatment has been used by at least 27,000 patients since it was first approved by the FDA in 2017, the agency said.
Cancer patients receiving CAR-T therapies tend to have few options left and are unlikely to change course even with the new warning, said Dr. John DiPercio, an oncologist at Washington University in St. Louis.
“The risk of not getting this treatment for most patients who receive it is rapid disease progression or certain death,” he said.
The FDA raised concerns about the treatments’ negative effects late last year.
In letters dated Jan. 19, the agency outlined warnings to be included by some of the companies making CAR-T therapies, which were also ordered to monitor patients for secondary cancers and report any to the FDA. Secondary cancers can lead to hospitalization or death, the agency noted, requiring drug companies to provide warnings on drug labels that secondary cancers “can occur as soon as weeks after the infusion and can include fatal outcomes.”
The FDA issued letters to these companies: Bristol-Myers Squibb, maker of Abecma. Juno Therapeutics, a Bristol-Myers Squibb Company, maker of Breyanzi. Johnson & Johnson’s Janssen Biotech, maker of Carvykti; Novartis, of Kymriah; and Kite Pharma, of Yescarta.
Given the dismal prognosis of patients considering CAR-T therapies, Dr. DiPersio, the new warning amounted to “much ado about nothing.” He said he hoped the news would not discourage further investment or study of treatments for other serious medical conditions. Some pharmacists are studying the use of CAR-T therapy to treat lupus, an autoimmune disease.
“We can’t create such a fearful environment where this approach is pushed away from companies and researchers because it’s seen as too dangerous — because it’s not,” he said.
Financial analysts also predicted little fallout, Brian Abrahams, an analyst at RBC Capital Markets, said in an email.
“It’s still a very rare side effect,” he said. “These are sick patients receiving these therapies, and most CAR-T therapies have shown very clear benefits, including prolonged survival — so we wouldn’t expect these label changes to substantially reduce the use of the agents.”
