Novartis' Kymriah first cell therapy to win USA green light

The FDA has approved Novartis' cancer immunotherapy tisagenleucel (CTL019) under the Kymriah brand name, setting a $475,000 price per course for the chimeric antigen receptor T cell (CAR-T) therapy.

USA regulators approved a revolutionary new treatment from Novartis that modifies the body's own immune-system cells and reinjects them to treat, and in many cases cure, patients with deadly blood cancers who have run out of other options.

Dr. Carl June, director of the center for cellular immunotherapies at University of Pennsylvania's Abramson Cancer Center, who pioneered the therapy, was still emotional an hour after receiving the news of the approval in an email. This is the first time the agency has greenlighted a gene therapy approach for use in the United States. The company also said it anticipates making additional filings beyond the US and European Union next year.

"As a breakthrough immunocellular therapy for children and young adults who desperately need new options, Kymriah truly embodies our mission to discover new ways to improve patient outcomes and the way cancer is treated", said Bruno Strigini, CEO of Novartis, the company that developed Kymriah.

These re-engineered T-cells are then transfused back into the patient, where they can begin attacking leukaemia. Now 12, she has been free of leukemia for more than five years.

Kymriah is one type of so-called CAR-T cancer therapies.

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.

Cool, Composed MS Dhoni Stretches India Lead in ODI Series
India were storming to another victory. "The board has been hugely supportive of everything that I do and what the players do". They were sensible, collected runs in singles and later on played their shots as they inched closer to the target.

The price per person is likely to be much higher when accounting for additional costs, such as drugs to prepare the patient for the treatment and medicines to control potential side-effects.

Once the cancer-killing cells are reprogrammed, they are inserted back inside the patient, finding their target and multiplying. That's what a researcher is saying following the FDA's approval of America's first-ever treatment that genetically alters a person's cells to fight cancer. Until now, treatments have focused on bringing in drugs, surgery or radiation to weaken the cancer. In trials, the therapy achieved remission in 83% of patients.

Instead, it harnesses a patient's own immune cells, called T-cells and white blood cells, and trains them to recognize and fight cancer.

The safety and effectiveness of the Kymriah have been demonstrated in a clinical trial with 63 patients pediatric and young adults who had not responded to other treatments or had relapsed. However, even patients who achieve an initial response to standard therapy will eventually relapse, and the prognosis is extremely poor for these patients.

According to Gottlieb, the FDA has already granted more than 550 active investigational new drug applications related to gene therapy products, including 76 active investigational new drug applications related to CAR-T cell products like Kymriah. When his blood was analyzed, it turned out that every single CAR-T cell he had had descended from a single T-cell.

Shares of Juno Therapeutics Inc, which past year reported a handful of patient deaths during trials of its CAR-T therapy, were down 8.9 percent at $39.92.


Popular
  • 5 potential ripple effects from the Kyrie Irving trade

    5 potential ripple effects from the Kyrie Irving trade

    The Cavs have the next-best odds to win a championship at 9/2, followed by the Celtics at 15/2 and the Rockets at 9/1. If you haven't heard, the Cleveland Cavaliers traded Irving , their superstar point guard to the Boston Celtics .
    Rare total eclipse mostly visible from Plumas

    Rare total eclipse mostly visible from Plumas

    As the eclipse approaches, the local weather forecast will be a more accurate indicator of the visibility of the eclipse . Kids will be able to read books about eclipses and art supplies will be available for anyone inspired by the eclipse.
    Venezuela's voter turnout figures tampered with

    Venezuela's voter turnout figures tampered with

    With the opposition boycotting the election, virtually all the candidates were supporters of Mr Maduro's ruling socialist party. Any sanctions by the United States - aside from the dubious moral authority of the Trump administration - feed Mr.
  • Uber Picks New CEO, Expedia's Dara Khosrowshahi

    Uber Picks New CEO, Expedia's Dara Khosrowshahi

    Khosrowshahi is not oblivious to the transportation industry as he has been heading a travel company itself. According to Tech Crunch , a person to take over CEO role is Dara Khosrowshahi , former CEO of Expedia .
    ECLIPSE SAFETY: How to safely watch the Great American Eclipse of 2017

    ECLIPSE SAFETY: How to safely watch the Great American Eclipse of 2017

    If you put them on and look at a very bright light in your house and you can not see that light at all, you're getting close. If you are looking for a place to stay during the eclipse , be careful if you are booking online through a third-party site.

    After Health Care Collapse, GOP Seeks Redemption With Tax Cuts

    He added that the Republican Party has a "once-in-a-generation opportunity" to redesign the nation's tax laws. Though Republicans occupy 52 seats, they were able to garner only 49 yes votes on the health care bill.
  • Hurricane Harvey to Have Impact on Gas Prices Nationwide

    Hurricane Harvey to Have Impact on Gas Prices Nationwide

    Houston and Dallas have set up shelters in convention centers and Austin was preparing to house as many as 7,000 evacuees. Houston Mayor Sylvester Turner said that there are already 5,500 people in shelters and that number is expected to rise.
    Hurricane Harvey downgraded to tropical storm, recovery will take 'years'

    Hurricane Harvey downgraded to tropical storm, recovery will take 'years'

    If the state decides to send the team, they would take two boat systems and a logistics trailer with them to Texas, Papagni said. More than 300,000 people are now without power, according to Electric Reliability Council of Texas company's Twitter account .
    Eclipse event in Osoyoos sold out, but still welcoming guests

    Eclipse event in Osoyoos sold out, but still welcoming guests

    When the dark orb of the moon-man mated with the sun-woman's bright circle of light, her fire was temporarily obscured. If Tecumseh South parents want to keep their kids home to watch the eclipse on Monday , absences will be excused.
  • A Tropical Storm is Developing Northeast of Jacksonville

    A Tropical Storm is Developing Northeast of Jacksonville

    If a cyclone does form, they said, it will likely happen over the next day as the storm slides along the North Carolina coast. EDT on August 30 or earlier when it interacts with a mid- to upper-level trough (elongated area) of low pressure.
    Kisner clings to PGA lead; Hideki lurks

    Kisner clings to PGA lead; Hideki lurks

    Two holes and two double bogeys later, Points tumbled down the leaderboard. " I just didn't believe Bud Cauley stayed around". He tied for fourth in the PGA Championship a year ago , and it felt as though he'd make a run for the top spot this weekend.
    Can Ind recover from late blows on day 1?

    Can Ind recover from late blows on day 1?

    They were all out for 28.2 overs against Pakistan in Kandy in 1994 and in 34.2 overs against Australia in Pallekele a year ago . Now Sri-Lanka is set to take the reins to chase down a challenging score target as the second innings of the match begins.

CONNECT