FDA OKs ‘living drug’ for childhood leukemia

The Food and Drug Administration approved a groundbreaking cancer treatment for childhood leukemia.

The Food and Drug Administration on Wednesday approved a groundbreaking cancer treatment for childhood leukemia that uses patients’ own genetically altered immune cells to fight the disease. It is the first gene therapy to be allowed in the United States - a “historic” approval, the agency said.

The decision clears the Novartis drug, called Kymriah, for treatment with children and young adults whose illness doesn’t respond to traditional approaches. That group numbers only 600 or so patients a year in this country. But the customized treatment is also being tested for a range of diseases from non-Hodgkin lymphoma and multiple myeloma to solid tumors, and its approval signals a new chapter in treating cancer by mobilizing the body’s own immune system.

“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 Scott Gottlieb said 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.”

Unlike traditional gene therapy, the new treatment doesn’t replace diseasecausing genes with healthy ones. Instead, in a complicated process developed at the University of Pennsylvania, white blood cells called T cells are extracted from the patient and sent to a manufacturing center to be modified to include a new gene that directs the T cells to target and kill leukemia cells. Those T cells then are infused back into the patient.

The treatment is approved for patients up to 25 years of age with B-cell acute lymphoblastic leukemia that doesn’t respond to treatment or that returns after initial treatment - as happens in up to 20 percent of patients, according to the FDA.

In a pivotal study of five dozen patients, the drug induced remission in more than 80 percent. But some patients relapsed, and doctors are still monitoring the rest to see how long the drug’s effect will last.

The treatment, which is expected to be costly, can cause serious side effects including high fevers and neurological problems. A drug called tocilizumab can often successfully treat those complications.

Because of the risks involved, the FDA is requiring that hospitals and medical staff using Kymriah be specially trained.

Novartis executives said in a call with reporters that the therapy would cost $475,000 per treatment and that the company expects to have 20 certified medical centers ready within a month for patients to begin undergoing treatment. The company will introduce a pricing model based on how well the drug works; a patient will not be charged for the therapy if it doesn’t show signs of working within a month.

Of the 600 patients in the United States who are eligible for Kymriah, most are children who have insurance coverage through their parents’ insurance, Medicaid or the federal Children’s Health Insurance Program. Novartis has set up a patient-assistance program that can be used to help families who lack insurance or have coverage that falls short for this treatment. It also has a program to assist with travel expenses.

Moving forward, Chief Executive Joe Jimenez said, the company plans to price the drug differently depending on which specific disease it is treating. If Kymriah is later approved for another type of cancer in which it is less effective, the price could theoretically be lower.

Novartis plans to file for FDA approval of Kymriah for a type of non-Hodgkin lymphoma that affects adults later this year.

California-based Kite Pharma, which is being acquired by Gilead Sciences, also has applied for FDA approval for use of its own CAR T-cell therapy in lymphoma patients.