Groundbreaking Penn Cancer Therapy Gets FDA Approval

To start, the first-of-its-kind gene therapy will be used in the treatment of leukemia.

In what the FDA is a calling a “historic action,” the federal administration on Wednesday approved the country’s first gene therapy treatment that can be used to fight cancer and other life-threatening diseases.

Researchers at the University of Pennsylvania and CHOP have been developing the therapy, called CAR T, for years. It’s been deemed the next frontier of cancer treatment because it uses patients’ own genetically immune cells to attack the disease. Standard immunotherapies have involved injections of synthetic substances intended to create various cancer-attacking reactions in the immune system. 

The FDA’s decision clears a Novartis drug called Kymriah, which was specifically approved to treat children and young adults up to age 25 suffering from a form of acute lymphoblastic leukemia. In 2012, Penn and Novartis launched a partnership to research and bring Kymriah to market.

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” said FDA commissioner Scott Gottlieb in a statement. “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.”

The CAR T therapy was first used by a team of researchers led by Dr. Carl June to treat Emily Whitehead, who’s been free of leukemia for the five years since. In a recent clinical study of 63 pediatric and young adult patients, Kymriah induced remission in 83 percent of patients within three months.

“Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease,” said Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research. “Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials.”

On Wednesday, Novartis said the drug will be pricey, but the company is working on ways to ensure that all patients who need it can afford it. The one-time treatment will cost $475,000 for patients who respond. Those who do not respond within a month will not be charged. And though the drug may be cancer’s newest miracle cure, it isn’t without severe side effects. The FDA warns that the therapy can cause very high fevers, serious infections, and low blood pressure.

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