FDA approves first genetically modified 'living drug' for cancer treatment

Emily Whitehead is seen in May five years after she became the first pediatric patient in the world to receive an experimental therapy at Children's Hospital of Philadelphia that has put her leukemia into long-term remission

"This is the first-ever true gene therapy treatment made available to the U.S. population and will help accelerate the speed at which we will see even more gene-based therapies come to fruition".

A rapidly emerging immunotherapy approach is called adoptive cell transfer (ACT): collecting and using patients' own immune cells to treat their cancer.

A brand new way of treating leukemia is on its way to the US market, and it could change how doctors fight cancer.

An FDA advisory committee had recommended the therapy for approval in July to treat the relapse of a blood cancer known as B-cell acute lymphoblastic leukemia, or ALL.

What is proposed in the Kymriah, this is a single treatment, with a remission rate of 83% in the three months following treatment.

The new therapy, which would cost just under €400,000, turns a patient's cells into a "living drug" and trains them to recognise and attack the disease. The illness is the most common cancer for children in the USA with the National Cancer Institute estimating around 3,100 patients less than 20 years of age diagnosed each year. Novartis said it hopes to have an initial network of 20 treatment centers within a month with plans to expand that to 32 by the end of the year.

So-called honesty app Sarahah being used to bully peers
For users who are anxious about their privacy on Sarahah , you can go to the Sarahah website and remove your account from the app. This essentially means that the company is collecting data from users for a new feature which doesn't even exists as of today.

Doctors remove T cells from each cancer victim and ship the material to its factory in Morris Plains, New Jersey. The genetic changes wrought through the customized process creates coding for a protein known as a chimeric antigen receptor, or vehicle. Penn and Novartis are also investigating the next generation of auto therapies for multiple myeloma, and for solid tumors, through trials in glioblastoma, mesothelioma, and ovarian and pancreatic cancer. Along with the Kymriah approval, the FDA also expanded the approval of a drug named Actemra (tocilizumab) used to offset the side effects like these, related to a cytokine release syndrome.

In CAR-T cell treatment the Patients own blood cells or immune cells are filtered out and sent to lab where they get reprogrammed into a CAR (chimeric antigen receptor) which makes the T cells super active or turbocharge and after that the T cells are again infused into the body so that they could combat with the cancerous cells more efficiently. Indication-specific pricing is an approach pharmacy benefits manager Express Scripts has been pushing for other cancer drugs. According to him, a price of $475,000 per treatment was "excessive".

Despite the high price tag, it's worth remembering that other leukemia treatments, like bone marrow transplants, can be even more expensive.

For now, Kymriah is only available to a small subsection of cancer patients.

Other side effects include serious infections, low blood pressure (hypotension), acute kidney injury, fever, and decreased oxygen (hypoxia). Novartis CEO Joseph Jimenez, however, says developing the treatment cost more than $1 billion. In a pivotal trial, one-quarter of patients who responded initially later saw their disease progress.

"We're at the very beginning of what's going to be a big new field of medicine", David Epstein, who helped license Kymriah in from the University of Pennsylvania while at Novartis.

Related news:

Hot News

isis-killer-beheading-video-story-top Controversial Milwaukee County sheriff David Clarke resigns
Sep 01, 2017 - 01:34
Clarke submitted his resignation to the Milwaukee County clerk, noting in his letter that it would be effective as of 11:59 p.m. Milwaukee County Sheriff David Clarke has resigned , county clerk George Christenson confirmed to TPM Thursday.

isis-killer-beheading-video-story-top Diamond bath in Taylor Swift's new music video cost $13 million
Aug 31, 2017 - 01:24
For all the details on Swift's new music and her life now, pick up the latest issue of PEOPLE, on newsstands Friday . According to Music Business Worldwide, the hit quickly became the most streamed song on Spotify in it's first day .

isis-killer-beheading-video-story-top Investor suit against former Uber CEO moved to arbitration
Aug 31, 2017 - 01:23
Khosrowshahi, 48, is an Iranian immigrant who came to the United States with his parents in 1978 during the Iranian Revolution. Whitman recently stepped off the board of directors of HP Inc which fuelled rumours that she might make the move to Uber.

isis-killer-beheading-video-story-top Israeli lawmakers visit Al-Aqsa Mosque compound after two-year ban
Aug 30, 2017 - 01:41
Guterres is expected to visit the Gaza Strip on Wednesday, the third leg in his visit to Israel and the Palestinian Territories. President Donald Trump, in the past, has said he hopes to mediate the "ultimate deal" between Israel and the Palestinians.

isis-killer-beheading-video-story-top Lions resign Stafford to 5-year extension
Aug 30, 2017 - 01:40
There has been media and fan speculation about the terms of Stafford's next deal, a mystery that came to an end Monday night. Since then, the eight-year veteran has posted over 30,000 passing yards, 187 touchdowns and 108 interceptions.

isis-killer-beheading-video-story-top Uber Said to Pick Expedia CEO to Lead Company Out of Crisis
Aug 30, 2017 - 01:39
Lawyers for the two stockholders sent a letter to Uber's board demanding to look at Uber's books, Business Insider reported . However, Whitman has publicly and staunchly rejected the possibility of becoming Uber's CEO, so her chances are slim.

isis-killer-beheading-video-story-top Kim Kardashian Says Saint Cries When North Won't Play with Him
Aug 29, 2017 - 00:34
Check out the cover below and see what you think of the budding controversy. When I'm out and people call North's name, that's when it's weird for me.

Game of Thrones Season Finale Had to Have One Shocking Death
Aug 29, 2017 - 00:33
There was obvious excitement for " The Dragon and the Wolf " given the type of season finales that we've gotten in the past. For all of the power that the Night King and the Army of the Dead had, the one thing stopping them was The Wall.

isis-killer-beheading-video-story-top Alex Rodriguez, Jennifer Lopez Flew To Vegas With Robert Kraft
Aug 29, 2017 - 00:32
The quarterback didn't watch the fight Saturday night, in which Mayweather won to increase his record to 50-0. With post on his social media accounts, dos Anjos called McGregor "McChicken" in the first of many hashtags.

isis-killer-beheading-video-story-top Two Lawsuits Filed Over Trump's Transgender Military Ban
Aug 29, 2017 - 00:31
Also named in the ACLU lawsuit are each of the civilian heads of the military departments. The lawsuit is at least the third filed against Trump's transgender military ban.

isis-killer-beheading-video-story-top Fitbit's New Ionic Smartwatch Is Here
Aug 29, 2017 - 00:31
Three color combinations will be offered, and an Adidias version with special training programs will also be available next year. Ionic is priced at $299.95 and is now up for pre-ordering via Fitbit's website , shipping is expected to starts in 3 to 4 weeks.

isis-killer-beheading-video-story-top Bethesda's next game might be a Skyrim-like Game of Thrones title
Aug 29, 2017 - 00:31
One of those projects is trademarked as " Starfield " and is expected to be the first of the two to be developed and released. The inevitable next step would be to give us a chance to freely explore that world on our own in a massive video game.

US Open Tennis Preview
Aug 28, 2017 - 00:38
Two years before that, at 17, Sharapova won her first major title at Wimbledon . The British No 1 is in the same quarter of the draw as Sharapova or Halep.

isis-killer-beheading-video-story-top England v Windies: Watch pick of second day's action at Headingley
Aug 27, 2017 - 00:23
Stokes was dropped on 98 before moving to his sixth Test hundred and being dismissed by Gabriel without adding to his century. West Indies' Darren Sammy and South Africa pair Hashim Amla and Morne Morkel are also in the squad.

isis-killer-beheading-video-story-top Donald Trump slammed for pardoning controversial former sheriff Joe Arpaio
Aug 27, 2017 - 00:21
He had gained notoriety for backing Mr Trump's " birther " campaign and for his massive roundups of suspected illegal immigrants. ET, the White House announced that Trump granted a presidential pardon to former Arizona Sheriff Joe Arpaio .