The Food and Drug Administration announced Wednesday that it has approved the first CAR T immunotherapy in the United States, a huge milestone for the group of breakthrough cancer treatments.
The drug, which genetically alters immune cells to fight cancer, was developed by Switzerland-based biomedical giant Novartis and was approved to treat children and young adults with a subset of acute lymphoblastic leukemia, or ALL. It will go on the market under the name Kymriah.
The approval is huge for those working in immunotherapy, including Seattle’s Fred Hutchinson Cancer Research Center, which has been researching immunotherapy treatments for decades; and its spin-out Juno Therapeutics, a public biotech company working to develop and commercialize many of those treatments.
“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” FDA Commissioner Dr. Scott Gottlieb said in a press release. “New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses. At the FDA, we’re committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving.”
Immunotherapies have produced incredible results in some patients. The FDA said a major factor behind approving Kymriah was its success rate: 83 percent of patients treated with the drug in trials went into remission, a staggering number for the kinds of extremely sick patients that took it.
Juno has also seen incredible success rates with some of its therapies, including a 60 percent remission rate in an early trial of JCAR017. That drug is aimed at Lymphoma, another blood cancer.
GeekWire has reached out to Juno for comment on the approval and will update this story when we hear back. Juno’s stock dipped just over one percent following the announcement of Kymriah’s approval. It was given a 40 percent boost this week when Kite Pharma, another major immunotherapy developer, announced it had reached a deal to be acquired for $11.9 billion.
Despite some successes, there are many questions surrounding the treatments. Although they have been successfully applied to blood cancers, using them against solid tumors is much more tricky, and they can often have severe side effects.
The company pulled its most advanced drug candidate earlier this year following five patient deaths in clinical trials, which put it behind in the race to be first to market with a CAR T immunotherapy.
Researchers at Fred Hutch are tackling many of the persistent issues with CAR T therapies, and have already begun joint clinical trials with Juno to test therapies for some solid tumor cancers.
Access is also an issue: CAR T immunotherapies are very complex and costly to make, meaning they will be astronomically pricey.
“Approvals are an important step, but they’re just the beginning,” Fred Hutch immunotherapy leader Dr. David Maloney said in an emailed statement. “The safety, availability and affordability of these new immunotherapies can be improved, but to achieve these goals we need an increase in industry partnerships, more corporate investments and continued government funding.”
He added, “This increased collaboration and funding will allow us to make further investments in fundamental research and development into how the immune system interacts with cancer, and to pursue advances in cell-engineering techniques so that clinical trials can be accelerated and these experimental therapies be made available to more patients.”