Cancer Gene Therapy Gets Green Light - And $475000 Price Tag

US clears first 'living drug' for childhood leukaemia

The new cells would then continue to multiply to fight disease for months or years. "New technologies such as gene and cell therapies hold out the potential to transform medicine". The Food and Drug Administration called the approval historic, this being the first gene therapy on the American market, ABC News reports.

It's a type of cancer immunotherapy, which harnesses the body's immune system to take on cancer cells.

Those reprogrammed cells are sent back to the patient and administered, at which point the new cells try to kill off the cancer cells.

The FDA's move to approve the auto T-cell therapy, which will be marketed under the name "Kymriah" by Novartis Pharmaceuticals, opens the door to a new era of cancer medicine, and medicine more broadly.

Actually, the gene therapy techniques of the CAR-T treatment procedure is not meant to fix the disease causing genes rather these make the T cells get turbocharged and hence cancer can not evade the immune system.

About 3,100 Americans ages 20 and younger are diagnosed with acute lymphoblastic leukemia each year, and 15 to 20 percent of them don't respond to the initial treatment or have their disease return, according to the National Cancer Institute.

"There has been an urgent need for novel treatment options that improve outcomes for patients with relapsed or refractory B-cell precursor ALL", Novartis, the drug company that makes Kymriah, said in a statement.

The treatment is personalized for every patient and Novartis Pharmaceuticals is planning to charge 475,000$ for it.

Although the price of the therapy is high by any standard, Cooper stated that Novartis is "committed to doing everything we can to ensure that children and young adults who can benefit from [tisagenlecleucel] have access to the therapy and we are working with payers to ensure they fully understand the value of [it] and provide coverage for patients. There are also over 800 cell therapy clinical trials now underway, and a considerable pool of research and pre-clinical work right across the cell therapy sector", said Bruce Levine, PhD, ISCT Commercialization and Immuno & Gene Therapy Committees, Barbara and Edward Netter Professor in Cancer Gene Therapy, University of Pennsylvania Perelman School of Medicine.

There are other severe side effects that can pop up as well that can require hospitalization. Both CRS and neurological events can be life-threatening. And the drug comes with serious side-effects, including a reaction known as cytokine release syndrome, that can prove fatal if not controlled with immunosuppressant drugs. Right now, that's where most of the big successes have come from, but one day that could expand out to solid tumors, and maybe even auto-immune diseases like type 1 diabetes, Epstein said. Since the CD19 antigen is also present on normal B-cells, and Kymriah will also destroy those normal B cells that produce antibodies, there may be an increased risk of infections for a prolonged period of time.

"We've never seen anything like this before", said Stephan Grupp, director of the cancer immunotherapy frontier program at Children's Hospital of Philadelphia, the first medical center to study Kymriah in children.

Related:

  • This is what the new Kermit the Frog voice sounds like

    This is what the new Kermit the Frog voice sounds like

    The iconic Muppet character was originally voiced by Jim Henson , who brought the muppet's green and down to earth leader to life. Matt Vogel , who replaced longtime Kermit the Frog puppeteer Steve Whitmire , made his debut as the beloved Muppet this week.
    It doesn't matter if Taylor's new song is any good

    It doesn't matter if Taylor's new song is any good

    KATY PERRY and Calvin Harris preferred to laugh it off rather than Shake It Off after being mocked in Taylor Swift's new video. Taylor knew her haters were going to be talking about her being dramatic and just stirring up trouble.
    Kardashian family pledges $500000 to Harvey relief efforts

    Kardashian family pledges $500000 to Harvey relief efforts

    The family seems to have been inspired by actor Kevin Hart , who called on celebrities to contribute to his relief fund . She told the Houston Chronicle in a statement on Monday that she was lending a hand, but didn't specify a dollar amount.
  • Harvey drives up gas prices

    Harvey drives up gas prices

    Nearly 2 million barrels of refining capacity was taken off line because storm shakes Texas with unprecedented flooding and rain. Eight refineries in Texas were in the process of controlled shutdowns.
    EU's Juncker warns Britain to first agree with divorce terms

    EU's Juncker warns Britain to first agree with divorce terms

    Sitting in for Iain Dale, he said: "It's rather like a ransom, they're treating us like we're a hostage". Barnier said: "We need United Kingdom papers that are clear in order to have constructive negotiations".
    Myanmar Buddhists seek tougher action against Rohingya

    Myanmar Buddhists seek tougher action against Rohingya

    The Myanmar government called the Rohingya rebels terrorists, claiming that they were responsible for murders and arson. According to the Myanmar Times, Tun's statement came before the Minister of Home Affairs Lt.
  • U2 post new song The Blackout

    U2 post new song The Blackout

    Full confession: I may have had an early listen to maybe half a dozen songs from U2's upcoming album some time back. But with a band that has been putting out music for over 40 years, that is to be expected.
    Read the memo Uber's board sent employees announcing its new CEO

    Read the memo Uber's board sent employees announcing its new CEO

    In a following tweet she made this even clearer: "We have a lot of work still to do at HPE and I am not going anywhere". A DE judge says an investor lawsuit targeting Uber Technologies' former CEO must be moved to private arbitration.
    What You Should Know About Apple's Upcoming iPhone 8

    What You Should Know About Apple's Upcoming iPhone 8

    We are only few days away from the new iPhone 8, as it is expected to be released next month - after Apple all but confirmed it. We previously saw a video of the first generation iPhone being torture tested and now we have a teardown video for the handset.
  • Chelsea target Danny Drinkwater asks to leave Leicester City

    Chelsea target Danny Drinkwater asks to leave Leicester City

    A move to Stamford Bridge would see Premier League title victor Drinkwater reunited with former Leicester team-mate N'Golo Kante. The Blues take on Leicester City in their next Premier League game at the King Power Stadium after the worldwide break.
    Trump to seize populist mantle for pitching messy tax overhaul - Source Politico

    Trump to seize populist mantle for pitching messy tax overhaul - Source Politico

    The current tax code is too complicated, too costly and too time consuming for most families to understand and file their taxes. In terms of policy, though, there are signs that the Trump White House is continuing to defer to Republicans in Congress.
    North Korea's Kim has third child

    North Korea's Kim has third child

    The Kim family has ruled North Korea since the country's founding in 1948, but very little is known about its members . Though Kim Jong Un was the youngest, he soon appeared to be being groomed for succession.

Comments

Other news