Thursday 19 October 2017

New York Tines/Dennis Grady: F.D.A. Approves Second Gene-Altering Treatment for Cancer

HealthF.D.A. Approves Second Gene-Altering Treatment for Cancer

By DENISE GRADY OCT. 18, 2017
Continue reading the main story
Share This Page

    Share
    Tweet
    Email
    More
    Save

Photo
Cells taken from cancer patients are genetically engineered to fight cancer at a Kite processing facility. Credit Kite Pharma

The Food and Drug Administration on Wednesday approved the second in a radically new class of treatments that genetically reboot a patient’s own immune cells to kill cancer.

The new therapy, Yescarta, made by Kite Pharma, was approved for adults with aggressive forms of a blood cancer, non-Hodgkin’s lymphoma, who have undergone two regimens of chemotherapy that failed.

The treatment, considered a form of gene therapy, transforms the patient’s cells into what researchers call a “living drug” that attacks cancer cells. It 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 results are pretty remarkable,” said Dr. Frederick L. Locke, a specialist in blood cancers at the Moffitt Cancer Center in Tampa, and a leader of a study of the new treatment. “We’re excited. We think there are many patients who may need this therapy.”

He added, “These patients don’t have other options.”

About 3,500 people a year in the United States may be candidates for Yescarta. It is meant to be given once, infused into a vein, and must be manufactured individually for each patient. The cost will be $373,000.
Continue reading the main story

Advertisement
Continue reading the main story

The treatment 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.

Largely on the strength of the new drug and related research, the drug giant Gilead purchased Kite in August, for $11.9 billion.

“Today marks another milestone in the development of a whole new scientific paradigm for the treatment of serious diseases,” the F.D.A. commissioner, Dr. Scott Gottlieb, said in a statement. “In just several decades, gene therapy has gone from being a promising concept to a practical solution to deadly and largely untreatable forms of cancer.”

Side-effects can be life-threatening, however. They include high fevers, crashing blood pressure, lung congestion and neurological problems.In some cases, patients have required treatment in an intensive care unit. In the study that led to the approval, two patients died from side effects. Doctors have learned to manage them better, but it takes training and experience.

Partly for that reason, Yescarta, like Kymriah, will be introduced gradually, and will be available only at centers where doctors and nurses have been trained in using it.

“Ten to 15 authorized institutions will be ready to go at the time of the launch,” a spokeswoman for Kite, Christine Cassiano, said. “In 12 months, we expect to have 70 to 90. There’s a lot that goes into it, making sure each institution is ready to go.”

Companies have been racing to develop new forms of immunotherapy. The first cell-based cancer treatment — Kymriah, made by Novartis — was approved in August for children and young adults with an aggressive type of acute leukemia. It will cost $475,000, but the company has said it will not charge patients who do not respond within the first month after treatment. Novartis is expected to ask the F.D.A. to approve Kymriah for lymphoma and other blood cancers as well, and may vary its price depending on how well it works for those diseases.

Advertisement
Continue reading the main story

Kite also plans to seek approval for other blood cancers, but does not plan to vary Yescarta’s price, said Ms. Cassiano.

The company also hopes that Yescarta will eventually be approved for earlier stages of lymphoma, rather than being limited to patients with advanced disease who have been debilitated by multiple types of chemotherapy that did not work, said Dr. David D. Chang, Kite’s chief medical officer and executive vice president for research and development.

“This is the beginning of many developments in cell therapy in the next few years,” Dr. Chang said in an interview.
Newsletter Sign Up
Continue reading the main story
The Well Newsletter

Get the best of Well, with the latest on health, fitness and nutrition, delivered to your inbox every week.
You agree to receive occasional updates and special offers for The New York Times's products and services.

    See Sample Manage Email Preferences Privacy Policy Opt out or contact us anytime

He said the F.D.A. had “embraced” the concept of cell therapy, designating it a breakthrough and accelerating the approval process to speed its availability to cancer patients, many of whom do not have time to wait.

Kite and Novartis also hope to produce cell therapies for so-called solid tumors — like those of the lung, prostate, breast and colon — which account for about 90 percent of all deaths from cancer.

Before it was approved and named Yescarta, Kite’s treatment was known by other names: axi-cel, axicabtagene ciloleucel, or KTE-C19.

The study that led to approval enrolled 111 patients at 22 hospitals; 101 of them received Yescarta. They had one of three diseases: diffuse large B-cell lymphoma, primary mediastinal B-cell lymphoma or transformed follicular lymphoma.

Initially, 54 percent had complete remissions, meaning that their tumors disappeared. Another 28 percent had partial remissions, in which tumors shrank or appeared less active on scans. After six months, 80 percent of the 101 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 than achieved with earlier treatments — and 5 percent still had partial remissions.

Advertisement
Continue reading the main story

“Many patients were seriously contemplating their own mortality,” said Dr. Caron A. Jacobson, who helped conduct the study at the Dana-Farber Cancer Institute and Brigham and Women’s Cancer Center in Boston. “We would be talking to them about other clinical trials, but also about hospice care and quality of life and comfort. 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 requires removing millions of a patient’s T-cells — a type of white blood cell that is critical to the immune system — freezing them and shipping them to Kite to be genetically engineered to kill cancer cells. The process reprograms the T-cells to attack B-cells, normal parts of the immune system that turn malignant in certain blood cancers. The revved-up T-cells — now known as “CAR-T cells” — are then frozen again and shipped back to the hospital to be dripped into the patient. The turnaround time is about 17 days.

Kite’s cell-processing facility, in El Segundo, Calif., can provide the treatment for 4,000 to 5,000 patients a year, Ms. Cassiano said, adding that the company has applied for approval in Europe, and if it is granted, will probably build a plant there.

Tina Bureau, a fifth-grade teacher from Queensbury, N.Y., was one of the lymphoma patients in the study. Previously, she’d had several types of chemotherapy.

“The cancer would shrink but then it would come right back,” she said.

Last spring, she had the T-cell treatment at the Dana-Farber Cancer Institute and Brigham and Women’s Hospital in Boston. The side effects were ferocious.

“You don’t even recognize your family members,” Ms. Bureau said. “I had some bleeding on my brain, and had to be put in intensive care. The week it was happening, I don’t remember a lot. It was much more difficult for my family than me.”

Within a month, she had a complete remission, which has continued. She is back at work, full time.

“Yes, it can pose life threatening problems,” Ms. Bureau said. “But when you’re in a situation where your life’s threatened anyway, I don’t feel you have anything to lose.”
Continue reading the main story
Trending

    Feature: When the Revolution Came for Amy Cuddy
    Trump’s Condolence Call to Soldier’s Widow Ignites an Imbroglio
    Inside a Secretive Group Where Women Are Branded
    Fallen Troops’ Families Tell of Meeting Presidents: Sympathy and Sometimes Discomfort
    Stream These 11 Underappreciated TV Shows on Hulu
    Jann Wenner and His Biographer Have a Falling Out
    F.D.A. Approves Second Gene-Altering Treatment for Cancer
    Contributing Op-Ed Writer: Yes, This Is a Witch Hunt. I’m a Witch and I’m Hunting You.
    Feature: Why Are More American Teenagers Than Ever Suffering From Severe Anxiety?
    A Marine Attacked an Iraqi Restaurant. But Was It a Hate Crime or PTSD?

View More Trending Stories »
What's Next
Loading...

Go to Home Page »
Site Index The New York Times
Site Index Navigation
News

    World
    U.S.
    Politics
    N.Y.
    Business
    Tech
    Science
    Health
    Sports
    Education
    Obituaries
    Today's Paper
    Corrections

Opinion

    Today's Opinion
    Op-Ed Columnists
    Editorials
    Op-Ed Contributors
    Letters
    Sunday Review
    Video: Opinion

Arts

    Today's Arts
    Art & Design
    Books
    Dance
    Movies
    Music
    N.Y.C. Events Guide
    Television
    Theater
    Video: Arts

Living

    Automobiles
    Crossword
    Food
    Education
    Fashion & Style
    Health
    Jobs
    Magazine
    N.Y.C. Events Guide
    Real Estate
    T Magazine
    Travel
    Weddings & Celebrations

Listings & More

    Reader Center
    Classifieds
    Tools & Services
    N.Y.C. Events Guide
    Multimedia
    Photography
    Video
    NYT Store
    Times Journeys
    Subscribe
    Manage My Account
    NYTCo

    Subscribe Home Delivery Digital Subscriptions Crossword

    Email Newsletters Alerts Gift Subscriptions Corporate Subscriptions Education Rate

    Mobile Applications Replica Edition

Site Information Navigation

    © 2017 The New York Times Company
    Home
    Search
    Accessibility concerns? Email us at accessibility@nytimes.com. We would love to hear from you.
    Contact Us
    Work With Us
    Advertise
    Your Ad Choices
    Privacy
    Terms of Service
    Terms of Sale

Site Information Navigation

    Site Map
    Help
    Site Feedback
    Subscriptions

No comments: