</s>WASHINGTON (AP) - U.S. health officials have approved a breakthrough treatment that genetically engineers patients' own blood cells into an army of assassins to seek and destroy childhood leukemia.

The Food and Drug Administration calls the approval historic, the first gene therapy to hit the U.S. market. Made from scratch for every patient, it's one of a wave of "living drugs" under development to fight additional blood cancers and other tumors, too.

Novartis Pharmaceuticals has set the price for its one-time infusion of so-called "CAR-T cells" at $475,000, but says there would be no charge for patients who didn't show a response within a month.

In this July 9, 2015, photo, provided by Novartis Pharmaceuticals Corp., human T cells belonging to cancer patients arrive at Novartis Pharmaceuticals Corp.'s Morris Plains, N.J., facility. The Food and Drug Administration (FDA) approved the first treatment that genetically engineers patients' own blood cells into an army of leukemia-fighting assassins. Manufacturer Novartis will create those turbocharged cells in this facility and ship them back to hospitals to infuse into patients. (Brent Stirton/Novartis Pharmaceuticals Corp. via AP)

This photo provided by the Children's Hospital of Philadelphia, taken in May 2017, shows Emily Whitehead five years after she became the first pediatric patient in the world to receive an experimental therapy at the hospital that has put her leukemia into long-term remission. Opening a new era in cancer care, the Food and Drug Administration (FDA) approved the first treatment that genetically engineers patients' own blood cells into an army of assassins to seek and destroy childhood leukemia. (Children's Hospital of Philadelphia via AP)

This undated image made available by Novartis on Wednesday, Aug. 31, 2017 shows an IV bag of their drug Kymriah. On Wednesday, the Food and Drug Administration approved this first treatment that genetically engineers patients' own blood cells to seek and destroy childhood leukemia. (Novartis via AP)

This photo provided by the Children's Hospital of Philadelphia, taken in May 2017, shows Dr. Stephan Grupp who led a key study of a newly approved treatment for childhood leukemia. Opening a new era in cancer care, the Food and Drug Administration (FDA) approved the first treatment that genetically engineers patients' own blood cells into an army of assassins to seek and destroy cancer. (Children's Hospital of Philadelphia via AP)</s>WASHINGTON (AP) -- U.S. health officials have approved a breakthrough treatment that genetically engineers patients' own blood cells into an army of assassins to seek and destroy childhood leukemia.

The Food and Drug Administration calls the approval historic, the first gene therapy to hit the U.S. market. Made from scratch for every patient, it's one of a wave of "living drugs" under development to fight additional blood cancers and other tumors, too.

Novartis Pharmaceuticals has set the price for its one-time infusion of so-called "CAR-T cells" at $475,000, but says there would be no charge for patients who didn't show a response within a month.</s>WASHINGTON (AP) – The Food and Drug Administration has approved the first treatment that genetically engineers patients’ own blood cells to seek and destroy childhood leukemia.

The move opens a new era in cancer care.

FDA’s action Wednesday makes Novartis Pharmaceutical’s CAR-T cell treatment the first type of gene therapy to hit the U.S. market.

It’s one in a wave of “living drugs” being developed for blood cancers and maybe other tumors, too.

The Novartis therapy is for children and young adults with acute lymphoblastic leukemia, or ALL, who have relapsed despite today’s best treatments.

It’s made from scratch, an expensive process that takes about three weeks.

Despite some serious side effects, a key test found a one-time infusion put about 80 percent of hard-to-treat patients into remission.</s>A revolutionary cancer therapy that uses genetically engineered immune cells has been approved by the U.S. Food and Drug Administration, ushering in a new era of cancer treatment.

The FDA calls the treatment, marketed by Novartis, the “first gene therapy” to be available in the U.S. The therapy is designed to treat an often-lethal type of blood and bone marrow cancer that affects children and young adults. Known as a CAR-T therapy, the approach has shown remarkable results in patients. The one-time treatment will cost $475,000, according to Novartis.

“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.

The therapy, which will be marketed as Kymriah by Novartis, is a customized treatment that uses a patient’s own T cells, a type of immune cell. A patient’s T cells are extracted and cryogenically frozen so that they can be transported to Novartis’s manufacturing center in New Jersey. There, the cells are genetically altered to have a new gene that codes for a protein—called a chimeric antigen receptor, or CAR. This protein directs the T cells to target and kill leukemia cells with a specific antigen on their surface. The genetically modified cells are then infused back into the patient.

In a clinical trial of 63 children and young adults with a type of acute lymphoblastic leukemia, 83 percent of patients that received the CAR-T therapy had their cancers go into remission within three months. At six months, 89 percent of patients who received the therapy were still living, and at 12 months, 79 percent had survived.

An estimated 3,100 patients aged 20 and younger in the U.S. are diagnosed with acute lymphoblastic leukemia each year, making it the most common childhood cancer, according to the National Cancer Institute. Current treatment options include chemotherapy and stem-cell patients, but about 600 pediatric and young adult patients with the disease relapse each year, and many patients remain incurable.

The historic approval bodes well for rival companies Kite Pharma and Juno Therapeutics, which are also developing CAR-T therapies. Kite Pharma, which is awaiting FDA approval for its CAR-T therapy to treat a form of blood cancer in adults, was this week bought out by Gilead in a deal worth $11.9 billion.

In reaction to the FDA approval, David Maloney, medical director of the Fred Hutchinson Cancer Research Center, said in a statement that the Novartis therapy is the “first of what will soon be many” such treatments for a variety of cancers.

Though the Novartis therapy has shown extraordinary results in patients, questions remain about how the company will be able to manufacture personalized therapies quickly enough to get them to patients across the country. Novartis says it takes an average of 22 days to create the therapy, from the time a patient's cells are removed to when they are infused back into the patient. Kymriah will initially be available at 20 U.S. hospitals within a month, Novartis says. Eventually, 32 total sites will offer the therapy.

CAR-T therapy has also been known to cause potentially life-threatening side effects in some patients, including neurological problems and a reaction called cytokine release syndrome. Juno Therapeutics ended a CAR-T study earlier this year after patient died from cerebral edema, or swelling in the brain. No patients treated with Novartis's CAR-T therapy have experienced that complication, according to the company.

The FDA defines gene therapy as a medicine that “introduces genetic material into a person’s DNA to replace faulty or missing genetic material” to treat a disease or medical condition. This is the first such therapy to be available in the U.S. Two gene therapies for rare, inherited diseases have already been approved in Europe.</s>Opening a new era in cancer care, the Food and Drug Administration on Wednesday approved the first treatment that genetically engineers patients' own blood cells into an army of assassins to seek and destroy childhood leukemia.

The CAR-T cell treatment developed by Noartis and the University of Pennsylvania is the first type of gene therapy to hit the U.S. market — and one in a powerful but expensive wave of custom-made "living drugs" being tested against blood cancers and some other tumors, too.

"This is a brand new way of treating cancer," said Dr. Stephan Grupp of Children's Hospital of Philadelphia, who treated the first child with CAR-T cell therapy — a girl who'd been near death but now is cancer-free for five years and counting. "That's enormously exciting."

CAR-T treatment uses gene therapy techniques not to fix disease-causing genes but to turbocharge T cells, immune system soldiers that cancer too often can evade. Researchers filter those cells from a patient's blood, reprogram them to harbor a "chimeric antigen receptor" that zeroes in on cancer, and grow hundreds of millions of copies. Returned to the patient, the revved-up cells can continue multiplying to fight disease for months or years.

Just the fact that I get a future is completely remarkable - I shouldn't be alive right now," Mitchell Carbon, 19, told CBS News' Jim Axelrod. After just one infusion, he is now cancer-free.

"It's a pretty amazing new treatment," Dr. David Agus, director of the USC Norris Westside Cancer Center and CBS News medical contributor, told "CBS News This Morning" in July, when an FDA panel voted to recommend approval. "They take the white [blood] cells out of a child with cancer, they send them to [a lab in] New Jersey, and they put in a gene to reprogram these cells to attack the cancer."

Novartis didn't immediately disclose the therapy's price but it is expected to cost hundreds of thousands of dollars. It's made from scratch for every patient.

"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.

This first use of CAR-T therapy is aimed at patients desperately ill with a common pediatric cancer — acute lymphoblastic leukemia — that strikes more than 3,000 children and young adults in the U.S. each year. While most survive, about 15 percent relapse despite today's best treatments, and their prognosis is bleak.

In a key study of 63 advanced patients, 83 percent went into remission. It's not clear how long that benefit lasts: Some patients did relapse months later. The others still are being tracked to see how they fare long-term.</s>Opening a new era in cancer care, the Food and Drug Administration on Wednesday approved the first treatment that genetically engineers patients' own blood cells into an army of assassins to seek and destroy childhood leukemia.

The CAR-T cell treatment developed by Novartis and the University of Pennsylvania is the first type of gene therapy to hit the U.S. market — and one in a powerful but expensive wave of custom-made "living drugs" being tested against blood cancers and some other tumors, too.

"This is a brand new way of treating cancer," said Dr. Stephan Grupp of Children's Hospital of Philadelphia, who treated the first child with CAR-T cell therapy — a girl who'd been near death but now is cancer-free for five years and counting. "That's enormously exciting."

CAR-T treatment uses gene therapy techniques not to fix disease-causing genes but to turbocharge T cells, immune system soldiers that cancer too often can evade. Researchers filter those cells from a patient's blood, reprogram them to harbor a "chimeric antigen receptor" that zeroes in on cancer, and grow hundreds of millions of copies. Returned to the patient, the revved-up cells can continue multiplying to fight disease for months or years.

Novartis didn't immediately disclose the therapy's price, but it is expected to cost hundreds of thousands of dollars. It's made from scratch for every patient.

"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.

This first use of CAR-T therapy is aimed at patients desperately ill with a common pediatric cancer — acute lymphoblastic leukemia — that strikes more than 3,000 children and young adults in the U.S. each year. While most survive, about 15 percent relapse despite today's best treatments, and their prognosis is bleak.

In a key study of 63 advanced patients, 83 percent went into remission. It's not clear how long that benefit lasts: Some patients did relapse months later. The others still are being tracked to see how they fare long-term.

Still, "a far higher percentage of patients go into remission with this therapy than anything else we've seen to date with relapsed leukemia," said Dr. Ted Laetsch of the University of Texas Southwestern Medical Center, one of the study sites. "I wouldn't say we know for sure how many will be cured yet by this therapy. There certainly is a hope" that some will be.

Most patients suffered side effects that can be grueling, even life-threatening. An immune overreaction called "cytokine release syndrome" can trigger high fevers, plummeting blood pressure and in severe cases organ damage, requiring special care to tamp down those symptoms without blocking the cancer attack. Also Wednesday, the FDA designated a treatment for those side effects.

For many ALL patients, the new CAR-T therapy might replace bone marrow transplants that cost more than half a million dollars, noted Grupp, who led the Novartis study.

"I don't want to be an apologist for high drug prices in the U.S.," Grupp stressed. But if it's the last treatment they need, "that's a really significant one-time investment in their wellness, especially in kids who have a whole lifetime ahead of them."

Initially, Novartis' CAR-T version — to be sold under the brand name Kymriah — will be available only through certain medical centers specially trained to handle the sophisticated therapy and its side effects. Patients' collected immune cells will be frozen and shipped to a Novartis factory in New Jersey that creates each dose, a process the company says should take about three weeks.

While this first use of CAR-T therapy only is aimed at a few hundred U.S. patients a year — relapsed ALL patients up to age 25 — it's being tested as a treatment for thousands more. Kite Pharma's similar CAR-T brand, developed by the National Cancer Institute, is expected to win approval later this year to treat aggressive lymphoma, and Juno Therapeutics and other companies are studying their own versions against blood cancers including multiple myeloma.

Scientists around the country also are trying to make CAR-T therapies that could fight more common solid tumors such as brain, breast or pancreatic cancers — a harder next step.</s>Aug 30 (Reuters) - Novartis AG on Wednesday won highly anticipated U.S. approval for the first of a new type of potent gene-modifying immunotherapy for leukemia, a $475,000 treatment that marks the start of a potential new treatment paradigm for some cancers.

The approval was widely expected after an FDA advisory panel last month unanimously recommended the action, and Novartis shares closed virtually unchanged in Swiss trading on Wednesday.

Novartis announced an agreement with the U.S. Centers for Medicare and Medicaid Services under which payment for the therapy will be based on clinical outcomes achieved.

The treatment, to be sold under the name Kymriah, was approved for patients up to 25 years of age who have relapsed or were not helped by initial treatment for B-cell acute lymphoblastic leukemia (ALL).

Kymriah belongs to a new class of drugs called chimeric antigen receptor T-cell, or CAR-T therapies, that involves removing disease-fighting T cells from a patient, genetically modifying them to better recognize and attack cancer and then replacing them, where they can circulate for years seeking out the disease.

"We´re entering a new frontier in medical innovation with the ability to reprogram a patient´s own cells to attack a deadly cancer," U.S. Food and Drug Administration Commissioner Scott Gottlieb said in a statement.

U.S. biotech Gilead Sciences Inc jumped into the field this week with its announcement of an $11.9 billion deal to buy Kite Pharma. Gilead shares were up more than 5 percent on Wednesday as Kite is widely seen as likely to receive the next U.S. approval of a CAR-T therapy.

Shares of Bluebird Bio Inc, which is also developing a treatment in the emerging field, were up 11.8 percent at $114.47 on the Nasdaq.

"Two years ago many people would have told you these types of treatments were science fiction," said Brad Loncar, Chief Executive of Loncar Investments, which runs the Loncar Cancer Immunotherapy ETF

In clinical trials, CAR-T therapies have shown remarkable efficacy against blood cancers. In the pivotal Novartis trial, 83 percent of patients achieved remission with a disease that has historically poor outcomes.

"We've never seen anything like this before and I believe this therapy may become the new standard of care for this patient population," Dr Stephan Grupp of Children's Hospital of Philadelphia said in a statement.

However, this type of therapy is not without risk of severe side effects as the immune system goes into high gear. Kymriah will carry a boxed warning for cytokine release syndrome, a potentially lethal systemic response to the activation and proliferation of CAR-T cells, causing high fever and potential for neurological problems.

Juno Therapeutics Inc last year reported a handful of patient deaths during trials of its CAR-T therapy for leukemia. Its shares were down 5.7 percent at $41.30. (Reporting by Bill Berkrot and Mike Erman in New York, Natalie Grover in Bengaluru, Deena Beasley in Los Angeles and Julie Steenhuysen in Chicago; Editing by Shounak Dasgupta and Matthew Lewis)</s>The Food and Drug Administration (FDA) has approved the latest Novartis AG (ADR) (NYSE: ) cancer treatment.

The drug is considered a groundbreaking approach to treating the deadly illness and it is called CAR-T, consisting of advanced gene therapy. It harvests patients’ white blood cells and rewires them to target tumors.

Novartis’ new product is the first CAR-T therapy to arrive before the FDA, proving to be the first in many innovative treatments that will revolutionize the way cancer is approached. However, the treatment is not exactly cheap.

The price tag comes in at $475,000 for the treatment, which is considerably lower than analysts expected despite the fact that it seems extremely high. Experts predicted the price to go for as high as $750,000, while U.S. regulators said that a fair price for it would be $700,000.

CAR-T is designed to treat young adults and children with relapsed acute lymphoblastic leukemia, and it is being marketed as Kymriah. “I think this is most exciting thing I’ve seen in my lifetime,” said Dr. Tim Cripe, an oncologist with Nationwide Children’s Hospital, at an FDA meeting on Kymriah in July.

Novartis added that it is working alongside Medicare on a system in which the government would only pay for the treatment if patients respond within a month.</s>The University of Chicago Medicine is one of a limited number of U.S. sites working to offer a breakthrough gene therapy for pediatric acute lymphoblastic leukemia (ALL), which was just approved by the U.S. Food and Drug Administration.

Chimeric antigen receptor T-cell, or CAR T-cell, therapy, targets two common forms of cancer and has been used in clinical trials at UChicago Medicine to treat both ALL and diffuse large B-cell lymphoma (DLBCL) in adults.

"CAR T-cell therapy has revolutionized the treatment of acute lymphoblastic leukemia," according to cancer specialist John Cunningham, MD, chair of pediatrics at the University of Chicago and physician-in-chief at Comer Children's Hospital. "I came to the United States 30 years ago with the intent to work on therapies such as this. We need focused treatments like this that can eradicate particular types of cancer."

One of the cancers, B-cell acute lymphoblastic leukemia, is the most common childhood cancer. ALL is a rapidly progressive leukemia that primarily affects children and young adults, from age 3 to 25. Nearly 3,100 patients under 21 years old in the U.S. will be diagnosed with ALL this year.

Used to supplement forms of cancer treatment like chemotherapy, radiation and stem cell transplants, CAR T-cell therapy works by using modified versions of a patient's own blood cells to target and destroy cancer cells.

UChicago Medicine is working with Novartis, the maker of Kymriah, to be part of a national network offering the treatment to pediatric patients in Chicago. The effort for pediatric patients is being spearheaded by Cunningham. The FDA approved the use of Kymriah in people up to age 25. Novartis announced it plans to use clinical outcomes to help determine the cost of treatment.

Ninety percent of pediatric patients with ALL enrolled in early CAR T-cell clinical trials throughout the country went into lasting remission. DLBCL patients often relapse after standard treatments, but those treated with CAR T-cell therapy have doubled the long-term survival rate. Now, about 50 percent of those patients appear to have lasting complete remissions.

The FDA approved Kymriah for use in pediatric ALL patients and is expected to approve a CAR T-cell therapy for treatment of DLBCL and ALL in adults being developed by Kite Pharma Inc.

Sam Tinaglia, 18, of Park Ridge was one of the first 100 children in the country to participate in clinical trials of CAR T-cell therapy for acute lymphoblastic leukemia. Diagnosed in 2003 at age 5, Tinaglia relapsed for the first time five years after initial treatment. Over the next seven years, doctors tried more rounds of chemotherapy and a bone marrow transplant. With each recurrence his chances of survival lessened.

"We thought we had reached the end, " said Sam's mother, Suzie. "But then Dr. Cunningham told us about the CAR T-cell clinical trial that was giving hope to kids, like Sam, who had no other options."

In 2015, during his junior year of high school, Sam underwent CAR T-cell therapy at Children's Hospital of Philadelphia. "Six weeks later, they sent me home and that was pretty much it," said Sam, who just started his freshman year of college. "I'm obviously very happy, because my cancer, hopefully is gone forever."
The Food and Drug Administration has approved the first treatment that genetically engineers patients' own blood cells to destroy acute lymphoblastic leukemia. The approval makes Novartis the proprietor of the first gene therapy to hit the U.S. market.