US approves first gene therapy treatment

By Barry Eitel

SAN FRANCISCO

The U.S. announced Wednesday it had approved a cell-based gene therapy in the United States for the first time.

The “historic action” by the Food and Drug Administration (FDA) permits a new treatment that uses a leukemia patient’s own blood to engineer a method to attack the cancer.

Scientists often refer to this type of gene therapy as a “living drug” because it involves using genetically modified immune cells from patients.

The drug Kymriah was developed by Novartis Pharmaceuticals and the University of Pennsylvania. The treatment was approved for certain children and young adult up to the age of 25 who suffer from a form of acute lymphoblastic leukemia (ALL) -- a cancer of blood and bone marrow that is the most common childhood cancer in the United States.

“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, who was appointed to the position by President Donald Trump earlier this year. “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.”

Novartis said the treatment has an 83 percent success rate for a form of leukemia that has proven very hard to treat by other methods.

“Five years ago, we began collaborating with the University of Pennsylvania and invested in further developing and bringing what we believed would be a paradigm-changing immunocellular therapy to cancer patients in dire need,” Novartis CEO Joseph Jimenez said in a statement.

The National Cancer Institute estimates 3,100 patients are diagnosed each year with ALL, which can come in several forms. Less than 10 percent of patients with the most common form of ALL survive longer than five years under current treatment regimens, Novartis said.