$373,000 per regimen for new treatment

FDA gives its okay to a second gene therapy that can send blood cancer into remission

The FDA has approved a new gene therapy for adults with aggressive non-Hodgkin's lymphoma.

According to a recent story by Denise Grady in The New York Times, Yescarta was okayed for patients who've undergone two regimens of chemotherapy that failed.

The Times article notes that the therapy "is part of the rapidly growing field of immunotherapy, which uses drugs or genetic tinkering to turbocharge the immune system to fight disease. In some cases the treatments have led to long remissions."

The therapy, which turns cells into "living drugs," is made by Kite Pharma.

Approval by the Federal Drug Administration was its "second in a radically new class of treatments that genetically reboot a patient's own immune cells to kill cancer."

The first was Kymriah, made by Novartis. It was okayed in August "for children and young adults with an aggressive type of acute leukemia."

That one costs $475,000 per dose, although the company has said "it will not charge patients who do not respond within the first month after treatment."

Yescarta isn't quite as expensive — $373,000 per treatment.

Dr. Frederick L. Locke

Dr. Frederick L. Locke, a leader of a study of Yescarta (and a specialist at the Moffitt Cancer Center in Tampa in blood cancers), is quoted as saying, "We're excited. We think there are many patients who may need this therapy."

Grady's story says "about 3,500 people a year in the United States may be candidates for Yescarta," which is intended "to be given once, infused into a vein, and must be manufactured individually for each patient."

Side effects, as with many therapies, can be daunting.

Life-threatening issues that stem from the Yescarta treatment can include "high fevers, crashing blood pressure, lung congestion and neurological problems," the story indicates.

According to Grady, two patients in the study that led to the drug's approval, "died from side effects," although doctors — through training and experience — apparently "have learned to manage them better."

The study, which was conducted at the Dana-Farber Cancer institute and Brigham and Women's Cancer Center in Boston, involved 111 patients at 22 hospitals.

"Initially," Grady's piece delineates, "54 percent [of the 101 who received Yescarta] had complete remissions…Another 28 percent had partial remissions [and] after six months, 80 percent…were still alive. With a median follow-up of 8.7 months, 39 percent of the 101 were still in complete remission — a much higher rate that achieved with earlier treatments — and 5 percent still had partial remissions."

Dr. Caron A. Jacobson

Says Dr. Caron A. Jacobson, who helped do the study: "You're really seeing people get their life back. After a couple weeks in the hospital and a couple weeks at home, they go back to work. On its face, it's quite remarkable and revolutionary."

The treatment, the Times explains, "was originally developed at the National Cancer Institute, by a team Dr. Steven Rosenberg led. The institute entered an agreement with Kite in 2012, in which the company helped pay for research and received rights to commercialize the results."

The drug giant Gilead reportedly bought Kite two months ago for $11.9 billion.

"In just several decades, said Dr. Scott Gottlieb, the FDA commissioner, "gene therapy has gone from being a promising concept to a practical solution to deadly and largely untreatable forms of cancer."

A look at many treatments and clinical trials can be found in "Rollercoaster: How a man can survive his partner's breast cancer," a VitalityPress book I, Woody Weingarten, aimed at caregivers.

Prediction: Price of drug therapies will skyrocket

New FDA-approved cancer gene therapy to cost $475,000 for a single course, reports say

Are pharmaceutical companies gouging the public?

Very possibly, as evidenced by a new gene therapy for childhood leukemia that will cost almost half a million dollars for a single course.

Reports yesterday about Federal Drug Administration approval for Novartis' Kymriah, which is the first gene therapy for cancer that it's okayed, say it will cost at least $475,000.

Although some oncologists have described the CAR-T drug as revolutionary, critics fear the price tag could pave the way for future drug costs to skyrocket.

According to a story by Catherine Ho in the San Francisco Chronicle, the way the treatment works is this: A patient's cells are extracted from his or her blood, then sent to a site where they'll be genetically modified. The altered cells are then re-infused into the patient's body.

The FDA this week approved Kymriah as a treatment for childhood B-cell acute lymphoblastic leukemia, a cancer of the blood and bone marrow that's the most common childhood cancer.

The National Cancer Institute has estimated 3,100 patients up to 20 years old are diagnosed with the disease annually. Although the survival rate is 90 percent, that figure drops to as low as 10 percent for those who relapse after a bone marrow transplant. It is that subset of patients who are eligible for cell therapy, whose rate of success is said to be between 70 and 80 percent.

Only a few hundred children and young adults are expected to be treated with Kymriah each year.

Bruno Strigini

A Guardian story, supplemented by Associated Press reporting, said that Bruno Strigini, Novartis' head of oncology, has explained that the high cost of the treatment is an attempt to balance patient access to the drug with ensuring a return on the company's investment.

The Guardian piece also cited a Novartis statement to the effect that the company "was collaborating with Medicare on a plan in which the government would only pay for the treatment if patients responded to them by the end of the first month."

Side effects of the drug include high fevers, diarrhea, vomiting, delirium, loss of balance, and difficulty speaking and understanding.

More information about the exorbitant costs of cancer treatments can be found in  "Rollercoaster: How a man survive his partner's breast cancer," a VitalityPress book I, Woody Weingarten, aimed at male caregivers.